How many clinical trials are tracked for spinal muscular atrophy?

28 trials are tracked, 6 with published results.

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Spinal muscular atrophy

Part of Motor neuron disease

1 published article · Updated continuously

Clinical Trial Landscape

Clinical Trials for spinal muscular atrophy

28 trials tracked for spinal muscular atrophy: 9 in phase 3 or 4 and 6 with published results. The most-cited published study has 1417 citations.

28Trials tracked

9Phase 3 & 4

0Recruiting

6With published results

Phase distribution

Phase 4 2 Phase 3 7 Phase 2 15 Phase 1 2 Other / NA 2

Phase 3 A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) Completed · 1,417 cited
Phase 3 Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 Completed · 293 cited
Phase 3 Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Completed · 29 cited
Phase 3 A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies Completed · 20 cited
Phase 3 Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Completed · 20 cited
Phase 3 Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam Completed · 8 cited

Show 22 more trials

Phase 4 Flu Vaccine Study in Neuromuscular Patients 2011 Completed
Phase 4 Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Completed
Phase 3 Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients Completed
Phase 2 A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients Completed
Phase 2 A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients Completed
Phase 2 A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy Completed
Phase 2 Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy Completed
Phase 2 A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) Completed
Phase 2 A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Completed
Phase 2 A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA) Completed
Phase 2 A Study of CK-2127107 in Patients With Spinal Muscular Atrophy Completed
Phase 2 An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy Completed
Phase 2 An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Completed
Phase 2 A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy Completed
Phase 2 Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy Completed
Phase 2 Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Completed
Phase 2 Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy Completed
Phase 2 An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) Completed
Phase 1 A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function Completed
Phase 1 A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants Completed
N/A Progressive Strength Training in Spinal Muscular Atrophy Completed
N/A WREX Outcome Study Completed

Showing the 28 most-cited and recently-updated of 28 trials. Browse the full registry →

Trial data sourced from ClinicalTrials.gov. Counts describe the research landscape and are not a treatment recommendation. Informational only — not medical advice.