Clinical Trial

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Study acronym: ONYX
Active, Not Recruiting Phase 3
View on ClinicalTrials.gov →
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.
Protocol Amendment History 28 amendments
This ClinicalTrials.gov record has been amended 28 times since 2022-11-15; most recent amendment 2025-05-23.
Status change: Not Yet Recruiting → Active, Not Recruiting 2023-04-21
Trial Details
NCT Number NCT05626855
Lead Sponsor Scholar Rock, Inc.
Conditions Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal +2 more
Enrollment 238 participants
Start Date 2023-04-17
Primary Completion 2026-11-01 (estimated)
Study Completion 2029-05-02 (estimated)
Updated on ClinicalTrials.gov 2025-05-25