Collaboration aims to improve design of FSHD clinical trials
FSHD Society teams with other groups to speed treatment development
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A new collaboration aims to improve the design of clinical trials testing potential treatments for facioscapulohumeral muscular dystrophy (FSHD).
The FSHD Society, a patient-driven advocacy organization, is teaming with two other groups — Solve FSHD, a venture philanthropic organization focused on speeding the development of new FSHD treatments, and the FSHD Clinical Trial Research Network, a consortium of academic researchers focused on identifying useful biomarkers and outcome measures in FSHD — to address challenges and develop standardized measures for trials.
“Aligning patient experience, scientific rigor, and regulatory expectations is essential for progress in FSHD,” Lucienne Ronco, chief science officer of the FSHD Society, said in an organization press release.
FSHD is a form of muscular dystrophy characterized by muscle weakness mainly affecting the face, shoulders, and upper arms. There are no approved treatments for the disease in the U.S.
Clinical trials are the gold standard for testing whether a new experimental medication works. But running clinical trials in FSHD is difficult, in part because there aren’t widely agreed-upon measures that can be objectively used to measure FSHD severity and the disease is highly variable from person to person. And if it’s hard to run clinical trials, it’s riskier for drug developers considering investing millions in developing a new treatment.
Finding reliable measures
The collaborative aims to leverage shared clinical trial and natural history data with the aim of identifying objective measures that can reliably provide useful information about disease severity and determining what patient factors lead to variability in these outcomes. The ultimate goal is to develop a framework that standardizes FSHD clinical trials and reduces the risk for drug developers to invest.
“SOLVE FSHD is pleased to provide foundational funding support to launch this Industry Collaborative and advance biomarker development in FSHD,” said Eva Chin, executive director of Solve FSHD. “By combining natural history data and existing clinical trial data, and best-in-class clinical data partner input, we are building the tools and infrastructure needed to reduce drug development risk and accelerate meaningful progress for the FSHD community.”
The three organizations are contributing key resources to make the project work, with the FSHD CTRN leveraging academic expertise and the FSHD Society providing support through its Global FSHD Innovation Hub. Pharmaceutical companies including Scholar Rock, Sanofi, and Fulcrum Therapeutic are also supporting the initiative. Fulcrum has donated clinical trial data from studies of its experimental FSHD treatment losmapimod, which was discontinued after failing to show benefit in late-stage clinical testing.
“Access to high-quality clinical trial and natural history data is critical for improving trial design for rare diseases,” said collaborative director Amanda Rickard, managing director of the Global FSHD Innovation Hub. “This research collaboration creates a rigorous framework for generating insights that individual FSHD programs cannot achieve in isolation.”
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