NCT03124459

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease Types 1 and X

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 63 (actual)
• Sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Part 1 (non-randomized, open-label, dose-escalation) Part 1 will consist of up to 3 cohorts of 6 patients each and will evaluate multiple ascending dose levels of ACE-083 administered bilaterally once every 3 weeks for up to 5 doses in the tibialis anterior (TA) muscle. Patients in each cohort will be enrolled in a 4-week screening period before beginning treatment. Part 2 (randomized, double-blind, placebo-controlled) Prior to the initiation of Part 2, a review of safety and efficacy data from Part 1 will be conducted by the Safety Review Team (SRT) to determine the recommended dose level (maximum 250 mg/muscle). A total of up to 40 new patients may be enrolled and randomized (1:1 randomization) to receive either ACE 083 (n=20) or placebo (n=20) bilaterally by injection into both TA muscles once every 3 weeks for up to 17 doses. Study duration for Parts 1 and 2 for each patient will be approximately 24 weeks, including a 4-week screening period, a 12-week treatment period, and an 8-week follow-up period after the last dose. Study duration for Part 2 will be 15 months, including 4-week screening, 6 months double blind placebo-controlled, 6 months open-label and 8 week follow-up.

Conditions

• Charcot-Marie-Tooth Disease

Interventions

Type	Name	Description
DRUG	ACE-083	Part 1 - Recombinant fusion protein. Part 2 - Recombinant fusion protein or buffer solution.
DRUG	Placebo	Recombinant fusion protein or buffer solution

Timeline

• Start date: 2017-07-31
• Primary completion: 2020-03-11
• Completion: 2020-03-11
• First posted: 2017-04-21
• Last updated: 2022-09-26
• Results posted: 2021-07-28

Locations

16 sites across 1 country: United States

Regulatory

• FDA-regulated drug study