Campbell 2017 — ACE-031 in Duchenne Muscular Dystrophy

Muscle & Nerve·April 1, 2017

Craig Campbell, Hugh J. McMillan, Jean K. Mah, et al.

Summary

ACE-031 increased lean mass signals in DMD boys but development was limited by safety/tolerability concerns.

Study Details

Study Design

Randomized double-blind placebo-controlled ascending-dose trial

Indication

Duchenne muscular dystrophy

Intervention

Subcutaneous ACE-031 every 2–4 weeks

Species

Human

Risk of Bias Assessment

Trial halted historically over safety concerns; rare disease sample