Status:
RECRUITING
Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
Lead Sponsor:
University of Kansas Medical Center
Collaborating Sponsors:
National Institute of Neurological Disorders and Stroke (NINDS)
FSHD Society
Conditions:
Facioscapulohumeral Muscular Dystrophy
Eligibility:
All Genders
18-75 years
Brief Summary
The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatment...
Detailed Description
The overall aim of this study is to hasten drug development for facioscapulohumeral muscular dystrophy (FSHD). Recent breakthroughs in FSHD research have identified the primary disease mechanism as th...
Eligibility Criteria
Inclusion
- Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
- Patients with symptomatic limb weakness
- Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
- If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
Exclusion
- Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
- Patients with orthopedic conditions that preclude safe testing of muscle function
- Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
- Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
- Patients that are pregnant
Key Trial Info
Start Date :
March 5 2018
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2027
Estimated Enrollment :
320 Patients enrolled
Trial Details
Trial ID
NCT03458832
Start Date
March 5 2018
End Date
December 1 2027
Last Update
April 23 2025
Active Locations (14)
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1
University of California Los Angeles
Los Angeles, California, United States, 90095
2
University of Kansas Medical Center
Kansas City, Kansas, United States, 66160
3
Kennedy Krieger Institute
Baltimore, Maryland, United States, 21205
4
University of Rochester Medical Center
Rochester, New York, United States, 14642