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

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ClinicalTrials.gov Identifier: NCT03254199
Recruitment Status : Active, not recruiting
First Posted : August 18, 2017
Last Update Posted : June 14, 2018
Sponsor:
Information provided by (Responsible Party):
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.


Condition or disease Intervention/treatment Phase
Charcot-Marie-Tooth Disease Drug: FLX-787-ODT (orally disintegrating tablet) Drug: Placebo ODT Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Masking Description: Double-blind
Primary Purpose: Treatment
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
Actual Study Start Date : October 16, 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : August 2018


Arm Intervention/treatment
Experimental: Experimental Drug: FLX-787-ODT (orally disintegrating tablet)
FLX-787-ODT taken three times daily for 28 days

Placebo Comparator: Placebo Comparator Drug: Placebo ODT
Placebo ODT taken three times daily for 28 days




Primary Outcome Measures :
  1. Cramp frequency [ Time Frame: 28 days ]
    Cramp frequency measured over the 28-day treatment period



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03254199


Locations
United States, Arizona
Mayo Clinic
Scottsdale, Arizona, United States, 85259
United States, Colorado
University of Colorado
Aurora, Colorado, United States, 80045
United States, Connecticut
Hospital for Special Care
New Britain, Connecticut, United States, 06053
United States, Florida
University of Florida
Gainesville, Florida, United States, 32607
Mayo Clinic
Jacksonville, Florida, United States, 32224
University of South Florida
Tampa, Florida, United States, 33612
United States, Iowa
University of Iowa
Iowa City, Iowa, United States, 52242
United States, Kansas
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Brighman and Women's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
University of Minnesota
Minneapolis, Minnesota, United States, 55455
United States, Missouri
Saint Louis University
Saint Louis, Missouri, United States, 63104
United States, New York
Columbia University Medical Center
New York, New York, United States, 10032
University of Rochester Medical Center
Rochester, New York, United States, 14642
United States, Oregon
Oregon Health Science University
Portland, Oregon, United States, 97239
United States, Pennsylvania
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States, 17033
Temple University
Philadelphia, Pennsylvania, United States, 19140
United States, Utah
The University of Utah
Salt Lake City, Utah, United States, 84112
United States, Vermont
University of Vermont Medical Center
Burlington, Vermont, United States, 05405
United States, Washington
Saint Luke's Rehabilitation Institute
Spokane, Washington, United States, 99202
Sponsors and Collaborators
Flex Pharma, Inc.
Investigators
Principal Investigator: Nicolas Johnson, MD University of Utah

Responsible Party: Flex Pharma, Inc.
ClinicalTrials.gov Identifier: NCT03254199     History of Changes
Other Study ID Numbers: FLX-787-204
First Posted: August 18, 2017    Key Record Dates
Last Update Posted: June 14, 2018
Last Verified: June 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Flex Pharma, Inc.:
Charcot-Marie-Tooth
CMT
Hereditary Sensory and Motor Neuropathy
Muscle Cramps
Muscle Cramping
FLX-787
Neurogenetic disorder
Nervous System Malformations
Nervous System Diseases
Neurodegenerative Diseases
Neuromuscular Diseases

Additional relevant MeSH terms:
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Tooth Diseases
Muscle Cramp
Spasm
Stomatognathic Diseases
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Signs and Symptoms