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NCT05626855 · ClinicalTrials.gov registry record · Phase 3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
A Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
- Active
- Registry status
- Phase 3
- Development phase
- 238
- Enrollment target
- 20
- Study locations
NCT05626855: Active Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, sponsored by Scholar Rock.
NCT05626855 is a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases that is active but no longer recruiting, run by Scholar Rock. The registered enrollment target is 238 participants, above the 98-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (143% higher). The trial reports 20 study locations across 18 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT05626855, a Phase 3 study of Spinal Muscular Atrophy and Neuromuscular Diseases, is active but no longer recruiting, sponsored by Scholar Rock.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 3
- Development phase
- 238 participants
- Enrollment target
- 20
- Study locations
Study Summary
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.
Primary Outcome
Incidence of TEAEs and SAEs by severity
Conditions Studied
Interventions
- DRUG Apitegromab
Study Locations (20)
California
- UCSD Altman Clinical and Translational Research - La Jolla
- Children's Hospital Los Angeles - Los Angeles
- Stanford Neuroscience Health Center - Palo Alto
Arizona
- Phoenix Childrens Hospital - Phoenix
Colorado
- Children's Hospital Colorado - Aurora
Florida
- Nemours Biomedical Research - Orlando
Illinois
- Lurie Children's Hospital of Chicago - Chicago
Iowa
- University of Iowa - Iowa City
Kansas
- University of Kansas Medical Center - Fairway
Maryland
- Johns HopkinsHospital - Baltimore
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 238 participants |
| Start Date | 2023-04-17 |
| Est. Completion | 2029-05-02 |
| Phase | Phase 3 |
What the registry record for NCT05626855 still lists
NCT05626855 is an interventional study that assigns participants to a tested intervention. The registered 238 participants enrollment target is mid-sized for trials with a published cap, above the 98-participant average among 22 other Spinal Muscular Atrophy trials with a reported enrollment target (143% higher).
The record links to 10 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention - of which Apitegromab is the first listed.
NCT05626855 names 20 study sites across 18 states, led by California, Arizona, Colorado.
Frequently Asked Questions
What is clinical trial NCT05626855 about?
NCT05626855 is a clinical study titled "Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab". 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.
What is the current status of trial NCT05626855?
This trial is currently active not recruiting. It is a Phase 3 study. The enrollment target is 238 participants. The study started on 2023-04-17. Estimated completion is 2029-05-02.
What conditions does trial NCT05626855 study?
This clinical trial studies the following conditions: Spinal Muscular Atrophy, Neuromuscular Diseases, Muscular Atrophy, Spinal, Spinal Muscular Atrophy Type 3, Neuromuscular Manifestations.
What interventions are being tested in trial NCT05626855?
The interventions under investigation include: Apitegromab (DRUG).
Who is sponsoring clinical trial NCT05626855?
This trial is sponsored by Scholar Rock, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05626855 being conducted?
This trial has 20 study locations across Arizona, California, Colorado, Florida, Illinois. 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.
Where NCT05626855's enrollment target sits among peer trials
238 3rd of 22 higher than 20 of 22 other Spinal Muscular Atrophy trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Spinal Muscular Atrophy trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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