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About the Facioscapulohumeral Muscular Dystrophy Clinical Trial Research Network

The Facioscapulohumeral Muscular Dystrophy Clinical Trial Research Network (FSHD CTRN) is a consortium of eleven academic research centers (8 in the United States, and 3 in Europe) with expertise in FSHD clinical research, or in conducting neuromuscular clinical trials. These centers leverage existing clinical trials infrastructure: including clinical investigators, research coordinators, study evaluators, and institutional resources like Clinical and Translational Science Research Awards (CTSAs) and local Research Institutes for clinical trial budget and contracting, and Clinical Research Centers, which provide subsidized clinical research space and facilities.

A major hurdle to development of trial strategies and validation of outcome measures for FSHD clinical trials is the lack of an existing clinical trial network infrastructure with common standard operating procedures (SOPs). The FSHD CTRN surmounts this challenge by:

  • creating a streamlined system for regulatory/ethical oversight;
  • developing standards for what data is collected and how it is collected;
  • creating a network of well-trained and knowledgeable clinical evaluators, essential for clinical trials;
  • creating a network of trained study coordinators with strong patient engagement, recruitment, and retention skills;
  • ensuring the participation of all major stakeholders;
  • validating new outcome measures for drug registration studies; and
  • training the next generation of FSHD clinical researchers.

The FSHD CTRN helps close gaps in trial readiness, and also provides 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.

Diagram of FSHD Trial Readiness pathway

Last modified: Apr 22, 2019
Events

FSHD Society Chapter Meeting
March 28, 2020
1 p.m. - 3 p.m.
4330 Shawnee Mission Pkwy.
3rd floor Conference Room

Voice of the Patient Forum for drug development on FSHD
April 21, 2020
webcast and in-person attendance info

27th Annual FSHD International Research Congress
June 25-26, 2020
Washington, DC
more information

2020 FSHD Connect conference
June 27-28, 2020
Washington, DC
more information

Clinical Trials
Contact Us

Kiley Higgs
Project Manager
913-945-9922
ksims2@kumc.edu

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