Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Status:
Terminated
Trial end date:
2019-10-09
Target enrollment:
Participant gender:
Summary
Study A083-02 is a multi-center, Phase 2 study to evaluate the safety, tolerability,
pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of locally-acting ACE-083 in
patients with Facioscapulohumeral muscular dystrophy (FSHD) to be conducted in two parts.
Part 1 is open-label, dose-escalation and Part 2 is randomized, double-blind, and
placebo-controlled.