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A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
NCT05337553 · View on ClinicalTrials.gov ↗
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 |
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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. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 269 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Biohaven Pharmaceuticals, which has 5 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT05337553 reports 20 study locations spanning 16 distinct geographic areas — top geographies include California, New York, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05337553?
The interventions under investigation include: Placebo (DRUG), taldefgrobep alfa (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05337553?
This trial is sponsored by Biohaven Pharmaceuticals, which has 5 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
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