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


This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with 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. Visit for more details. 


  • Drug: ACE-083

For study details, please contact Amanda Cowsert:


  • 18 years and older
  • Left and right ankle dorsiflexion Medical Research Council (MRC) manual muscle testing (MMT) grade 4- to 4+, inclusive
  • Females of childbearing potential must have negative urine pregnancy test prior to enrollment and use highly effective birth control methods during study participation and for 8 weeks following the last dose of ACE-083. Males must agree to use a condom during any sexual contact with females of childbearing potential while participating in the study and for 8 weeks following the last dose of ACE-083, even if he has undergone a successful vasectomy.
  • Ability to adhere to the study visit schedule/procedures, and to understand and comply with protocol requirements
  • Phase 1 and Phase 2 have specific eligibility requirements, contact the study team for more details

Additional criteria apply, please contact Amanda Cowsert:


18 to 65
65 and over



Can be done from home



Charcot-Marie Tooth, Charcot-Marie-Tooth disease, Neurology

Principal Investigator

S.H. Subramony, MD



Contact Information


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