NCT03254199

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.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Oct 2017

Shorter than P25 for phase_2

Geographic Reach
1 country

20 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

August 16, 2017

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 18, 2017

Completed
2 months until next milestone

Study Start

First participant enrolled

October 16, 2017

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 27, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 27, 2018

Completed
Last Updated

August 29, 2018

Status Verified

August 1, 2018

Enrollment Period

9 months

First QC Date

August 16, 2017

Last Update Submit

August 28, 2018

Conditions

Keywords

Charcot-Marie-ToothCMTMuscle CrampsMuscle CrampingFLX-787Hereditary Sensory and Motor NeuropathyNeurogenetic disorderNervous System MalformationsNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Diseases

Outcome Measures

Primary Outcomes (1)

  • Cramp frequency

    Cramp frequency measured over the 28-day treatment period

    28 days

Study Arms (2)

Experimental

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

Placebo Comparator

PLACEBO COMPARATOR
Drug: Placebo ODT

Interventions

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

Experimental

Placebo ODT taken three times daily for 28 days

Placebo Comparator

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Presence of symptoms of CMT since at least 6 months prior to Screening, and confirmed diagnosis of CMT as defined by:
  • Genetic confirmation of a mutation known to cause CMT, or
  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (20)

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

University of Colorado

Aurora, Colorado, 80045, United States

Location

Hospital for Special Care

New Britain, Connecticut, 06053, United States

Location

University of Florida

Gainesville, Florida, 32607, United States

Location

Mayo Clinic

Jacksonville, Florida, 32224, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

University of Iowa

Iowa City, Iowa, 52242, United States

Location

University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02114, United States

Location

Brighman and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

Saint Louis University

St Louis, Missouri, 63104, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

Oregon Health Science University

Portland, Oregon, 97239, United States

Location

Penn State Milton S. Hershey Medical Center

Hershey, Pennsylvania, 17033, United States

Location

Temple University

Philadelphia, Pennsylvania, 19140, United States

Location

The University of Utah

Salt Lake City, Utah, 84112, United States

Location

University of Vermont Medical Center

Burlington, Vermont, 05405, United States

Location

Saint Luke's Rehabilitation Institute

Spokane, Washington, 99202, United States

Location

MeSH Terms

Conditions

Charcot-Marie-Tooth DiseaseMuscle CrampSpasmHereditary Sensory and Motor NeuropathyNervous System MalformationsNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Diseases

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemPolyneuropathiesPeripheral Nervous System DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, InbornMuscular DiseasesMusculoskeletal DiseasesNeuromuscular ManifestationsNeurologic ManifestationsSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • Nicolas Johnson, MD

    University of Utah

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Masking Details
Double-blind
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 16, 2017

First Posted

August 18, 2017

Study Start

October 16, 2017

Primary Completion

July 27, 2018

Study Completion

July 27, 2018

Last Updated

August 29, 2018

Record last verified: 2018-08

Locations