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

Phase 2

Terminated

• Conditions: Charcot-Marie-Tooth Disease
• Interventions: Drug: FLX-787-ODT (orally disintegrating tablet); Drug: Placebo ODT

• Registration Number: NCT03254199
• Lead Sponsor: Flex Pharma, Inc.

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2017-08-16
• Study First Submitted QC: 2017-08-16
• Study First Posted: 2017-08-18
• Study Start: 2017-10-16
• Primary Completion: 2018-07-27
• Study Completion: 2018-07-27
• Status Verified: 2018-08
• Last Update Submitted: 2018-08-28
• Last Update Posted: 2018-08-29

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 27

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Experimental	FLX-787-ODT (orally disintegrating tablet)	-
Placebo Comparator	Placebo ODT	-

Primary Outcome Measures

Name	Time	Method
Cramp frequency	28 days	Cramp frequency measured over the 28-day treatment period

Trial Locations

Locations (19)

University of Colorado; 🇺🇸 Aurora, Colorado, United States

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

University of Rochester Medical Center; 🇺🇸 Rochester, New York, United States

The University of Utah; 🇺🇸 Salt Lake City, Utah, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States

Oregon Health Science University; 🇺🇸 Portland, Oregon, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Saint Louis University; 🇺🇸 Saint Louis, Missouri, United States

Massachusetts General Hospital; 🇺🇸 Boston, Massachusetts, United States

Brighman and Women's Hospital; 🇺🇸 Boston, Massachusetts, United States

Temple University; 🇺🇸 Philadelphia, Pennsylvania, United States

Hospital for Special Care; 🇺🇸 New Britain, Connecticut, United States

Mayo Clinic; 🇺🇸 Jacksonville, Florida, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

Penn State Milton S. Hershey Medical Center; 🇺🇸 Hershey, Pennsylvania, United States

Saint Luke's Rehabilitation Institute; 🇺🇸 Spokane, Washington, United States

University of Vermont Medical Center; 🇺🇸 Burlington, Vermont, United States

University of Kansas Medical Center; 🇺🇸 Kansas City, Kansas, United States