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Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
NCT05626855 · View on ClinicalTrials.gov ↗
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05626855
The ClinicalTrials.gov registry entry for NCT05626855 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 238 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Scholar Rock, 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 10 conditions, with Spinal Muscular Atrophy appearing as the primary indexed condition, and to 1 intervention — of which Apitegromab 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: NCT05626855 reports 20 study locations spanning 18 distinct geographic areas — top geographies include California, Arizona, Colorado. 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 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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT05626855?
The interventions under investigation include: Apitegromab (DRUG). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT05626855?
This trial is sponsored by Scholar Rock, 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 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.
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