A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
NCT ID: NCT03254199
Last Updated: 2018-08-29
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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TERMINATED
PHASE2
27 participants
INTERVENTIONAL
2017-10-16
2018-07-27
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Experimental
FLX-787-ODT (orally disintegrating tablet)
FLX-787-ODT taken three times daily for 28 days
Placebo Comparator
Placebo ODT
Placebo ODT taken three times daily for 28 days
Interventions
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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
Eligibility Criteria
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Inclusion Criteria
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 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
18 Years
ALL
No
Sponsors
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Flex Pharma, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Nicolas Johnson, MD
Role: PRINCIPAL_INVESTIGATOR
University of Utah
Locations
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Mayo Clinic
Scottsdale, Arizona, United States
University of Colorado
Aurora, Colorado, United States
Hospital for Special Care
New Britain, Connecticut, United States
University of Florida
Gainesville, Florida, United States
Mayo Clinic
Jacksonville, Florida, United States
University of South Florida
Tampa, Florida, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Massachusetts General Hospital
Boston, Massachusetts, United States
Brighman and Women's Hospital
Boston, Massachusetts, United States
University of Minnesota
Minneapolis, Minnesota, United States
Saint Louis University
St Louis, Missouri, United States
Columbia University Medical Center
New York, New York, United States
University of Rochester Medical Center
Rochester, New York, United States
Oregon Health Science University
Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, United States
Temple University
Philadelphia, Pennsylvania, United States
The University of Utah
Salt Lake City, Utah, United States
University of Vermont Medical Center
Burlington, Vermont, United States
Saint Luke's Rehabilitation Institute
Spokane, Washington, United States
Countries
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Other Identifiers
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FLX-787-204
Identifier Type: -
Identifier Source: org_study_id
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