Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Description

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. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.

Conditions

Facioscapulohumeral Muscular Dystrophy

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Study Overview

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Study Details

Study overview

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. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Condition
Facioscapulohumeral Muscular Dystrophy
Intervention / Treatment

-

Contacts and Locations

Los Angeles

University of California Los Angeles, Los Angeles, California, United States, 90095

Kansas City

University of Kansas Medical Center, Kansas City, Kansas, United States, 66160

Baltimore

Kennedy Krieger Institute, Baltimore, Maryland, United States, 21205

Rochester

University of Rochester Medical Center, Rochester, New York, United States, 14642

Columbus

The Ohio State University, Columbus, Ohio, United States, 43210

Salt Lake City

University of Utah, Salt Lake City, Utah, United States, 84132

Richmond

Virginia Commonwealth University, Richmond, Virginia, United States, 23298

Seattle

University of Washington, Seattle, Washington, United States, 98195

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
  • * Patients with symptomatic limb weakness
  • * Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
  • * If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
  • * Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
  • * Patients with orthopedic conditions that preclude safe testing of muscle function
  • * Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
  • * Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
  • * Patients that are pregnant

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Kansas Medical Center,

Jeffrey Statland, MD, PRINCIPAL_INVESTIGATOR, University of Kansas Medical Center

Rabi Tawil, MD, PRINCIPAL_INVESTIGATOR, University of Rochester

Study Record Dates

2027-03