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TerminatedNCT03254199COMMITUpdated Aug 29, 2018

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

A Phase 2 interventional study of FLX-787-ODT (orally disintegrating tablet) and Placebo ODT in Charcot-Marie-Tooth Disease, sponsored by Flex Pharma, Inc.. Terminated at 20 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2018-08-29.

Sponsored by Flex Pharma, Inc. · Phase 2, Interventional, and Treatment

Why this study was terminated
Sponsor
Phase
Phase 2
Study type
Interventional
Enrollment
27
Allocation
Randomized
Ages
18 Years and older
Sex
All
01

Study 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.

02

Conditions studied

  • Charcot-Marie-Tooth Disease

Keywords

  • Charcot-Marie-Tooth
  • CMT
  • Muscle Cramps
  • Muscle Cramping
  • FLX-787
  • Hereditary Sensory and Motor Neuropathy
  • Neurogenetic disorder
  • Nervous System Malformations
  • Nervous System Diseases
  • Neurodegenerative Diseases
  • Neuromuscular Diseases
03

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
27 participants (actual)

Study arms

  • Experimental
    Experimental

    Drug: FLX-787-ODT (orally disintegrating tablet)

  • Placebo comparator
    Placebo Comparator

    Drug: Placebo ODT

Interventions

  • DrugFLX-787-ODT (orally disintegrating tablet)

    FLX-787-ODT taken three times daily for 28 days

  • DrugPlacebo ODT

    Placebo ODT taken three times daily for 28 days

05

What researchers measure

Primary outcomes

  1. Cramp frequency

    Cramp frequency measured over the 28-day treatment period

    Time frame: 28 days

06

Study locations

20 sites
  • Mayo Clinic
    Scottsdale, Arizona 85259, United States
  • University of Colorado
    Aurora, Colorado 80045, United States
  • Hospital for Special Care
    New Britain, Connecticut 06053, United States
  • University of Florida
    Gainesville, Florida 32607, United States
  • Mayo Clinic
    Jacksonville, Florida 32224, United States
  • University of South Florida
    Tampa, Florida 33612, United States
  • University of Iowa
    Iowa City, Iowa 52242, United States
  • University of Kansas Medical Center
    Kansas City, Kansas 66160, United States
  • Massachusetts General Hospital
    Boston, Massachusetts 02114, United States
  • Brighman and Women's Hospital
    Boston, Massachusetts 02115, United States
  • University of Minnesota
    Minneapolis, Minnesota 55455, United States
  • Saint Louis University
    Saint Louis, Missouri 63104, United States
  • Columbia University Medical Center
    New York, New York 10032, United States
  • University of Rochester Medical Center
    Rochester, New York 14642, United States
  • Oregon Health Science University
    Portland, Oregon 97239, United States
  • Penn State Milton S. Hershey Medical Center
    Hershey, Pennsylvania 17033, United States
  • Temple University
    Philadelphia, Pennsylvania 19140, United States
  • The University of Utah
    Salt Lake City, Utah 84112, United States
  • University of Vermont Medical Center
    Burlington, Vermont 05405, United States
  • Saint Luke's Rehabilitation Institute
    Spokane, Washington 99202, United States
07

References and documents

Individual participant data

Plan to share: Undecided

No publications or documents are linked to this record.

08

Registry details

Key details

Study ID
NCT03254199
Lead sponsor
Flex Pharma, Inc.
Responsible party
Sponsor
First posted
Aug 18, 2017
Start date
Oct 16, 2017
Primary completion
Jul 27, 2018
Completion
Jul 27, 2018
Last update
Aug 29, 2018

Study contacts

Nicolas Johnson, MD
principal investigator · University of Utah

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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