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).
What is the full name of this clinical trial?
A Multi-center, Randomized, Double-blind, Placebo-controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth Type 1A