Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Status:
Terminated
Trial end date:
2020-03-11
Target enrollment:
Participant gender:
Summary
This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics
(PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with Charcot-Marie-Tooth
Disease Type 1 and Type X (CMT1 and CMTX), to be conducted in two parts. Part 1 is
non-randomized, open-label, dose-escalation and Part 2 is randomized, double-blind, and
placebo-controlled.