A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.

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 Motor Neuron Disease

The COMMEND Study will assess the safety and effectiveness of FLX-787 in men and women with Motor Neuron Disease [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)] 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 approximately 30 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.

Study Overview

• Status: Terminated
• Conditions: Motor Neuron Disease
• Intervention / Treatment: Drug: FLX-787-ODT (orally disintegrating tablet); Drug: Placebo ODT

• Study Type: Interventional
• Enrollment (Actual): 54
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Scottsdale, Arizona, United States, 85259
      • Mayo Clinic
    • Scottsdale, Arizona, United States, 85251
      • Honor Health Research Institute
  • California
    • Sacramento, California, United States, 95817
      • University of California - Davis
    • Sacramento, California, United States, 94115
      • California Pacific Medical Center
    • San Francisco, California, United States, 94143
      • University of California San Francisco
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Connecticut
    • New Britain, Connecticut, United States, 06053
      • Hospital for Special Care
  • District of Columbia
    • Washington, District of Columbia, United States, 20037
      • GW Medical Faculty Associates Inc.
  • Florida
    • Jacksonville, Florida, United States, 32224
      • Mayo Clinic
    • Tampa, Florida, United States, 33612
      • University of South Florida Health
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Emory University
  • Indiana
    • Indianapolis, Indiana, United States, 46202
      • Indiana University Neuroscience Center
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Maryland
    • Baltimore, Maryland, United States, 21205
      • Johns Hopkins Hospital
  • Massachusetts
    • Burlington, Massachusetts, United States, 01805
      • Lahey Hospital and Medical Center
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • University of Minnesota
  • Missouri
    • Saint Louis, Missouri, United States, 63104
      • Saint Louis University
  • New York
    • New York, New York, United States, 10021
      • Hospital for Special Surgery
  • North Carolina
    • Greensboro, North Carolina, United States, 27405
      • Guilford Neurologic Associates
    • Winston-Salem, North Carolina, United States, 27157
      • Wake Forest University
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health Sciences University
    • Portland, Oregon, United States, 97213
      • Providence Brain and Spine Institute
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center
    • Philadelphia, Pennsylvania, United States, 19140
      • Temple University
    • Pittsburgh, Pennsylvania, United States, 15213
      • University of Pittsburgh
  • South Carolina
    • Charleston, South Carolina, United States, 29425
      • Medical University of South Carolina
  • Texas
    • Austin, Texas, United States, 78756
      • Austin Neuromuscular Center
    • Round Rock, Texas, United States, 78665
      • Baylor Scott and White Health
    • San Antonio, Texas, United States, 78229
      • UT Health San Antonio
  • Utah
    • Salt Lake City, Utah, United States, 84112
      • The University of Utah
  • Vermont
    • Burlington, Vermont, United States, 05405
      • University of Vermont Medical Center
  • Virginia
    • Richmond, Virginia, United States, 23298
      • Virginia Commonwealth University
  • Washington
    • Spokane, Washington, United States, 99202
      • Saint Luke's Rehabilitation Institute
  • Wisconsin
    • Milwaukee, Wisconsin, United States, 53226
      • Medical College of Wisconsin

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Documented diagnosis of Motor Neuron Disease (MND) [including Amyotrophic Lateral Sclerosis (ALS), Primary Lateral Sclerosis (PLS) or Progressive Muscular Atrophy (PMA)]
• Expected survival > 6 months
• 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 laryngospasm or significant swallowing problems
• Presence of percutaneous endoscopic gastrostomy, esophagogastroduodenoscopy, or G-tube
• 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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental	Drug: FLX-787-ODT (orally disintegrating tablet); FLX-787-ODT taken three times daily for 28 days
Placebo Comparator: Placebo Comparator	Drug: Placebo ODT; Placebo ODT taken three times daily for 28 days

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cramp frequency	Cramp frequency measured over the 28-day treatment period	28 days

Collaborators and Investigators

• Sponsor: Flex Pharma, Inc.
• Investigators: Principal Investigator: Bjorn Oskarsson, MD, Mayo Clinic Jacksonville Florida

Study record dates

Study Major Dates

• Study Start (Actual): July 28, 2017
• Primary Completion (Actual): July 27, 2018
• Study Completion (Actual): July 27, 2018

Study Registration Dates

• First Submitted: June 20, 2017
• First Submitted That Met QC Criteria: June 20, 2017
• First Posted (Actual): June 22, 2017

Study Record Updates

• Last Update Posted (Actual): August 29, 2018
• Last Update Submitted That Met QC Criteria: August 28, 2018
• Last Verified: August 1, 2018

More Information

Terms related to this study

• Keywords: Amyotrophic Lateral Sclerosis; ALS; Motor Neuron Disease; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Diseases; MND; FLX-787; Primary Lateral Sclerosis; PLS; Progressive Muscular Atrophy; PMA; Muscle cramps; Muscle cramping
• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Musculoskeletal Diseases; Muscular Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Neuromuscular Manifestations; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Spasm; Muscle Cramp
• Other Study ID Numbers: FLX-787-203

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No