Key Insights

Highlights

Success Rate

93% trial completion (above average)

Published Results

21 trials with published results (14%)

Research Maturity

71 completed trials (48% of total)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 40/100

Termination Rate

3.4%

5 terminated out of 149 trials

Success Rate

93.4%

+6.9% vs benchmark

Late-Stage Pipeline

11%

16 trials in Phase 3/4

Results Transparency

30%

21 of 71 completed with results

Key Signals

21 with results93% success

Data Visualizations

Phase Distribution

86Total
Not Applicable (42)
Early P 1 (1)
P 1 (12)
P 2 (15)
P 3 (12)
P 4 (4)

Trial Status

Completed71
Recruiting24
Unknown24
Active Not Recruiting18
Terminated5
Withdrawn3

Trial Success Rate

93.4%

Benchmark: 86.5%

Based on 71 completed trials

Clinical Trials (149)

Showing 20 of 20 trials
NCT07047144Phase 2RecruitingPrimary

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

NCT03689660Not ApplicableCompleted

Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback

NCT07547189Not ApplicableActive Not RecruitingPrimary

Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy

NCT07444476Phase 3RecruitingPrimary

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

NCT05232929Phase 4Active Not RecruitingPrimary

Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

NCT05866419Not ApplicableRecruitingPrimary

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

NCT05794139Phase 2Active Not RecruitingPrimary

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

NCT07448610Phase 4Not Yet RecruitingPrimary

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)

NCT07478172Not ApplicableRecruiting

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

NCT05219487CompletedPrimary

Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration

NCT04139343CompletedPrimary

Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)

NCT06955897RecruitingPrimary

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

NCT05337553Phase 3Active Not RecruitingPrimary

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

NCT06321965Not ApplicableRecruitingPrimary

Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

NCT03505099Phase 3CompletedPrimary

Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2

NCT03381729Phase 1TerminatedPrimary

Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy

NCT05156320Phase 3CompletedPrimary

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

NCT05386680Phase 3CompletedPrimary

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

NCT00961103CompletedPrimary

Motor Development and Orthoses in Spinal Muscular Atrophy (SMA)

NCT04773470Not ApplicableCompletedPrimary

Implementation of FEES in Spinal Muscle Atrophy

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