RARE Daily

FSHD Society and Critical Path Institute Collaborate to Collect Clinical Trial Data

May 23, 2022

The FSHD Society, a research-focused patient advocacy nonprofit dedicated to bringing treatments to patients affected by facioscapulohumeral muscular dystrophy, said it has partnered with the independent nonprofit Critical Path Institute to facilitate the integration of clinical trial data from the control arm of various FSHD-related clinical studies into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform.

Photo: Alexandre Bétourné, RDCA-DAP scientific director

These data will come from several companies that have sponsored clinical trials in FSHD.

The integration of these data makes it possible to analyze information from a larger number of patients to generate a more robust and granular understanding of the natural disease progression in FSHD. This is particularly important in rare diseases like FSHD that progress at slow and unpredictable rates.

“RDCA-DAP promotes the sharing of existing patient-level data and encourages the standardization of new data collection,” said Alexandre Bétourné, RDCA-DAP scientific director. “By integrating such data in a regulatory-grade format suitable for analytics, RDCA-DAP helps accelerate the understanding of disease progression, clinical outcome measures, and biomarkers, and facilitates the development of mathematical models of disease and innovative clinical trial designs.”

Organizations participating to date in the FSHD Society’s initiative include Acceleron Pharma and the University of Rochester. Acceleron, based in Cambridge, Mass., is contributing control data from its phase 2, randomized, placebo-controlled trial of ACE-083. This therapy was designed to increase the strength and function of specific muscles in FSHD patients. Ninety-five patients were enrolled in the study, which terminated in October of 2019. Acceleron has agreed to provide data collected from the control arm of the study, including total muscle volume (measured by MRI) and quantitative testing of muscle strength.

The University of Rochester trial enrolled 90 patients to assess the effectiveness of albuterol, a beta-adrenergic agonist, to increase the strength of FSHD patients. The study concluded in 2015. The functional data collected in this study included quantitative voluntary isometric contraction testing and muscle mass assessments by dual energy x-ray absorptiometry.

“This is a great example of how we can collaborate to help accelerate drug development for rare diseases,” Bétourné said. “The FSHD society included their community in discussions, landscaped the accessible data and simultaneously engaged C-Path and multiple data contributors, which expedited the sharing of these two datasets.”

Author: Rare Daily Staff

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