8 completed, 4 recruiting, 2 active not recruiting, 2 terminated. 5 Phase 2, 4 Not applicable, 2 Phase 3, 1 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 5 | Completed 2, Recruiting 2, Terminated 1 |
| Not applicable | 4 | Completed 2, Terminated 1, Unknown 1 |
| Phase 3 | 2 | Active not recruiting 1, Completed 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 |
|---|---|---|---|---|---|---|
| Gait and Bone Health in SMA Spinal Muscular Atrophy Type 3, Ambulatory Spinal Muscular Atrophy | Recruiting | Jacqueline Montes | 22 | 2026-03-05 | 2026-03-18 | |
| 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 |
| Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads) - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Not applicable | Completed | Marco Capogrosso | 3 | 2024-05-31 | 2026-10-23 |
| 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 |
| Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy (DMD) | Recruiting | Columbia University | 106 | 2024-05-01 | 2026-04-09 | |
| Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab Apitegromab - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Phase 3 | Active not recruiting | Scholar Rock, Inc. | 238 | 2023-04-17 | 2026-09-01 |
| The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy Aerobic Exercise Training, Home exercise program - Neuromuscular Diseases, Spinal Muscular Atrophy Type 3 | Not applicable | Unknown | Istanbul Medipol University Hospital | 20 | 2022-07-28 | 2023-09-28 |
| Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam Apitegromab, Placebo - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3 | Phase 3 | Completed | Scholar Rock, Inc. | 188 | 2022-04-14 | 2026-01-22 |
| Spinal Cord Stimulation in Spinal Muscular Atrophy Spinal Cord Stimulator (octopolar Medtronic Vectris Leads) - Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4 | Not applicable | Completed | Marco Capogrosso | 3 | 2022-04-05 | 2026-03-16 |
| Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease Beck depression inventory fast screen (Questionnaire), Brief Pain Inventory (BPI) (Questionnaire), German Pain Inventory (Questionnaire) - Pompe Disease (Late-onset), Inclusion Body Myositis | Unknown | LMU Klinikum | 95 | 2022-03-31 | 2023-10-10 | |
| Musculoskeletal Nociceptive Pain in Participants With Neuromuscular Disorders Beck depression inventory fast screen, German Pain Inventory, Brief Pain Inventory - Pompe Disease (Late-onset), Myotonic Dystrophy Type 1 (DM1) | Completed | LMU Klinikum | 82 | 2021-04-15 | 2022-08-24 | |
| Wearable Technology to Assess Gait Function in SMA and DMD Observational - Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy | Completed | Columbia University | 39 | 2019-11-25 | 2024-09-23 | |
| 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 |
| Spinraza in Adult Spinal Muscular Atrophy Observational study to examine safety, tolerability, and effectiveness of SPINRAZA® prescribed as part of standard of care, One time survey - Spinal Muscular Atrophy, Spinal Muscular Atrophy Type II | Active not recruiting | Washington University School of Medicine | 148 | 2018-08-16 | 2024-11-20 | |
| Whole Body Vibration Therapy in Children With Spinal Muscular Atrophy whole body vibration therapy - Spinal Muscular Atrophy Type 3 | Not applicable | Terminated | The Hong Kong Polytechnic University | 1 | 2017-08-01 | 2019-07-26 |
| Oxidative Capacity and Exercise Tolerance in Ambulatory SMA Spinal Muscular Atrophy Type 3, Mitochondrial Myopathy | Completed | Columbia University | 42 | 2016-11-01 | 2022-03-08 | |
| 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 (18) · Muscular Atrophy, Spinal (12) · Neuromuscular Diseases (7) · Neuromuscular Manifestations (4) · Atrophy (3) · Muscular Atrophy (3) · Glycogen Storage Disease Type II (2) · Muscular Dystrophy, Duchenne (2) · Myositis, Inclusion Body (2) · Fractures, Bone (1) · Mitochondrial Myopathies (1) · Motor Activity (1) · Myotonic Dystrophy (1) · Nociceptive Pain (1) · Spinal muscular atrophy 4 (1)
Central Nervous System Diseases (18) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (18) · Genetic Diseases, Inborn (18) · Heredodegenerative Disorders, Nervous System (18) · Motor Neuron Disease (18) · Nervous System Diseases (18) · Neurodegenerative Diseases (18) · Spinal Cord Diseases (18) · Neuromuscular Diseases (11) · Muscular Atrophy, Spinal (6) · Muscular Diseases (5) · Musculoskeletal Diseases (5) · Neurologic Manifestations (5) · Pathological Conditions, Signs and Symptoms (5) · Signs and Symptoms (5)
apitegromab (4) · Vital Signs (2) · Walk Test (2) · Amifampridine (1) · Blood Specimen Collection (1) · Pyridostigmine Bromide (1) · Risdiplam (1) · Tablets (1) · Watchful Waiting (1) · nusinersen (1)
Scholar Rock, Inc. (4) · Columbia University (3) · LMU Klinikum (2) · Marco Capogrosso (2) · Catalyst Pharmaceuticals, Inc. (1) · Centre Hospitalier Régional de la Citadelle (1) · GeneCradle Inc (1) · Istanbul Medipol University Hospital (1) · Jacqueline Montes (1) · The Hong Kong Polytechnic University (1) · Washington University School of Medicine (1)
United States (11) · Italy (5) · Belgium (4) · Germany (4) · Netherlands (4) · Spain (4) · France (3) · United Kingdom (3) · Poland (2) · Canada (1) · China (1) · Hong Kong (1) · Turkey (Türkiye) (1)
Jacqueline Montes, PT, EdD (2) · Marco Capogrosso (2) · Stephan Wenninger, PD Dr. med. (2) · Craig Zaidman, MD (1) · Fatma Karantay Mutluay,, Professor (1) · Lorenzo Maggi, MD (1) · Stephanie Delstanche (1) · Tamis W Pin, PhD (1) · Thomas O. Crawford, MD (1)
PMID 38330285, PMID 39910271, PMID 40557477, PMID 40818473
K01HD084690 (1), R01HD112908 (1)