For research and educational purposes only. Not medical advice.
ACE-031 Reference
Educational, not medical advice reference for ACE-031: Growth Factor; regulatory status, evidence posture, source review, and schedule notes. Also known as R…
Reference summary
Campbell 2017 Muscle Nerve published the abbreviated Phase 2 dataset (24 ambulatory boys with DMD). Despite the early termination, the trial reported a non-significant trend toward increased lean body mass and 6-minute walk distance; the safety signals (epistaxis, telangiectasia) drove the program halt. There is no Phase 3 trial and no later follow-up program.
Regulatory and posture
- Categories
- Growth Factor
- Aliases
- Ramatercept, Soluble activin receptor type IIB-Fc (ACVR2B-Fc) fusion protein, Acceleron Pharma development code
- Evidence posture
- human - Phase 2 trial halted early due to vascular-pattern safety signals; development discontinued.
- Regulatory status
- Not FDA-approved. Development discontinued. ACE-031 was a soluble activin receptor type IIB-Fc fusion protein developed by Acceleron Pharma as a systemic myostatin pathway inhibitor for muscular dystrophy. The Phase 2 trial in ambulatory boys with Duchenne muscular dystrophy was halted in 2010 due to non-clinical safety signals (epistaxis, gum bleeding, telangiectasia) consistent with off-target effects on vascular biology. Acceleron discontinued the program. WADA-prohibited at all times as a myostatin-pathway agent (S4).
- Content review status
- research reference