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

This is an interventional treatment trial for Charcot-Marie-Tooth Disease focused on measuring Charcot-Marie-Tooth, CMT, Muscle Cramps, Muscle Cramping, FLX-787, Hereditary Sensory and Motor Neuropathy, Neurogenetic disorder, Nervous System Malformations, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases Read More

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