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

Phase 2 Terminated 63 enrolled Acceleron Pharma, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

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.

Timeline

Start: 2017-07-31
Primary completion: 2020-03-11
Completion: 2020-03-11

Outcome

Met primary endpoint

Stopped: “(Investigation of ACE-083 for use in patients with CMT is being discontinued as it did not achieve functional secondary endpoints in the A083-03 trial.”

registry analysis (superiority test); Part 2 (Double-blind Placebo Controlled) Placebo Arm vs Part 2 (Double-blind Placebo Controlled) ACE-083 Arm; p = 0.01; Mean Difference (Final Values) 13.5 (90% CI 4.9 to 22.1) NCT03124459 ↗

paper there was significantly greater change in TMV with ACE-083 compared with placebo (least-squares mean difference 13.5%; p = 0.0096). PMID 35545446 ↗

Publications

Thomas FP, Brannagan TH 3rd, Butterfield RJ, Desai U, Habib AA, Herrmann DN, Eichinger KJ, Johnson NE, Karam C, Pestronk A, Quinn C, Shy ME, Statland JM, Subramony SH, Walk D, Stevens-Favorite K, Miller B, Leneus A, Fowler M, van de Rijn M, Attie KM. Randomized Phase 2 Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease. Neurology. 2022 Jun 6;98(23):e2356-e2367. doi: 10.1212/WNL.0000000000200325.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	EFMITERMANT ALFA	Protein / enzyme biologic	150 mg	Intramuscular
Subject	EFMITERMANT ALFA	Protein / enzyme biologic	200 mg	Intramuscular
Subject	EFMITERMANT ALFA	Protein / enzyme biologic	240 mg	Intramuscular
Subject	EFMITERMANT ALFA	Protein / enzyme biologic	250 mg	Intramuscular