Trial to Test Drug Targeting Muscle Cramps in CMT Seeks Participants

Researchers are looking for people with Charcot-Marie-Tooth disease (CMT) to participate in a phase 2 clinical trial to evaluate the safety and effectiveness of the investigational drug FLX-787-ODT.

FLX-787-ODT, under development by Flex Pharma, is taken in tablet form and is designed to reduce muscle cramps in adults with CMT.

Trial length is approximately three months, during which participants will visit with study investigators approximately 20 times. For the duration of the study, participants will record muscle cramp frequency in a diary over the 28-day treatment period.

In order to be eligible to participate, individuals must:

  • be at least 18 years old
  • have a documented genetic confirmation of a mutation known to cause CMT or clinical evidence of CMT and a genetic confirmation in a family member
  • experience weekly muscle cramping
  • meet additional criteria

The trial is taking place at more than a dozen study sites across the United States, and support for travel costs may be available.

To learn more about this trial, visit and enter NCT03254199 into the “Other Terms” search box.