Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
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 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.
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.
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 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05337553
The ClinicalTrials.gov registry entry for NCT05337553 describes a study currently listed as active not recruiting, categorized as Phase 3. The registered enrollment target is 269 participants, a figure that helps gauge the scale of data the investigators plan to collect, above the 97-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (177% higher). The listed sponsor is Biohaven Pharmaceuticals, which has 12 total studies on file at ClinicalTrials.gov.
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 reports 20 study locations spanning 16 distinct geographic areas - top geographies include 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.
-
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
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.