在中国招募中的罕见病临床试验

系统性硬化症 68 项在招目录 第1批 · 112

• 招募中NCT06417502
  Observational Study of Pediatric Rheumatic and Immunologic Diseases in China: The CAPRID Registry
  观察性 · 2022/04/29Peking Union Medical College Hospital
  中国研究中心 9 个：Beijing、Changsha、Chongqing、Hangzhou、Nanjing、Shenzhen
• 仅邀请入组NCT05859997
  Universal CAR-T Cells (BRL-301) in Relapse or Refractory Autoimmune Diseases
  不适用 · 干预性 · 2023/05/17Bioray Laboratories
  中国研究中心 1 个：Shanghai
• 招募中NCT06056921
  Safety and Efficacy of CD19 Targeted CAR-T Therapy for Refractory Autoimmune Disease
  I 期 · 干预性 · 2023/08/31Chongqing Precision Biotech Co., Ltd
  中国研究中心 1 个：Taiyuan
• 招募中NCT05878717
  A Study of the Efficacy and Safety of Belimumab in Adults With Systemic Sclerosis Associated Interstitial Lung Disease
  II 期、III 期 · 干预性 · 2023/09/13GlaxoSmithKline
  中国研究中心 11 个：Beijing、Changchun、Chengdu、Luzhou、Mianyang、Nanjing 等 11 地
• 招募中NCT06058091
  RY_SW01 Cell Injection Therapy in Systemic Sclerosis
  I 期、II 期 · 干预性 · 2023/09/22Jiangsu Renocell Biotech Company
  中国研究中心 1 个：Nanjing
• 招募中NCT06189495
  A Randomized, Double-blind, Placebo-controlled Phase II Clinical Study to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic Profile of Genakumab Injection in Patients With Connective Tissue Disease-associated Interstitial Lung Disease
  II 期 · 干预性 · 2023/12/30Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 4 个：Beijing、Chengdu、Jinan、Wuhan
• 招募中NCT06249438
  A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
  I 期 · 干预性 · 2024/03/20RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06503224
  An Exploratory Clinical Study of SCAR02 Targeting BCMA and CD19 for the Treatment of Refractory Autoimmune Diseases
  不适用 · 干预性 · 2024/04/09The First Affiliated Hospital of University of Science and Technology of China
  中国研究中心 1 个：Changsha
• 招募中NCT06379646
  An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease
  不适用 · 干预性 · 2024/04/24China Immunotech (Beijing) Biotechnology Co., Ltd.
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT06420154
  The Safety and Efficacy of Anti-CD19 CAR-T Cells in Patients With Relapsed/Refractory Autoimmune Diseases
  早期 I 期 · 干预性 · 2024/05/27First Affiliated Hospital of Wenzhou Medical University
  中国研究中心 1 个：Wenzhou
• 招募中NCT06546540
  The Safety and Efficacy of Telitacicept in the Treatment of Systemic Sclerosis
  IV 期 · 干预性 · 2024/06/08Peking University Third Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06414135
  Relmacabtagene Autoleucel for the Treatment of Systemic Sclerosis
  I 期 · 干预性 · 2024/06/12Liangjing Lu
  中国研究中心 1 个：Shanghai

另有 56 项未列出，见该病详情页。

血栓性血小板减少性紫癜 66 项在招目录 第2批 · 79

• 招募中NCT04518475
  Eltrombopag Combining Rituximab Versus Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults
  IV 期 · 干预性 · 2020/08/10Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT04993885
  Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies
  II 期 · 干预性 · 2021/09/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05214391
  A Prospective, One-arm and Open Clinical Study of Zanubrutinib in the Treatment of Immune Thrombocytopenia
  II 期 · 干预性 · 2022/02/15Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05522465
  Short-course High-dose Prednisone and Dexamethasone in Children With ITP
  IV 期 · 干预性 · 2022/10/11Fujian Medical University Union Hospital
  中国研究中心 1 个：Fujian
• 招募中NCT05571774
  Construction of a Database for TTP
  观察性 · 2022/10/20The First Affiliated Hospital of Soochow University
  中国研究中心 1 个：Suzhou
• 招募中NCT05995054
  A Prospective, One-arm and Open Clinical Study of Obinutuzumab in the Treatment of Immune Thrombocytopenia
  II 期 · 干预性 · 2023/08/28Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06577909
  Prednisone Plus IVIg vs. Prednisone for ITP During Pregnancy
  II 期 · 干预性 · 2023/10/19Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06107582
  Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
  观察性 · 2023/11/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06094881
  A Prospective, One-arm and Open Clinical Study of Obinutuzumab in the Treatment of Pediatric Primary Immune Thrombocytopenia (ITP)
  II 期 · 干预性 · 2023/12/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06168851
  Anti-CD38 Antibody Treating Pediatric Primary Immune Thrombocytopenia (ITP)
  II 期 · 干预性 · 2023/12/28Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06352281
  Efficacy and Safety of CAR-T Cells Therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP)
  I 期、II 期 · 干预性 · 2024/02/01920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
  中国研究中心 1 个：Kunming
• 招募中NCT06281327
  Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia
  II 期 · 干预性 · 2024/03/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin

另有 54 项未列出，见该病详情页。

黑色素瘤 65 项在招目录 第2批 · 48

• 招募中NCT03340506
  Dabrafenib and/or Trametinib Rollover Study
  IV 期 · 干预性 · 2018/01/26Novartis Pharmaceuticals
  中国研究中心 1 个：Beijing
• 招募中NCT03755739
  Trans-Artery/Intra-Tumor Infusion of Checkpoint Inhibitors Plus Chemodrug for Immunotherapy of Advanced Solid Tumors
  II 期、III 期 · 干预性 · 2018/11/01Second Affiliated Hospital of Guangzhou Medical University
  中国研究中心 1 个：Guanzhou
• 招募中NCT04616443
  OH2 Injection in Combination With HX008 for Melanoma.
  I 期、II 期 · 干预性 · 2020/12/01Binhui Biopharmaceutical Co., Ltd.
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT04622566
  Lenvatinib and Pembrolizumab in Resectable Mucosal Melanoma
  II 期 · 干预性 · 2020/12/30Peking University Cancer Hospital & Institute
  中国研究中心 1 个：Beijing
• 招募中NCT04331093
  Neoadjuvant SHR-1210 Plus Apatinib for Resectable Stage III-IV Acral Melanoma
  II 期 · 干预性 · 2021/03/01Di Wu
  中国研究中心 1 个：Changchun
• 招募中NCT05098184
  A Study of GC101 TIL in Advanced Melanoma (10hospital)
  早期 I 期 · 干预性 · 2021/09/26Shanghai Juncell Therapeutics
  中国研究中心 1 个：Shanghai
• 招募中NCT05366478
  A Clinical Study of LM103 Injection in the Treatment of Advanced Solid Tumors
  I 期 · 干预性 · 2022/05/30Suzhou BlueHorse Therapeutics Co., Ltd.
  中国研究中心 1 个：Tianjin
• 尚未开始招募NCT05539118
  Interferon-α1b Combined With Toripalimab and Anlotinib Hydrochloride in Advanced Unresectable Melanoma
  I 期、II 期 · 干预性 · 2022/09/30Xijing Hospital
  中国研究中心 1 个：Xi'an
• 招募中NCT05538130
  A Study to Learn About the Study Medicine Called PF-07799544 as Monotherapy or in Combination in People With Advanced Solid Tumors
  I 期 · 干预性 · 2022/11/30Pfizer
  中国研究中心 3 个：Fuzhou、Hangzhou、Wenzhou
• 招募中NCT05868707
  OH2 Injection in Melanoma
  III 期 · 干预性 · 2023/03/08Binhui Biopharmaceutical Co., Ltd.
  中国研究中心 30 个：Beijing、Changchun、Changsha、Chengdu、Chongqing、Dalian 等 26 地
• 招募中NCT05789043
  Camrelizumab in Combination With Apatinib and Temozolomide as First-line Treatment in Advanced Acral Melanoma
  III 期 · 干预性 · 2023/03/21Peking University Cancer Hospital & Institute
  中国研究中心 1 个：Beijing
• 招募中NCT07644897
  Thymosin Alpha 1 Combined With Anti-PD-1 Monoclonal Antibody in Elderly Patients With Advanced Melanoma
  II 期 · 干预性 · 2023/05/30Sun Yat-sen University
  中国研究中心 2 个：Guangzhou、Shenzhen

另有 53 项未列出，见该病详情页。

胶质母细胞瘤 57 项在招目录 第2批 · 38

• 招募中NCT03005132
  Integrative Analysis of Human Glioblastoma Multiforme
  观察性 · 2016/01Shanghai 10th People's Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT05990244
  Magnetic Resonance Elastography in Glioma: Exploring Tumor Stiffness and Adhesion
  不适用 · 干预性 · 2017/01/01Shengjing Hospital
  中国研究中心 1 个：Shenyang
• 仅邀请入组NCT03170141
  Immunogene-modified T (IgT) Cells Against Glioblastoma Multiforme
  I 期 · 干预性 · 2017/05/31Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 招募中NCT04267978
  Study of Recombinant Human Endostatin Combined with Temozolomide and Irinotecan in Recurrent Gliomas
  II 期 · 干预性 · 2020/02/13Beijing Sanbo Brain Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06595186
  JK-1201I Combined with Adjuvant Temozolomide in Patients with Newly Diagnosed Glioblastoma Multiforme (GBM)
  II 期 · 干预性 · 2022/10/31JenKem Technology Co., Ltd.
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT05541042
  Radiogenomics in Glioblastoma: Correlation Between Multiparametric Imaging Biomarkers and Genetic Biomarkers
  观察性 · 2022/12/01Henan Provincial People's Hospital
  中国研究中心 1 个：Zhengzhou
• 尚未开始招募NCT05502991
  Sintilimab (One Anti-PD-1 Antibody) Plus Low-dose Bevacizumab for ctDNA-level-relapse and Clinical-relapse Glioblastoma
  II 期 · 干预性 · 2022/12/11Henan Provincial People's Hospital
  中国研究中心 1 个：Zhengzhou
• 招募中NCT05957679
  MRE Evaluation for Spinal Cord Tumor Surgery: Stiffness and Adhesion Assessment
  不适用 · 干预性 · 2023/01/01Shengjing Hospital
  中国研究中心 1 个：Shenyang
• 尚未开始招募NCT05842746
  Elemene Plus Stupp Protocol Versus Stupp Protocol Alone for Newly-diagnosed Glioblastoma
  II 期 · 干预性 · 2023/05Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05303519
  SIGMA (Safusidenib in IDH1 Mutant Glioma Maintenance)
  III 期 · 干预性 · 2023/06/05Nuvation Bio Inc.
  中国研究中心 9 个：Beijing、Changsha、Chengdu、Fuzhou、Guangzhou、Shanghai
• 招募中NCT05577091
  Tris-CAR-T Cell Therapy for Recurrent Glioblastoma
  I 期 · 干预性 · 2023/09/30Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT05540275
  Tislelizumab (One Anti-PD-1 Antibody) Plus Low-dose Bevacizumab for Bevacizumab Refractory Recurrent Glioblastoma
  II 期 · 干预性 · 2023/10/05Henan Provincial People's Hospital
  中国研究中心 1 个：Zhengzhou

另有 45 项未列出，见该病详情页。

多发性内分泌腺瘤病 48 项在招目录 第2批 · 51

• 招募中NCT03093116
  A Study of Repotrectinib (TPX-0005) in Patients With Advanced Solid Tumors Harboring ALK, ROS1, or NTRK1-3 Rearrangements
  I 期、II 期 · 干预性 · 2017/03/07Turning Point Therapeutics, Inc.
  中国研究中心 31 个：Beijing、Changchun、Changsha、Chengdu、Chongqing、Daping 等 23 地
• 招募中NCT05406999
  Neoadjuvant Intense Endocrine Therapy for High Risk and Locally Advanced Prostate Cancer
  II 期 · 干预性 · 2020/02/01The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School
  中国研究中心 1 个：Nanjing
• 招募中NCT06540937
  Phase II Clinical Study of Leflunomide in the Treatment of MEN-1 Neuroendocrine Tumor
  II 期 · 干预性 · 2020/07/01Cancer Institute and Hospital, Chinese Academy of Medical Sciences
  中国研究中心 1 个：Beijing
• 招募中NCT04686682
  A First-in-Human, JAB-8263 in Adult Patients With Advanced Tumors
  I 期、II 期 · 干预性 · 2021/05/07Jacobio Pharmaceuticals Co., Ltd.
  中国研究中心 1 个：Tianjin
• 招募中NCT04924075
  Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
  II 期 · 干预性 · 2021/08/12Merck Sharp & Dohme LLC
  中国研究中心 4 个：Beijing、Chengdu、Guangzhou、Shanghai
• 招募中NCT04520074
  Adjuvant Chemotherapy of Three-step Regimen in BRCA1/2 Wide Type Ovarian Cancer (ACTS-2)
  III 期 · 干预性 · 2021/10/08Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT05123482
  A Phase I/IIa Study of AZD8205 Given Alone or Combined, in Participants With Advanced/Metastatic Solid Malignancies
  I 期、II 期 · 干预性 · 2021/10/18AstraZeneca
  中国研究中心 6 个：Beijing、Changsha、Chongqing、Guangzhou、Kunming、Shandong
• 招募中NCT04921527
  Chiauranib Plus Weekly Paclitaxel in Patients with Platinum-refractory or Platinum-resistant Recurrent Ovarian Cancer
  III 期 · 干预性 · 2021/12/20Chipscreen Biosciences, Ltd.
  中国研究中心 1 个：Shanghai
• 招募中NCT05251883
  A Study to Analyze Data on Metastatic Ovarian Cancer Using Multi-omics
  观察性 · 2022/01/01Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT05236686
  Survival Effect of Hepato-celiac Lymphadenectomy In Primary or Relapsed Ovarian Cancer
  不适用 · 干预性 · 2022/02/15Shanghai Gynecologic Oncology Group
  中国研究中心 1 个：Shanghai
• 招募中NCT06286631
  Prediction of Lymph Node Metastasis in Patients With Thyroid Malignancy by a New Scale
  观察性 · 2023/05/01Second Affiliated Hospital of Xi'an Jiaotong University
  中国研究中心 1 个：Xi'an
• 招募中NCT05803018
  A Study of BL-B01D1 in Patients With Multiple Solid Tumors, Including Recurrent or Metastatic Gynecological Malignancies
  I 期、II 期 · 干预性 · 2023/06/25Sichuan Baili Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Shanghai

另有 36 项未列出，见该病详情页。

全身型重症肌无力 44 项在招目录 第1批 · 32

• 招募中NCT04182984
  TheRapeutic Effect of Different immunosuppressAnts on Non-Thymoma Ocular Myasthenia Gravis: a Real-world Study
  观察性 · 2019/11/04Tang-Du Hospital
  中国研究中心 1 个：Xi'an
• 招募中NCT04202341
  Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)
  观察性 · 2019/12/02Alexion Pharmaceuticals, Inc.
  中国研究中心 8 个：Beijing、Changchun、Chengdu、Guangzhou、Jinan、Nanchang 等 8 地
• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT04837625
  Study of Myasthenic Crisis in China
  观察性 · 2020/10/01Huashan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT04951622
  A Study of Nipocalimab Administered to Adults With Generalized Myasthenia Gravis
  III 期 · 干预性 · 2021/07/15Janssen Research & Development, LLC
  中国研究中心 14 个：Beijing、Changchun、Changsha、Chengdu、Fuzhou、Guangzhou 等 11 地
• 招募中NCT05067348
  Efficacy and Safety of Tocilizumab in the Treatment of Generalized Myasthenia Gravis
  II 期 · 干预性 · 2022/07/21Tang-Du Hospital
  中国研究中心 6 个：Beijing、Changsha、Chengdu、Shanghai、Tianjin、Xi'an
• 招募中NCT06006832
  A Prospective Cohort Study of Myasthenia Gravis in China
  观察性 · 2023/08/20Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07146425
  Construction of A Multimodal Digital Assessment Model for Myasthenia Gravis
  观察性 · 2023/08/31Huashan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT04535843
  Precision Diagnosis and Prospective Cohort Study for Myasthenia Gravis: Multicenter Analysis in China
  观察性 · 2024/02/01Huashan Hospital
  中国研究中心 4 个：Shanghai
• 招募中NCT06241521
  Myasthenia Gravis Registry in China
  观察性 · 2024/02/01Huashan Hospital
  中国研究中心 16 个：Beijing、Changchun、Changsha、Chengdu、Chongqing、Guangzhou 等 13 地
• 招募中NCT06249438
  A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
  I 期 · 干预性 · 2024/03/20RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT07653984
  Cohort Study on Neuroimmune Diseases in the Reproductive Age
  观察性 · 2024/04/21Third Affiliated Hospital, Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou

另有 32 项未列出，见该病详情页。

ANCA相关性血管炎 42 项在招目录 第2批 · 7

• 招募中NCT05315141
  Multicenter Cohort Study of AAV in Hunan of China
  观察性 · 2022/03/16Xiangya Hospital of Central South University
  中国研究中心 1 个：Changsha
• 招募中NCT05965284
  Efficacy and Safety for Telitacicept in the Remission Maintenance Treatment of ANCA-associated Vasculitis (TTCAZAREM)
  IV 期 · 干预性 · 2023/03/09Chinese SLE Treatment And Research Group
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT05859997
  Universal CAR-T Cells (BRL-301) in Relapse or Refractory Autoimmune Diseases
  不适用 · 干预性 · 2023/05/17Bioray Laboratories
  中国研究中心 1 个：Shanghai
• 招募中NCT05962840
  Efficacy and Safety for Rituximab Combined With Telitacicept in the Treatment of ANCA-associated Vasculitis (TTCAAVREM)
  IV 期 · 干预性 · 2023/06/29Chinese SLE Treatment And Research Group
  中国研究中心 1 个：Beijing
• 招募中NCT06056921
  Safety and Efficacy of CD19 Targeted CAR-T Therapy for Refractory Autoimmune Disease
  I 期 · 干预性 · 2023/08/31Chongqing Precision Biotech Co., Ltd
  中国研究中心 1 个：Taiyuan
• 招募中NCT05979051
  A Study to Evaluate the Efficacy and Safety of SHR-1703 in Subjects With Eosinophilic Granulomatosis With Polyangiitis （EGPA）
  II 期、III 期 · 干预性 · 2023/11/16Guangdong Hengrui Pharmaceutical Co., Ltd
  中国研究中心 2 个：Beijing、Hangzhou
• 招募中NCT06277427
  Refractory ANCA Associated Vasculitis and Lupus Nephritis Treated With BCMA-targeting CAR-T Cells
  不适用 · 干预性 · 2024/02/05Lingli Dong
  中国研究中心 1 个：Wuhan
• 招募中NCT06285279
  The BCMA/CD19 Dual Targeted CAR-T Cell in Participants With Autoimmune Kidney Diseases
  I 期 · 干预性 · 2024/03/04Nanjing University School of Medicine
  中国研究中心 1 个：Nanjing
• 招募中NCT06379646
  An Clinical Study of YTS109 Cell Injection in Subjects With Recurrent/Refractory Autoimmune Disease
  不适用 · 干预性 · 2024/04/24China Immunotech (Beijing) Biotechnology Co., Ltd.
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT06420154
  The Safety and Efficacy of Anti-CD19 CAR-T Cells in Patients With Relapsed/Refractory Autoimmune Diseases
  早期 I 期 · 干预性 · 2024/05/27First Affiliated Hospital of Wenzhou Medical University
  中国研究中心 1 个：Wenzhou
• 招募中NCT06350110
  Fourth-gen CAR T Cells Targeting BCMA/CD19 for Refractory Systemic Lupus Erythematosus (SLE)
  I 期、II 期 · 干预性 · 2024/07/10Essen Biotech
  中国研究中心 1 个：Beijing
• 招募中NCT06548607
  Clinical Study on Targeted CD19 or CD19-BCMA CAR-T Therapy for Autoimmune Diseases
  早期 I 期 · 干预性 · 2024/09/14Nanjing Bioheng Biotech Co., Ltd.
  中国研究中心 1 个：Bengbu

另有 30 项未列出，见该病详情页。

多发性硬化 37 项在招目录 第1批 · 76

• 招募中NCT04106830
  Clinical and Imaging Cohort of Neuroinflammation Diseases in China (CLUE)
  观察性 · 2019/01/01Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT05154370
  China National Registry of Neuro-Inflammatory Diseases
  观察性 · 2021/12/15Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07353216
  Exploring the Efficacy and Safety of Ofatumumab in Patients With Relapsing Multiple Sclerosis (RMS) and Its Impact on Serum Neurofilament Light Chain (sNfL) Levels
  观察性 · 2023/12/01Third Affiliated Hospital, Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 仅邀请入组NCT06643858
  Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)
  观察性 · 2023/12/01Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06541626
  Sun Yat-Sen Cohort of CNS Idiopathic Inflammatory Demyelinating Diseases
  观察性 · 2024/01/01Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06249438
  A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
  I 期 · 干预性 · 2024/03/20RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT07653984
  Cohort Study on Neuroimmune Diseases in the Reproductive Age
  观察性 · 2024/04/21Third Affiliated Hospital, Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 仅邀请入组NCT06782958
  Safety, Tolerability, and Pharmacokinetics of FHND1002 Granules in Healthy Adults
  I 期 · 干预性 · 2024/07/05Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Lanzhou
• 招募中NCT06502015
  Biomarkers in Autoimmune Disease of Nervous System
  观察性 · 2024/07/31Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06474520
  Efficacy and Safety of Calculus Bovis Sativus (CBS) for Idiopathic Inflammatory Demyelinating Disease (CBSinIIDD)
  不适用 · 干预性 · 2024/08/08Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06671236
  Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases
  I 期 · 干预性 · 2024/11/21Novabio Therapeutics
  中国研究中心 1 个：Zhengzhou

另有 25 项未列出，见该病详情页。

骨肉瘤 35 项在招目录 第2批 · 57

• 招募中NCT04072042
  BIOmarker Driven Trial of VEGFR2 Inhibitor in Advanced or Metastatic Sarcoma
  II 期 · 干预性 · 2019/10/30Ruijin Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT04589741
  Toripalimab Combined With CAV/IE Regimen
  II 期 · 干预性 · 2020/10/23Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 尚未开始招募NCT05590572
  A Study of Sulfatinib on Relapsed or Refractory Drug Resistant Osteosarcoma
  I 期、II 期 · 干预性 · 2023/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 4 个：Beijing、Jinan、Shanghai
• 招募中NCT05617859
  Lenvatinib for Advanced Bone and Soft Tissue Sarcoma
  II 期 · 干预性 · 2023/04/30Henan Cancer Hospital
  中国研究中心 1 个：Zhengzhou
• 招募中NCT06074692
  PARP Inhibition, Stereotactic Body Radiotherapy and Immunotherapy for Metastatic or Advanced Sarcoma (PRIMA)
  II 期 · 干预性 · 2023/06/01Ruijin Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06114225
  Pulmonary Resectable Osteosarcoma Treated by Metastasectomy and Pre-operative Immunotherapy and Stereotactic Body Radiotherapy (PROMIS): a Prospective Clinical Trial
  II 期 · 干预性 · 2023/06/01Ruijin Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT05830123
  ARTEMIS-002: HS-20093 in Patients With Relapsed or Refractory Osteosarcoma and Other Sarcomas
  II 期 · 干预性 · 2023/06/08Hansoh BioMedical R&D Company
  中国研究中心 11 个：Beijing、Changsha、Guangzhou、Hangzhou、Nanjing、Shanghai 等 8 地
• 招募中NCT06126510
  Clinical Study of VG161 in the Treatment of Advanced Bone and Soft Tissue Sarcoma
  II 期 · 干预性 · 2023/07/18CNBG-Virogin Biotech (Shanghai) Ltd.
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06041490
  Adjuvant Therapy for High-risk Hepatocellular Carcinoma Post Liver Transplantation
  II 期 · 干预性 · 2023/09Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06778603
  B7-H3 NIR Imaging for Osteosarcoma Surgery
  I 期、II 期 · 干预性 · 2024/01/01Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06111612
  Intensive Conditioning withTHI/Bu/Flu/Ara-C in Allo-HSCT for Myeloid Malignancies With Extramedullary Involvement
  观察性 · 2024/01/20Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 招募中NCT06340204
  Weekly Irinotecan Liposomes in Recurrent or Refractory Ewing Sarcoma
  I 期 · 干预性 · 2024/03/25Peking University People's Hospital
  中国研究中心 3 个：Beijin、Beijing、Jina

另有 23 项未列出，见该病详情页。

视神经脊髓炎 34 项在招目录 第1批 · 81

• 招募中NCT04106830
  Clinical and Imaging Cohort of Neuroinflammation Diseases in China (CLUE)
  观察性 · 2019/01/01Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT05154370
  China National Registry of Neuro-Inflammatory Diseases
  观察性 · 2021/12/15Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05145361
  Clinical Study of B001 Injection in Subjects With Neuromyelitis Optic Spectrum Disorder (NMOSD)
  早期 I 期 · 干预性 · 2022/04/07Shanghai Pharmaceuticals Holding Co., Ltd
  中国研究中心 4 个：Beijing、Taiyuan、Tianjin、Xi’an
• 招募中NCT05504694
  Ofatumumab in AQP4-IgG Seropositive NMOSD
  I 期、II 期 · 干预性 · 2022/06/28Tang-Du Hospital
  中国研究中心 1 个：Xi'an
• 招募中NCT06697535
  A Study to Evaluate the Efficacy and Safety of JYP0061 in Patients With Acute Neuromyelitis Spectrum Disease (NMOSD)
  II 期 · 干预性 · 2023/05/24Guangzhou JOYO Pharma Co., Ltd
  中国研究中心 1 个：Changsha
• 仅邀请入组NCT06643858
  Normal Chinese Lifespan Brain Charts Initiative (NCLBCI)
  观察性 · 2023/12/01Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06541626
  Sun Yat-Sen Cohort of CNS Idiopathic Inflammatory Demyelinating Diseases
  观察性 · 2024/01/01Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT05966467
  Registry of Patients With AQP4+ NMOSD Treated With Alexion C5 Inhibitor Therapies
  观察性 · 2024/02/01Alexion Pharmaceuticals, Inc.
  中国研究中心 6 个：Beijing、Guangzhou、Henan、Shanghai、Sichuan、Wuhan
• 招募中NCT06249438
  A Study of C-CAR168 in the Treatment of Autoimmune Diseases Refractory to Standard Therapy
  I 期 · 干预性 · 2024/03/20RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT07653984
  Cohort Study on Neuroimmune Diseases in the Reproductive Age
  观察性 · 2024/04/21Third Affiliated Hospital, Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06413654
  A Clinical Study of B001 Injection in the Treatment of Neuromyelitis Optica Spectrum Disorders(NMOSD)
  II 期、III 期 · 干预性 · 2024/07/17Shanghai Jiaolian Drug Research and Development Co., Ltd
  中国研究中心 51 个：Beijing、Cangzhou、Changsha、Chengdu、Fuzhou、Guangzhou 等 33 地

另有 22 项未列出，见该病详情页。

血友病 32 项在招目录 第1批 · 36

• 招募中NCT04645199
  National Longitudinal Cohort of Hematological Diseases
  观察性 · 2020/12/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT04728841
  Gene Therapy for Chinese Hemophilia A
  不适用 · 干预性 · 2021/03/04Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05145127
  Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
  III 期 · 干预性 · 2021/11/17Pfizer
  中国研究中心 6 个：Beijing、Guangzhou、Guiyang、Nanchang、Tianjin、Wuhan
• 招募中NCT05641610
  A Study to Evaluate the Safety and Efficacy of ZS801 in Adult Hemophilia B Patients
  I 期、II 期 · 干预性 · 2022/02/16Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05454774
  A Study of FVIII Gene Therapy for Hemophilia A
  早期 I 期 · 干预性 · 2022/07/19Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05611801
  A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
  III 期 · 干预性 · 2022/12/09Pfizer
  中国研究中心 7 个：Beijing、Guangzhou、Guiyang、Nanchang、Tianjin、Wuhan
• 招募中NCT05685238
  A Research Study Looking at Long-term Treatment With Mim8 in People With Haemophilia A
  III 期 · 干预性 · 2023/02/13Novo Nordisk A/S
  中国研究中心 13 个：Beijing、Changsha、Chengdu、Guangzhou、Huzhou、Jinan 等 8 地
• 招募中NCT05709288
  Gene Therapy for Hemophilia B Patients Aged 12-18 Years Old
  I 期 · 干预性 · 2023/03/23Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05630651
  The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
  不适用 · 干预性 · 2023/04/19Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05849740
  An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A
  IV 期 · 干预性 · 2023/05/22Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT07205523
  High-Altitude Hematology Observation-Stem Cell Transplantation (HALO-SCT)
  观察性 · 2023/09/01Yigeng Cao,MD,PhD
  中国研究中心 1 个：Xining
• 招募中NCT05888870
  ITI Using SCT800 Alone or Combining Daratumumab in Hemophilia A Adolescents and Adults With High Titer Inhibitor
  IV 期 · 干预性 · 2023/11/22Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin

另有 20 项未列出，见该病详情页。

原发性轻链型淀粉样变 28 项在招目录 第1批 · 96

• 仅邀请入组NCT04210791
  Autologous Stem Cell Transplantation for Patients With AL Amyloidosis
  观察性 · 2010/07/01Nanjing University School of Medicine
  中国研究中心 1 个：Nanjing
• 招募中NCT04924998
  Clinical Study for AL-CM
  观察性 · 2021/06/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 招募中NCT06376214
  Daratumumab for Patients With Light Chain Amyloidosis
  不适用 · 干预性 · 2023/01/01Nanjing University School of Medicine
  中国研究中心 1 个：Nanjing
• 招募中NCT06192979
  Optimize First-line Treatment for AL Amyloidosis With t (11; 14)
  不适用 · 干预性 · 2024/01/05Jin Lu, MD
  中国研究中心 7 个：Beijing、Nanjing、Qingdao、Xi’an
• 招募中NCT06342466
  Bortezomib, Pomalidomide, Dexamethasone for Systemic AL Amyloidosis
  II 期 · 干预性 · 2024/05/06Jin Lu, MD
  中国研究中心 1 个：Beijing
• 招募中NCT06418477
  Daratumumab, Bortezomib, Cyclophosphamide, and Dexamethasone Therapy for Patients with MIDD
  II 期 · 干预性 · 2024/06/15Peking University People's Hospital
  中国研究中心 3 个：Beijing、Guangzhou
• 招募中NCT06803082
  The Impact of Systemic Light Chain Amyloidosis on Eyes
  观察性 · 2024/09/04Zhongshan Ophthalmic Center, Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06627309
  Rapid dFLC Response Predict CHR in AL Amyloidosis
  观察性 · 2024/10/26Peking University People's Hospital
  中国研究中心 8 个：Beijing、Harbin、Nanjing、Shenyang、Xi'an
• 招募中NCT07626476
  Autologous BCMA-targeted CAR-T Cell Injection for Relapsed/Refractory Light Chain Amyloidosis
  早期 I 期 · 干预性 · 2024/12/16Beijing Boren Hospital
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT07103863
  Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
  观察性 · 2025/02/13Beijing Anzhen Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06983951
  Exploratory Clinical Study on the Safety and Efficacy of Targeted BCMA Autologous CART Cell Injection in Subjects With Recurrent/Refractory Light Chain Amyloidosis
  不适用 · 干预性 · 2025/02/20Beijing Boren Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06998875
  A Prospective Cohort Study on Primary Cutaneous Amyloidosis
  IV 期 · 干预性 · 2025/03/01Army Medical University, China
  中国研究中心 1 个：Chongqing

另有 16 项未列出，见该病详情页。

神经母细胞瘤 27 项在招目录 第2批 · 53

• 招募中NCT05027386
  Apatinib Mesylate Combined With IT Regimen for the Treatment of Recurrent or Refractory Pediatric Neuroblastoma: A Single-arm, Phase I/II，Multi-center, Clinical Study.
  I 期、II 期 · 干预性 · 2021/08/26Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT05069220
  18F-MFBG PET/CT in the Evaluation of Neural Crest Tumor
  早期 I 期 · 干预性 · 2021/09/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07516678
  Serial ctDNA and Molecular Residual Disease Monitoring in Neuroblastoma
  观察性 · 2022/01/01Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT07064746
  Clinical Application Study of [123I]Metaiodobenzylguanidine and Somatostatin Receptor-Targeted Imaging in the Diagnosis and Staging of Neuroblastoma
  观察性 · 2022/07/10Nanjing First Hospital, Nanjing Medical University
  中国研究中心 1 个：Nanjing
• 尚未开始招募NCT05303727
  Allogeneic Hematopoietic Stem Cell Transplantation for 4/M Neuroblastoma
  II 期 · 干预性 · 2022/08Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT05562024
  TAA06 Injection in the Treatment of Patients With B7-H3-positive Relapsed/ Refractory Neuroblastoma
  I 期 · 干预性 · 2022/12/30PersonGen BioTherapeutics (Suzhou) Co., Ltd.
  中国研究中心 2 个：Jinan、Tianjin
• 招募中NCT05701306
  APG-115 Alone or in Combination With APG-2575 in Children With Recurrent or Refractory Neuroblastoma or Solid Tumors
  I 期 · 干预性 · 2023/02/28Ascentage Pharma Group Inc.
  中国研究中心 3 个：Guangzhou、Tianjin、Wuhan
• 招募中NCT06013618
  Clinical Analysis of Naxitamab (hu3F8) in the Treatment of Pediatric High Risk or Refractory/ Relapsed Neuroblastoma
  II 期 · 干预性 · 2023/06/19Sun Yat-Sen University Cancer Center
  中国研究中心 1 个：Guangzhou
• 招募中NCT06255210
  Efficacy and Safety of Induction Chemotherapy for Olfactory Neuroblastoma (ESICON)
  II 期 · 干预性 · 2024/03/01Hongmeng Yu
  中国研究中心 1 个：Shanghai
• 招募中NCT06574698
  Adding Of Naxitamb In Induction Therapy For High Risk Neuroblastoma
  观察性 · 2024/05/06Guangzhou Women and Children's Medical Center
  中国研究中心 1 个：Guanzhou
• 招募中NCT06818916
  Hetrombopag in Promoting Platelet Engraftment After Auto-HSCT in Children With Neuroblastoma
  不适用 · 干预性 · 2024/06/30Beijing Children's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06631391
  Phase I Study of Umbilical Cord Blood Natural Killer (NK) Cell Therapy for Children With High-risk, R/R Neuroblastoma.
  I 期 · 干预性 · 2024/11/01Sun Yat-sen University
  中国研究中心 1 个：Guangzhou

另有 15 项未列出，见该病详情页。

胃肠间质瘤 26 项在招目录 第2批 · 32

• 招募中NCT03594422
  A Study of HQP1351 in Patients With GIST or Other Solid Tumors
  I 期 · 干预性 · 2018/07/11Ascentage Pharma Group Inc.
  中国研究中心 6 个：Beijing、Guangzhou、Shanghai、Wuhan、Zhengzhou
• 招募中NCT03716089
  Comparison of Tumor Efficacy Safety in Laparoscopic Resection of Gastrointestinal Stromal Tumors Between Favorable and Unfavorable Site
  观察性 · 2018/10/11Fujian Medical University
  中国研究中心 1 个：Fuzhou
• 招募中NCT04924075
  Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
  II 期 · 干预性 · 2021/08/12Merck Sharp & Dohme LLC
  中国研究中心 4 个：Beijing、Chengdu、Guangzhou、Shanghai
• 招募中NCT04933669
  Prospective Multicenter Clinical Study of Neoadjuvant Imatinib Mesylate for Gastrointestinal Stromal Tumors
  II 期 · 干预性 · 2021/09/07First Affiliated Hospital of Zhejiang University
  中国研究中心 1 个：Hangzhou
• 招募中NCT05408897
  Prediction of Postoperative Treatment Efficacy and Recurrence Risk of High-risk GIST Based on Liquid Biopsy MRD
  观察性 · 2022/01/01Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05464875
  A Multicenter Study of Avapritinib Efficacy and Safety of Metastatic or Unresectable Gastrointestinal Stromal Tumors
  观察性 · 2022/07/09Xinhua Zhang, MD
  中国研究中心 1 个：Guangzhou
• 招募中NCT05461664
  Avapritinib in the Treatment of Unresectable or Recurrent Metastatic GIST Non-exon18 Mutations of PDGFRA
  观察性 · 2022/07/10Xinhua Zhang, MD
  中国研究中心 1 个：Guangzhou
• 招募中NCT05751733
  Apatinib Mesylate Versus Standard Second-line TKI in the Treatment of Advanced GIST
  不适用 · 干预性 · 2023/02/01Xiangya Hospital of Central South University
  中国研究中心 1 个：Changsha
• 招募中NCT05938309
  A Cohort Study on the Safety of Laparoscopic Resection of 5cm or Larger Gastric Gastrointestinal Stromal Tumors
  观察性 · 2023/05/01Fujian Medical University
  中国研究中心 1 个：Fuzhou
• 招募中NCT05867901
  Clinical Research of Drug Holiday Based on MRD Detection in GIST Patients at High Risk of Recurrence
  观察性 · 2023/06/01Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT05970900
  Preoperative Imatinib Mesylate Combined With Rectal-sparing Surgery in Patients With c-KIT Gene-mutant Rectal GIST
  III 期 · 干预性 · 2023/10/01Fujian Medical University Union Hospital
  中国研究中心 1 个：Fuzhou
• 招募中NCT06748690
  MCB vs EUS-FNA for Preoperative Pathological Evaluation of Gastric SMT
  不适用 · 干预性 · 2024/01/01Peking University People's Hospital
  中国研究中心 1 个：Beijing

另有 14 项未列出，见该病详情页。

多系统萎缩 24 项在招目录 第1批 · 77

• 招募中NCT04912843
  Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
  II 期、III 期 · 干预性 · 2021/06/18Wuhan Neurophth Biotechnology Limited Company
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT06101940
  Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
  观察性 · 2021/08/01Huashan Hospital
  中国研究中心 22 个：Beijing、Changchun、Chengdu、Chifeng、Chongqing、Fuzhou 等 18 地
• 招募中NCT05824169
  Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
  I 期、II 期 · 干预性 · 2023/02/25GeneCradle Inc
  中国研究中心 4 个：Beijing、Shenyang、Suzhou
• 招募中NCT07392411
  Study on Artificial Intelligence-Based Facial and Speech-Related Patterns in Parkinson's Disease and Their Digital Biomarkers
  观察性 · 2023/07/25Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06421831
  Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
  I 期、II 期 · 干预性 · 2024/05/10GeneCradle Inc
  中国研究中心 1 个：Beijing
• 招募中NCT06949865
  AI-Enhanced Optimization of Acute Levodopa Challenge Test
  观察性 · 2024/05/28Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07353463
  Shanghai Clinical Cohort - Parkinson's Disease (Reserve)
  观察性 · 2025/04/01Ruijin Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06765733
  The Study of Safety and Preliminary Efficacy of Aleeto in Patients With MultIple System Atrophy
  早期 I 期 · 干预性 · 2025/05/31Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07644013
  Prospective Multicenter Registry Study of Multiple System Atrophy in China
  观察性 · 2025/06/01Peking University First Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07036939
  Efficacy and Safety of Bilateral Cervical Lymphatic-Venous Anastomosis in the Treatment of Multiple System Atrophy
  不适用 · 干预性 · 2025/07/01Ruijin Hospital
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT06607900
  hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases
  I 期 · 干预性 · 2025/07/01Xuanwu Hospital, Beijing
  中国研究中心 1 个：Beijing
• 招募中NCT07570212
  Individualized Transcranial Magnetic Stimulation in Parkinsonian Disorders
  不适用 · 干预性 · 2025/12/22Peking University First Hospital
  中国研究中心 1 个：Beijing

另有 12 项未列出，见该病详情页。

特发性肺纤维化 21 项在招目录 第1批 · 55

• 招募中NCT06125327
  SC1011 Twice Daily vs Placebo in Patients Diagnosed With Idiopathic Pulmonary Fibrosis (IPF)
  II 期、III 期 · 干预性 · 2023/06/06Guangzhou JOYO Pharma Co., Ltd
  中国研究中心 1 个：Beijing
• 招募中NCT06267183
  A Clinical Study to Evaluate Safety, Tolerability and Pharmacokinetics of SV001 in Chinese Healthy Adult Volunteers.
  早期 I 期 · 干预性 · 2024/01/12Shanghai Synvida Biotechnology Co.,Ltd.
  中国研究中心 1 个：Shanghai
• 招募中NCT06238622
  A Follow-up Study to Test Long-term Treatment With Nerandomilast in People With Pulmonary Fibrosis Who Took Part in a Previous Study With Nerandomilast
  III 期 · 干预性 · 2024/05/06Boehringer Ingelheim
  中国研究中心 37 个：Beijing、Changsha、Chengdu、Guangzhou、Hangzhou、Hefei 等 23 地
• 仅邀请入组NCT06629623
  Current Status of Diagnosis, Treatment and Quality of Life for Patients with Idiopathic Pulmonary Fibrosis
  观察性 · 2024/07/30Chu Hongling
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06922916
  Perioperative Risk Factors Related to the Prognosis of Lung Transplantation: A Retrospective Study
  观察性 · 2025/04/22Zhejiang University
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT07019714
  Daoyin Technique for Improving Exercise Endurance in IPF
  不适用 · 干预性 · 2025/06/30Affiliated Hospital of Liaoning University of Traditional Chinese Medicine
  中国研究中心 1 个：Shenyang
• 尚未开始招募NCT06914713
  Jin-shui Huan-xian Formula for Retarding the Decline of Pulmonary Function in IPF
  不适用 · 干预性 · 2025/08/01Henan University of Traditional Chinese Medicine
  中国研究中心 1 个：Zhengzhou
• 尚未开始招募NCT07131150
  Menstrual Blood-Derived Mesenchymal Stem Cell Injection (SC01009) in the Treatment of Idiopathic Pulmonary Fibrosis
  II 期 · 干预性 · 2025/08/31Ruijin Hospital
  中国研究中心 22 个：Beijing、Changchun、Changsha、Chengdu、Guangzhou、Hebei 等 15 地
• 尚未开始招募NCT07162870
  Idiopathic Pulmonary Fibrosis Cohort of TCM
  观察性 · 2025/09/05Henan University of Traditional Chinese Medicine
  中国研究中心 1 个：Zhengzhou
• 尚未开始招募NCT07178392
  Disease-syndrome Characteristics of IPF
  观察性 · 2025/09/15Henan University of Traditional Chinese Medicine
  中国研究中心 1 个：Zhengzhou
• 招募中NCT07082842
  Confirmatory Clinical Study of HEC585 Tablets in Patients With IPF
  III 期 · 干预性 · 2025/10/27Sunshine Lake Pharma Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT07121413
  A Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Immunogenicity of SV001 in Patients With Idiopathic Pulmonary Fibrosis
  II 期 · 干预性 · 2025/11/04Shanghai Synvida Biotechnology Co.,Ltd.
  中国研究中心 12 个：Chengdu、Guangzhou、Hangzhou、Nanchang、Nanjing、Neimeng 等 10 地

另有 9 项未列出，见该病详情页。

肌萎缩侧索硬化 20 项在招目录 第1批 · 4

• 招募中NCT04008329
  A Registered Cohort Study on Amyotrophic Lateral Sclerosis
  观察性 · 2019/07/05First Affiliated Hospital of Fujian Medical University
  中国研究中心 1 个：Fuzhou
• 招募中NCT04454892
  Natural History and Clinical Features of Amyotrophic Lateral Sclerosis (ALS)
  观察性 · 2019/11/01Peking University Third Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT04885374
  China Amyotrophic Lateral Sclerosis Registry of Patients With Traditional Chinese Medicine
  观察性 · 2021/03/01Dongzhimen Hospital, Beijing
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT04961450
  Explore Biomarkers of Motor Neuron Disease/Frontal Dementia Spectrum Disease in China
  观察性 · 2021/06/24Peking University Third Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05923905
  Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients
  IV 期 · 干预性 · 2023/01/18Peking University Third Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06765564
  Clinical Study of Induced Pluripotent Stem Cells Derived Motor Neuron Precursor Cell Therapy for Amyotrophic Lateral Sclerosis (ALS)
  不适用 · 干预性 · 2024/03/13Shanghai East Hospital
  中国研究中心 1 个：Shanghai
• 仅邀请入组NCT06782958
  Safety, Tolerability, and Pharmacokinetics of FHND1002 Granules in Healthy Adults
  I 期 · 干预性 · 2024/07/05Jiangsu Chia Tai Fenghai Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Lanzhou
• 尚未开始招募NCT06645197
  An IIT Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Intrathecal Injection of SNUG01 in Patients with Amyotrophic Lateral Sclerosis
  早期 I 期 · 干预性 · 2024/10/16Peking University Third Hospital
  中国研究中心 5 个：Beijing、Chengdu、Fujian、Hangzhou、Nanjing
• 招募中NCT06671236
  Clinical Study of Regulatory T Cells (Tregs) in the Treatment of Neurodegenerative Diseases
  I 期 · 干预性 · 2024/11/21Novabio Therapeutics
  中国研究中心 1 个：Zhengzhou
• 招募中NCT06598202
  Exploring Nasal Drop Therapy With Small Extracellular Vesicles for ALS
  I 期、II 期 · 干预性 · 2024/12/01Xuanwu Hospital, Beijing
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06689982
  Tofacitinib in Patients With Amyotrophic Lateral Sclerosis
  早期 I 期 · 干预性 · 2024/12/01Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06556394
  A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation
  I 期 · 干预性 · 2024/12/24Ractigen Therapeutics.
  中国研究中心 3 个：Beijing、Chengdu、Hangzhou

另有 8 项未列出，见该病详情页。

地中海贫血（重型） 20 项在招目录 第2批 · 78

• 招募中NCT04064060
  A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
  III 期 · 干预性 · 2019/08/12Celgene
  中国研究中心 6 个：Beijing、Chengdu、Guangzhou、Hangzhou、Shanghai、Tianjin
• 招募中NCT04143724
  Study of Safety & PK of Luspatercept (ACE-536) in Pediatric Participants With Beta (β)-Thalassemia
  II 期 · 干预性 · 2019/11/07Celgene
  中国研究中心 6 个：Chengdu、Guangzhou、Nanning、Shenzhen
• 招募中NCT05745532
  Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients
  早期 I 期 · 干预性 · 2020/12/01Shenzhen Hemogen
  中国研究中心 1 个：Shenzhen
• 招募中NCT06314529
  Long-term Follow-up Study of BHC001 for TDT
  观察性 · 2022/12/14Bioray Laboratories
  中国研究中心 2 个：Changsha、Nanning
• 招募中NCT05762510
  A Study Evaluating the Safety and Efficacy of LentiRed Drug Product in Transfusion-dependent β-Thalassemia [TDT]
  早期 I 期 · 干预性 · 2023/02/22First Affiliated Hospital of Guangxi Medical University
  中国研究中心 1 个：Nanning
• 招募中NCT05991336
  Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy
  观察性 · 2023/06/05Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT05776173
  Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia
  不适用 · 干预性 · 2023/08/10Shanghai BDgene Co., Ltd.
  中国研究中心 1 个：Shanghai
• 招募中NCT05773729
  Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent Beta-thalassemia
  不适用 · 干预性 · 2023/09/15Shanghai BDgene Co., Ltd.
  中国研究中心 1 个：Shanghai
• 招募中NCT06219239
  Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients
  不适用 · 干预性 · 2024/01/04Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06465550
  A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major
  I 期 · 干预性 · 2024/01/05Shanghai BDgene Co., Ltd.
  中国研究中心 3 个：Guangzhou、Nanning、Shanghai
• 仅邀请入组NCT06328764
  CS-101 in Patients With β-thalassemia
  早期 I 期 · 干预性 · 2024/03/19CorrectSequence Therapeutics Co., Ltd
  中国研究中心 1 个：Nanning
• 招募中NCT06280378
  Β-Thalassemia Treatment with KL003 Cell Injection
  I 期、II 期 · 干预性 · 2024/04/05Kanglin Biotechnology (Hangzhou) Co., Ltd.
  中国研究中心 2 个：Shanghai、Tianjin

另有 8 项未列出，见该病详情页。

特发性肺动脉高压 19 项在招目录 第1批 · 54

• 招募中NCT02061787
  the Application of Cardiopulmonary Exercise Testing in Assessment Outcome of Patients With Pulmonary Hypertension
  观察性 · 2010/09Chinese Pulmonary Vascular Disease Research Group
  中国研究中心 1 个：Beijing
• 招募中NCT07184814
  Clinical Features and Prognosis of Takayasu's Arteritis With Pulmonary Arteries Involvement
  观察性 · 2016/01/01China-Japan Friendship Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06954727
  China Pulmonary Vascular Disease Intervention Diagnosis and Management Study-right Heart Catheterization
  观察性 · 2018/01/01China-Japan Friendship Hospital
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT03683186
  A Study Evaluating the Long-Term Efficacy and Safety of Ralinepag in Subjects With PAH Via an Open-Label Extension
  III 期 · 干预性 · 2019/09/23United Therapeutics
  中国研究中心 5 个：Beijing、Changsha、Guangzhou、Shanghai
• 招募中NCT05368467
  National Registry and Cohort Study of Pulmonary Vascular Disease
  观察性 · 2022/03/29Nanjing First Hospital, Nanjing Medical University
  中国研究中心 13 个：Beijing、Changsha、Chendu、Guangzhou、Nanjing、Shenyang 等 10 地
• 招募中NCT05179876
  A Study Providing Treatment Access in Participants With Pulmonary Hypertension Completing a Parent Study and Having no Other Option
  III 期 · 干预性 · 2022/05/04Actelion
  中国研究中心 7 个：Beijing、Changsha、Nanjing、Qingdao、Shanghai、Xi'an
• 招募中NCT05566002
  Artificial Intelligence-assisted Evaluation of Pulmonary HYpertension
  观察性 · 2022/06/01Chinese Pulmonary Vascular Disease Research Group
  中国研究中心 1 个：Beijing
• 招募中NCT06605794
  FreeFlow Percutaneous Atrial Septal Shunt for IPAH
  不适用 · 干预性 · 2023/09/11Shanghai Zhongshan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT07604805
  Efficacy and Safety of Qishen Yiqi Dropping Pills in Patients With Severe Pulmonary Hypertension
  不适用 · 干预性 · 2023/10/25Guangdong Provincial People's Hospital
  中国研究中心 1 个：Guangzhou
• 招募中NCT06528418
  Identification of Multiple Pulmonary Diseases Using Volatile Organic Compounds Biomarkers in Human Exhaled Breath
  观察性 · 2024/06/30ChromX Health
  中国研究中心 1 个：Guangzhou
• 招募中NCT07149935
  Continuous Effect Of Rehabilitation Training On Pulmonary Arterial Hypertension Patients
  不适用 · 干预性 · 2024/09/01RenJi Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06554301
  A Study on the Efficacy and Safety of Empagliflozin in the Treatment of Pulmonary Arterial Hypertension
  I 期 · 干预性 · 2024/10/20China National Center for Cardiovascular Diseases
  中国研究中心 1 个：Beijing

另有 7 项未列出，见该病详情页。

IgG4相关性疾病 19 项在招目录 第1批 · 56

• 招募中NCT01670695
  A Prospective Cohort Study of IgG4RD in China
  观察性 · 2012/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT03023371
  National Registry of IgG4-RD in China
  观察性 · 2016/12Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05789030
  Treatment Strategies for IgG4-RD Patients With Superficial Organ Involvement
  不适用 · 干预性 · 2022/07/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06663618
  Short-term Glucocorticoid Combined with MMF for IgG4-RD
  不适用 · 干预性 · 2023/09/01Wen Zhang
  中国研究中心 1 个：Beijing
• 招募中NCT06285279
  The BCMA/CD19 Dual Targeted CAR-T Cell in Participants With Autoimmune Kidney Diseases
  I 期 · 干预性 · 2024/03/04Nanjing University School of Medicine
  中国研究中心 1 个：Nanjing
• 招募中NCT06497387
  Safety and Efficacy of PRG-1801 for Refractory Lupus Nephritis and IgG4-Related Disease
  早期 I 期 · 干预性 · 2024/07Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06497361
  Safety and Efficacy of PRG-2311 for Refractory Lupus Nephritis and IgG4-Related Disease
  早期 I 期 · 干预性 · 2024/07Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06794008
  BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases
  II 期 · 干预性 · 2024/12/26Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06978738
  UCAR T-cell Therapy Targeting CD19/ BCMA in Patients With Relapse/ Refractory Autoimmune Diseases
  I 期 · 干预性 · 2025/05Changzhou No.2 People's Hospital
  中国研究中心 1 个：Changzhou
• 招募中NCT07104058
  A Study on the Effect of MRD Detected by FAPI PET / CT on the Recurrence of IgG4-RD
  不适用 · 干预性 · 2025/05/10Luo Yaping
  中国研究中心 1 个：Beijing
• 招募中NCT06978647
  A Clinical Study of YTS109 Cell in R/R Autoimmune Diseases
  I 期 · 干预性 · 2025/05/19China Immunotech (Beijing) Biotechnology Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT07068165
  A Randomized Controlled Study of the Efficacy and Safety of Lenalidomide in the Treatment of Active IgG4-related Disease
  II 期、III 期 · 干预性 · 2025/07/07Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing

另有 7 项未列出，见该病详情页。

朗格汉斯组织细胞增生症 19 项在招目录 第1批 · 60

• 招募中NCT03491605
  Surveillance and Tracking the Outcomes of Chronic Latent EBV Infection
  观察性 · 2019/07/01Huazhong University of Science and Technology
  中国研究中心 1 个：Wuhan
• 招募中NCT05936086
  A Randomized Controlled Study of High-dose Cyclophosphamide Induction Therapy in Adult Patients With HLH
  不适用 · 干预性 · 2023/04/20shifeng Lou
  中国研究中心 1 个：Chongqing
• 尚未开始招募NCT05775705
  L-DEP Regimen Combined With PD-1 Antibody as Induction Therapy for Epstein-Barr Virus-positive LA-HLH
  III 期 · 干预性 · 2023/08/01Beijing Friendship Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT05988177
  Efficacy and Safety of Carrimycin Tablets in HLH Patients With Carbapenem-resistant Infections
  不适用 · 干预性 · 2023/08/30Beijing Friendship Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05997602
  To Evaluate the Efficacy, Safety, and PK Characteristics of FCN-159 in Pediatric Patients With Refractory/Recurrent LCH
  II 期 · 干预性 · 2023/09/28Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
  中国研究中心 11 个：Beijing、Chengdu、Chongqing、Guangzhou、Hangzhou、Shenzhen 等 9 地
• 招募中NCT06078969
  Oral Prednisone in Treating LCH of Bone in Childhood and Adolescence
  II 期 · 干预性 · 2023/11/04Shanghai Changzheng Hospital
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT06131489
  Segmental Resection Combined With DEP Regimen for EBV-HLH Patients With Intestinal Involvement
  观察性 · 2023/12/01Beijing Friendship Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06614998
  Establishment of an Early Warning Screening System for Hemophagocytic Lymphohistiocytosis a Multi-center, Prospective Study
  观察性 · 2024/05/01The Affiliated Hospital of Xuzhou Medical University
  中国研究中心 1 个：Xuzhou
• 仅邀请入组NCT06902792
  Adebrelimab Combined With Trametinib in the Treatment of Refractory Recurrent Langerhans Cell Histiocytosis in Children and Adolescents
  I 期 · 干预性 · 2025/02/06Second Affiliated Hospital of Wenzhou Medical University
  中国研究中心 1 个：Wenzhou
• 招募中NCT07187167
  Efficacy and Safety of the RD Regimen(Lenalidomide, Dexamethasone) for Rosai-Dorfman Disease
  II 期 · 干预性 · 2025/04/12Cancer Institute and Hospital, Chinese Academy of Medical Sciences
  中国研究中心 1 个：Beijing
• 招募中NCT07187193
  Efficacy and Safety of Low-Dose Cytarabine Combined With Thalidomide in Adult Patients With Untreated LCH
  II 期 · 干预性 · 2025/04/12Cancer Institute and Hospital, Chinese Academy of Medical Sciences
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06951971
  Dose-modified Emapalumab and Ruxolitinib (E-Ru) Regimens for Hemophagocytic Lymphohistiocytosis
  II 期、III 期 · 干预性 · 2025/06/01The First Affiliated Hospital of Soochow University
  中国研究中心 1 个：Suzhou

另有 7 项未列出，见该病详情页。

阵发性睡眠性血红蛋白尿 18 项在招目录 第1批 · 88

• 招募中NCT04645199
  National Longitudinal Cohort of Hematological Diseases
  观察性 · 2020/12/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT07256301
  A Phase Ib, Multicenter, Open-Label Study of Multiple-Dose EA5 in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH)
  I 期 · 干预性 · 2024/01/03Shanghai Lanyi Therapeutics Co., Ltd.
  中国研究中心 2 个：Hangzhou、Tianjin
• 尚未开始招募NCT06543459
  To Evaluate the Effect of Single Oral Dose of MY008211A Tablets on QTc Interval in Healthy Subjects
  I 期 · 干预性 · 2024/08/10Wuhan Createrna Science and Technology Co., Ltd
  中国研究中心 1 个：Beijing
• 招募中NCT06932471
  Study of Safety and Efficacy of MY008211A in Patients With Residual Anemia Despite Anti-C5 Antibody Treatment
  III 期 · 干预性 · 2024/08/30Wuhan Createrna Science and Technology Co., Ltd
  中国研究中心 1 个：Beijing
• 招募中NCT06932744
  Study of Safety and Efficacy of MY008211A in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients Who Are Naive to Complement Inhibitor Therapy
  III 期 · 干预性 · 2024/08/30Wuhan Createrna Science and Technology Co., Ltd
  中国研究中心 2 个：Beijing、Tianjin
• 招募中NCT06933914
  Long-term Safety and Tolerability of MY008211A Tablets in Patients With Paroxysmal Nocturnal Hemoglobinuria
  II 期、III 期 · 干预性 · 2024/11/30Wuhan Createrna Science and Technology Co., Ltd
  中国研究中心 1 个：Tianjin
• 招募中NCT06745622
  Long-term Safety and Tolerability of HSK39297 in Patients With Paroxysmal Nocturnal Hemoglobinuria
  II 期 · 干预性 · 2025/01/03Haisco Pharmaceutical Group Co., Ltd.
  中国研究中心 1 个：Nanjing
• 招募中NCT06978699
  A Phase II Study Evaluating the Efficacy and Safety of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
  II 期 · 干预性 · 2025/04/30S-INFINITY Pharmaceuticals Co., Ltd
  中国研究中心 1 个：Tianjin
• 招募中NCT07152288
  Investigation of Pharmacokinetics,Safety,and Pharmacodynamics of HSK39297 in Subjects With Hepatic Impairment
  I 期 · 干预性 · 2025/05/29Haisco Pharmaceutical Group Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT06931691
  A Multi-center, Ambispective Cohort Study to Evaluate the Impact of Iptacopan for Adult Patients With PNH in China
  观察性 · 2025/06/10Novartis Pharmaceuticals
  中国研究中心 10 个：Changchun、Guangzhou、Hefei、Jinan、Nanchang、Nantong 等 10 地
• 仅邀请入组NCT07657052
  A Multicenter, Open-label Study Evaluating the Long-term Safety and Efficacy of XH-S003 Capsules in Patients With Paroxysmal Nocturnal Hemoglobinuria
  不适用 · 干预性 · 2025/08/15S-INFINITY Pharmaceuticals Co., Ltd
  中国研究中心 1 个：Tianjin
• 尚未开始招募NCT07177859
  A Phase III Study of NTQ5082 Capsules in the Treatment of Paroxysmal Nocturnal Hemoglobinuria Patients
  III 期 · 干预性 · 2025/11Nanjing Chia-tai Tianqing Pharmaceutical
  中国研究中心 1 个：Tianjin

另有 6 项未列出，见该病详情页。

原发性胆汁性胆管炎 15 项在招目录 第2批 · 63

• 招募中NCT06365424
  Fenofibrate in Patients With Primary Biliary Cholangitis (PBC)
  II 期、III 期 · 干预性 · 2017/04/08Xijing Hospital of Digestive Diseases
  中国研究中心 1 个：Xi'an
• 招募中NCT04650243
  Clinical Research of Tapering UDCA in PBC Patients With a Complete Response
  IV 期 · 干预性 · 2020/01/21Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT05050136
  A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
  II 期 · 干预性 · 2021/09/22Mirum Pharmaceuticals, Inc.
  中国研究中心 7 个：Beijing、Changchun、Fuzhou、Hangzhou、Wuhan
• 招募中NCT05749822
  Fenofibrate for Compensated Cirrhosis Patients With Primary Biliary Cholangitis
  II 期、III 期 · 干预性 · 2023/02/17Xijing Hospital of Digestive Diseases
  中国研究中心 12 个：Beijing、Changsha、Chengdu、Guangzhou、Kunming、Nanjing 等 11 地
• 招募中NCT05751967
  Fenofibrate Combined With Ursodeoxycholic Acid in Subjects With Primary Biliary Cholangitis
  III 期 · 干预性 · 2023/02/22Xijing Hospital of Digestive Diseases
  中国研究中心 7 个：Chengdu、Guangzhou、Lanzhou、Nanjing、Shenyang、Tianjin 等 7 地
• 招募中NCT06051617
  Seladelpar in Subjects With Primary Biliary Cholangitis (PBC) and Compensated Cirrhosis
  III 期 · 干预性 · 2023/09/07Gilead Sciences
  中国研究中心 20 个：Beijing、Changchun、Chengdu、Chongqing、Fuzhou、Guangzhou 等 17 地
• 招募中NCT06755151
  Fenofibrate in Primary Biliary Cholangitis: a Real World Study
  III 期 · 干预性 · 2024/01/17Xijing Hospital of Digestive Diseases
  中国研究中心 1 个：Xi'an
• 招募中NCT06755541
  Fenofibrate in Combination With Ursodeoxycholic Acid in Primary Biliary Cholangitis: a Real World Study
  III 期 · 干预性 · 2024/01/30Xijing Hospital of Digestive Diseases
  中国研究中心 1 个：Xi'an
• 仅邀请入组NCT06591468
  A Multicenter, Randomized, Controlled Trial of Prednisone Combined With Ursodeoxycholic Acid in the Treatment of Primary Biliary Cholangitis With Moderate to Severe Interface Hepatitis Characteristics
  II 期、III 期 · 干预性 · 2024/10/30Han Ying
  中国研究中心 2 个：Guangzhou、Xi'an
• 招募中NCT07149675
  PBC Long-term Outcomes Study
  观察性 · 2025/05/18Beijing Friendship Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07282353
  A Study of CS0159 in Patients With PBC With Inadequate Response or Intolerance to UDCA
  III 期 · 干预性 · 2025/12/01Cascade Pharmaceuticals, Inc
  中国研究中心 42 个：Baoji、Beijing、Changchun、Changsha、Chengdu、Chongqing 等 31 地
• 招募中NCT07296458
  FIREFLY Trial: Fenofibrate Intervention---Randomized Evaluation in First-Line PBC Therapy
  III 期 · 干预性 · 2025/12/15Xijing Hospital of Digestive Diseases
  中国研究中心 1 个：Xi'an

另有 3 项未列出，见该病详情页。

自身免疫性脑炎 14 项在招目录 第1批 · 9

• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06173076
  A Prospective Study to Evaluate Clinical Outcomes in Anti-LGI1 Encephalitis
  观察性 · 2022/05/18Shen Chun-Hong
  中国研究中心 1 个：Hangzhou
• 招募中NCT06388161
  Neural Autoantibody Prevalence in New-onset Focal Seizures of Unknown Etiology
  观察性 · 2023/08/01Shen Chun-Hong
  中国研究中心 1 个：Hangzhou
• 招募中NCT07653984
  Cohort Study on Neuroimmune Diseases in the Reproductive Age
  观察性 · 2024/04/21Third Affiliated Hospital, Sun Yat-Sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06502015
  Biomarkers in Autoimmune Disease of Nervous System
  观察性 · 2024/07/31Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06584045
  Efficacy and Safety of Calculus Bovis Sativus (CBS) for Adult Encephalitis (CBSinEncephalitis)
  不适用 · 干预性 · 2024/09/30Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06867991
  Safety and Efficacy of Combined B Cell Depleting theRapy And Daratumumab In Autoimmune Encephalitis
  III 期 · 干预性 · 2024/11/08The First People's Hospital of Changzhou
  中国研究中心 2 个：Changzhou、Zhengzhou
• 招募中NCT06510283
  Efficacy and Safety of Taitacept in Treatment of Refractory or Recurrent Anti-NMDAR/anti-LGI1 Encephalitis
  II 期 · 干预性 · 2024/12/01Beijing Tongren Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06760091
  A Multicentric Cohort of Autoimmune Encephalitis
  观察性 · 2025/02/01Xuanwu Hospital, Beijing
  中国研究中心 1 个：Beijing
• 招募中NCT06939166
  Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/06/17Tianjin Huanhu Hospital
  中国研究中心 1 个：Tianjin
• 招募中NCT07337785
  CD19/BCMA-Targeted UCAR-T for Patients With Neurological Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/12/12Tongji Hospital
  中国研究中心 1 个：Wuhan
• 尚未开始招募NCT07333196
  Tongji NADs Cohort
  观察性 · 2026/03/01Tongji Hospital
  中国研究中心 1 个：Wuhan

另有 2 项未列出，见该病详情页。

慢性炎性脱髓鞘性多发性神经根神经病 13 项在招目录 第2批 · 13

• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT05327114
  Efficacy and Safety Study of Nipocalimab for Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
  II 期、III 期 · 干预性 · 2022/09/23Janssen Research & Development, LLC
  中国研究中心 19 个：Beijing、Changchun、Changsha、Chengdu、Chifeng、Fuzhou 等 12 地
• 招募中NCT06502015
  Biomarkers in Autoimmune Disease of Nervous System
  观察性 · 2024/07/31Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT06290141
  A Study to Test the Efficacy and Safety of Riliprubart Against the Usual Treatment of Intravenous Immunoglobulin (IVIg) in People With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
  III 期 · 干预性 · 2024/08/21Sanofi
  中国研究中心 17 个：Beijing、Changsha、Chengdu、Fuzhou、Guangzhou、Hangzhou 等 12 地
• 招募中NCT06858579
  A Study to Evaluate the Efficacy and Safety of DNTH103 in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CAPTIVATE)
  III 期 · 干预性 · 2025/02/10Dianthus Therapeutics
  中国研究中心 14 个：Beijing、Changsha、Chengdu、Chifeng、Fujian、Guangdong 等 14 地
• 尚未开始招募NCT06485232
  Universal CAR-T Cells in Patients with Refractory Autoimmune Diseases of the Nervous System.
  早期 I 期 · 干预性 · 2025/02/28Xuanwu Hospital, Beijing
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT06859099
  Long-term Safety and Efficacy Study of Riliprubart in Participants With CIDP
  III 期 · 干预性 · 2025/04/01Sanofi
  中国研究中心 8 个：Beijing、Chengdu、Fuzhou、Guangzhou、Shanghai、Wuhan
• 招募中NCT07022197
  Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases
  I 期、II 期 · 干预性 · 2025/04/10Tianjin Medical University General Hospital
  中国研究中心 1 个：Tianjin
• 招募中NCT06939166
  Universal Chimeric Antigen Receptor T-Cell （UCAR T-cell） Therapy Targeting CD19/B Cell Maturation Antigen （CD19/BCMA） in Patients With r/r Neurological Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/06/17Tianjin Huanhu Hospital
  中国研究中心 1 个：Tianjin
• 招募中NCT07091630
  A Study to Assess the Efficacy and Safety of Empasiprubart in Adults With CIDP
  III 期 · 干预性 · 2025/09/16argenx
  中国研究中心 5 个：Beijing、Fuzhou、Guangzhou、Xi'an
• 招募中NCT07337785
  CD19/BCMA-Targeted UCAR-T for Patients With Neurological Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/12/12Tongji Hospital
  中国研究中心 1 个：Wuhan
• 尚未开始招募NCT07333196
  Tongji NADs Cohort
  观察性 · 2026/03/01Tongji Hospital
  中国研究中心 1 个：Wuhan

另有 1 项未列出，见该病详情页。

化脓性汗腺炎 13 项在招目录 第2批 · 40

• 招募中NCT06926192
  Efficacy and Safety of Secukinumab in Conjunction With Surgery in Moderate to Severe Hidradenitis Suppurativa
  观察性 · 2020/01/01Peking Union Medical College
  中国研究中心 1 个：Nanjing
• 招募中NCT06082323
  A Single and Multiple Ascending Dose Trial of LT-002-158 Tablets in Healthy Adult Volunteers
  I 期 · 干预性 · 2024/06/13Leadingtac Pharmaceutical (Shaoxing) Co., Ltd.
  中国研究中心 1 个：Shanghai
• 招募中NCT06707246
  A Randomized Trial Comparing the Efficacy and Safety of Secukinumab with Tofacitinib in the Treatment of Moderate to Severe Hidradenitis Suppurativa in Chinese Patients
  不适用 · 干预性 · 2024/12/15Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 招募中NCT06895499
  Efficacy, Safety, and Tolerability of HB0043 in Hidradenitis Suppurativa Patients.
  I 期、II 期 · 干预性 · 2025/03/19Shanghai Huaota Biopharmaceutical Co., Ltd.
  中国研究中心 1 个：Jinan
• 招募中NCT06946641
  A Phase I Study of QLS12010 Capsules: Safety, Tolerability, PK, PD, and Food Effects in Healthy Adults and Moderate to Severe Atopic Dermatitis Patients
  I 期 · 干预性 · 2025/03/31Shanghai Qilu Pharmaceutical Research and Development Center LTD
  中国研究中心 1 个：Beijing
• 招募中NCT07109765
  Secukinumab Treatment for Moderate to Severe Hidradenitis Suppurativa
  观察性 · 2025/06/01Peking Union Medical College
  中国研究中心 1 个：Beijing
• 招募中NCT07170917
  Brivekimig for the Treatment of Moderate to Severe Hidradenitis Suppurativa
  II 期 · 干预性 · 2025/11/06Sanofi
  中国研究中心 4 个：Chengdu、Guangzhou、Shanghai、Ürümqi
• 尚未开始招募NCT07261072
  Comparison of the Clinical Efficacy and Safety of Topical and Subcutaneous Injection of Secukinumab in HS
  IV 期 · 干预性 · 2025/12/01Peking Union Medical College
  中国研究中心 1 个：Nanjing
• 招募中NCT07225569
  A Study to Investigate Efficacy and Safety With SAR445399 in Adult Participants With Moderate to Severe Hidradenitis Suppurativa
  II 期 · 干预性 · 2025/12/29Sanofi
  中国研究中心 4 个：Beijing、Changsha、Shanghai、Wuhan
• 招募中NCT07755670
  Exploration of Brain Functional Networks in Patients With Hidradenitis Suppurativa Using fMRI
  观察性 · 2026/01/03Second Xiangya Hospital of Central South University
  中国研究中心 1 个：Changsha
• 招募中NCT07244263
  A Study of Zasocitinib in Adults With Hidradenitis Suppurativa
  II 期 · 干预性 · 2026/01/26Takeda
  中国研究中心 5 个：Beijing、Guangzhou、Hangzhou、Shanghai、Wuhan
• 尚未开始招募NCT07554014
  A Single-center, Prospective, Self-controlled, Observational Real-world Study of the Efficacy and Safety of Anti-IL-17A/F Monoclonal Antibody Combined With Non-ablative Dot Laser for the Treatment of Mild to Moderate Hidradenitis Suppurativa
  IV 期 · 干预性 · 2026/04/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou

另有 1 项未列出，见该病详情页。

原发性骨髓纤维化 12 项在招目录 第2批 · 67

• 招募中NCT04064060
  A Study to Evaluate Long-term Safety in Participants Who Have Participated in Other Luspatercept (ACE-536) Clinical Trials
  III 期 · 干预性 · 2019/08/12Celgene
  中国研究中心 6 个：Beijing、Chengdu、Guangzhou、Hangzhou、Shanghai、Tianjin
• 招募中NCT06164561
  18F-FAPI PET/MRI Imaging in Myelofibrosis: a Prospective Observational Study.
  观察性 · 2023/11/21First Affiliated Hospital of Zhejiang University
  中国研究中心 1 个：Hangzhou
• 招募中NCT06151119
  68Ga-FAPI PET/CT Imaging for Diagnosis, Grading, and Efficacy Evaluation of Myelofibrosis.
  观察性 · 2023/11/22The First Affiliated Hospital of Xiamen University
  中国研究中心 1 个：Xiamen
• 招募中NCT06122831
  A Clinical Trial of TQ05105 Tablets Combined With TQB3617 Capsules in the Treatment of Myelofibrosis (MF)
  I 期、II 期 · 干预性 · 2023/12/12Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
  中国研究中心 22 个：Cangzhou、Changchun、Changzhi、Chengde、Guangzhou、Harbin 等 18 地
• 招募中NCT06674382
  Haplo-HSCT for Myelofibrosis
  不适用 · 干预性 · 2024/01/01Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06378437
  A Study of GLB-001 in Patients With Myeloid Malignancies
  I 期 · 干预性 · 2024/05/24Hangzhou GluBio Pharmaceutical Co., Ltd.
  中国研究中心 14 个：Beijing、Chongqing、Hangzhou、Hefei、Nanchang、Shanghai 等 13 地
• 招募中NCT07317700
  A Clinical Trial of Flonoltinib Maleate for Intermediate or High-Risk Myelofibrosis
  III 期 · 干预性 · 2026/02/13Chengdu Zenitar Biomedical Technology Co., Ltd
  中国研究中心 2 个：Chengdu、Tianjin
• 招募中NCT07443306
  A Clinical Trial of Flonoltinib Maleate Tablets in the Treatment of JAK Inhibitor Refractory/Relapsed/Intolerant Patients With Intermediate to High Risk Myelofibrosis
  II 期 · 干预性 · 2026/03/18Chengdu Zenitar Biomedical Technology Co., Ltd
  中国研究中心 2 个：Chengdu、Tianjin
• 招募中NCT07480824
  To Evaluate the Pharmacokinetics and Safety of TQ05105 Tablet in Hepatic Impairment Subjects
  I 期 · 干预性 · 2026/04/01Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
  中国研究中心 2 个：Jinan、Luoyang
• 尚未开始招募NCT07539779
  Luspatercept in Preventing Poor Erythroid Engraftment for Hematological Malignancies With Moderate to Severe Myelofibrosis
  II 期、III 期 · 干预性 · 2026/05/01Nanfang Hospital, Southern Medical University
  中国研究中心 1 个：Guangzhou
• 招募中NCT07357727
  A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
  III 期 · 干预性 · 2026/06/16Novartis Pharmaceuticals
  中国研究中心 15 个：Beijing、Changchun、Chongqing、Fuzhou、Guangzhou、Hangzhou 等 15 地
• 招募中NCT07551427
  A Clinical Trial to Evaluate the Efficacy, Safety, and Pharmacokinetics of TQ05105 Tablets in Subjects With Intermediate/High-risk Myelofibrosis
  II 期 · 干预性 · 2026/06/18Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
  中国研究中心 24 个：Cangzhou、Changchun、Changzhi、Chengde、Chengdu、Fuzhou 等 22 地

肝豆状核变性 10 项在招目录 第1批 · 37

• 招募中NCT04965558
  Clinical Features and Outcome in Patients With Osseomuscular Type of Wilson's Disease
  观察性 · 2004/01/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 招募中NCT04965545
  Role for Biochemical Assays and Kayser-Fleischer Rings in Diagnosis of Wilson Disease
  观察性 · 2004/01/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 招募中NCT03131427
  China Registry for Genetic / Metabolic Liver Diseases
  观察性 · 2015/06/13Beijing Friendship Hospital
  中国研究中心 12 个：Beijing、Chengdu、Guangzhou、Shanghai、Shijiazhuang、Tianjin 等 8 地
• 招募中NCT04012658
  A Registered Cohort Study on Wilson's Disease
  观察性 · 2019/07/01Wan-Jin Chen
  中国研究中心 1 个：Fuzhou
• 尚未开始招募NCT06196931
  Clinical Value of DWI-ADC Matching in the Short-term Prognosis of Wilson's Disease
  观察性 · 2024/01/01Hospital Affiliated to the Institute of Neurology, Anhui University of Chinese Medicine
  中国研究中心 1 个：Hefei
• 招募中NCT06650319
  A Clinical Study to Evaluate the Safety and Efficacy of LY-M003 Injection in Patients With Wilson Disease
  早期 I 期 · 干预性 · 2024/09/24Chaohui Yu
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT07173933
  Phase I/II Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of GC310 Injection in Patients With Wilson's Disease (WD)
  I 期、II 期 · 干预性 · 2025/10GeneCradle Inc
  中国研究中心 1 个：Beijing
• 招募中NCT07240896
  A Clinical Study on the Treatment of Wilson Disease With ATP7B mRNA/LNP (DSL101)
  早期 I 期 · 干预性 · 2025/12/31DSciLab Co., Ltd.
  中国研究中心 1 个：Hefei
• 尚未开始招募NCT07641140
  Phase I/II Clinical Study to Evaluate the Safety, Tolerability and Efficacy of LY-M003 Injection in Adult Patients With Wilson's Disease
  I 期、II 期 · 干预性 · 2026/06/15Lingyi Biotech Co., Ltd.
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT07465718
  Trientine Tetrahydrochloride Administered Once a Day for the First Line Treatment of Wilson's Disease Patients.
  III 期 · 干预性 · 2026/08Orphalan
  中国研究中心 3 个：Beijing、Hangzhou

脊髓延髓肌萎缩症（肯尼迪病） 10 项在招目录 第1批 · 109

• 尚未开始招募NCT05953168
  T-DXd in the First-Line Treatment of Locally Advanced or Metastatic TNBC-LAR HER2-low Patients
  II 期 · 干预性 · 2023/08Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT05954442
  Everolimus With Investigator's Choice of Chemotherapy in Advanced Triple-Negative Breast Cancer (TNBC) With Luminal Androgen Receptor (LAR) Subtype
  III 期 · 干预性 · 2023/09/13Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06348264
  Adjuvant Rezvilutamide in Combination With Androgen Deprivation Therapy in Androgen Receptor-positive, High-risk Salivary Duct Carcinoma
  II 期 · 干预性 · 2024/10/10Peking University First Hospital
  中国研究中心 2 个：Beijing
• 招募中NCT06854250
  Prostate Cryoablation Combined With Metronomic Cyclophosphamide for Metastatic Prostate Cancer
  II 期 · 干预性 · 2025/03/01Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06764485
  A Study to Compare the Efficacy and Safety of BMS-986365 Versus the Investigator's Choice of Therapy in Participants With Metastatic Castration-resistant Prostate Cancer
  III 期 · 干预性 · 2025/03/13Celgene
  中国研究中心 40 个：Beijing、Changsha、Changzhou、Chengdu、Chongqing、Fuzhou 等 25 地
• 尚未开始招募NCT06838520
  Effect of ADT and ARPI on Bone Loss of Patients with Prostate Cancer
  观察性 · 2025/04/01The First Affiliated Hospital with Nanjing Medical University
  中国研究中心 4 个：Changzhou、Nanjing、Suzhou、Yangzhou
• 招募中NCT06855277
  Study Comparing AAA817+ARPI Versus Standard of Care in Adult Participants With PSMA-positive mCRPC
  III 期 · 干预性 · 2025/07/01Novartis Pharmaceuticals
  中国研究中心 10 个：Beijing、Chengdu、Fuzhou、Guangzhou、Hangzhou、Nanjing 等 10 地
• 招募中NCT07190300
  TulmiSTAR-02: A Phase I/II Open-label Study of Tulmimetostat in Combination With Darolutamide vs. Darolutamide, and Tulmimetostat With Abiraterone in Patients With Metastatic Hormone-sensitive Prostate Cancer (mHSPC)
  I 期、II 期 · 干预性 · 2026/01/13Novartis Pharmaceuticals
  中国研究中心 1 个：Guangzhou
• 招募中NCT07336446
  A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
  I 期、II 期 · 干预性 · 2026/01/27AstraZeneca
  中国研究中心 1 个：Chengdu
• 招募中NCT07311694
  A Phase III Study Comparing HRS-4357 With Novel Androgen Receptor Pathway Inhibitors in Patients With Progressive, PSMA-Positive Metastatic Castration-Resistant Prostate Cancer
  III 期 · 干预性 · 2026/02/02Jiangsu HengRui Medicine Co., Ltd.
  中国研究中心 1 个：Shanghai

皮肤T细胞淋巴瘤 10 项在招目录 第2批 · 20

• 招募中NCT02848274
  ID Of Prognostic Factors In Mycosis Fungoides/Sezary Syndrome
  观察性 · 2016/10Stanford University
  中国研究中心 1 个：Beijing
• 招募中NCT05518851
  TreAtments and outComes in paTients With prImary CutAneous Lymphoma
  观察性 · 2022/08/01Peking University First Hospital
  中国研究中心 5 个：Beijing、Guandong、Jinan、Nanjing
• 招募中NCT06436677
  A Study of Molecular Subtyping-based Therapeutic Strategies for Cutaneous T-cell Lymphoma
  观察性 · 2024/05/09Peking University First Hospital
  中国研究中心 3 个：Beijing
• 招募中NCT06716658
  JAK1 Inhibitor Golidocitnib for the Treatment of Relapsed/Refractory Indolent T/NK-cell Lymphomas
  II 期 · 干预性 · 2024/12/25Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 2 个：Tianjin
• 招募中NCT06914037
  A Clinical Study of CHT101 in CD70-Positive Relapsed or Refractory Hematological Malignancies
  I 期 · 干预性 · 2025/03/31Tianjin Medical University Cancer Institute and Hospital
  中国研究中心 1 个：Tianjin
• 招募中NCT06925464
  CD7 CAR T-Cell Therapy for r/r T-cell Lymphomas
  I 期、II 期 · 干预性 · 2025/04/01Beijing GoBroad Hospital
  中国研究中心 4 个：Beijing、Shanghai、Zhanjiang
• 招募中NCT07022964
  CD5 CAR T-Cell Therapy for r/r T-cell Lymphomas
  I 期、II 期 · 干预性 · 2025/06/01Beijing GoBroad Hospital
  中国研究中心 3 个：Beijing、Shanghai
• 招募中NCT07754890
  Golidocitinib and Chidamide for Cutaneous T-Cell Lymphoma
  I 期、II 期 · 干预性 · 2025/08/26Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07535710
  Aclarubicin Plus Cyclophosphamide, Vincristine, and Prednisone (CAOP) in Patients With Previously Treated Cutaneous T-cell Lymphoma
  I 期、II 期 · 干预性 · 2026/03/30Shanghai Jiao Tong University School of Medicine
  中国研究中心 2 个：Shanghai、Wuxi
• 尚未开始招募NCT07717580
  Efficacy and Safety of Brentuximab Vedotin Combined With Lisaftoclax in CD30+ Cutaneous T-cell Lymphoma (CTCL)
  II 期 · 干预性 · 2026/07/31Peking University First Hospital
  中国研究中心 1 个：Beijing

大动脉炎/多发性大动脉炎 10 项在招目录 第2批 · 76

• 招募中NCT05388682
  Treatment of Patients With Takayasu Arteritis Complicated With Pulmonary Artery Involvement
  观察性 · 2014/01/19Chinese Pulmonary Vascular Disease Research Group
  中国研究中心 1 个：Beijing
• 招募中NCT03372980
  A Registry Study on Biomarkers of Takayasu's Arteritis (ARSBTA)
  观察性 · 2016/01Beijing Institute of Heart, Lung and Blood Vessel Diseases
  中国研究中心 1 个：Beijing
• 招募中NCT07184814
  Clinical Features and Prognosis of Takayasu's Arteritis With Pulmonary Arteries Involvement
  观察性 · 2016/01/01China-Japan Friendship Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT04300686
  A Pilot Study in Severe Patients With Takayasu Arteritis.
  IV 期 · 干预性 · 2020/03/01Shanghai Zhongshan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT05102448
  Comparison of Tofacitinib and Methotrexate in Takayasu's Arteritis
  IV 期 · 干预性 · 2021/11/01Shanghai Zhongshan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06178419
  Remote Ischemic Conditioning for Cerebral Ischemia in Patients With Takayasu Arteritis (TARIC-1)
  不适用 · 干预性 · 2024/01/01Xuanwu Hospital, Beijing
  中国研究中心 1 个：Beijing
• 招募中NCT06498089
  A Randomized, Controlled, Open-label, Multicenter Clinical Trial Comparing the Efficacy and Safety of a Precision Treatment Regimen Based on Clinical-molecular Phenotypes with a Conventional Treatment Regimen in the Treatment of Patients with Active Takayasu's Arteritis
  IV 期 · 干预性 · 2024/06/28Shanghai Zhongshan Hospital
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT06766552
  A Registry Study Assessing PRO, Dosing Patterns, and Safety of Vunakizumab in Patients With General Rheumatic Diseases.
  观察性 · 2025/01/30Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT07013838
  The Efficacy and Safety of Deucravacitinib in Takayasu's Arteritis
  IV 期 · 干预性 · 2025/06/15Chinese SLE Treatment And Research Group
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07451665
  Efficacy and Safety of Vunakizumab and Ivarmacitinib in the Treatment of Active Takayasu's Arteritis
  IV 期 · 干预性 · 2026/04/01Chinese SLE Treatment And Research Group
  中国研究中心 20 个：Baotou、Beijing、Chengdu、Ch’ang-ch’un、Guanzhou、Hangzhou 等 17 地

转甲状腺素蛋白淀粉样变性 10 项在招目录 第2批 · 80

• 招募中NCT07336394
  Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
  观察性 · 2010/01/01Chinese Academy of Medical Sciences, Fuwai Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT07130565
  Pan-Amyloid PET/CT in Various Amyloid-Related Disease
  观察性 · 2023/11/01Tianjin Medical University
  中国研究中心 2 个：Hefei、Tianjin
• 招募中NCT06082050
  Efficacy and Safety of Intravenous YOLT-201 for Transthyretin Amyloidosis Cardiomyopathy
  早期 I 期 · 干预性 · 2023/11/02Zhejiang University
  中国研究中心 1 个：Hangzhou
• 招募中NCT06539208
  A Phase I/IIa，Open-label, Single Ascending Dose and Dose-expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of YOLT-201 in Patients With Transthyretin Amyloidosis Polyneuropathy (ATTR-PN) or Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM)
  I 期、II 期 · 干预性 · 2024/05/23YolTech Therapeutics Co., Ltd
  中国研究中心 3 个：Beijing、Changsha、Hanzhou
• 招募中NCT06465810
  Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
  观察性 · 2024/06/25AstraZeneca
  中国研究中心 19 个：Beijing、Changsha、Chengdu、Chongqing、Dalian、Fuzhou 等 19 地
• 仅邀请入组NCT07103863
  Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
  观察性 · 2025/02/13Beijing Anzhen Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06940336
  To Evaluate the Efficacy and Safety of Tafamidis Meglumine Soft Capsules in the Treatment of Adult Patients With Transthyretin Amyloid Polyneuropathy
  IV 期 · 干预性 · 2025/06/19Qilu Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Jinan
• 招募中NCT07052903
  TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy
  III 期 · 干预性 · 2025/07/02Alnylam Pharmaceuticals
  中国研究中心 12 个：Binhai、Changsha、Dongcheng、Fuzhou、Haidian、Hangzhou 等 12 地
• 招募中NCT07232459
  [18F]FT8 PET Imaging in Immunoglobulin Light Chain Amyloidosis
  观察性 · 2025/07/21Tianjin Medical University
  中国研究中心 2 个：Hefei、Tianjin
• 招募中NCT07207811
  CLEOPATTRA: A Research Study to Look at the Effects of Treatment With a Medicine Called Coramitug (NNC6019-0001) in People With Heart Failure Due to Transthyretin Amyloid (ATTR) Amyloidosis
  III 期 · 干预性 · 2025/10/02Novo Nordisk A/S
  中国研究中心 32 个：Beijing、Binhai、Changchun、Changsha、Chongqing、Fuzhou 等 22 地

肺囊性纤维化 9 项在招目录 第1批 · 101

• 招募中NCT05289245
  A National Registry on Chinese Patients With Cystic Fibrosis
  观察性 · 2022/04/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06528418
  Identification of Multiple Pulmonary Diseases Using Volatile Organic Compounds Biomarkers in Human Exhaled Breath
  观察性 · 2024/06/30ChromX Health
  中国研究中心 1 个：Guangzhou
• 招募中NCT06660992
  A Study to Assess the Efficacy and Safety of HSK31858 in Participants With Non-Cystic Fibrosis Bronchiectasis
  III 期 · 干预性 · 2024/09/30Haisco Pharmaceutical Group Co., Ltd.
  中国研究中心 2 个：Guangzhou、Yixingcun
• 尚未开始招募NCT06715956
  Observational Study of Sputum Inflammation Phenotypes and Clinical Outcomes in Non-Cystic Fibrosis Bronchiectasis
  观察性 · 2024/12/01Guangzhou Institute of Respiratory Disease
  中国研究中心 1 个：Guangzhou
• 招募中NCT06775340
  Study of RSS0343 Tablets in Patients With Non-cystic Fibrosis Bronchiectasis
  II 期 · 干预性 · 2025/01/22Jiangsu HengRui Medicine Co., Ltd.
  中国研究中心 1 个：Guangzhou
• 尚未开始招募NCT06981091
  A Multicenter, Randomized, Double-blind, Placebo-controlled Parallel-group Phase 2 Clinical Trial to Evaluate the Efficacy and Safety of XH-S004 Tablets in Patients With Non-cystic Fibrosis Bronchiectasis
  II 期 · 干预性 · 2025/05/16S-INFINITY Pharmaceuticals Co., Ltd
  中国研究中心 1 个：Shanghai
• 招募中NCT07616375
  A Study to Assess the Safety of HSK31858 in Participants With Non-Cystic Fibrosis Bronchiectasis
  III 期 · 干预性 · 2025/09/28Haisco Pharmaceutical Group Co., Ltd.
  中国研究中心 1 个：Yixingcun
• 招募中NCT07245407
  A Translational Study for Phenotyping and Endotyping Chinese Patients With NCFBE
  观察性 · 2025/10/27AstraZeneca
  中国研究中心 38 个：Anning、Beijing、Changchun、Changsha、Chengdu、Chongqing 等 25 地
• 尚未开始招募NCT07696091
  Study on Drug Interactions Affecting the Pharmacokinetics of RSS0343 Tablet
  I 期 · 干预性 · 2026/07Reistone Biopharma Company Limited
  中国研究中心 1 个：Guangzhou

进行性肌营养不良 8 项在招目录 第1批 · 98

• 招募中NCT04369209
  A Registered Cohort Study on FSHD1
  观察性 · 2001/01Ning Wang, MD., PhD.
  中国研究中心 1 个：Fuzhou
• 招募中NCT04012671
  A Registered Cohort Study on Duchenne Muscular Dystrophy
  观察性 · 2019/07/01Ning Wang, MD., PhD.
  中国研究中心 1 个：Fuzhou
• 仅邀请入组NCT04989751
  A Multicenter Phenotype-Genotype Analysis of LGMD Patients in China
  观察性 · 2021/07/07Huashan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06641895
  Evaluation of the Safety and Efficacy of BBM-D101 to Treat Patients with Duchenne Muscular Dystrophy
  早期 I 期 · 干预性 · 2024/07/25Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 招募中NCT06900049
  Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
  早期 I 期 · 干预性 · 2024/10/24Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 招募中NCT07058662
  A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy.
  I 期、II 期 · 干预性 · 2025/07/31Belief BioMed (Beijing) Co., Ltd
  中国研究中心 1 个：Beijing
• 招募中NCT07188012
  Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
  早期 I 期 · 干预性 · 2025/08/06Shanghai Siponuoyin Biotechnology Co Ltd
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT07511920
  A Multicenter Cohort Study of Duchenne and Becker Muscular Dystrophy in Western Chinese Children
  观察性 · 2026/04/20West China Second University Hospital
  中国研究中心 1 个：Chengdu

视网膜色素变性 8 项在招目录 第1批 · 102

• 招募中NCT05874310
  Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa
  早期 I 期 · 干预性 · 2023/02/01Frontera Therapeutics
  中国研究中心 2 个：Beijing、Shanghai
• 招募中NCT05853107
  Pilot Study of AuTNA I
  不适用 · 干预性 · 2023/05/12Eye & ENT Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06292650
  Safety and Efficacy Study of Novel Gene Therapy ZM-02 for Retinitis Pigmentosa Patients
  早期 I 期 · 干预性 · 2024/02/25Zhongmou Therapeutics
  中国研究中心 1 个：Beijing
• 招募中NCT06936787
  An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa
  I 期 · 干预性 · 2025/04/20Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT07082855
  A Multicenter, Randomized, Double-Blind, Controlled Clinical Study of Minocycline for the Treatment of Retinitis Pigmentosa
  III 期 · 干预性 · 2025/07/15Zhongshan Ophthalmic Center, Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06952842
  Safety and Efficacy of ZVS203e in the Treatment of Retinitis Pigmentosa Caused by RHO Gene Mutation
  I 期、II 期 · 干预性 · 2025/08/04Chigenovo Co., Ltd
  中国研究中心 1 个：Beijing
• 招募中NCT07126470
  Impact of Capsular Tension Ring on Intraocular Lens Position in Retinitis Pigmentosa Cataract Patients
  不适用 · 干预性 · 2025/08/25Zhongshan Ophthalmic Center, Sun Yat-sen University
  中国研究中心 3 个：Guangzhou、Shenzhen、Wuhan
• 尚未开始招募NCT07311863
  UGX202 Injection in Patients With Advanced Retinitis Pigmentosa
  早期 I 期 · 干预性 · 2026/01Suzhou UgeneX Therapeutics Co., Ltd.
  中国研究中心 1 个：Shanghai

恶性胸膜间皮瘤 8 项在招目录 第2批 · 47

• 招募中NCT03917043
  APG-2449 in Patients With Advanced Solid Tumors
  I 期 · 干预性 · 2019/05/27Ascentage Pharma Group Inc.
  中国研究中心 9 个：Beijing、Changsha、Fuzhou、Guangzhou、Hangzhou、Wuhan 等 7 地
• 招募中NCT06726564
  A Study of MT027 in Patients with Pleural Malignant Tumors
  I 期 · 干预性 · 2024/05/15Suzhou Maximum Bio-tech Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT06535607
  Study of Volrustomig as Monotherapy or in Combination With Anti- Cancer Agents in Participants With Advanced/Metastatic Solid Tumors
  II 期 · 干预性 · 2024/08/22AstraZeneca
  中国研究中心 25 个：Anyang、Beijing、Bengbu、Changchun、Changsha、Chengdu 等 25 地
• 招募中NCT05188859
  First Line Sintilimab Combined With Anlotinib and Platinum Doublet Chemotherapy in Malignant Pleural Mesothelioma
  II 期 · 干预性 · 2024/09/01Cancer Institute and Hospital, Chinese Academy of Medical Sciences
  中国研究中心 2 个：Beijing、Taiyuan
• 招募中NCT06566079
  Study of ISM6331 in Participants With Advanced/Metastatic Malignant Mesothelioma or Other Solid Tumors
  I 期 · 干预性 · 2024/12/27InSilico Medicine Hong Kong Limited
  中国研究中心 4 个：Beijing、Guangzhou、Shanghai、Zhengzhou
• 招募中NCT06875076
  Multicenter Single-Arm Study of Ivonescimab (AK112) Combined with Chemotherapy in Pretreated Pleural Mesothelioma
  II 期 · 干预性 · 2025/03/08The First Hospital of Jilin University
  中国研究中心 1 个：Changchun
• 尚未开始招募NCT07282873
  Study of TYK-01054 Capsules in Patients With Advanced Solid Tumors
  I 期、II 期 · 干预性 · 2026/01/01TYK Medicines, Inc
  中国研究中心 1 个：Shanghai
• 招募中NCT07510815
  Dual-Target MSLN/FAP CAR-NK Cells for Pleural and Peritoneal Mesothelioma
  I 期、II 期 · 干预性 · 2026/03/02Beijing Biotech
  中国研究中心 1 个：Shenzhen

嗜铬细胞瘤 8 项在招目录 第2批 · 60

• 招募中NCT04924075
  Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
  II 期 · 干预性 · 2021/08/12Merck Sharp & Dohme LLC
  中国研究中心 4 个：Beijing、Chengdu、Guangzhou、Shanghai
• 招募中NCT05069220
  18F-MFBG PET/CT in the Evaluation of Neural Crest Tumor
  早期 I 期 · 干预性 · 2021/09/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06813742
  18F-mFBG PET Imaging in the Evaluation of Pheochromocytoma
  观察性 · 2023/05/01First Affiliated Hospital of Zhejiang University
  中国研究中心 2 个：Hangzhou
• 招募中NCT07288931
  Al18F-NOTA-LM3 PET/CT in Patients With Pheochromocytoma and Paraganglioma
  不适用 · 干预性 · 2024/08/06Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT07182175
  Comparative Study of Perioperative Cardiac Injury in Patients With Functional Adrenal Tumors
  观察性 · 2024/09/19Peking University First Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06683846
  Ivonescimab in the Treatment of Multiple Advanced Tumors
  II 期 · 干预性 · 2024/11/20Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT07195500
  Clinical Application of Somatostatin Receptor and Norepinephrine Transporter Targeted Imaging for Diagnosis and Staging of Neuroblastoma and Pheochromocytoma/Paraganglioma
  观察性 · 2024/12/25Nanjing First Hospital, Nanjing Medical University
  中国研究中心 1 个：Nanjing
• 尚未开始招募NCT06429397
  Anlotinib Combined With Benmelstobart for Advanced Pheochromocytoma
  II 期 · 干预性 · 2025/05/01Sun Yat-sen University
  中国研究中心 1 个：Guangzhou

真性红细胞增多症 8 项在招目录 第2批 · 62

• 招募中NCT05870475
  Pegylated Interferon α-2b in Combination With Ruxolitinib for Treating Hydroxyurea-resistant/Intolerant PV
  II 期 · 干预性 · 2023/06/30Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06752746
  A Study of Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of 9MW3011 in Patients With Polycythemia Vera
  I 期 · 干预性 · 2024/03/21Mabwell (Shanghai) Bioscience Co., Ltd.
  中国研究中心 6 个：Hangzhou、Nanchang、Tianjin、Wuhan、Zhengzhou
• 招募中NCT06378437
  A Study of GLB-001 in Patients With Myeloid Malignancies
  I 期 · 干预性 · 2024/05/24Hangzhou GluBio Pharmaceutical Co., Ltd.
  中国研究中心 14 个：Beijing、Chongqing、Hangzhou、Hefei、Nanchang、Shanghai 等 13 地
• 招募中NCT06734637
  Efficacy and Safety of Peginterferon in ET and PV.
  不适用 · 干预性 · 2024/11/20Zhenya Hong
  中国研究中心 1 个：Wuhan
• 招募中NCT07012109
  Study of BEBT-507 Injection in Subjects With Polycythemia Vera (PV)
  I 期 · 干预性 · 2025/06/17BeBetter Med Inc
  中国研究中心 1 个：Tianjin
• 招募中NCT07232290
  Phase IIa Study of Flonoltinib Maleate Tablets in the Treatment of Patients With Polycythemia Vera
  II 期 · 干预性 · 2025/09/23Chengdu Zenitar Biomedical Technology Co., Ltd
  中国研究中心 1 个：Tianjin
• 招募中NCT07445893
  A Clinical Study of Gecacitinib Combined With Pegylated Interferon in Patients With PV
  不适用 · 干预性 · 2026/04/02Duan Minghui
  中国研究中心 1 个：Beijing
• 招募中NCT07357727
  A Phase 3 Study of Pelabresib (DAK539) and Ruxolitinib in Myelofibrosis (MF)
  III 期 · 干预性 · 2026/06/16Novartis Pharmaceuticals
  中国研究中心 15 个：Beijing、Changchun、Chongqing、Fuzhou、Guangzhou、Hangzhou 等 15 地

冠状动脉扩张病 7 项在招目录 第1批 · 24

• 招募中NCT04573660
  Abbott Vascular Medical Device Registry
  观察性 · 2020/10/25Abbott Medical Devices
  中国研究中心 3 个：Jinhua、Ningbo、Xi'an
• 招募中NCT06993636
  Pharmacometrics Analysis of Rivaroxaban in Chinese Children Aged Over 2 Years
  观察性 · 2023/01/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06978439
  Model-informed Dose Optimization for Rivaroxaban in Children With Giant Coronary Artery Aneurysm After Kawasaki Disease
  IV 期 · 干预性 · 2024/01/10Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06822712
  Efficacy and Safety of Sirolimus-coated Coronary Balloon Dilatation Catheter for De Novo Coronary Bifurcation Lesions
  不适用 · 干预性 · 2025/03/03BrosMed Medical Co., Ltd
  中国研究中心 1 个：Lanzhou
• 招募中NCT05643651
  Rivaroxaban for Children Aged Over 2 Years With Giant Coronary Artery Aneurysms After Kawasaki Disease
  IV 期 · 干预性 · 2025/06/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT07415902
  Coronary Artery Drug-Coated Balloon Dilatation Catheter Clinical Trial Protocol
  观察性 · 2026/02/28DK Medical Technology (Suzhou) Co., Ltd.
  中国研究中心 1 个：Zhengzhou
• 招募中NCT07764081
  Progression, Management, and Clinical Outcomes of Coronary Artery Aneurysms
  观察性 · 2026/07/31Nanjing First Hospital, Nanjing Medical University
  中国研究中心 1 个：Nanjing

法布雷病 7 项在招目录 第1批 · 27

• 招募中NCT00196742
  Fabry Disease Registry & Pregnancy Sub-registry
  观察性 · 2001/07/31Genzyme, a Sanofi Company
  中国研究中心 6 个：Beijing、Hangzhou、Jinan、Nanjing、Shanghai
• 招募中NCT07336394
  Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
  观察性 · 2010/01/01Chinese Academy of Medical Sciences, Fuwai Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06207552
  Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
  早期 I 期 · 干预性 · 2024/02/20Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06539624
  Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
  不适用 · 干预性 · 2024/10/16The Children's Hospital of Zhejiang University School of Medicine
  中国研究中心 2 个：Hangzhou、Shanghai
• 招募中NCT06512571
  CVI Alterations in FD: a Prospective, Multicenter, Observational Cohort Study
  观察性 · 2024/12/31China National Center for Cardiovascular Diseases
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06819514
  The Safety and Efficacy of Intravenous EXG110 in Patients With Fabry Disease
  I 期、II 期 · 干预性 · 2025/03/01Guangzhou Jiayin Biotech Ltd
  中国研究中心 3 个：Hangzhou、Nanjing
• 招募中NCT07187440
  A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
  观察性 · 2025/11/01Takeda
  中国研究中心 18 个：Beijing、Chengdu、Fuzhou、Hangzhou、Harbin、Hefei 等 14 地

泛发性脓疱型银屑病 7 项在招目录 第2批 · 33

• 招募中NCT06231381
  Efficacy and Safety of HB0034 in Patients with Generalized Pustular Psoriasis (GPP)
  II 期 · 干预性 · 2024/02/29Shanghai Huaota Biopharmaceutical Co., Ltd.
  中国研究中心 2 个：Beijing、Shanghai
• 招募中NCT06477536
  Long-Term Safety and Efficacy of HB0034 in Subjects With Generalized Pustular Psoriasis
  II 期、III 期 · 干预性 · 2024/09/02Shanghai Huaota Biopharmaceutical Co., Ltd.
  中国研究中心 3 个：Beijing、Hangzhou
• 招募中NCT07270003
  A Prospective, Single-arm, Open-label Clinical Trial to Evaluate the Efficacy and Safety of Ivarmacitinib in the Treatment of Palmoplantar Pustulosis
  不适用 · 干预性 · 2025/10/01Xijing Hospital
  中国研究中心 1 个：Xi'an
• 招募中NCT07219420
  A Study to Evaluate the Efficacy and Safety of Bimekizumab in Study Participants With Palmoplantar Pustulosis
  III 期 · 干预性 · 2025/11/14UCB Biopharma SRL
  中国研究中心 15 个：Beijing、Chengdu、Chongqing、Guangzhou、Hangzhou、Jinan 等 11 地
• 招募中NCT07503652
  Clinical Observation of Xeligekimab in the Treatment of Moderate to Severe Palmoplantar Pustulosis
  IV 期 · 干预性 · 2026/03First Hospital of China Medical University
  中国研究中心 1 个：Shenyang
• 招募中NCT07552454
  Picankibart in Palmoplantar Pustulosis
  不适用 · 干预性 · 2026/04/15Xijing Hospital
  中国研究中心 1 个：Xi'an
• 尚未开始招募NCT07530367
  Tofacitinib Plus Imatinib in Moderate-to-Severe Palmoplantar Pustulosis
  III 期 · 干预性 · 2026/08/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou

家族性地中海热 6 项在招目录 第1批 · 28

• 招募中NCT07077473
  Observing the Efficacy and Safety of Different Drugs Used in Real-world Familial Mediterranean Fever (FMF) Cases
  观察性 · 2018/01/01Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT05200715
  AutoInflammatory Disease Alliance Registry (AIDA)
  观察性 · 2020/08/06University of Siena
  中国研究中心 2 个：Beijing、Chongqing
• 招募中NCT05845723
  Tocilizumab and Tofacitinib in the Treatment of Vascular Behçet's Syndrome
  II 期 · 干预性 · 2023/06/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06145893
  A Study of Efficacy and Safety of Hemay005 Tablets in Patients With Behçet's Disease
  III 期 · 干预性 · 2023/11/13Ganzhou Hemay Pharmaceutical Co., Ltd
  中国研究中心 22 个：Beijing、Changchun、Guangzhou、Guilin、Hangzhou、Linyi 等 16 地
• 招募中NCT06849908
  Baricitinib in the Treatment of Intestinal Behçet's Syndrome
  II 期 · 干预性 · 2024/11/08Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06794008
  BCMA-CD19 CAR-T Therapy for Refractory Autoimmune Diseases
  II 期 · 干预性 · 2024/12/26Peking University People's Hospital
  中国研究中心 1 个：Beijing

视网膜母细胞瘤 6 项在招目录 第1批 · 103

• 招募中NCT05080010
  Adjuvant Chemotherapy for High-risk Postenucleation Retinoblastoma
  III 期 · 干预性 · 2020/11/01Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT06369181
  Neuroendocrine Transformation in RB1/TP53 Inactivated NSCLC
  观察性 · 2021/01/01Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT04799002
  Topotecan and Melphalan for Retinoblastoma
  III 期 · 干预性 · 2021/03/11Sun Yat-sen University
  中国研究中心 1 个：Guangzhou
• 招募中NCT04903678
  Intrathecal Chemotherapy for Central Nervous System Metastasis in Retinoblastoma
  不适用 · 干预性 · 2021/05/01Eye & ENT Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06424301
  Targeting NUDT21 siRNA Drugs for Patients With Refractory Retinoblastoma
  早期 I 期 · 干预性 · 2024/12/20Eye & ENT Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06972602
  High-dose Topotecan for Retinoblastoma With Recurrent or Refractory Vitreous Seed
  早期 I 期 · 干预性 · 2025/04/01Eye & ENT Hospital of Fudan University
  中国研究中心 1 个：Shanghai

发作性睡病 6 项在招目录 第2批 · 52

• 招募中NCT06279247
  Proteomics and Metabolomics of Body Fluid in Patients With Narcolepsy
  观察性 · 2022/09/01Qilu Hospital of Shandong University
  中国研究中心 1 个：Jinan
• 招募中NCT06797284
  The Impact of Light, Electrical, and Magnetic Neuroregulation Interventions on Sleep-wake Disorders
  不适用 · 干预性 · 2025/02/01Second Affiliated Hospital of Soochow University
  中国研究中心 1 个：Suzhou
• 招募中NCT06952699
  A Study of TAK-360 in Adults With Narcolepsy Without Cataplexy (NT2)
  II 期 · 干预性 · 2025/05/06Takeda
  中国研究中心 4 个：Beijing、Changchun、Guangzhou、Shanghai
• 招募中NCT07493265
  A Study to Evaluate the Efficacy and Safety of E2086 in Adults With Narcolepsy
  II 期 · 干预性 · 2026/03/26Eisai Inc.
  中国研究中心 8 个：Beijing、Changchun、Guangdong、Guangzhou、Jinan、Nanchang 等 8 地
• 招募中NCT07633301
  A Study of TAK-360 in Adults With Narcolepsy Type 1 (Narcolepsy With Cataplexy)
  II 期 · 干预性 · 2026/06/04Takeda
  中国研究中心 3 个：Beijing、Guangzhou、Zhengzhou
• 招募中NCT07727239
  Temporal Interference Stimulation of Hypothalamus in Patients With Narcolepsy Type 1
  不适用 · 干预性 · 2026/08/01Xijing Hospital
  中国研究中心 1 个：Xi'an

全身型幼年特发性关节炎 6 项在招目录 第2批 · 74

• 招募中NCT03773965
  A Study of Baricitinib in Participants From 1 Year to Less Than 18 Years Old With Juvenile Idiopathic Arthritis
  III 期 · 干预性 · 2019/04/05Eli Lilly and Company
  中国研究中心 4 个：Beijing、Chongqing、Nanjing、Suzhou
• 招募中NCT05200715
  AutoInflammatory Disease Alliance Registry (AIDA)
  观察性 · 2020/08/06University of Siena
  中国研究中心 2 个：Beijing、Chongqing
• 招募中NCT06417502
  Observational Study of Pediatric Rheumatic and Immunologic Diseases in China: The CAPRID Registry
  观察性 · 2022/04/29Peking Union Medical College Hospital
  中国研究中心 9 个：Beijing、Changsha、Chongqing、Hangzhou、Nanjing、Shenzhen
• 招募中NCT05609630
  Study of Oral Upadacitinib and Subcutaneous/Intravenous Tocilizumab to Evaluate Change in Disease Activity, Adverse Events and How Drug Moves Through the Body of Pediatric and Adolescent Participants With Active Systemic Juvenile Idiopathic Arthritis.
  III 期 · 干预性 · 2023/10/02AbbVie
  中国研究中心 5 个：Chongqing、Hangzhou、Shanghai、Suzhou、Xi'an
• 招募中NCT05925452
  To Evaluate the Efficacy and Safety of Genalumab for Injection in the Treatment of Active Systemic Juvenile Idiopathic Arthritis.
  不适用 · 干预性 · 2023/12/01Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 10 个：Beijing、Changsha、Chengdu、Chongqing、Hangzhou、Nanjing 等 10 地
• 招募中NCT07184450
  Clinical Study of BCMA/CD70-targeted CAR-T Therapy for Refractory Pediatric Rheumatic Diseases
  I 期 · 干预性 · 2025/09/01Chongqing Precision Biotech Co., Ltd
  中国研究中心 1 个：Hangzhou

戈谢病 5 项在招目录 第1批 · 31

• 招募中NCT00358943
  International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry
  观察性 · 1991/04/01Genzyme, a Sanofi Company
  中国研究中心 10 个：Beijing、Guangzhou、Hangzhou、Nanjing、Shanghai
• 招募中NCT06272149
  An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease
  早期 I 期 · 干预性 · 2023/03/01Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 招募中NCT06162338
  A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I
  不适用 · 干预性 · 2023/11/15He Huang
  中国研究中心 1 个：Hanzhou
• 招募中NCT06818838
  A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease
  I 期、II 期 · 干预性 · 2024/07/05Lingyi Biotech Co., Ltd.
  中国研究中心 3 个：Guangzhou、Taiyuan、Tianjin
• 招募中NCT04637503
  4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
  I 期、II 期 · 干预性 · 2026/06/01Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

糖原累积病（I型、Ⅱ型） 5 项在招目录 第1批 · 35

• 招募中NCT00231400
  Pompe Disease Registry Protocol
  观察性 · 2004/09/15Genzyme, a Sanofi Company
  中国研究中心 8 个：Beijing、Guangzhou、Jinan、Shanghai
• 招募中NCT07336394
  Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
  观察性 · 2010/01/01Chinese Academy of Medical Sciences, Fuwai Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06178432
  Evaluation of the Safety, Tolerability and Efficacy of Gene Therapy Drug for Late Onset Pompe Disease (LOPD)
  早期 I 期 · 干预性 · 2023/12Huashan Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06391736
  Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
  I 期、II 期 · 干预性 · 2024/04/19GeneCradle Inc
  中国研究中心 1 个：Beijing
• 招募中NCT06666413
  China Post-approval Commitment (PAC) Study of Avalglucosidase Alfa in Participants With IOPD
  IV 期 · 干预性 · 2025/05/07Genzyme, a Sanofi Company
  中国研究中心 1 个：Shanghai

纯合子家族性高胆固醇血症 5 项在招目录 第1批 · 46

• 招募中NCT06125847
  NGGT006 Gene Therapy for Homozygous Familial Hypercholesterolemia
  早期 I 期 · 干预性 · 2023/10/29First Affiliated Hospital Xi'an Jiaotong University
  中国研究中心 1 个：Xi'an
• 招募中NCT06597006
  Study to Evaluate Safety, Tolerability and Efficacy of Inclisiran in Children With Homozygous Familial Hypercholesterolemia
  III 期 · 干预性 · 2025/02/28Novartis Pharmaceuticals
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07133815
  Evaluate the Long-term Efficacy and Safety of SHR-1918 in Patients With Homozygous Familial Hypercholesterolemia
  II 期 · 干预性 · 2025/08Beijing Suncadia Pharmaceuticals Co., Ltd
  中国研究中心 1 个：Changsha
• 招募中NCT07489209
  A Dose-exploration Study of EDP167 in HoFH
  II 期 · 干预性 · 2026/02/06Eddingpharm (Zhuhai) Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT07591298
  Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants With Homozygous Familial Hypercholesterolemia
  II 期 · 干预性 · 2026/05/24CSPC Zhongnuo Pharmaceutical (Shijiazhuang) Co., Ltd.
  中国研究中心 1 个：Changsha

POEMS综合征 5 项在招目录 第1批 · 91

• 招募中NCT04561557
  Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System
  早期 I 期 · 干预性 · 2020/09/22Tongji Hospital
  中国研究中心 1 个：Wuhan
• 招募中NCT05283993
  A Cohort Study of Plasma Cell Disorders (PCDs) in PKUFH
  观察性 · 2021/07/01Peking University First Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT06518876
  A Study of KQ-2003 CAR-T Cell Therapy for Patients With Relapsed or Refractory POEMS Syndrome
  I 期 · 干预性 · 2024/08/31Novatim Immune Therapeutics (Zhejiang) Co., Ltd.
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07115654
  BCMA/CD3 BsAb Therapy for POEMS Syndrome
  II 期 · 干预性 · 2025/08/10Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT07652879
  KCD (Carfilzomib/Cyclophosphamide/Dexamethasone) Regimen for the Treatment of Newly Diagnosed POEMS Syndrome
  II 期 · 干预性 · 2026/06/15Shanghai Changzheng Hospital
  中国研究中心 1 个：Shanghai

先天性胆道闭锁 5 项在招目录 第2批 · 16

• 招募中NCT05072626
  High Medium-chain Triglyceride Nutritional Support in Infants With Biliary Atresia
  观察性 · 2021/10/11Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 仅邀请入组NCT05426733
  An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
  III 期 · 干预性 · 2022/07/05Albireo, an Ipsen Company
  中国研究中心 2 个：Guangdong、Shanghai
• 招募中NCT05925309
  Preventive Effect of Prophylactic Oral Antibiotics Against Cholangitis After Kasai Portoenterostomy
  不适用 · 干预性 · 2023/07/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT05848310
  Preoperative Serum FGF19 in the Prognosis of Biliary Atresia
  观察性 · 2023/10/15Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai
• 招募中NCT06764082
  Nutritional Intervention for Biliary Atresia
  不适用 · 干预性 · 2025/02/01Tongji Hospital
  中国研究中心 1 个：Wuhan

骨巨细胞瘤 5 项在招目录 第2批 · 36

• 招募中NCT05595603
  Zoledronic Acid-loaded Bone Cement as a Local Adjuvant Therapy for Giant Cell Bone Tumor After Intralesional Curettage
  II 期 · 干预性 · 2023/03Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT05813665
  A Study to Evaluate the Efficacy and Safety of Narlumosbart (JMT103) in Patients With Giant Cell Tumor of Bone
  III 期 · 干预性 · 2023/04Shanghai JMT-Bio Inc.
  中国研究中心 1 个：Beijing
• 招募中NCT06302465
  Narlumosbartmab Combined With Neoadjuvant Chemotherapy in Bone-derived Malignancies With Osteolytic Lesions and Multinucleated Giant Cells
  不适用 · 干预性 · 2024/05/01Peking University People's Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06647901
  Efficacy of ICG-based NIR Imaging in Intralesional Curettage of Giant Cell Tumors of Bone in Limbs: a Prospective, Single-center, Single-arm, Open Study
  I 期、II 期 · 干预性 · 2024/08/23Tang Xiaodong
  中国研究中心 1 个：Beijing
• 招募中NCT07315828
  Accuracy of Indocyanine Green (ICG) Fluorescence Imaging in Giant Cell Tumor of Bone Surgery
  IV 期 · 干预性 · 2026/01/02Shanghai Jiao Tong University Affiliated Sixth People's Hospital
  中国研究中心 1 个：Shanghai

原发性免疫缺陷 5 项在招目录 第2批 · 66

• 招募中NCT03217617
  SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
  I 期、II 期 · 干预性 · 2024/06/30Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 招募中NCT06679881
  Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
  III 期 · 干预性 · 2025/02/01Pharvaris Netherlands B.V.
  中国研究中心 1 个：Beijing
• 招募中NCT06960213
  STOP-HAE: A Phase 3 Study of ADX-324 in HAE
  III 期 · 干预性 · 2025/08/28ADARx Pharmaceuticals, Inc.
  中国研究中心 3 个：Beijing、Harbin、Zhengzhou
• 招募中NCT07371663
  An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
  I 期、II 期 · 干预性 · 2025/12/03Beijing Tide Pharmaceutical Co., Ltd
  中国研究中心 3 个：Beijing、Hangzhou、Zhengzhou
• 招募中NCT07428499
  Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
  III 期 · 干预性 · 2026/04/02ADARx Pharmaceuticals, Inc.
  中国研究中心 3 个：Beijing、Harbin、Zhengzhou

短肠综合征 5 项在招目录 第2批 · 73

• 招募中NCT07691125
  Phase IV Study of rhGH Injection in Adult Short Bowel Syndrome
  IV 期 · 干预性 · 2024/07/31Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 2 个：Nanjing、Xiamen
• 招募中NCT06512584
  Efficacy and Safety of PJ009 in Patients With Short Bowel Syndrome Requiring Parenteral Nutrition
  III 期 · 干预性 · 2024/09/03Chongqing Peg-Bio Biopharm Co., Ltd.
  中国研究中心 8 个：Beijing、Changsha、Chongqing、Guangzhou、Nanjing、Qingdao 等 8 地
• 招募中NCT06973304
  A Study of Teduglutide in Chinese Adults With Short Bowel Syndrome
  III 期 · 干预性 · 2025/06/26Takeda
  中国研究中心 4 个：Changping、Dongcheng、Nanjing、Siming
• 招募中NCT07319832
  A Study of Teduglutide in Chinese Children and Teenagers With Short Bowel Syndrome
  观察性 · 2026/03/05Takeda
  中国研究中心 4 个：Guangzhou、Hangzhou、Shanghai
• 招募中NCT07704047
  A Clinical Study of FT1 in Patients With Short Bowel Syndrome
  II 期 · 干预性 · 2026/08/30Chongqing Peg-Bio Biopharm Co., Ltd.
  中国研究中心 1 个：Nanjing

范可尼贫血 4 项在招目录 第1批 · 29

• 可获取（拓展性用药）NCT01995305
  Use Massive Parallel Sequencing and Exome Capture Technology to Sequence the Exome of Fanconi Anemia Children and Their Patents
  拓展性用药Xiaofan Zhu
  中国研究中心 1 个：Tianjin
• 招募中NCT06287541
  The Necessity of a Second Transurethral Resection in High-risk Non-muscle-invasive Bladder Cancer Patients With Negative Urine Biomarker After Initial Transurethral Resection
  不适用 · 干预性 · 2023/07/01The First Affiliated Hospital with Nanjing Medical University
  中国研究中心 1 个：Nanjing
• 招募中NCT07036731
  A Study Comparing the Necessity of a Second Transurethral Resection in High-Risk Non-Muscle-Invasive Bladder Cancer Patients With Negative Results From Post-Initial Resection Urine Genome-Wide Low-Depth Sequencing
  不适用 · 干预性 · 2025/02/01The First Affiliated Hospital with Nanjing Medical University
  中国研究中心 1 个：Nanjing
• 招募中NCT03351868
  FANCA Gene Transfer for Fanconi Anemia Using a High-safety, High-efficiency, Self-inactivating Lentiviral Vector
  不适用 · 干预性 · 2026/06/01Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

高苯丙氨酸血症 4 项在招目录 第1批 · 49

• 招募中NCT06687733
  Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria
  I 期、II 期 · 干预性 · 2024/07/16NGGT (Suzhou) Biotechnology Co., Ltd.
  中国研究中心 2 个：Bengbu、Shanghai
• 招募中NCT07672756
  A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
  I 期 · 干预性 · 2025/07/20Chongqing Peg-Bio Biopharm Co., Ltd.
  中国研究中心 1 个：Hefei
• 尚未开始招募NCT07318909
  To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
  早期 I 期 · 干预性 · 2025/12/31Gritgen Therapeutics Co., Ltd.
  中国研究中心 1 个：Hefei
• 招募中NCT07685210
  GenSci144 Tablets Phase I Clinical Trial
  I 期 · 干预性 · 2026/06/22Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Jinan

帕金森病（青年型、早发型） 4 项在招目录 第1批 · 87

• 招募中NCT03508960
  Chinese EOPD Registry
  观察性 · 2017/02/01Xiangya Hospital of Central South University
  中国研究中心 1 个：Changsha
• 招募中NCT06608355
  The Safety, Tolerability and Preliminary Efficacy of NouvNeu001 for Early-onset Parkinson's Disease
  I 期 · 干预性 · 2024/08/23iRegene Therapeutics Co., Ltd.
  中国研究中心 1 个：Wuhan
• 招募中NCT07166757
  Clinical Study to Evaluate XS411 in Treatment of Early-onset Parkinson's Disease
  I 期、II 期 · 干预性 · 2025/09/22XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT07741045
  Phase Ⅲ Clinical Trial to Evaluate the Efficacy and Safety of TJ0113 Capsules in Patients With Early-Onset Parkinson's Disease
  III 期 · 干预性 · 2026/08/03Hangzhou PhecdaMed Co., Ltd.
  中国研究中心 21 个：Beijing、Changsha、Chengdu、Chongqing、Guangzhou、Hangzhou 等 15 地

苯丙酮尿症 4 项在招目录 第1批 · 90

• 招募中NCT06687733
  Safety and Efficacy Study of NGGT002 in Adult Patients With Phenylketonuria
  I 期、II 期 · 干预性 · 2024/07/16NGGT (Suzhou) Biotechnology Co., Ltd.
  中国研究中心 2 个：Bengbu、Shanghai
• 招募中NCT07672756
  A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
  I 期 · 干预性 · 2025/07/20Chongqing Peg-Bio Biopharm Co., Ltd.
  中国研究中心 1 个：Hefei
• 尚未开始招募NCT07318909
  To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria
  早期 I 期 · 干预性 · 2025/12/31Gritgen Therapeutics Co., Ltd.
  中国研究中心 1 个：Hefei
• 招募中NCT07685210
  GenSci144 Tablets Phase I Clinical Trial
  I 期 · 干预性 · 2026/06/22Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Jinan

婴儿严重肌阵挛性癫痫(Dravet综合征) 4 项在招目录 第1批 · 105

• 招募中NCT06660394
  A Phase 3, Placebo-Controlled Study to Investigate LP352 in Children and Adults With Dravet Syndrome (DS)
  III 期 · 干预性 · 2024/09/25Longboard Pharmaceuticals
  中国研究中心 6 个：Beijing、Guangzhou、Shanghai、Shenzhen Shi、Wuhan
• 仅邀请入组NCT06908226
  A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
  III 期 · 干预性 · 2025/02/12Longboard Pharmaceuticals
  中国研究中心 5 个：Beijing、Guangzhou、Shanghai、Wuhan
• 招募中NCT06872125
  A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome
  III 期 · 干预性 · 2025/06/04Stoke Therapeutics, Inc
  中国研究中心 3 个：Beijing、Guangzhou、Wuhan
• 招募中NCT07675746
  A Study to Evaluate the Safety and Pharmacokinetics of RC001 in Children With Dravet Syndrome
  早期 I 期 · 干预性 · 2025/12/22Second Affiliated Hospital of Guangzhou Medical University
  中国研究中心 1 个：Guangzhou

镰刀型细胞贫血病 4 项在招目录 第1批 · 106

• 招募中NCT06399107
  Investigation Into the Use of BAH243 Lentiviral Vector for Gene Therapy in Treating Sickle Cell Disease
  I 期、II 期 · 干预性 · 2024/08/01Essen Biotech
  中国研究中心 1 个：Beijing
• 招募中NCT06565026
  CS-206 in Patients With Sickle Cell Disease
  早期 I 期 · 干预性 · 2024/09/02CorrectSequence Therapeutics Co., Ltd
  中国研究中心 1 个：Nanning
• 尚未开始招募NCT07190001
  YOLT-204 in Patients With Hemoglobinopathies
  早期 I 期 · 干预性 · 2025/09/30Guangzhou Women and Children's Medical Center
  中国研究中心 1 个：Guangzhou
• 尚未开始招募NCT07000318
  CS-206 in Patients With Sickle Cell Disease
  早期 I 期 · 干预性 · 2026/09/30Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai

胃肠胰神经内分泌肿瘤 4 项在招目录 第2批 · 31

• 招募中NCT05884255
  An Open-label Phase 3 Study of Lutetium (177Lu) Oxodotreotide Injection in Subjects With Advanced Gastrointestinal Pancreatic Neuroendocrine Tumors.
  III 期 · 干预性 · 2023/07/06Jiangsu HengRui Medicine Co., Ltd.
  中国研究中心 2 个：Shanghai、Tianjin
• 招募中NCT06840821
  Evaluating the Safety, Tolerability, and Preliminary Anti-tumor Activity of MB0151 in Adult Subjects With Advanced Solid Tumors Expressing Somatostatin Receptors
  I 期、II 期 · 干预性 · 2025/05/08Mainline Biosciences (Shanghai) Co., Ltd
  中国研究中心 1 个：Jinan
• 招募中NCT06784752
  Study to Evaluate the Efficacy and Safety of [177Lu]Lu-DOTA-TATE in Patients With Grade 1 and Grade 2 Advanced GEP-NET
  III 期 · 干预性 · 2025/05/30Novartis Pharmaceuticals
  中国研究中心 2 个：Beijing、Shanghai
• 招募中NCT07165886
  Sirolimus for Injection (Albumin Bound) Combined With Octreotide Long-acting Injection in Patients With Metastatic Gastroenteropancreatic Neuroendocrine Tumors
  II 期、III 期 · 干预性 · 2025/08/29CSPC ZhongQi Pharmaceutical Technology Co., Ltd.
  中国研究中心 1 个：Beijing

神经纤维瘤病 4 项在招目录 第2批 · 54

• 招募中NCT04085159
  Immunotherapy Based on Antigen-specific Immune Effector Cells Targeting Neurofibromatosis or Schwannomatosis
  I 期、II 期 · 干预性 · 2026/06/01Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 尚未开始招募NCT07707947
  Selumetinib for NF2-Related Schwannomatosis
  II 期 · 干预性 · 2026/07/15Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07713745
  Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2)
  II 期 · 干预性 · 2026/07/18Beijing Tiantan Hospital
  中国研究中心 5 个：Beijing、Changchun、Shanghai
• 尚未开始招募NCT07708285
  Luvometinib in Combination With Serplulimab for NF2-Related Tumors
  I 期、II 期 · 干预性 · 2026/07/31Beijing Tiantan Hospital
  中国研究中心 5 个：Beijing、Changchun、Shanghai

Alport 综合征 3 项在招目录 第1批 · 3

• 招募中NCT04947813
  Genotype-Phenotype Correlations in Patients With Alport Syndrome
  观察性 · 2021/01/01Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT05133050
  Safety and Efficacy of ACEI in Alport Syndrome Patients With COL4A3/COL4A4/COL4A5 Variants
  不适用 · 干预性 · 2022/01/01Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
  中国研究中心 1 个：Shanghai
• 招募中NCT07211685
  A Study to Learn About How Well BAY 3401016 Works in Adults With Alport Syndrome
  II 期 · 干预性 · 2025/11/19Bayer
  中国研究中心 5 个：Beijing、Chongqing、Guangzhou、Hangzhou、Wuhan

遗传性血管性水肿 3 项在招目录 第1批 · 38

• 招募中NCT06679881
  Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
  III 期 · 干预性 · 2025/02/01Pharvaris Netherlands B.V.
  中国研究中心 1 个：Beijing
• 招募中NCT06960213
  STOP-HAE: A Phase 3 Study of ADX-324 in HAE
  III 期 · 干预性 · 2025/08/28ADARx Pharmaceuticals, Inc.
  中国研究中心 3 个：Beijing、Harbin、Zhengzhou
• 招募中NCT07428499
  Phase 3 Extension Study of ADX-324 in Participants With Hereditary Angioedema (HAE)
  III 期 · 干预性 · 2026/04/02ADARx Pharmaceuticals, Inc.
  中国研究中心 3 个：Beijing、Harbin、Zhengzhou

遗传性痉挛性截瘫 3 项在招目录 第1批 · 43

• 招募中NCT06478238
  Calcium Folinate Treatment of Spastic Paraplegia 56
  早期 I 期 · 干预性 · 2024/07/01Shanghai 6th People's Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06844734
  A Prospective Cohort Study of ITB Treatment for HSP
  观察性 · 2025/01/01Shanghai 6th People's Hospital
  中国研究中心 1 个：Shanghai
• 招募中NCT06936163
  A Prospective Cohort Study of Surgical Treatment for Foot Deformities in HSP
  观察性 · 2025/03/01Shanghai 6th People's Hospital
  中国研究中心 1 个：Shanghai

亨廷顿舞蹈病 3 项在招目录 第1批 · 47

• 招募中NCT07326709
  A Study to Investigate the Efficacy, Safety and Tolerability of Votoplam in Participants With Huntington's Disease
  III 期 · 干预性 · 2026/03/24Novartis Pharmaceuticals
  中国研究中心 4 个：Changsha、Chengdu、Hangzhou、Wuhan
• 招募中NCT07513844
  Optimizing Parameters of Low-Intensity Focused Ultrasound for Pallidal Modulation in Huntington's Disease
  不适用 · 干预性 · 2026/04/20First Affiliated Hospital of Fujian Medical University
  中国研究中心 1 个：Fuzhou
• 尚未开始招募NCT07516899
  Mass Balance Study of [14C] LPM3770164 in Healthy Participants
  I 期 · 干预性 · 2026/04/30Luye Pharma Group Ltd.
  中国研究中心 1 个：Wuxi

原发性联合免疫缺陷 3 项在招目录 第1批 · 94

• 招募中NCT03645460
  Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
  不适用 · 干预性 · 2024/06/30Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 招募中NCT03217617
  SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
  I 期、II 期 · 干预性 · 2024/06/30Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 招募中NCT07371663
  An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
  I 期、II 期 · 干预性 · 2025/12/03Beijing Tide Pharmaceutical Co., Ltd
  中国研究中心 3 个：Beijing、Hangzhou、Zhengzhou

脊髓小脑性共济失调 3 项在招目录 第1批 · 111

• 招募中NCT07092358
  Hereditary Ataxia Research on Multi-Omics and Neuroclinical Insights in the Yangtze Delta
  观察性 · 2025/06/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 2 个：Hangzhou、Shanghai
• 招募中NCT07371663
  An Phase Ib/II Clinical Trial of TCC1727 Combination Therapy in Advanced Solid Tumors
  I 期、II 期 · 干预性 · 2025/12/03Beijing Tide Pharmaceutical Co., Ltd
  中国研究中心 3 个：Beijing、Hangzhou、Zhengzhou
• 尚未开始招募NCT07743866
  Optimizing Parameters of Transcranial Temporal Interference Stimulation for Spinocerebellar Ataxia Type 3
  不适用 · 干预性 · 2026/08/07First Affiliated Hospital of Fujian Medical University
  中国研究中心 1 个：Fuzhou

X-连锁肾上腺脑白质营养不良 3 项在招目录 第1批 · 120

• 招募中NCT05939232
  Registry of X-linked Adrenoleukodystrophy
  观察性 · 2023/07/20Beijing Tiantan Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06796920
  Epidural Spinal Cord Stimulation for Lower-limb Impairment in Adrenomyeloneuropathy
  不适用 · 干预性 · 2025/02/10Third Military Medical University
  中国研究中心 4 个：Beijing、Chongqing、Jingmen
• 招募中NCT03727555
  IT and IV Lentiviral Gene Therapy for X-ALD
  不适用 · 干预性 · 2025/08/31Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

上皮样肉瘤 3 项在招目录 第2批 · 24

• 招募中NCT04390737
  Evaluate the Safety and Clinical Activity of HH2853
  I 期、II 期 · 干预性 · 2020/09/08Haihe Biopharma Co., Ltd.
  中国研究中心 18 个：Beijing、Changsha、Chengdu、Guangzhou、Hangzhou、Hefei 等 15 地
• 招募中NCT06277154
  MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma
  II 期 · 干预性 · 2024/02/21HRYZ Biotech Co.
  中国研究中心 1 个：Guangzhou
• 招募中NCT06683846
  Ivonescimab in the Treatment of Multiple Advanced Tumors
  II 期 · 干预性 · 2024/11/20Fudan University
  中国研究中心 1 个：Shanghai

腱鞘巨细胞瘤/色素沉着绒毛结节性滑膜炎 3 项在招目录 第2批 · 77

• 招募中NCT04192344
  A Study to Assess the Safety, Tolerability, and Pharmacokinetics of ABSK-021 in Patients With Advanced Solid Tumor
  I 期 · 干预性 · 2020/01/20Abbisko Therapeutics Co, Ltd
  中国研究中心 13 个：Beijing、Chengdu、Guangdong、Hangzhou、Nanjing、Shanghai 等 10 地
• 招募中NCT07315841
  Accuracy of Indocyanine Green (ICG) Fluorescence Imaging in Tenosynovial Giant Cell Tumor Surgery
  IV 期 · 干预性 · 2026/01/02Shanghai Jiao Tong University Affiliated Sixth People's Hospital
  中国研究中心 1 个：Shanghai
• 尚未开始招募NCT07707882
  Neoadjuvant Pimicotinib Combined With Surgery Versus Upfront Surgery for Diffuse Tenosynovial Giant Cell Tumor
  II 期 · 干预性 · 2027/10/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou

Von Hippel-Lindau综合征 3 项在招目录 第2批 · 83

• 招募中NCT04924075
  Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
  II 期 · 干预性 · 2021/08/12Merck Sharp & Dohme LLC
  中国研究中心 4 个：Beijing、Chengdu、Guangzhou、Shanghai
• 招募中NCT06553339
  A Study of HS-10516 in Patients With VHL Syndrome Associated Tumors
  I 期 · 干预性 · 2024/07/19Jiangsu Hansoh Pharmaceutical Co., Ltd.
  中国研究中心 1 个：Beijing
• 招募中NCT07557225
  18F-T2 PET/CT Imaging for CAIX Positive Solid Tumors
  不适用 · 干预性 · 2026/04/27Peking University First Hospital
  中国研究中心 1 个：Beijing

非典型溶血性尿毒症 2 项在招目录 第1批 · 8

• 招募中NCT05935215
  Efficacy and Safety of Switching From Anti-C5 Antibody Treatment to Iptacopan Treatment in Study Participants With Atypical Hemolytic Uremic Syndrome (aHUS)
  III 期 · 干预性 · 2024/02/28Novartis Pharmaceuticals
  中国研究中心 3 个：Beijing、Nanjing、Shanghai
• 招募中NCT05795140
  Evaluate Long-term Safety, Tolerability and Efficacy of Iptacopan in Study Participants With aHUS
  III 期 · 干预性 · 2024/05/08Novartis Pharmaceuticals
  中国研究中心 1 个：Beijing

Castleman病 2 项在招目录 第1批 · 16

• 招募中NCT05200715
  AutoInflammatory Disease Alliance Registry (AIDA)
  观察性 · 2020/08/06University of Siena
  中国研究中心 2 个：Beijing、Chongqing
• 招募中NCT05283993
  A Cohort Study of Plasma Cell Disorders (PCDs) in PKUFH
  观察性 · 2021/07/01Peking University First Hospital
  中国研究中心 1 个：Beijing

腓骨肌萎缩症 2 项在招目录 第1批 · 17

• 招募中NCT04010188
  A Registered Cohort Study on Charcot-Marie-Tooth Disease
  观察性 · 2019/07/01Ning Wang, MD., PhD.
  中国研究中心 1 个：Fuzhou
• 招募中NCT07625020
  Ultrasound-Guided Nerve Hydrodissection for Diabetic Lower Limb Entrapment Neuropathy
  不适用 · 干预性 · 2026/05/20Chongqing Medical University
  中国研究中心 1 个：Chongqing

遗传性多发脑梗死性痴呆 2 项在招目录 第1批 · 42

• 招募中NCT04310098
  CADASIL Registry Study
  观察性 · 2020/03/09Bin Cai
  中国研究中心 1 个：Fuzhou
• 尚未开始招募NCT07692399
  Safety and Efficacy of Edaravone Dexborneol Sublingual Tablets for Blood-Brain Barrier Dysfunction in CADASIL
  II 期 · 干预性 · 2026/07Huashan Hospital
  中国研究中心 1 个：Shanghai

特发性低促性腺激素性性腺功能减退症 2 项在招目录 第1批 · 53

• 招募中NCT05569577
  An Open Labeled RCT on the Effect of Additional hCG Injection for LPS on Pregnancy Outcomes in IHH Patients
  不适用 · 干预性 · 2021/01/01Fudan University
  中国研究中心 2 个：Shanghai
• 尚未开始招募NCT06561594
  To Evaluate Recombinant Human Follicle Stimulating Hormone-CTP Fusion Protein Injection or Placebo Combined With Chorionic Gonadotropin for Injection
  III 期 · 干预性 · 2024/09/20Changchun GeneScience Pharmaceutical Co., Ltd.
  中国研究中心 17 个：Beijing、Chengdu、Guangzhou、Hefei、Jining、Nanchang 等 12 地

肌强直性营养不良 2 项在招目录 第1批 · 78

• 仅邀请入组NCT06979024
  A Registered Observational Cohort Study of Myotonic Dystrophy Type 1
  观察性 · 2008/01First Affiliated Hospital of Fujian Medical University
  中国研究中心 1 个：Fuzhou
• 仅邀请入组NCT06101940
  Chinese Multicenter Clinical Outcome Cohort Study of Myotonic Dystrophy Type 1 (C-DMCOS-DM1)
  观察性 · 2021/08/01Huashan Hospital
  中国研究中心 22 个：Beijing、Changchun、Chengdu、Chifeng、Chongqing、Fuzhou 等 18 地

新生儿糖尿病 2 项在招目录 第1批 · 80

• 招募中NCT07492004
  China Monogenic Diabetes Registry
  观察性 · 2021/02/28Tianjin Medical University General Hospital
  中国研究中心 1 个：Tianjin
• 尚未开始招募NCT07314502
  Prenatal Exercise Including Perineal Massage and Maternal-Neonatal Outcomes
  不适用 · 干预性 · 2026/01/01South China Normal University
  中国研究中心 1 个：Guangzhou

软骨发育不全 2 项在招目录 第2批 · 1

• 招募中NCT07301463
  A Study in Children With Achondroplasia
  观察性 · 2025/06/20Abbisko Therapeutics Co, Ltd
  中国研究中心 8 个：Beijing、Chengdu、Guangzhou、Hangzhou、Shanghai、Wuhan 等 7 地
• 尚未开始招募NCT07297875
  A Study of ABSK061 to Assess Safety, Tolerability, Pharmacokinetics, and Efficacy in Children With Achondroplasia
  I 期、II 期 · 干预性 · 2025/12/10Abbisko Therapeutics Co, Ltd
  中国研究中心 7 个：Beijing、Chengde、Chengdu、Hangzhou、Shanghai、Wuhan 等 7 地

获得性血友病 2 项在招目录 第2批 · 2

• 招募中NCT05849740
  An Exploratory Study of Cluster of Differentiation 38 (CD38) Monoclonal Antibody Combined Corticosteroid in Acquired Hemophilia A
  IV 期 · 干预性 · 2023/05/22Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 招募中NCT06727669
  Longitudinal Cohort of Thrombosis and Hemostasis Diseases
  观察性 · 2024/11/01Peking University People's Hospital
  中国研究中心 5 个：Beijing、Chongqing、Zhengzhou

冷凝集素病 2 项在招目录 第2批 · 15

• 招募中NCT07019038
  National Longitudinal Cohort of Hematological Diseases- Autoimmune Hemolytic Anemia
  观察性 · 2001/01/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin
• 尚未开始招募NCT06847607
  A Multicenter Prospective Study of Iptacopan in the Treatment of Refractory/Relapsed Autoimmune Hemolytic Anemia (AIHA)
  不适用 · 干预性 · 2025/03/01Bing Han
  中国研究中心 1 个：Beijing

先天性凝血因子VII缺乏症 2 项在招目录 第2批 · 17

• 招募中NCT07644832
  An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
  I 期、II 期 · 干预性 · 2024/05/31Shanghai RAAS Blood Products Co., Ltd.
  中国研究中心 9 个：Changsha、Hefei、Jinan、Lanzhou、Shanghai、Taiyuan 等 9 地
• 尚未开始招募NCT07711158
  A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
  II 期 · 干预性 · 2026/07Shanghai RAAS Blood Products Co., Ltd.
  中国研究中心 1 个：Tianjin

家族性腺瘤性息肉病 2 项在招目录 第2批 · 27

• 尚未开始招募NCT04531930
  Colorectal Adenoma Canceration in FAP
  观察性 · 2020/10/01Changhai Hospital
  中国研究中心 1 个：Shanghai
• 仅邀请入组NCT06649825
  Efficacy of Vitamin B1 in Familial Adenomatous Polyposis Patients.
  不适用 · 干预性 · 2024/01/08Shanghai Zhongshan Hospital
  中国研究中心 1 个：Shanghai

巨细胞动脉炎 2 项在招目录 第2批 · 35

• 招募中NCT06172361
  Induction and Tapering Therapy With Tofacitinib and Glucocorticoid in Patients With Polymyalgia Rheumatica
  III 期 · 干预性 · 2024/01/20Zhejiang University
  中国研究中心 1 个：Hangzhou
• 尚未开始招募NCT06766552
  A Registry Study Assessing PRO, Dosing Patterns, and Safety of Vunakizumab in Patients With General Rheumatic Diseases.
  观察性 · 2025/01/30Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou

异染性脑白质营养不良 2 项在招目录 第2批 · 49

• 招募中NCT03725670
  Direct Lentiviral Injection Gene Therapy for MLD
  不适用 · 干预性 · 2025/05/31Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 招募中NCT07046338
  Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
  不适用 · 干预性 · 2025/06/01Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

West综合征/婴儿痉挛综合征 2 项在招目录 第2批 · 86

• 招募中NCT06528197
  Construction of Clinical Model of Primary Sjogren's Syndrome Combined Traditional Chinese and Western Medicine
  观察性 · 2024/07/04China-Japan Friendship Hospital
  中国研究中心 1 个：Beijing
• 仅邀请入组NCT06519617
  Study on Optimization and Evaluation of Integrated Chinese and Western Medicine for pSS Glandular Damage
  II 期 · 干预性 · 2024/07/13Qingwen Tao
  中国研究中心 1 个：Beijing

先天性脊柱侧弯 1 项在招目录 第1批 · 23

• 招募中NCT06315933
  Anesthesia Neurodevelopmental Impact in Congenital Scoliosis Children
  观察性 · 2020/07/13Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing

Gitelman 综合征 1 项在招目录 第1批 · 33

• 招募中NCT06922370
  The Osteoarticular Manifestations in Patients With Gitelman Syndrome
  观察性 · 2021/11/01Second Affiliated Hospital, Zhejiang University, School of Medicine
  中国研究中心 1 个：Hangzhou

戊二酸血症I型 1 项在招目录 第1批 · 34

• 招募中NCT06217861
  A Study to Evaluate the Tolerability, Safety and Efficacy of VGM-R02b
  I 期 · 干预性 · 2024/04/29Shanghai Vitalgen BioPharma Co., Ltd.
  中国研究中心 1 个：Hangzhou

特发性心肌病 1 项在招目录 第1批 · 52

• 招募中NCT07336394
  Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
  观察性 · 2010/01/01Chinese Academy of Medical Sciences, Fuwai Hospital
  中国研究中心 1 个：Beijing

Leber遗传性视神经病变 1 项在招目录 第1批 · 62

• 招募中NCT04912843
  Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
  II 期、III 期 · 干预性 · 2021/06/18Wuhan Neurophth Biotechnology Limited Company
  中国研究中心 1 个：Beijing

淋巴管肌瘤病 1 项在招目录 第1批 · 64

• 招募中NCT03193892
  A National Registry on Chinese Patients With Lymphangioleiomyomatosis
  观察性 · 2017/01/01Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing

马凡综合征 1 项在招目录 第1批 · 68

• 招募中NCT07672210
  PregnAncy-Related Aortic DISsEction in China
  观察性 · 2026/03/17DeltaHealth Hospital · Shanghai
  中国研究中心 4 个：Beijing、Guangzhou、Shanghai、Wuhan

黏多糖贮积症 1 项在招目录 第1批 · 73

• 招募中NCT06519552
  A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
  I 期 · 干预性 · 2024/07/22West China Hospital
  中国研究中心 1 个：Chengdu

Noonan综合征 1 项在招目录 第1批 · 84

• 招募中NCT07336394
  Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
  观察性 · 2010/01/01Chinese Academy of Medical Sciences, Fuwai Hospital
  中国研究中心 1 个：Beijing

鸟氨酸氨甲酰基转移酶缺乏症 1 项在招目录 第1批 · 85

• 招募中NCT04573660
  Abbott Vascular Medical Device Registry
  观察性 · 2020/10/25Abbott Medical Devices
  中国研究中心 3 个：Jinhua、Ningbo、Xi'an

成骨不全症（脆骨病） 1 项在招目录 第1批 · 86

• 招募中NCT07557446
  A Dose REgimen-Finding Study of AGA2115 in Chinese Patients With Osteogenesis ImpeRfecta (EIR)
  II 期 · 干预性 · 2026/06Angitia Biopharmaceuticals Guangzhou Limited
  中国研究中心 4 个：Beijing、Shanghai、Shenzhen、Suzhou

黑斑息肉综合征 1 项在招目录 第1批 · 89

• 招募中NCT06722534
  Celecoxib for Prevention of Progression in Peutz-Jeghers Syndrome
  不适用 · 干预性 · 2025/02/01Air Force Military Medical University, China
  中国研究中心 1 个：Xi'an

Prader-Willi综合征 1 项在招目录 第1批 · 93

• 招募中NCT05791604
  The Intervention of Obesity in Children With Prader-Willi Syndrome Using Prebiotics and Probiotics
  不适用 · 干预性 · 2023/04/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai

进行性家族性肝内胆汁淤积症 1 项在招目录 第1批 · 97

• 招募中NCT07588880
  A Study of the Effectiveness, Safety and the Long-term Outcomes of Participants With Progressive Familial Intrahepatic Cholestasis (PFIC) Who Take Odevixibat (Bylvay) in China
  观察性 · 2026/04/16Ipsen
  中国研究中心 5 个：Beijing、Hunan、Nanjing、Shanghai、Xiamen

威廉姆斯综合征 1 项在招目录 第1批 · 117

• 尚未开始招募NCT07509879
  Research on the Molecular Mechanism of Cognitive Differences Between Williams Syndrome and Autism Spectrum Disorder
  观察性 · 2026/04/01Qilu Hospital of Shandong University
  中国研究中心 1 个：Jinan

X-连锁淋巴增生症 1 项在招目录 第1批 · 121

• 招募中NCT04645199
  National Longitudinal Cohort of Hematological Diseases
  观察性 · 2020/12/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin

肢端肥大症 1 项在招目录 第2批 · 3

• 招募中NCT03158090
  The Longitudinal Approach to Acromegaly: A Pattern of Treatment and Comparative Effectiveness Research
  观察性 · 2017/12/15Ginkgo Leaf Center for Rare Disorders
  中国研究中心 1 个：Guangzhou

成人斯蒂尔病 1 项在招目录 第2批 · 4

• 招募中NCT07191444
  A Study of Firsekibart Versus Anakinra in Adult-Onset Still's Disease
  观察性 · 2025/09/24Ruijin Hospital
  中国研究中心 1 个：Shanghai

肾透明细胞肉瘤 1 项在招目录 第2批 · 14

• 招募中NCT06683846
  Ivonescimab in the Treatment of Multiple Advanced Tumors
  II 期 · 干预性 · 2024/11/20Fudan University
  中国研究中心 1 个：Shanghai

嗜酸性粒细胞性胃肠炎 1 项在招目录 第2批 · 23

• 招募中NCT07257835
  Dupilumab Therapy for EGIDs
  观察性 · 2025/01/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai

面肩肱型肌营养不良症 1 项在招目录 第2批 · 25

• 招募中NCT04369209
  A Registered Cohort Study on FSHD1
  观察性 · 2001/01Ning Wang, MD., PhD.
  中国研究中心 1 个：Fuzhou

进行性骨化性纤维发育不良 1 项在招目录 第2批 · 28

• 招募中NCT05090891
  To Assess the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva
  II 期 · 干预性 · 2022/05/05Incyte Corporation
  中国研究中心 4 个：Beijing、Shanghai

脆性X综合征 1 项在招目录 第2批 · 29

• 招募中NCT05120505
  Metformin in Children With Fragile X Syndrome
  IV 期 · 干预性 · 2021/12/29Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai

Leber先天性黑矇 1 项在招目录 第2批 · 43

• 招募中NCT05906953
  Safety and Efficacy Trial of HG004 for Leber Congenital Amaurosis Related to Rpe65 Gene Mutations (STAR)
  I 期、II 期 · 干预性 · 2023/10/31HuidaGene Therapeutics Co., Ltd.
  中国研究中心 1 个：Shanghai

Lennox-Gastaut 综合征 1 项在招目录 第2批 · 44

• 仅邀请入组NCT06908226
  A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
  III 期 · 干预性 · 2025/02/12Longboard Pharmaceuticals
  中国研究中心 5 个：Beijing、Guangzhou、Shanghai、Wuhan

恶性高热 1 项在招目录 第2批 · 46

• 招募中NCT05402839
  Screening of Malignant Hyperthermia Susceptible Individuals
  观察性 · 2022/02/22Peking University Third Hospital
  中国研究中心 1 个：Beijing

神经营养性角膜炎 1 项在招目录 第2批 · 56

• 招募中NCT06975748
  A Phase II Study of STSP-0902 Ophthalmic Solution in Patients With Neurotrophic Keratitis
  II 期 · 干预性 · 2025/06/12Staidson (Beijing) Biopharmaceuticals Co., Ltd
  中国研究中心 1 个：Jinan

天疱疮 1 项在招目录 第2批 · 58

• 招募中NCT07681388
  CD19 CAR-T Therapy for Refractory Pemphigus Vulgaris
  不适用 · 干预性 · 2026/01/15Jinbo Chen
  中国研究中心 1 个：Wuhan

新生儿持续肺动脉高压 1 项在招目录 第2批 · 59

• 招募中NCT06832163
  Close Loop Smart Weaning for INO With PPHN
  不适用 · 干预性 · 2025/10/01Children's Hospital of Fudan University
  中国研究中心 1 个：Shanghai

原发性硬化性胆管炎 1 项在招目录 第2批 · 68

• 尚未开始招募NCT07678645
  The Safety and Efficacy of Upadacitinib in Refractory Autoimmune Related Cholangitis and Atopic Dermatitis With Moderate to Severe Itching
  II 期 · 干预性 · 2026/06/01RenJi Hospital
  中国研究中心 1 个：Shanghai

进行性纤维化性间质性肺疾病 1 项在招目录 第2批 · 69

• 招募中NCT05139719
  A Study to Explore the Therapeutic Effect of HEC585 on Delaying Forced Vital Capacity (FVC) Decline and Tolerance in Progressive Fibrosing Interstitial Lung Disease (PF-ILD) Patients
  II 期 · 干预性 · 2023/02/15Sunshine Lake Pharma Co., Ltd.
  中国研究中心 1 个：Beijing

早产儿视网膜病 1 项在招目录 第2批 · 71

• 招募中NCT06717412
  Efficacy and Safety of Low-dose Conbercept for Retinopathy of Prematurity Therapy
  不适用 · 干预性 · 2024/11/29Wang Yusheng
  中国研究中心 3 个：Xi'an、Xianyang

Rett综合征 1 项在招目录 第2批 · 72

• 仅邀请入组NCT06739434
  GCB-002 in Treatment of Patients With Rett Syndrome
  不适用 · 干预性 · 2024/11/11Genecombio Ltd.
  中国研究中心 1 个：Shanghai

系统性肥大细胞增多症 1 项在招目录 第2批 · 75

• 招募中NCT07562542
  Peripheral Blood KIT-D816V Mutation in Adult Systemic Mastocytosis
  观察性 · 2025/05/19The First Affiliated Hospital of Soochow University
  中国研究中心 1 个：Suzhou

肿瘤相关骨软化症 1 项在招目录 第2批 · 82

• 招募中NCT07366099
  Al18F-NOTA-LM3 PET/CT in Patients With TIO
  不适用 · 干预性 · 2023/03/30Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing