罕见病知识库 RareSeen

肌萎缩侧索硬化

Amyotrophic lateral sclerosis

ORPHA:803疾病中国目录 第1批 · 4

定义 英文原文（暂无中文）

A rare neurodegenerative disease characterized by progressive muscular paralysis reflecting degeneration of motor neurons in the primary motor cortex, corticospinal tracts, brainstem and spinal cord.

别名

Lou Gehrig病

基本事实

遗传方式
常染色体显性、常染色体隐性、多基因/多因素、X 连锁显性
发病年龄
成年期
患病率
1-9 / 100 000（Europe）

相关基因 33

基因名称关联类型
LRP12LDL receptor related protein 12Disease-causing germline mutation(s) in
ATXN2ataxin 2Major susceptibility factor in
CHMP2Bcharged multivesicular body protein 2BDisease-causing germline mutation(s) in
SOD1superoxide dismutase 1Disease-causing germline mutation(s) in
TREM2triggering receptor expressed on myeloid cells 2Major susceptibility factor in
VAPBVAMP associated protein B and CDisease-causing germline mutation(s) in
VCPvalosin containing proteinDisease-causing germline mutation(s) in
NEFHneurofilament heavy chainMajor susceptibility factor in
OPTNoptineurinDisease-causing germline mutation(s) in
DCTN1dynactin subunit 1Candidate gene tested in
TARDBPTAR DNA binding proteinDisease-causing germline mutation(s) in
FIG4FIG4 phosphoinositide 5-phosphataseDisease-causing germline mutation(s) in
FUSFUS RNA binding proteinDisease-causing germline mutation(s) in
ANGangiogeninDisease-causing germline mutation(s) in
GLE1GLE1 RNA export mediatorDisease-causing germline mutation(s) in
MATR3matrin 3Disease-causing germline mutation(s) in
PRPHperipherinMajor susceptibility factor in
DAOD-amino acid oxidaseDisease-causing germline mutation(s) in
NEK1NIMA related kinase 1Major susceptibility factor in
SQSTM1sequestosome 1Disease-causing germline mutation(s) in
UBQLN2ubiquilin 2Disease-causing germline mutation(s) in
C9ORF72C9orf72-SMCR8 complex subunitDisease-causing germline mutation(s) in
PFN1profilin 1Disease-causing germline mutation(s) in
TBK1TANK binding kinase 1Major susceptibility factor in
HNRNPA1heterogeneous nuclear ribonucleoprotein A1Disease-causing germline mutation(s) in
TAF15TATA-box binding protein associated factor 15Candidate gene tested in
ERBB4erb-b2 receptor tyrosine kinase 4Disease-causing germline mutation(s) in
UNC13Aunc-13 homolog AMajor susceptibility factor in
CHCHD10coiled-coil-helix-coiled-coil-helix domain containing 10Disease-causing germline mutation(s) in
CFAP410cilia and flagella associated protein 410Disease-causing germline mutation(s) in
CCNFcyclin FDisease-causing germline mutation(s) in
ANXA11annexin A11Disease-causing germline mutation(s) in
GLT8D1glycosyltransferase 8 domain containing 1Major susceptibility factor in

临床表型 47

必现 100%1

  • 肌萎缩侧索硬化 HP:0007354

极常见 99–80%3

  • 泛发性肌无力 HP:0003324
  • 运动神经元萎缩 HP:0007373
  • 神经变性 HP:0002180

常见 79–30%32

  • 肺功能检测结果异常 HP:0030878
  • 焦虑 HP:0000739
  • 非典型行为 HP:0000708
  • 巴彬斯基征 HP:0003487
  • 认知功能损害 HP:0100543
  • 抑郁 HP:0000716
  • 远端肌肉萎缩 HP:0003693
  • 流涎 HP:0002307
  • 构音障碍 HP:0001260
  • 吞咽困难 HP:0002015
  • 发音困难 HP:0001618
  • 呼吸困难 HP:0002094
  • 情绪不稳 HP:0000712
  • 肌束震颤 HP:0002380
  • 口咽部肌肉疲劳性无力 HP:0030192
  • 呼吸肌疲劳性无力 HP:0030196
  • 吞咽肌肉疲劳性无力 HP:0030195
  • 疲乏 HP:0012378
  • Hoffmann征 HP:0031993
  • 反射亢进 HP:0001347
  • 下肢肌肉无力 HP:0007340
  • 肌肉痉挛 HP:0003394
  • 疼痛 HP:0012531
  • 瘫痪 HP:0003470
  • 进行性远端肌萎缩 HP:0008955
  • 呼吸衰竭 HP:0002878
  • 骨骼肌萎缩 HP:0003202
  • 痉挛 HP:0001257
  • 舌萎缩 HP:0012473
  • 上肢肌无力 HP:0003484
  • 体重减轻 HP:0001824
  • 口腔干燥 HP:0000217

偶见 29–5%10

  • 恶病质 HP:0004326
  • 足背屈无力 HP:0009027
  • 额颞叶痴呆 HP:0002145
  • 下颌反射亢进 HP:0033683
  • 语言障碍 HP:0002463
  • 端坐式呼吸 HP:0012764
  • 睡眠异常 HP:0002360
  • 痉挛性双下肢瘫 HP:0002313
  • 跨阈步态 HP:0003376
  • 舌肌束震颤 HP:0001308

罕见 <4–1%1

  • 喉痉挛 HP:0025425

近两年的全球研究 16,835L2

2024/10 起在 Europe PMC 检索所得，按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务，尚未引入。

  • 2026-10
    Mdivi-1 regulates mitochondrial fission and function via the CaMK2/Drp1 pathway in amyotrophic lateral sclerosis
    Neuroscience · DOI · Europe PMC
  • 2026-10
    Nitrate-responsive Sialin2 couples stress granule assembly and clearance to maintain neuronal homeostasis
    Developmental cell · DOI · Europe PMC
  • 2026-10综述
    From patient cells to patient therapy: Twenty years of iPSC-based translation in neurodegenerative disease
    Cell reports. Medicine · DOI · Europe PMC
  • 2026-10综述
    Acidic cannabinoids in brain disorders: Neurobiological mechanisms, preclinical evidence, and translational challenges
    Neuroscience and biobehavioral reviews · DOI · Europe PMC
  • 2026-10综述病例报告
    Amyotrophic Lateral Sclerosis
    Continuum (Minneapolis, Minn.) · DOI · Europe PMC
  • 2026-10
    Associations Between Prediagnosis Plasma Neurofilament Light Polypeptide and Amyotrophic Lateral Sclerosis Risk in the Danish EPIC Cohort
    Annals of neurology · DOI · Europe PMC
  • 2026-10
    Insertion or deletion variants in TAF15 exon 15 are genetic factors impacting the prognosis of amyotrophic lateral sclerosis in a Japanese cohort
    European journal of human genetics : EJHG · DOI · Europe PMC
  • 2026-10
    The accessory makes the drug: ASO-delivered siRNA enters the human CNS for ALS
    Cell stem cell · DOI · Europe PMC
  • 2026-10
    A genetic screen for suppressors of neuron degeneration in a C. elegans sod-1G85R model of Amyotrophic Lateral Sclerosis
    G3 (Bethesda, Md.) · DOI · Europe PMC
  • 2026-10综述开放获取
    Proteostasis and ageing dissidence
    Philosophical transactions of the Royal Society of London. Series B, B · DOI · Europe PMC
  • 2026-10综述开放获取
    The microbiota-proteostasis axis: implications in neurodegenerative diseases
    Philosophical transactions of the Royal Society of London. Series B, B · DOI · Europe PMC
  • 2026-10综述开放获取
    Proteostasis (in)action: the role of co-pathologies in neurodegenerative disease
    Philosophical transactions of the Royal Society of London. Series B, B · DOI · Europe PMC
  • 2026-10综述开放获取
    Chaperone-mediated remodelling of mutant huntingtin
    Philosophical transactions of the Royal Society of London. Series B, B · DOI · Europe PMC
  • 2026-10综述开放获取
    Disruption of RNA metabolism and its impact on protein homeostasis
    Philosophical transactions of the Royal Society of London. Series B, B · DOI · Europe PMC
  • 2026-10综述开放获取
    Fyn kinase: a key mechanistic regulator and therapeutic target in tauopathy and neurodegenerative diseases
    Translational neurodegeneration · DOI · Europe PMC
  • 2026-10开放获取
    Glymphatic System Dysfunction and its Associated Factors in Wilson's Disease: A DTI-ALPS Study
    Brain and behavior · DOI · Europe PMC
  • 2026-10病例报告
    Recognition and Treatment of Concurrent Amyotrophic Lateral Sclerosis and Multiple Sclerosis
    Rhode Island medical journal (2013) · Europe PMC
  • 2026-10综述开放获取
    The role of IRF5 as an immune regulator
    Biochemical Society transactions · DOI · Europe PMC
  • 2026-10开放获取
    Motor Cortex Hyperexcitability Is Coupled to Neuromuscular Dysfunction in Aged Mice
    Aging cell · DOI · Europe PMC
  • 2026-10开放获取
    Ginsenosides Rb1 and Rg1 Suppress Lipopolysaccharide-Induced Inflammatory Gene Expression in Microglia and Astrocytes, Unlike Rb2
    Pharmacology research & perspectives · DOI · Europe PMC

国家医保药品目录中点名本病的药品 3L2

出自《国家基本医疗保险、生育保险和工伤保险药品目录（2025年）》（医保发〔2025〕33号，2026-01-01 起执行）。下列药品在药品名称或限定支付范围里出现了本病的名称。

匹配不到 ≠ 不能报销。目录里只有约一成药品设了限定支付范围，其余按适应症正常使用同样可报销；本区块只能回答「目录有没有点名这个病」，不能回答「这个病有没有药能报销」。各省执行细则、双通道与单独支付范围另有规定，请以当地医保部门口径为准。

  • 依达拉奉氯化钠注射液乙类西药
    限肌萎缩侧索硬化(ALS)。
  • 依达拉奉注射液乙类西药
    限肌萎缩侧索硬化(ALS)。
  • 利鲁唑口服混悬液乙类谈判药品
    限肌萎缩侧索硬化(ALS)。

境外已获批用于本病的药物 8L2

欧盟 2 项、美国 6 项。同一药物在两地各批一次的，会分别列出。

「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径，各有各的条件与费用。这一节能确定地告诉你的只有一件事：这个病在世界范围内已经有获得批准的药物，它叫什么名字。拿这个名字去问主治医生，是下一步最省力的做法。

药名一律保留英文原文，不作翻译——中国的药品通用名与英文名的音译经常不一致，译错会让人去找一个不存在的药。

尚未获批的在研药物（174 项）

这些药已被欧盟或美国的监管机构认定为罕见病用药（英文 orphan drug designation，中文行业里通称「孤儿药资格」——「孤儿」说的是这类药市场太小、没有厂商愿意认领，不是在说病人）。但这只是一种监管身份：意味着监管机构给予研发上的激励，不代表这个药已被证明有效，也不代表将来一定能上市，绝大多数最终不会成药。列在这里是为了看清有哪些方向正在被尝试。

  • filgrastim欧盟2008-04-01
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydrochlo欧盟2009-02-27
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • recombinant human vascular endothelial growth factor欧盟2010-01-29
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • smilagenin欧盟2011-09-27
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • S[+] apomorphine欧盟2012-02-09
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one (tirasemt欧盟2012-03-05
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • sodium chlorite欧盟2013-06-19
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • allogeneic motor neuron progenitor cells derived from human embryonic 欧盟2013-07-17
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • autologous bone-marrow-derived mesenchymal stromal cells secreting neu欧盟2013-07-17
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • edaravone欧盟2014-12-16
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Enoxacin欧盟2015-03-19
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate欧盟2015-07-28
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • recombinant human cerebral dopamine neurotrophic factor欧盟2016-04-28
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide)欧盟2016-05-30
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Alsitek; Masitinib AB Science欧盟2016-08-29
    Masitinib mesilate
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Ibudilast欧盟2016-12-12
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • tauroursodeoxycholic acid欧盟2017-02-27
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • recombinant human antibody directed against misfolded human superoxide欧盟2017-07-17
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • (R)-troloxamide quinone欧盟2017-10-12
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • adeno-associated viral vector serotype 9 encoding miRNA against human 欧盟2018-04-16
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • ambroxol hydrochloride欧盟2018-05-25
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • human culture expanded autologous mesenchymal stromal cells欧盟2019-04-24
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH欧盟2019-12-16
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • reldesemtiv欧盟2020-02-28
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Albrioza欧盟2020-06-04
    sodium phenylbutyrate;tauroursodeoxycholic acid
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • dextran sulfate low molecular weight欧盟2020-08-21
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide欧盟2020-11-13
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • celecoxib;ciprofloxacin欧盟2021-01-06
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • ganglioside GM1欧盟2021-04-13
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • trehalose欧盟2021-05-20
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • pridopidine hydrochloride欧盟2021-07-19
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • anti-CD38 IgG4 human monoclonal antibody欧盟2021-11-12
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • D-lactic acid, glycolic acid欧盟2021-11-12
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • fasudil hydrochloride欧盟2022-01-14
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • aldesleukin欧盟2022-02-24
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Icerguastat acetate欧盟2022-05-16
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Gold欧盟2022-08-10
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • sotuletinib欧盟2022-08-10
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • Oxygen, sodium chloride solution 0.9%欧盟2022-10-11
    Treatment of amyotrophic lateral sclerosis
    官方记录
  • crisdesalazine欧盟2023-02-15
    Treatment of amyotrophic lateral sclerosis
    官方记录

另有 134 项未列出。

数据来自欧洲药品管理局（EMA）的药品与罕见病用药资格公开导出表，以及美国 FDA 的罕见病用药资格数据库（Orphan Drug Product Designation Database）。两边口径不同：欧盟一侧取的是当前状态仍为「已授权」的药品；美国一侧记录的是「曾获批准」这一事实，FDA 的公开表不追踪药物此后是否退市（例如 Relyvrio 于 2024 年撤市，表中仍记为已获批）。请以官方记录页为准。

在中国开展的临床试验 53L2

按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实，并与主治医生商量。

登记为可入组 20

  • 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
  • 招募中NCT06849609
    A Study to Evaluate the Tolerability, Safety and Efficacy of VGN-R13 in Patients with ALS
    早期 I 期 · 干预性 · 2025/01/16Hong Chen
    中国研究中心 1 个：Wuhan
  • 招募中NCT06829212
    Research on Wireless Brain Implant System for General Control of External Devices
    不适用 · 干预性 · 2025/03Shanghai StairMed Technology Co., Ltd.
    中国研究中心 1 个：Shanghai
  • 招募中NCT06765499
    The Study Evaluating the Improvement of Nutritional Status and Frailty With Silkworm Pupa Powder Among Patients With Motor Neuron Disease
    不适用 · 干预性 · 2025/03/04First People's Hospital of Hangzhou
    中国研究中心 1 个：Hangzhou
  • 尚未开始招募NCT06607900
    hUC-MSC-sEV-001 Nasal Drops for Neurodegenerative Diseases
    I 期 · 干预性 · 2025/07/01Xuanwu Hospital, Beijing
    中国研究中心 1 个：Beijing
  • 招募中NCT07118319
    The Safety, Tolerability and Preliminary Efficacy of Derived Motor Neuron Progenitor Cells (XS228CN) in Subjects With Amyotrophic Lateral Sclerosis
    I 期 · 干预性 · 2025/09/15XellSmart Bio-Pharmaceutical (Suzhou) Co., Ltd.
    中国研究中心 1 个：Beijing
  • 尚未开始招募NCT07169175
    A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
    I 期、II 期 · 干预性 · 2025/12/01SineuGene Therapeutics Co., Ltd.
    中国研究中心 3 个：Beijing、Fuzhou、Hangzhou
  • 仅邀请入组NCT07539662
    Unrelated Umbilical Cord Blood Transplantation for the Treatment of Amyotrophic Lateral Sclerosis (ALS)
    不适用 · 干预性 · 2025/12/29Institute of Hematology & Blood Diseases Hospital, China
    中国研究中心 1 个：Tianjin
  • 招募中NCT06726577
    TP04HN106 in the Treatment of Patients With Amyotrophic Lateral Sclerosis
    I 期、II 期 · 干预性 · 2026/05/10Talengen Institute of Life Sciences, Shenzhen, P.R. China.
    中国研究中心 1 个：Suzhou
其他状态的试验（33 项）
  • 已完成NCT00397423
    G-CSF Treatment for Amyotrophic Lateral Sclerosis: A RCT Study Assessing Clinical Response
    II 期 · 干预性 · 2006/12Peking University
    中国研究中心 1 个：Beijing
  • 状态未知NCT00753571
    Cistanche Total Glycosides for Amyotrophic Lateral Sclerosis: A Randomized Control Trial (RCT) Study Assessing Clinical Response
    II 期 · 干预性 · 2008/01Peking University
    中国研究中心 1 个：Beijing
  • 已完成NCT03085706
    Transplantation of Autologous Peripheral Blood Mononuclear Cells for Amyotrophic Lateral Sclerosis
    不适用 · 干预性 · 2010/10The First Affiliated Hospital of Dalian Medical University
    中国研究中心 1 个：Dalian
  • 状态未知NCT01494480
    The Clinical Trial on the Use of Umbilical Cord Mesenchymal Stem Cells in Amyotrophic Lateral Sclerosis
    II 期 · 干预性 · 2012/03General Hospital of Chinese Armed Police Forces
    中国研究中心 1 个：Beijing
  • 状态未知NCT01935518
    A Clinical Trial of Safety and Efficacy of Fasudil in Subjects With Amyotrophic Lateral Sclerosis (ALS)
    II 期 · 干预性 · 2013/09Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 已完成NCT04952025
    Quantitative Analysis of Precise Brain Volume in Amyotrophic Lateral Sclerosis
    观察性 · 2015/01/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 已完成NCT04454840
    Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral Sclerosis
    早期 I 期 · 干预性 · 2016/05/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT03103815
    Trial of Amivita in Amyotrophic Lateral Sclerosis
    不适用 · 干预性 · 2017/04/24Wujin People's Hospital
    中国研究中心 1 个：Changzhou
  • 已完成NCT04455542
    Peripheral Neurofilament Levels and Amyotrophic Lateral Sclerosis
    观察性 · 2017/08/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 已完成NCT04948346
    Correlation Between Intestinal Microflora Metabolites and Amyotrophic Lateral Sclerosis
    观察性 · 2018/01/31Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04956822
    Application of Motor Unit Estimation Index in Amyotrophic Lateral Sclerosis and Related Diseases
    观察性 · 2018/06/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04960540
    Multimodal Brain Imaging Study
    观察性 · 2019/08/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04952155
    Low Dose IL-2 in the Treatment of Immune-associated ALS Syndrome
    II 期 · 干预性 · 2020/01/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04328675
    Registration Study of Amyotrophic Lateral Sclerosis (ALS) in Mainland China
    观察性 · 2020/03/20Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04950933
    The Treatment of Amyotrophic Lateral Sclerosis With Huollingshengji Granules
    II 期、III 期 · 干预性 · 2020/06/01Peking University Third Hospital
    中国研究中心 1 个：Beijin
  • 状态未知NCT04950647
    Efficacy and Safety of Nitrazine in the Treatment of ALS
    II 期 · 干预性 · 2020/07/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04953624
    Appetite and Related Factors in Patients With ALS
    观察性 · 2020/08/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04952038
    Sleep Disorders and the Onset and Progression of ALS
    观察性 · 2020/08/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 已完成NCT04950231
    Investigation on Home Care Needs of ALS Patients and Their Caregivers
    观察性 · 2020/09/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04518540
    Explore Neuroprotective Effect of Lipoic Acid in Amyotrophic Lateral Sclerosis
    不适用 · 干预性 · 2020/09/01Second Affiliated Hospital, Zhejiang University, School of Medicine
    中国研究中心 1 个：Hangzhou
  • 状态未知NCT04391361
    The Safety and Effectiveness of Cholinergic Receptor Block Therapy in the Treatment of ALS
    II 期 · 干预性 · 2020/11/01Ruijin Hospital
    中国研究中心 1 个：Shanghai
  • 状态未知NCT04953481
    Home Care Needs and Current Nursing Status of Patients With Amyotrophic Lateral Sclerosis
    观察性 · 2021/01/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT04953494
    Pleiotropy and Mechanism of Peripheral Nerve Related Genes in the Progression of Amyotrophic Lateral Sclerosis
    观察性 · 2021/08/01Peking University Third Hospital
    中国研究中心 1 个：Beijing
  • 已终止NCT05237284
    Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis (ALS)
    II 期 · 干预性 · 2022/04/13Sanofi
    中国研究中心 6 个：Beijing、Chengdu、Guangzhou、Hangzhou、Wuhan、Xi'an
  • 已完成NCT05903690
    Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
    早期 I 期 · 干预性 · 2023/05/24Beijing Tiantan Hospital
    中国研究中心 1 个：Beijing
  • 状态未知NCT05892822
    Amyotrophic Lateral Sclerosis Registry
    观察性 · 2023/06/15yilong Wang
    中国研究中心 1 个：Beijing
  • 进行中·不再招募NCT06454682
    An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS
    早期 I 期 · 干预性 · 2023/09/11RJK Biopharma Ltd
    中国研究中心 1 个：Fuzhou
  • 状态未知NCT06181526
    Safety and Preliminary Efficacy of Aleeto in Amyotrophic Lateral Sclerosis
    I 期 · 干预性 · 2023/12/15Beijing Tiantan Hospital
    中国研究中心 1 个：Beijing
  • 已完成NCT06849115
    Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations
    IV 期 · 干预性 · 2024/07/18ChaodongWang
    中国研究中心 1 个：Beijing
  • 进行中·不再招募NCT06493279
    Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
    I 期 · 干预性 · 2024/09/24RJK Biopharma Ltd
    中国研究中心 1 个：Beijing
  • 进行中·不再招募NCT06643481
    A Clinical Trial to Learn About the Effects of VHB937 in People With Amyotrophic Lateral Sclerosis (ALS)
    II 期 · 干预性 · 2024/10/17Novartis Pharmaceuticals
    中国研究中心 1 个：Beijing
  • 进行中·不再招募NCT07223723
    A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
    IV 期 · 干预性 · 2025/12/11Biogen
    中国研究中心 1 个：Beijing
  • 进行中·不再招募NCT07618585
    Safety and Efficacy of Intrathecal NTF001 Injection in ALS
    I 期 · 干预性 · 2026/04/01Ruijin Hospital
    中国研究中心 1 个：Shanghai

中国境外的在招试验 230L2

这些试验在中国没有研究中心，通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处：看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生，或据此进一步查该药是否已在境外获批、是否有拓展性用药（expanded access）通道。

美国72法国23意大利22加拿大14德国12荷兰11英国11比利时10西班牙9澳大利亚8波兰8瑞典6丹麦5爱尔兰5另有 21 个国家/地区

CT.gov 报告命中 230 项，此处取回并展示最近的 15 项。

  • 尚未开始招募NCT04667013
    A Multiple Dose Study to Investigate Safety, Tolerability and Pharmacokinetics of TBN
    I 期 · 干预性 · 2028/09/01Guangzhou Magpie Pharmaceuticals Co., Ltd.
  • 尚未开始招募NCT06704347
    Safety Study of XT-150 in Participants With ALS
    I 期 · 干预性 · 2027/03Xalud Therapeutics, Inc.
    美国
  • 尚未开始招募NCT02478450
    Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)
    I 期、II 期 · 干预性 · 2026/12Q Therapeutics, Inc.
  • 尚未开始招募NCT07698496
    Evaluation of the Feasibility and Efficacy of a Chronic Brain-computer Interface for Speech Rehabilitation in Patients With Locked-in-syndrome (LIS)
    不适用 · 干预性 · 2026/10/10University Hospital, Grenoble
    法国
  • 尚未开始招募NCT05470478
    iBCI Optimization for Veterans With Paralysis
    不适用 · 干预性 · 2026/10/02VA Office of Research and Development
    美国
  • 尚未开始招募NCT07589764
    Pilot Trial Utilizing β-hydroxy-β-methylbutyrate to Lower IGFBP7 Levels in People With ALS
    I 期 · 干预性 · 2026/10/01Duke University
    美国
  • 尚未开始招募NCT07819890
    Development in Home Mechanical Ventilation for Patient With Amyotrophic Lateral Sclerosis.
    观察性 · 2026/10/01Rigshospitalet, Denmark
  • 招募中NCT07823101
    Imaging Assessment of Swallowing and Speech Muscles in Inclusion Body Myositis
    观察性 · 2026/10Johns Hopkins University
    美国
  • 尚未开始招募NCT07813858
    Colchicine Effect on Amyotrophic Lateral Sclerosis Patients
    II 期 · 干预性 · 2026/10Azienda Ospedaliero-Universitaria di Modena
    意大利
  • 招募中NCT07521930
    Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)
    不适用 · 干预性 · 2026/09Johns Hopkins University
    美国
  • 招募中NCT07674667
    FUNCtion ALS: Aiming to Restore UNC13A Function in People Living With ALS
    I 期、II 期 · 干预性 · 2026/08/20Trace Neuroscience, Inc.
    德国、荷兰、英国
  • 招募中NCT07224269
    Effect of Terazosin on ATP Levels in People With Amyotrophic Lateral Sclerosis
    早期 I 期 · 干预性 · 2026/08/12University of Iowa
    美国
  • 尚未开始招募NCT07706270
    Serum Neurofilaments in the Diagnosis of Amyotrophic Lateral Sclerosis
    观察性 · 2026/08/01University Hospital, Montpellier
  • 尚未开始招募NCT07400393
    Healey ALS MyMatch Common Screening Protocol
    观察性 · 2026/08Massachusetts General Hospital
  • 尚未开始招募NCT07737977
    Developing a Comprehensive Biomarker Panel for Monitoring Progression and Early Detection in ALS Patients
    观察性 · 2026/08University Hospital, Montpellier
    法国、西班牙

外部标识与链接

OrphanetOMIM:105400OMIM:205250OMIM:300857MONDO:0004976GARD:5786ICD-10 G12.2ICD-11 8B60.0ClinicalTrials.gov 检索

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本页数据来源

  • 疾病定义、同义词、基因、表型、流行病学：Orphanet（CC BY 4.0）
  • 中文病名：Orphanet 中文包，冻结于 2020-06-01
  • 表型中文标签：HPO 简体中文翻译（CHPO 上游成果）