Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
OPAL: Apitegromab in Spinal Muscular Atrophy (SMA) <2 Years Old
NCT07047144Phase 2Recruiting
- Sponsor
- Scholar Rock
- Start
- Sep 2025
- Primary completion
- Nov 2028
- Modality
- Monoclonal Antibodies
- Countries
- 7
01
Overview
Phase 2, randomised, double-blind trial of apitegromab in spinal muscular atrophy (SMA) (2 arms), sponsored by Scholar Rock. Primary endpoints: Pharmacokinetics of apitegromab at 52 weeks, Pharmacodynamics of apitegromab at 52 weeks and 1 more. Started Sep 2025; status: recruiting.
02
Drugs
apitegromab
03
Conditions
spinal muscular atrophy (SMA)
Beyond the preview
Go deeper on NCT07047144.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on NCT07047144.