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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03254199
• Date of registration: 16/08/2017
• Prospective Registration: Yes
• Primary sponsor: Flex Pharma, Inc.
• Public title: A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps. COMMIT
• Scientific 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
• Date of first enrolment: October 16, 2017
• Target sample size: 27
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03254199
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Nicolas Johnson, MD
• Affiliation: University of Utah

Key inclusion & exclusion 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



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Charcot-Marie-Tooth Disease

Intervention(s)

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

Drug: Placebo ODT

Primary Outcome(s)

Cramp frequency [Time Frame: 28 days]

Secondary ID(s)

FLX-787-204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors