Investigators: Rabi Tawil MD, Jeffrey M Statland MD
Category: Research - Clinical
Clinical Trial Research Network (CTRN) international expansion.
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. To that end, an existing NIH-funded study, at 7 US sites will develop two new outcome measures and optimize eligibility criteria by testing 150 patients over a period of 18 months. The current proposal will allow the expansion of this study to include 2 additional European sites. This proposal will not only increase the number of individuals studied to 200 patients, but will also help grow the number of sites with the required expertise to run future FSHD drug trials.
Due to Covid-19, the study was extended to 24 months. End date adjusted.
See Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD
See support now also added by the MDA in a grant for CTRN U.S. sites
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