Condition: Charcot-Marie-Tooth type 1A


Key Inclusion Criteria

  • 16-65 years old with genetically proven diagnosis of Charcot-Marie-Tooth type 1A (CMT1A)
  • Mild to moderate severity
  • Muscle weakness in at least foot dorsiflexion (backward bending and contracting of the foot)

Key Exclusion Criteria

  • Previously enrolled in any PXT3003 study
  • Living in the same household with individual enrolled in a PXT3003 study
  • CMT of any subtype other than 1A

Full Study Name

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A (CMT1A) (IRB no. 000001460)


The purpose of this study is to evaluate the effectiveness of an investigational drug called PXT3003 for the treatment of Charcot-Marie-Tooth type 1A (CMT1A disease). Researchers will compare PXT3003 to placebo (inactive substance) on the disease progression and symptoms of CMT1A, as well as determine whether the drug is tolerable and safe for patients with CMT1A.

CMT1A is caused by inherited mutations (changes) in genes that cause the peripheral nerves to become damaged, which leads to muscle weakness, numbness, and sensory loss. Participants will be randomly assigned to one of two treatment groups. Group 1 will receive PXT3003 twice daily, and Group 2 will receive a placebo (twice daily).

Principal Investigator