Clinical trial NCT03254199: A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: 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
Lead sponsor: Flex Pharma, Inc.
Sponsor class: INDUSTRY
First posted: 2017-08-18
Enrollment: 27
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Cramp frequency
timeFrame: 28 days
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Aurora
country: United States
facility: University of Colorado
state: Colorado
city: New Britain
country: United States
facility: Hospital for Special Care
state: Connecticut
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Massachusetts General Hospital
state: Massachusetts
city: Boston
country: United States
facility: Brighman and Women's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: University of Minnesota
state: Minnesota
city: Saint Louis
country: United States
facility: Saint Louis University
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Portland
country: United States
facility: Oregon Health Science University
state: Oregon
city: Hershey
country: United States
facility: Penn State Milton S. Hershey Medical Center
state: Pennsylvania
city: Philadelphia
country: United States
facility: Temple University
state: Pennsylvania
city: Salt Lake City
country: United States
facility: The University of Utah
state: Utah
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Spokane
country: United States
facility: Saint Luke's Rehabilitation Institute
state: Washington
Study officials:
affiliation: University of Utah
name: Nicolas Johnson, MD
role: PRINCIPAL_INVESTIGATOR
References: