2 completed, 2 recruiting, 1 terminated. 5 Phase 2, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Recruiting 2, Terminated 1 |
| Phase 1 | 1 | Recruiting 1 |
Follow Spinal Muscular Atrophy Type 3: new trials by email Literature Review of Spinal Muscular Atrophy Type 3 trials Research Report on Spinal Muscular Atrophy Type 3
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 |
|---|---|---|---|---|---|---|
| A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy Apitegromab, Nusinersen, Risdiplam - Spinal Muscular Atrophy, SMA | Phase 2 | Recruiting | Scholar Rock, Inc. | 52 | 2025-09-15 | 2026-10-02 |
| Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients GC101 - Spinal Muscular Atrophy Type 3 | Phase 1 / Phase 2 | Recruiting | GeneCradle Inc | 21 | 2024-05-10 | 2025-07-03 |
| An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy SRK-015 - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Phase 2 | Completed | Scholar Rock, Inc. | 58 | 2019-04-22 | 2024-12-02 |
| Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3 Amifampridine Phosphate 10 MG Oral Tablet - Spinal Muscular Atrophy Type 3 | Phase 2 | Terminated | Catalyst Pharmaceuticals, Inc. | 13 | 2019-03-07 | 2023-11-30 |
| Safety and Efficacy Study of Pyridostigmine on Patients With Spinal Muscular Atrophy Type 3 Pyridostigmine Bromide - Spinal Muscular Atrophy Type 3 | Phase 2 | Completed | Centre Hospitalier Régional de la Citadelle | 4 | 2014-07-01 | 2023-10-11 |
Spinal Muscular Atrophies of Childhood (5) · Muscular Atrophy, Spinal (2) · Neuromuscular Manifestations (2) · Atrophy (1) · Muscular Atrophy (1) · Neuromuscular Diseases (1)
Central Nervous System Diseases (5) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Heredodegenerative Disorders, Nervous System (5) · Motor Neuron Disease (5) · Nervous System Diseases (5) · Neurodegenerative Diseases (5) · Spinal Cord Diseases (5) · Neuromuscular Diseases (4) · Muscular Atrophy, Spinal (3) · Neurologic Manifestations (2) · Pathological Conditions, Signs and Symptoms (2) · Signs and Symptoms (2) · Pathological Conditions, Anatomical (1)
apitegromab (2) · Amifampridine (1) · Pyridostigmine Bromide (1) · Risdiplam (1) · Tablets (1) · nusinersen (1)
Scholar Rock, Inc. (2) · Catalyst Pharmaceuticals, Inc. (1) · Centre Hospitalier Régional de la Citadelle (1) · GeneCradle Inc (1)
Italy (3) · Belgium (2) · Netherlands (2) · Spain (2) · United States (2) · China (1) · France (1) · United Kingdom (1)
Lorenzo Maggi, MD (1) · Stephanie Delstanche (1) · Thomas O. Crawford, MD (1)