Trials / Active Not Recruiting
Active Not RecruitingNCT05626855
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
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
- Status
- Active Not Recruiting
- Phase
- Phase 3
- Study type
- Interventional
- Enrollment
- 238 (actual)
- Sponsor
- Scholar Rock, Inc. · Industry
- Sex
- All
- Age
- 2 Years
- Healthy volunteers
- Not accepted
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
- Spinal Muscular Atrophy
- Spinal Muscular Atrophy Type 3
- Spinal Muscular Atrophy Type 2
- SMA
- Neuromuscular Diseases
- Muscular Atrophy
- Atrophy
- Muscular Atrophy, Spinal
- Neuromuscular Manifestations
- Anti-myostatin
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Apitegromab | Apitegromab (SRK-015) is an investigational, fully human immunoglobulin G4 monoclonal antibody that specifically binds to human proforms (i.e., inactive precursor forms) of myostatin, pro- and latent- myostatin, with high affinity, inhibiting activation of myostatin, a negative regulator of muscle growth and strength. |
Timeline
- Start date
- 2023-04-17
- Primary completion
- 2026-11-01
- Completion
- 2029-05-02
- First posted
- 2022-11-25
- Last updated
- 2025-05-25
Locations
49 sites across 9 countries: United States, Belgium, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT05626855. Inclusion in this directory is not an endorsement.