A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.

• Status: Terminated
• Trial end date: 2018-07-27
• Target enrollment: 0
• Participant gender: All

Summary

The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps. Participants will be asked to take two study products during the course of the study. One of these study products will be a placebo.

Approximately 120 participants in 20 study centers across the United States are expected to take part. Participants will be in the study for approximately 3 months and visit the study clinic 3 times.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Flex Pharma, Inc.

Criteria

Inclusion Criteria:

• Presence of symptoms of CMT since at least 6 months prior to Screening, and confirmed diagnosis of CMT as defined by:

1. Genetic confirmation of a mutation known to cause CMT, or

2. Clinical and electrophysiological evidence of CMT and a genetic confirmation in a family member. Clinical features include length dependent sensory and motor loss, with sensorimotor axonal or demyelinating changes on a nerve conduction study.

• Weekly muscle cramping (defined as: a sustained muscle contraction that's most often painful and lasts seconds to minutes)

Exclusion Criteria:

• Presence of major gastrointestinal disorders, such as inflammatory bowel disease, diverticulitis, active peptic ulcer disease, or significant gastroesophageal reflux disease (i.e., not well-controlled on antacids or proton pump inhibitors), or oral or esophageal lesions/ulcers

• Presence of significant swallowing problems

• Unable or unwilling to discontinue medications for cramps and/or opiates

• Inability to tolerate a spicy sensation in the mouth or stomach

• Actively using illicit drugs or history of chronic substance abuse within the past year prior to screening, including abuse of alcohol

• Intention to change the current level of tobacco use or use of nicotine-containing products (i.e., new smokers or those actively trying to quit may not enrolled)

• Participated in a clinical study (except natural history studies without administration of an investigational product) within 30 days prior to screening