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

Study Overview
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Brief Summary
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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.

Official Title
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A Randomized, Double-Blind, Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of FLX-787-ODT for Treatment of Muscle Cramps in Adult Subjects With Charcot-Marie-Tooth Disease

Conditions
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Charcot-Marie-Tooth Disease

Intervention / Treatment
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* Drug: FLX-787-ODT (orally disintegrating tablet)
* Drug: Placebo ODT


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Presence of symptoms of CMT since at least 6 months prior to Screening, and confirmed diagnosis of CMT as defined by: Genetic confirmation of a mutation known to cause CMT, or 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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Experimental<br> | Drug: FLX-787-ODT (orally disintegrating tablet)<br>* FLX-787-ODT taken three times daily for 28 days<br>|
| Placebo Comparator: Placebo Comparator<br> | Drug: Placebo ODT<br>* Placebo ODT taken three times daily for 28 days<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cramp frequency | Cramp frequency measured over the 28-day treatment period | 28 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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CMT, Charcot-Marie-Tooth, Muscle Cramps, Muscle Cramping, FLX-787, Hereditary Sensory and Motor Neuropathy, Neurogenetic disorder, Nervous System Malformations, Nervous System Diseases, Neurodegenerative Diseases, Neuromuscular Diseases