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

Charcot-Marie-Tooth Disease Clinical Trial

— COMMIT  

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03254199
• Other study ID #: FLX-787-204
• Status: Terminated
• Phase: Phase 2
• Start date: October 16, 2017
• Est. completion date: July 27, 2018

Study information

• Verified date: August 2018
• Source: Flex Pharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 27
• Est. completion date: July 27, 2018
• Est. primary completion date: July 27, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Charcot-Marie-Tooth Disease
• Hereditary Sensory and Motor Neuropathy
• Muscle Cramp
• Nerve Compression Syndromes
• Spasm
• Tooth Diseases

Intervention

Drug:
• FLX-787-ODT (orally disintegrating tablet)
FLX-787-ODT taken three times daily for 28 days
• Placebo ODT
Placebo ODT taken three times daily for 28 days

Locations

Country	Name	City	State
United States	University of Colorado	Aurora	Colorado
United States	Brighman and Women's Hospital	Boston	Massachusetts
United States	Massachusetts General Hospital	Boston	Massachusetts
United States	University of Vermont Medical Center	Burlington	Vermont
United States	University of Florida	Gainesville	Florida
United States	Penn State Milton S. Hershey Medical Center	Hershey	Pennsylvania
United States	University of Iowa	Iowa City	Iowa
United States	Mayo Clinic	Jacksonville	Florida
United States	University of Kansas Medical Center	Kansas City	Kansas
United States	University of Minnesota	Minneapolis	Minnesota
United States	Hospital for Special Care	New Britain	Connecticut
United States	Columbia University Medical Center	New York	New York
United States	Temple University	Philadelphia	Pennsylvania
United States	Oregon Health Science University	Portland	Oregon
United States	University of Rochester Medical Center	Rochester	New York
United States	Saint Louis University	Saint Louis	Missouri
United States	The University of Utah	Salt Lake City	Utah
United States	Mayo Clinic	Scottsdale	Arizona
United States	Saint Luke's Rehabilitation Institute	Spokane	Washington
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Flex Pharma, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Cramp frequency	Cramp frequency measured over the 28-day treatment period	28 days