A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
Primary Purpose
Motor Neuron Disease
Status
Terminated
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
FLX-787-ODT (orally disintegrating tablet)
Placebo ODT
Sponsored by
About this trial
This is an interventional treatment trial for Motor Neuron Disease focused on measuring Motor Neuron Disease, Neurodegenerative Diseases, Nervous System Diseases, Neuromuscular Diseases, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Muscle cramps, Muscle cramping, ALS, MND, PMA, PLS, FLX-787
Eligibility Criteria
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
Sites / Locations
- Honor Health Research Institute
- Mayo Clinic
- California Pacific Medical Center
- University of California - Davis
- University of California San Francisco
- University of Colorado
- Hospital for Special Care
- GW Medical Faculty Associates Inc.
- Mayo Clinic
- University of South Florida Health
- Emory University
- Indiana University Neuroscience Center
- University of Iowa Hospitals and Clinics
- University of Kansas Medical Center
- Johns Hopkins Hospital
- Lahey Hospital and Medical Center
- University of Minnesota
- Saint Louis University
- Hospital for Special Surgery
- Guilford Neurologic Associates
- Wake Forest University
- Providence Brain and Spine Institute
- Oregon Health Sciences University
- Penn State Milton S. Hershey Medical Center
- Temple University
- University of Pittsburgh
- Medical University of South Carolina
- Austin Neuromuscular Center
- Baylor Scott and White Health
- UT Health San Antonio
- The University of Utah
- University of Vermont Medical Center
- Virginia Commonwealth University
- Saint Luke's Rehabilitation Institute
- Medical College of Wisconsin
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Experimental
Placebo Comparator
Arm Description
Outcomes
Primary Outcome Measures
Cramp frequency
Cramp frequency measured over the 28-day treatment period
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03196375
Brief Title
A Study to Assess FLX-787 in Subjects With Motor Neuron 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 Motor Neuron Disease
Study Type
Interventional
2. Study Status
Record Verification Date
August 2018
Overall Recruitment Status
Terminated
Why Stopped
Sponsor
Study Start Date
July 28, 2017 (Actual)
Primary Completion Date
July 27, 2018 (Actual)
Study Completion Date
July 27, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Flex Pharma, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Motor Neuron Disease
Keywords
Motor Neuron Disease, Neurodegenerative Diseases, Nervous System Diseases, Neuromuscular Diseases, Amyotrophic Lateral Sclerosis, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Muscle cramps, Muscle cramping, ALS, MND, PMA, PLS, FLX-787
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
54 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Experimental
Arm Type
Experimental
Arm Title
Placebo Comparator
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
FLX-787-ODT (orally disintegrating tablet)
Intervention Description
FLX-787-ODT taken three times daily for 28 days
Intervention Type
Drug
Intervention Name(s)
Placebo ODT
Intervention Description
Placebo ODT taken three times daily for 28 days
Primary Outcome Measure Information:
Title
Cramp frequency
Description
Cramp frequency measured over the 28-day treatment period
Time Frame
28 days
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bjorn Oskarsson, MD
Organizational Affiliation
Mayo Clinic Jacksonville Florida
Official's Role
Principal Investigator
Facility Information:
Facility Name
Honor Health Research Institute
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85251
Country
United States
Facility Name
Mayo Clinic
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
California Pacific Medical Center
City
Sacramento
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
University of California - Davis
City
Sacramento
State/Province
California
ZIP/Postal Code
95817
Country
United States
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Hospital for Special Care
City
New Britain
State/Province
Connecticut
ZIP/Postal Code
06053
Country
United States
Facility Name
GW Medical Faculty Associates Inc.
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20037
Country
United States
Facility Name
Mayo Clinic
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
University of South Florida Health
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Indiana University Neuroscience Center
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
University of Iowa Hospitals and Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
University of Kansas Medical Center
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21205
Country
United States
Facility Name
Lahey Hospital and Medical Center
City
Burlington
State/Province
Massachusetts
ZIP/Postal Code
01805
Country
United States
Facility Name
University of Minnesota
City
Minneapolis
State/Province
Minnesota
ZIP/Postal Code
55455
Country
United States
Facility Name
Saint Louis University
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63104
Country
United States
Facility Name
Hospital for Special Surgery
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Guilford Neurologic Associates
City
Greensboro
State/Province
North Carolina
ZIP/Postal Code
27405
Country
United States
Facility Name
Wake Forest University
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Providence Brain and Spine Institute
City
Portland
State/Province
Oregon
ZIP/Postal Code
97213
Country
United States
Facility Name
Oregon Health Sciences University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Penn State Milton S. Hershey Medical Center
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Temple University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19140
Country
United States
Facility Name
University of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Medical University of South Carolina
City
Charleston
State/Province
South Carolina
ZIP/Postal Code
29425
Country
United States
Facility Name
Austin Neuromuscular Center
City
Austin
State/Province
Texas
ZIP/Postal Code
78756
Country
United States
Facility Name
Baylor Scott and White Health
City
Round Rock
State/Province
Texas
ZIP/Postal Code
78665
Country
United States
Facility Name
UT Health San Antonio
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States
Facility Name
The University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84112
Country
United States
Facility Name
University of Vermont Medical Center
City
Burlington
State/Province
Vermont
ZIP/Postal Code
05405
Country
United States
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23298
Country
United States
Facility Name
Saint Luke's Rehabilitation Institute
City
Spokane
State/Province
Washington
ZIP/Postal Code
99202
Country
United States
Facility Name
Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
A Study to Assess FLX-787 in Subjects With Motor Neuron Disease Experiencing Muscle Cramps.
We'll reach out to this number within 24 hrs