39 active trials 23 recruiting 8 Phase 3 27 sponsors 5 changes in 30 days
| Early Phase 1 | 1 | |
| Phase 1/2 | 4 | |
| Phase 2 | 1 | |
| Phase 2/3 | 1 | |
| Phase 3 | 7 | |
| Phase 4 | 1 |
| NCT ID | Title | Sponsor | Status | Updated |
|---|---|---|---|---|
| Phase 4 | ||||
| NCT05232929 | Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) | Genentech, Inc. | Active, Not Recruiting | 2026-07-16 |
| Phase 3 | ||||
| NCT07221669 | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When… | Biogen | Recruiting | 2026-08-18 |
| NCT07444450 | A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to… | Biogen | Recruiting | 2026-08-11 |
| NCT07444476 | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in… | Biogen | Recruiting | 2026-04-21 |
| NCT07265232 | Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal… | Lantu Biopharma | Recruiting | 2025-12-04 |
| NCT06971094 | Safety and Efficacy Evaluation of GC101 Gene Therapy Via Intrathecal (IT) Injectionin the… | GeneCradle Inc | Recruiting | 2025-06-05 |
| NCT05335876 | Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in… | Novartis Pharmaceuticals | Active, Not Recruiting | 2026-07-09 |
| NCT05067790 | A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as… | Biogen | Active, Not Recruiting | 2026-05-22 |
| Phase 2/3 | ||||
| NCT05115110 | A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam… | Hoffmann-La Roche | Active, Not Recruiting | 2026-06-11 |
| Phase 2 | ||||
| NCT07287982 | A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and… | argenx | Recruiting | 2026-08-27 |
| Phase 1/2 | ||||
| NCT05824169 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | GeneCradle Inc | Recruiting | 2025-07-03 |
| NCT06421831 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | GeneCradle Inc | Recruiting | 2025-07-03 |
| NCT05747261 | Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN… | Biocad | Recruiting | 2024-02-22 |
| NCT05901987 | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal… | GeneCradle Inc | Active, Not Recruiting | 2025-07-03 |
| Early Phase 1 | ||||
| NCT06888661 | Clinical Trial to Assess the Safety and Efficacy of EXG001-307 in Patients With Spinal… | Guangzhou Jiayin Biotech Ltd | Enrolling by Invitation | 2025-03-21 |
| Not applicable | ||||
| NCT07578454 | VRehab-SMA Phase 1.2 | University of Oxford | Recruiting | 2026-06-11 |
| NCT07321977 | Assessment of a Portable Digital Device for Quantified Analysis of Markerless Walking in… | Institut de Myologie, France | Recruiting | 2026-05-19 |
| NCT07488338 | HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness | Teachers College, Columbia University | Recruiting | 2026-03-23 |
| NCT07332702 | Long Read Analysis in Spinal Muscular Atrophy - LOREASI | University Hospital, Rouen | Recruiting | 2026-01-12 |
| NCT07286565 | Active NBS Study: Decentralised Monitoring Motor Development in Children With Duchenne… | Centre Hospitalier Universitaire de Liege | Recruiting | 2025-12-16 |
| NCT06363357 | The Effect of a Muscle-mimicking, Fabric-type Shoulder Orthosis on Functional Movements… | Seoul National University Hospital | Recruiting | 2025-12-03 |
| NCT06977269 | Safety and Tolerability of Low Motoneuron Stimulation Via Transcranial Magnetic… | Charitable Foundation Children with Spinal Muscular Atrophy | Recruiting | 2025-06-18 |
| NCT07617779 | A Study of the Safety and Preliminary Efficacy of NKG001 in Pediatric Patients With Type… | Nikegen Pharmaceutical (Hangzhou) Company Limited | Active, Not Recruiting | 2026-06-01 |
| NCT07356063 | Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children… | Aga Khan University | Not Yet Recruiting | 2026-01-22 |
| NCT07336602 | Neuroproprioceptive Equine-Assisted Physiotherapy for Spinal Muscular Atrophy | Charles University, Czech Republic | Not Yet Recruiting | 2026-01-13 |
Biogen (7) · GeneCradle Inc (4) · University of Oxford (3) · Novartis Pharmaceuticals (2) · Aga Khan University (1) · argenx (1) · Biocad (1) · Centre Hospitalier Universitaire de Liege (1) · Charitable Foundation Children with Spinal Muscular Atrophy (1) · Charles University, Czech Republic (1)