Researchers are looking for people with type 1A Charcot-Marie-Tooth disease (CMT1A) to participate in a new pivotal phase 3 clinical trial of the investigational drug PXT3003. The trial aims to determine whether PXT3003 is effective and well-tolerated in people with CMT1A.
PXT3003 is an oral low-dose combination of three different drugs designed to target the genetic cause of CMT1A by reducing levels of PMP22 protein. If shown to be effective, it could become the first approved treatment designed to stop or slow the progression of the disease.
The study, which expects to enroll 300 participants, is taking place at 28 trial sites in the United States (California, Connecticut, Florida, Massachusetts, Minnesota, Missouri, New York and Washington) and Europe (France, Germany, United Kingdom, Spain, the Netherlands and Belgium). Trial participants will be treated with one of two doses of PXT3003 or placebo for 15 months.
Participants may be male or female, 16 to 65 years old, with a proven genetic diagnosis of CMT1A and must meet additional eligibility requirements.
To learn more about this trial, including site locations and complete inclusion and exclusion criteria, visit ClinicalTrials.gov and enter NCT02579759 into the search box.