The FSHD Society has awarded $300,000 a year to expand the Facioscapulohumeral Muscular Dystrophy Clinical Trial Research Network (FSHD CTRN), with consideration of follow-on funding of $300,000 a year in 2021 and 2022.

The existing consortium in the United States is expanding by four sites: the University Florida in Gainesville, University of Texas Southwestern Medical Center in Dallas, University of Colorado in Denver, and the Stanford University School of Medicine in Stanford, California, according to a media release from FSHD Society.

“This funding aims to ensure better coverage and access to patients. It also provides resources for the effective management and coordination across the entire network. With the prospect of a growing number of FSHD clinical trials on the horizon, it will be critical for the CTRN to have the capacity and bandwidth to not only carry out the existing research studies but also successfully recruit and participate in clinical trials.”

— Jamshid Arjomand, PhD, chief science officer, FSHD Society

CTRN Aims to Accelerate FSHD Research

The CTRN is a consortium of academic research centers in the United States and Europe with expertise in FSHD clinical research or in neuromuscular clinical trials. It aims to help close gaps in trial readiness and provide a network of sites with a centralized streamlined regulatory process, specific, common expertise in FSHD, and an engaged patient population ready to conduct efficient, high-quality clinical trials.

It was formed to create an infrastructure of clinics with the expertise to accelerate therapeutic development for FSHD: by ensuring site training of key personnel; streamlining regulatory oversight and data capture; validating novel clinical outcome measures; and refining clinical trial strategies.

The network was initially financed with seed funding from the FSHD Society, as well as other non-profits, and has since been awarded millions in additional funding from the Muscular Dystrophy Association and biotechnology companies that are pursuing therapies for FSHD.

The current CTRN encompasses a total of 15 centers, with 12 sites in the United States and three collaborating sites in Europe. The central coordinating center is housed at the University of Kansas Medical Center, and the data and statistical coordinating center is located at the University of Rochester Medical Center.

Current Research

Currently, the network is running an NIH-funded study named “Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD (ReSolve),” an 18- to 24-month observational study following 220 individuals with FSHD.

Jeffrey Statland, MD, associate professor of neurology at the University of Kansas Medical Center, and Rabi Tawil, MD, University of Rochester Medical Center, serve as co-directors of the network, the release continues.

“We are grateful to the FSHD Society and like-minded organizations that have made it possible for the network to form and carry out studies to help bridge the gaps in clinical trial readiness. The additional sites will ensure that the CTRN will be able to meet the goal of trial preparedness and ensure well-trained FSHD clinical centers,” Statland says, in the release.

[Source(s): FSHD Society, PRWeb]

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