在中国招募中的罕见病临床试验

黑色素瘤 66 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
• 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NCT04913285
  A Study to Evaluate KIN-2787 in Participants With BRAF and/or NRAS Mutation Positive Solid Tumors
  I 期 · 干预性 · 2021/08/04Pierre Fabre Medicament
  中国研究中心 4 个：Beijing、Haerbin、Linyi、Shanghai
• 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
• 招募中NCT05512481
  Camrelizumab Plus Apatinib and Temozolomide as Neoadjuvant in High Risk Acral Melanoma
  II 期 · 干预性 · 2022/09/13Peking University Cancer Hospital & Institute
  中国研究中心 1 个：Beijing
• 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 54 项未列出，见该病详情页。

系统性硬化症 65 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
• 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
• 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
• 招募中NCT06470048
  A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis
  II 期 · 干预性 · 2024/10/09Novartis Pharmaceuticals
  中国研究中心 7 个：Beijing、Changchun、Chengdu、Nanning、Ningbo、Tianjin 等 7 地

另有 53 项未列出，见该病详情页。

血栓性血小板减少性紫癜 64 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
• 招募中NCT06478537
  TPO-RA Treatment on Immune Tolerance Induction of ITP Patients With Sustained Platelet Recovery After Treatment Termination
  II 期 · 干预性 · 2024/06/20Peking Union Medical College Hospital
  中国研究中心 1 个：Beijing
• 招募中NCT06838962
  Anti-CD38 Antibody Treating Children With Primary Immune Thrombocytopenia (ITP)
  II 期 · 干预性 · 2024/08/01Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin

另有 52 项未列出，见该病详情页。

胶质母细胞瘤 56 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
• 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
• 招募中NCT07145047
  Clinical Study of Oncolytic Virus in Glioblastoma
  I 期、II 期 · 干预性 · 2023/11/02Mianyang Central Hospital
  中国研究中心 1 个：Mianyang

另有 44 项未列出，见该病详情页。

多发性内分泌腺瘤病 49 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
• 招募中NCT06249308
  Liquid Biopsy-based Early Detection of Ovarian Cancer: a Proof-of-concept Study ( PROFOUND-OC )
  观察性 · 2023/12/18Fudan University
  中国研究中心 3 个：Guangzhou、Shanghai、Shenyang

另有 37 项未列出，见该病详情页。

全身型重症肌无力 46 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 34 项未列出，见该病详情页。

ANCA相关性血管炎 40 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
• 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
• 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
• 招募中NCT06775912
  Clinical Study on Targeted CD19/BCMA CAR-T Therapy for Autoimmune Diseases
  早期 I 期 · 干预性 · 2025/01/03Nanjing Bioheng Biotech Co., Ltd.
  中国研究中心 1 个：Lianyungang

另有 28 项未列出，见该病详情页。

多发性硬化 39 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
• 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
• 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 27 项未列出，见该病详情页。

骨肉瘤 36 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 24 项未列出，见该病详情页。

视神经脊髓炎 35 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 23 项未列出，见该病详情页。

血友病 30 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
• 招募中NCT06111638
  Evaluation of the Safety and Efficacy of Hemophilia A Gene Therapy Drugs
  II 期、III 期 · 干预性 · 2024/01/03Shanghai Xinzhi BioMed Co., Ltd.
  中国研究中心 10 个：Beijing、Changsha、Chengde、Guangzhou、Guiyang、Kunming 等 10 地

另有 18 项未列出，见该病详情页。

原发性轻链型淀粉样变 27 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
• 招募中NCT06699394
  Prospective Study of Teclistamab in the Treatment of Systemic AL Amyloidosis
  不适用 · 干预性 · 2025/03/29Peking University People's Hospital
  中国研究中心 2 个：Beijing

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
• 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
• 招募中NCT06507683
  Evaluate the Efficacy and Safety of Radiotherapy Combined With Ripretinib in the Treatment of Unresectable Advanced GIST
  不适用 · 干预性 · 2024/05/28First Affiliated Hospital of Chongqing Medical University
  中国研究中心 1 个：Chongqing

另有 14 项未列出，见该病详情页。

神经母细胞瘤 26 项在招目录 第2批 · 53

• 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 Recurrent or Refractory Neuroblastoma or Solid Tumors
  I 期、II 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
• 招募中NCT06684639
  A Study on the Efficacy of GD2-CAR T Cells in the Treatment of Neuroblastoma
  早期 I 期 · 干预性 · 2024/12/01The General Hospital of Western Theater Command
  中国研究中心 1 个：Chengdu

另有 14 项未列出，见该病详情页。

多系统萎缩 25 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
• 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
• 招募中NCT07805967
  Multi-Center Study of Natural History in Atypical Parkinsonian Syndromes (MAPS-NH)
  观察性 · 2025/11/12Second Affiliated Hospital of Soochow University
  中国研究中心 20 个：Changshu、Deyang、Huai'an、Huzhou、Jiaxing、Kunshan 等 17 地

另有 13 项未列出，见该病详情页。

特发性肺纤维化 22 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 10 项未列出，见该病详情页。

地中海贫血（重型） 21 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 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
• 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
• 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。

IgG4相关性疾病 20 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 8 项未列出，见该病详情页。

阵发性睡眠性血红蛋白尿 19 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
• 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
• 尚未开始招募NCT07266155
  Efficacy, Safety, and Pharmacokinetics of LP-005 Injection in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
  II 期 · 干预性 · 2025/12/05Longbio Pharma
  中国研究中心 1 个：Nanjing

另有 7 项未列出，见该病详情页。

特发性肺动脉高压 18 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 6 项未列出，见该病详情页。

朗格汉斯组织细胞增生症 18 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
• 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
• 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
• 招募中NCT07022834
  Real-world Study of Darafenib or Trametinib and Clofarabine for High-risk/Recurrent/Refractory Langerhans Cell Histiocytosis in Children
  II 期 · 干预性 · 2025/06/01West China Second University Hospital
  中国研究中心 1 个：Chengdu

另有 6 项未列出，见该病详情页。

自身免疫性脑炎 15 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 3 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
• 招募中NCT07449793
  Biochemical Response and Clinical Outcomes in Patients With PBC
  观察性 · 2026/02/01RenJi Hospital
  中国研究中心 1 个：Shanghai

另有 3 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 10 个：Beijing、Changsha、Chengdu、Fuzhou、Guangzhou、Hangzhou 等 8 地
• 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 6 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NCT06932003
  A Study to Explore the Safety, Tolerability, Efficacy, and Pharmacokinetics of LT-002-158 Tablets in Chinese Adult Patients With Hidradenitis Suppurativa
  I 期、II 期 · 干预性 · 2025/07/08Leadingtac Pharmaceutical (Shaoxing) Co., Ltd.
  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 13 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NCT07802405
  Evaluating Safety ,Tolerability, Pharmacokinetic,Efficacy of WJ01024 or WJ01024 Combined With Ruxolitinib in Patients With Myelofibrosis
  I 期 · 干预性 · 2023/11/21Henan Cancer Hospital
  中国研究中心 1 个：Zhengzhou
• 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 17 个：Beijing、Changchun、Chongqing、Fuzhou、Guangzhou、Hangzhou 等 17 地

另有 1 项未列出，见该病详情页。

转甲状腺素蛋白淀粉样变性 11 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 3 个：Hefei、Qingdao、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NCT06465810
  Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis
  观察性 · 2024/06/25AstraZeneca
  中国研究中心 20 个：Beijing、Changsha、Chengdu、Chongqing、Dalian、Fuzhou 等 20 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 18 个：Binhai、Changsha、Chengdu、Dongcheng、Fuzhou、Guangzhou 等 18 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 3 个：Hefei、Qingdao、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 33 个：Beijing、Binhai、Changchun、Changsha、Chongqing、Fuzhou 等 22 地
• 招募中NCT07654855
  Long-term Safety of Eplontersen Treated aTTR Patients and in Liver Transplant and Severely Hepatic Impaired Subpopulations
  观察性 · 2026/09/01AstraZeneca
  中国研究中心 1 个：Beijing
• 尚未开始招募NCT07789483
  An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of ALXN2220 in Adults With Transthyretin Amyloid Cardiomyopathy
  III 期 · 干预性 · 2027/11/23Alexion Pharmaceuticals, Inc.
  中国研究中心 13 个：Beijing、Changsha、Chongqing、Guangzhou、Guiyang、Hangzhou 等 13 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

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

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

肺囊性纤维化 8 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
• 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

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

脊髓延髓肌萎缩症（肯尼迪病） 8 项在招目录 第1批 · 109

• 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
• 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
• 尚未开始招募NCT07795177
  A Prospective Study Based on Spatial Multi-omics to Predict the Efficacy of ADT Combined With Second-generation Novel Hormonal Agents in Metastatic Hormone-sensitive Prostate Cancer.
  II 期 · 干预性 · 2026/10/01Anhui Medical University
  中国研究中心 1 个：Hefei

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 Mesothelioma or Other Advanced Solid Tumors
  I 期、II 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 17 个：Beijing、Changchun、Chongqing、Fuzhou、Guangzhou、Hangzhou 等 17 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

发作性睡病 7 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NCT07646678
  A Study of TAK-360 in People With Narcolepsy or Idiopathic Hypersomnia
  II 期、III 期 · 干预性 · 2026/06/11Takeda
  中国研究中心 3 个：Guangzhou、Xicheng、Zhenzhou
• 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

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

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
• 尚未开始招募NCT04637503
  4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma
  I 期、II 期 · 干预性 · 2026/12/31Shenzhen 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

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
• 尚未开始招募NCT03217617
  SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
  I 期、II 期 · 干预性 · 2027/06/30Shenzhen 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
• 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
• 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 · 2027/12/31Gritgen Therapeutics Co., Ltd.
  中国研究中心 1 个：Hefei

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
• 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
• 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 · 2027/12/31Gritgen Therapeutics Co., Ltd.
  中国研究中心 1 个：Hefei

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

先天性胆道闭锁 4 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
• 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

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

短肠综合征 4 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 3 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

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 5 个：Changsha、Chengdu、Guangzhou、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NCT03645460
  Gene Therapy for ADA-SCID Using an Improved Lentiviral Vector (Ivlv-ADA)
  不适用 · 干预性 · 2027/06/30Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 尚未开始招募NCT03217617
  SCID-X1 Gene Therapy Via Intravenous Lentiviral (Ivlv-X1) Injection
  I 期、II 期 · 干预性 · 2027/06/30Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

婴儿严重肌阵挛性癫痫(Dravet综合征) 3 项在招目录 第1批 · 105

• 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 Shi、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

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
• 尚未开始招募NCT03727555
  IT and IV Lentiviral Gene Therapy for X-ALD
  不适用 · 干预性 · 2027/06/01Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen

获得性血友病 3 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
• 尚未开始招募NCT07789587
  A Study of Bometase Alfa for Bleeding Control in Acquired Hemophilia A
  不适用 · 干预性 · 2026/08Institute of Hematology & Blood Diseases Hospital, China
  中国研究中心 1 个：Tianjin

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

神经纤维瘤病 3 项在招目录 第2批 · 54

• 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

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

Alport 综合征 2 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

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

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

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

Lennox-Gastaut 综合征 2 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 Shi、Wuhan
• 招募中NCT05066217
  An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
  III 期 · 干预性 · 2025/04/09Epygenix
  中国研究中心 16 个：Beijing、Changchun、Changsha、Chengdu、Chongqing、Guangzhou 等 13 地

异染性脑白质营养不良 2 项在招目录 第2批 · 49

• 尚未开始招募NCT03725670
  Direct Lentiviral Injection Gene Therapy for MLD
  不适用 · 干预性 · 2027/05/31Shenzhen Geno-Immune Medical Institute
  中国研究中心 1 个：Shenzhen
• 尚未开始招募NCT07046338
  Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD
  不适用 · 干预性 · 2027/06/01Shenzhen 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

特发性低促性腺激素性性腺功能减退症 1 项在招目录 第1批 · 53

• 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

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 6 个：Beijing、Chongqing、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

皮肤神经内分泌癌（梅克尔细胞癌） 1 项在招目录 第2批 · 19

• 招募中NCT07070232
  A Clinical Study to Test if an Investigational Treatment Called BNT326 is Safe and Potentially Beneficial When Used Alone or in Combination With Other Investigational Treatments Such as BNT327, for People With Advanced Malignant Tumors
  I 期、II 期 · 干预性 · 2025/08/12BioNTech SE
  中国研究中心 4 个：Hangzhou、Tianjin、Wuhan

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

巨细胞动脉炎 1 项在招目录 第2批 · 35

• 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

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

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