🏛️ Verified NIH Record • Updated 2026-05-29
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy (NCT05337553)
A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants With Spinal Muscular Atrophy With Open-Label Extension
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Biohaven Pharmaceuticals, Inc.
🩺 Plain-English Summary (8th-Grade Level)
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.
Who can join: Review the eligibility criteria below.
Location: Phoenix Children's — Phoenix, Arizona
Age range: 4 Years to 21 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (53 Sites)
Showing the first 25 of 53 sites.
- Phoenix Children's — Phoenix, Arizona
- UCSD & Rady Children's — La Jolla, California
- Children's Hospital of Los Angeles — Los Angeles, California
- UCSF Benioff Children's Hospital, Medical Center — San Francisco, California
- Children's Hospital Colorado — Aurora, Colorado
- UF Health, Shands Hospital — Gainesville, Florida
- Rare Disease Research — Atlanta, Georgia
- Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- Indiana University -Riley Research — Indianapolis, Indiana
- University of Iowa — Iowa City, Iowa
- University of Kansas Medical Center — Fairway, Kansas
- Boston Children's Hospital - Harvard — Boston, Massachusetts
- BSHS Office of Research — Grand Rapids, Michigan
- Washington University in St. Louis — St Louis, Missouri
- Columbia University Medical Center — New York, New York
- Stony Brook University Hospital — Stony Brook, New York
- Duke University Medicine — Durham, North Carolina
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Nationwide Children's Hospital — Columbus, Ohio
- Penn State College of Medicine — Hershey, Pennsylvania
- CHOP Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- UPMC Children's Hospital of Pittsburgh — Pittsburgh, Pennsylvania
- Vanderbilt University Medical Center — Nashville, Tennessee
- UT Pediatric Neurosciences/Dell Children's Medical Center — Austin, Texas
- Neurology Rare Disease Center — Denton, Texas
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Spinal Muscular Atrophy
- Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) — Phoenix, Arizona
- Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb — Pittsburgh, Pennsylvania
- 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) — Phoenix, Arizona
- Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab — Phoenix, Arizona
- Spinraza in Adult Spinal Muscular Atrophy — Phoenix, Arizona
- Early Check: Expanded Screening in Newborns — Research Triangle Park, North Carolina
Explore by condition: All Spinal Muscular Atrophy clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.