A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Running, not enrolling · Phase 3 · Has a placebo group
Conditions studied: Spinal Muscular Atrophy, Neuromuscular Diseases, SMA
In brief
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.
Key facts
- Study ID
- NCT05337553
- Run by
- Biohaven Pharmaceuticals, Inc.
- People needed
- 269
- Starts
- 2022-07-06
- Expected to finish
- 2027-12-01
- Last updated by the study team
- 2026-06-01
Who can join
Age: 4 and older, up to 21. Sex: any. Healthy volunteers: not accepted.
Where it is running
- UCSD & Rady Children's — La Jolla, California, United States
- Children's Hospital of Los Angeles — Los Angeles, California, United States
- UCSF Benioff Children's Hospital, Medical Center — San Francisco, California, United States
- Children's Hospital Colorado — Aurora, Colorado, United States
- UF Health, Shands Hospital — Gainesville, Florida, United States
- Rare Disease Research — Atlanta, Georgia, United States
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
- Indiana University -Riley Research — Indianapolis, Indiana, United States
- University of Iowa — Iowa City, Iowa, United States
- University of Kansas Medical Center — Fairway, Kansas, United States
- Boston Children's Hospital - Harvard — Boston, Massachusetts, United States
- BSHS Office of Research — Grand Rapids, Michigan, United States
- Washington University in St. Louis — St Louis, Missouri, United States
- Columbia University Medical Center — New York, New York, United States
- Stony Brook University Hospital — Stony Brook, New York, United States
- Duke University Medicine — Durham, North Carolina, United States
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States
- Nationwide Children's Hospital — Columbus, Ohio, United States
- Penn State College of Medicine — Hershey, Pennsylvania, United States
- CHOP Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania, United States
- Vanderbilt University Medical Center — Nashville, Tennessee, United States
- UT Pediatric Neurosciences/Dell Children's Medical Center — Austin, Texas, United States
- Neurology Rare Disease Center — Denton, Texas, United States
- Phoenix Children's — Phoenix, Arizona, United States
Full record on ClinicalTrials.gov
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