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NCT05337553 · ClinicalTrials.gov registry record · Phase 3
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
A Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Biohaven Pharmaceuticals.
- Active
- Registry status
- Phase 3
- Development phase
- 269
- Enrollment target
- 20
- Study locations
NCT05337553: Active Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Biohaven Pharmaceuticals.
NCT05337553 is a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases that is active but no longer recruiting, run by Biohaven Pharmaceuticals. The registered enrollment target is 269 participants, above the 97-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (177% higher). The trial reports 20 study locations across 16 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05337553, a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, is active but no longer recruiting, sponsored by Biohaven Pharmaceuticals.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 269 participants
- Enrollment target
- 20
- Study locations
Study Summary
This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.
Primary Outcome
Change in MFM-32 total score from baseline to Week 48. Scores range from 0-3 on each item. The scores from the 32 items are summed and transformed to a 0-100 scale, with higher scores reflecting higher levels of functional abilities.
Conditions Studied
Interventions
- DRUG Placebo
- DRUG taldefgrobep alfa
Study Locations (20)
California
- UCSD & Rady Children's - La Jolla
- Children's Hospital of Los Angeles - Los Angeles
- UCSF Benioff Children's Hospital, Medical Center - San Francisco
New York
- Columbia University Medical Center - New York
- Stony Brook University Hospital - Stony Brook
Ohio
- Cincinnati Children's Hospital Medical Center - Cincinnati
- Nationwide Children's Hospital - Columbus
Arizona
- Phoenix Children's - Phoenix
Colorado
- Children's Hospital Colorado - Aurora
Florida
- UF Health, Shands Hospital - Gainesville
Georgia
- Rare Disease Research - Atlanta
Illinois
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 269 participants |
| Start Date | 2022-07-06 |
| Est. Completion | 2026-06 |
| Phase | Phase 3 |
What the registry record for NCT05337553 still lists
NCT05337553 is an interventional study that assigns participants to a tested intervention. The registered 269 participants enrollment target is mid-sized for trials with a published cap, above the 97-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (177% higher).
The record links to 3 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 2 interventions - of which Placebo is the first listed.
NCT05337553 names 20 study sites across 16 states, led by California, New York, Ohio.
Frequently Asked Questions
What is clinical trial NCT05337553 about?
NCT05337553 is a clinical study titled "A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy". This trial will study the efficacy and safety of taldefgrobep alfa as an adjunctive therapy for participants who are already taking a stable dose of nusinersen and/or risdiplam and/or have a history of onasemnogene abeparvovec, compared to placebo.
What is the current status of trial NCT05337553?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 269 participants. The study started on 2022-07-06. Estimated completion is 2026-06.
What conditions does trial NCT05337553 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy, Neuromuscular Diseases, SMA.
What interventions are being tested in trial NCT05337553?
The interventions under investigation include: Placebo (DRUG), taldefgrobep alfa (DRUG).
Who is sponsoring clinical trial NCT05337553?
This trial is sponsored by Biohaven Pharmaceuticals, which has 12 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05337553 being conducted?
This trial has 20 study locations across Arizona, California, Colorado, Florida, Georgia. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Spinal Muscular Atrophy
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT05337553's enrollment target sits among peer trials
269 2nd of 22 the highest of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
-
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
ACTIVE NOT RECRUITING · Phase 3
-
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
ACTIVE NOT RECRUITING · Phase 3
-
Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam
COMPLETED · Phase 3
-
Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
RECRUITING
-
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
RECRUITING · NA
-
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
RECRUITING · Phase 2
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