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

Flex Pharma

Status and phase: Terminated; Phase 2

Conditions: Charcot-Marie-Tooth Disease

Treatments: Drug: Placebo ODT; Drug: FLX-787-ODT (orally disintegrating tablet)

Study type: Interventional

Funder types: Industry

Identifiers

NCT03254199

FLX-787-204

Details and patient eligibility

About

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.

Enrollment: 27 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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27 participants in 2 patient groups, including a placebo group

Experimental

Experimental group

Treatment:

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

Placebo Comparator

Placebo Comparator group

Treatment:

Drug: Placebo ODT