162 completed, 64 recruiting, 47 unknown, 36 active not recruiting. 87 Not applicable, 71 Phase 2, 46 Phase 1, 32 Phase 3, 10 Phase 4.
Follow Muscular Atrophy, Spinal: new trials by email Literature Review of Muscular Atrophy, Spinal trials Research Report on Muscular Atrophy, Spinal
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT) INTENT Neural Interface System - Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS) | Not applicable | Recruiting | Johns Hopkins University | 5 | 2027-01-01 | 2026-10-09 |
| SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE) SPX-01 - Spinal Muscular Atrophy (SMA) | Early Phase 1 | Not yet recruiting | Exir Gene Pharmed | 1 | 2027-01-01 | 2026-10-29 |
| A Real-World Medical Chart Review of Spinal Muscular Atrophy Patients Treated With Onasemnogene Abeparvovec in Saudi Arabia Spinal Muscular Atrophy | Not yet recruiting | Novartis Pharmaceuticals | 6 | 2026-11-30 | 2026-11-10 | |
| A Registry of Cases of Spinal Muscular Atrophy Spinal Muscular Atrophy | Recruiting | St. Jude Children's Research Hospital | 30 | 2026-11-01 | 2026-10-22 | |
| A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec Salanersen, Sham Procedure - Muscular Atrophy, Spinal | Phase 3 | Recruiting | Biogen | 42 | 2026-10-31 | 2026-10-15 |
| Effects of Different Stiffness Settings During Robotic Isokinetic Training in Children With SMA Portable Isokinetic Knee Training Robot With an Individually Optimized Compliant Setting, Portable Isokinetic Knee Training Robot With a Rigid Setting - Spinal Muscular Atrophy (SMA) | Not applicable | Recruiting | Peking University Third Hospital | 13 | 2026-10-22 | 2026-10-23 |
| A Study of the Safety and Effectiveness of Onasemnogene Abeparvovec (Zolgensma) Intrathecal Injection in Spinal Muscular Atrophy Patients Spinal Muscular Atrophy | Recruiting | Novartis Pharmaceuticals | 80 | 2026-10-07 | 2026-10-22 | |
| Register for Medical Data, Functional Scales, and Care Outcomes in ALS, SMA, and Other Motor System Disorders Standard of Care (Investigator Choice) - ALS (Amyotrophic Lateral Sclerosis), SMA - Spinal Muscular Atrophy | Recruiting | Ambulanzpartner Soziotechnologie APST GmbH | 2000 | 2026-10-01 | 2026-10-28 | |
| An Open Label Individual Patient (OLIP)-02 Study of Nusinersen (BIIB058) Nusinersen Injectable Product - SMA - Spinal Muscular Atrophy | Phase 3 | Not yet recruiting | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | 1 | 2026-09-17 | 2026-09-29 |
| An OLIP Study (Open Label Individual Patient) of Nusinersen in a Participant With Spinal Muscular Atrophy Who Previously Participated in an Investigational Study With Nusinersen Nusinersen Injectable Product - SMA - Spinal Muscular Atrophy | Not applicable | Not yet recruiting | London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's | 1 | 2026-09-17 | 2026-09-24 |
| A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam Salanersen - Spinal Muscular Atrophy | Phase 3 | Recruiting | Biogen | 90 | 2026-09-08 | 2026-10-02 |
| ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) Onasemnogene Abeparvovec-brve - Spinal Muscular Atrophy | Phase 4 | Recruiting | Novartis Pharmaceuticals | 36 | 2026-08-27 | 2026-10-29 |
| Self-Efficacy Enhancement Among Online Support Group of Primary Caregivers of Children With Neuro Muscular Dystrophy Multicomponent Intervention - Duchenne Muscular Dystrophy (DMD), Spinal Muscular Atrophy (SMA) | Not applicable | Active not recruiting | Aga Khan University | 34 | 2026-08-15 | 2026-10-09 |
| Spinal Muscular Atrophy and School Transitions Spinal Muscular Atrophy | Not yet recruiting | Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust | 20 | 2026-08-10 | 2026-07-22 | |
| A Study to Learn About the Investigational Drug Rinzimetostat (ORIC-944) in Patients With mCRPC Who Were Previously Treated With Abiraterone Acetate (Himalayas-1) Rinzimetostat, Darolutamide, Enzalutamide - Metastatic Castration Resistant Prostate Cancer | Phase 3 | Recruiting | ORIC Pharmaceuticals | 600 | 2026-08-03 | 2026-11-03 |
| Service Evaluation for Newborn Screening for Spinal Muscular Atrophy Spinal Muscular Atrophy (SMA) | Recruiting | University of Oxford | 750000 | 2026-08-01 | 2026-10-22 | |
| A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA) Spinal Muscle Atrophy | Recruiting | Hoffmann-La Roche | 30 | 2026-07-22 | 2026-11-04 | |
| SMAdult - Monitoring of Movement on Spinal Muscular Atrophy wearable sensors - Spinal Amyotrophy | Not applicable | Recruiting | Centre Hospitalier Universitaire de Liege | 60 | 2026-07-17 | 2026-10-26 |
| A Phase I/II Clinical Study to Evaluate SKG0201 Injection in Subjects With Spinal Muscular Atrophy Type I SKG0201 Injection - Spinal Muscular Atrophy 1 | Phase 1 / Phase 2 | Recruiting | Lanyue Biotech (Hangzhou) Co., Ltd. | 11 | 2026-07-15 | 2026-10-14 |
| Exploring Lived Experiences of Families of Children With Spinal Muscular Atrophy(SMA) Type 1 Regarding Feeding and Communication Spinal Muscular Atrophy 1 | Not yet recruiting | Guy's and St Thomas' NHS Foundation Trust | 15 | 2026-06-19 | 2026-06-02 |
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Spinal Muscular Atrophies of Childhood (61) · Bulbo-Spinal Atrophy, X-Linked (57) · Neuromuscular Diseases (37) · Amyotrophic Lateral Sclerosis (32) · Motor Neuron Disease (28) · Muscular Dystrophy, Duchenne (16) · Prostatic Neoplasms (15) · Muscular Dystrophies (13) · Cerebral Palsy (12) · Spinal Cord Injuries (10) · Motor Activity (9) · Muscular Atrophy (9) · Muscular Diseases (9) · Glycogen Storage Disease Type II (6)
Central Nervous System Diseases (358) · Spinal Cord Diseases (358) · Neurodegenerative Diseases (356) · Nervous System Diseases (354) · Motor Neuron Disease (331) · Neuromuscular Diseases (322) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (155) · Genetic Diseases, Inborn (151) · Heredodegenerative Disorders, Nervous System (131) · Genetic Diseases, X-Linked (74) · Musculoskeletal Diseases (47) · Pathological Conditions, Signs and Symptoms (46) · Metabolic Diseases (40) · Nutritional and Metabolic Diseases (40)
nusinersen (27) · Risdiplam (18) · Zolgensma (13) · Blood Specimen Collection (6) · Exercise (5) · Surveys and Questionnaires (5) · Valproic Acid (5) · bicalutamide (5) · enzalutamide (5) · Carnitine (4) · apitegromab (4) · 4-phenylbutyric acid (3) · Hydroxyurea (3) · Watchful Waiting (3) · darolutamide (3)
Biogen (27) · Hoffmann-La Roche (17) · Novartis Pharmaceuticals (16) · Assistance Publique - Hôpitaux de Paris (9) · Columbia University (9) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (9) · National Institute of Neurological Disorders and Stroke (NINDS) (7) · Novartis Gene Therapies (7) · MarsiBionics (6) · University of Utah (6) · Copenhagen University Hospital, Rigshospitalet (5) · Institut de Myologie, France (5) · National Taiwan University Hospital (5) · University of Oxford (5) · Duke University (4)
United States (151) · Italy (52) · France (47) · Germany (44) · United Kingdom (43) · China (36) · Canada (32) · Belgium (30) · Spain (30) · Japan (23) · Poland (21) · Taiwan (21) · Turkey (Türkiye) (21) · Netherlands (20) · Australia (16)
Laurent Servais, MD, PhD (4) · Jacqueline Montes, PT, EdD (3) · Kenneth H Fischbeck, M.D. (3) · Richard Finkel, MD (3) · Bakri Elsheikh, MD (2) · Christopher Grunseich, M.D. (2) · Darryl C De Vivo, MD (2) · Dr Ching H. Wang (2) · Eugenio Mercuri, MD (2) · Ferdinand Knieling, MD (2) · Kathryn J Swoboda, M.D. (2) · Lorenzo Maggi, MD (2)
PMID 31420846, PMID 34383289, PMID 37148485, PMID 27939059, PMID 38705943, PMID 19440247, PMID 26247043, PMID 26823478, PMID 28284873, PMID 29091570, PMID 29443664, PMID 30048507, PMID 30879249, PMID 42016922, PMID 10540002, PMID 10826123, PMID 11508406, PMID 12641825, PMID 13997180, PMID 16096237, PMID 16267188, PMID 16849828, PMID 16900450, PMID 17029646, PMID 17030772, PMID 17136445, PMID 17364435, PMID 17385269, PMID 17709710, PMID 17980667, PMID 18506712, PMID 19846582, PMID 19922132, PMID 20697104, PMID 20808854, PMID 21216197, PMID 21435220, PMID 21706151, PMID 21754985, PMID 22003934, PMID 22077695, PMID 22327252, PMID 22507681, PMID 22558076, PMID 22558154, PMID 23421600, PMID 23456136, PMID 23562111, PMID 23599814, PMID 23628801
2023 (11), 2025 (6), 2014 (4), 2017 (4), 2015 (3), 2016 (3), U54NS092091 (3), 2018 (2), 2019 (2), N01NS42361 (2), 2005 (1), K01HD084690 (1), R01HD054599 (1), R01HD069045 (1), R01HD112908 (1)