Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)

Summary

This is an open-label, multicenter, phase 2 extension study to evaluate the safety, tolerability, PK, PD, and efficacy of ACE-083 in subjects with FSHD previously enrolled in Study A083-02 and subjects with CMT1 and CMTX previously enrolled in Study A083-03. This study will be conducted in two Parts: Part 1, which is a loading phase of 6 months' duration, and Part 2, the maintenance phase, which will last up to 24 months.

Timeline

Start

2019-05-10

Primary completion

2020-03-11

Completion

2020-03-11

Outcome

Outcome not reported

Stopped: “Investigation of ACE-083 for use in patients with CMT and FSHD is being discontinued in this extension study as functional secondary endpoints were not achieved in the A083-023 or A083-03 trial (the parent trials).”

Drugs

Indications