🏛️ Verified NIH Record • Updated 2025-05-23
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab (NCT05626855)
An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Scholar Rock, Inc.
🩺 Plain-English Summary (8th-Grade Level)
The ONYX study is an Open-Label, Multicenter, Extension study that will evaluate the long-term safety and efficacy of Apitegromab in Patients with Type 2 and Type 3 SMA who have completed TOPAZ or SAPPHIRE.
Who can join: Review the eligibility criteria below.
Location: Phoenix Childrens Hospital — Phoenix, Arizona
Age range: 2 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 (49 Sites)
Showing the first 25 of 49 sites.
- Phoenix Childrens Hospital — Phoenix, Arizona
- UCSD Altman Clinical and Translational Research — La Jolla, California
- Children's Hospital Los Angeles — Los Angeles, California
- Stanford Neuroscience Health Center — Palo Alto, California
- Children's Hospital Colorado — Aurora, Colorado
- Nemours Biomedical Research — Orlando, Florida
- Lurie Children's Hospital of Chicago — Chicago, Illinois
- University of Iowa — Iowa City, Iowa
- University of Kansas Medical Center — Fairway, Kansas
- Johns HopkinsHospital — Baltimore, Maryland
- Boston Children's Hospital — Boston, Massachusetts
- Helen DeVos Children's Hospital — Grand Rapids, Michigan
- Gillette Children's Specialty Healthcare — Saint Paul, Minnesota
- Washington University Medical Campus — St Louis, Missouri
- Columbia University Medical Center — New York, New York
- Wake Forest University School of Medicine — Winston-Salem, North Carolina
- Nationwide Children's Hospital — Columbus, Ohio
- Oregon Health & Science University — Portland, Oregon
- The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- St. Jude Children's Research Hospital — Memphis, Tennessee
- University of Texas Southwestern - Pediatric Neurology — Dallas, Texas
- University of Utah — Salt Lake City, Utah
- Children's Specialty Group PLLC (Children's Hospital of The King's Daughters) — Newport News, Virginia
- Seattle Children's Hospital — Seattle, Washington
- University of Wisconsin Hospital — Madison, Wisconsin
💰 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
- 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.