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Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
Sponsor: University of Kansas Medical Center
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 treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.
Key Details
Gender
All
Age Range
18 Years - 75 Years
Study Type
OBSERVATIONAL
Enrollment
320
Start Date
2018-03-05
Completion Date
2027-12
Last Updated
2025-04-23
Healthy Volunteers
No
Conditions
Interventions
FSHD-specific functional rating scale
The FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance.
Electrical Impedance Myography
EIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs.
Locations (14)
University of California Los Angeles
Los Angeles, California, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kennedy Krieger Institute
Baltimore, Maryland, United States
University of Rochester Medical Center
Rochester, New York, United States
The Ohio State University
Columbus, Ohio, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
University of Washington
Seattle, Washington, United States
Chu De Nice
Nice, France
Institut de Myologie
Paris, France
Ludwig-Maximilians-Universität München
München, Germany
Centro Clinico NeMO
Milan, Italy
Radboud Unviersity
Nijmegen, Netherlands
University of College London - Queens Square
London, United Kingdom