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

Overview

Información sobre este estudio

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.

Elegibilidad para la participación

Los requisitos de elegibilidad de los participantes incluyen la edad, el sexo, el tipo y el estadio de la enfermedad, y los problemas de salud o tratamientos previos. Las pautas difieren de un estudio a otro e identifican quiénes pueden o no pueden participar. No hay garantía de que cada persona elegible que desee participar en un ensayo se inscribirá. Comunícate con el equipo del estudio para analizar la elegibilidad del estudio y la posible participación.

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

More information

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CLS-20425271

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