Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Running, not enrolling

Conditions studied: Facioscapulohumeral Muscular Dystrophy

In brief

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 facts

Study ID

NCT03458832

Run by

University of Kansas Medical Center

People needed

324

Starts

2018-03-05

Expected to finish

2027-12-01

Last updated by the study team

2026-04-13

Who can join

Age: 18 and older, up to 75. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• 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

Full record on ClinicalTrials.gov

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