RECRUITING

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Quick Facts

Study Start:2018-03-05
Study Completion:2027-03
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT03458832

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.

Ages Eligible for Study:18 Years to 75 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Patients with genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring
  2. * Patients with symptomatic limb weakness
  3. * Patients must be able to walk 30 feet without the support of another person or assistance (canes, walking sticks, and braces allowed; no walker).
  4. * If taking over the counter supplements, willing to remain consistent with supplement regimen throughout the course of the study
  1. * Patients with cardiac or respiratory dysfunction (deemed clinically unstable, or would interfere with safe testing, in the opinion of the Investigator)
  2. * Patients with orthopedic conditions that preclude safe testing of muscle function
  3. * Patients that regularly use available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists
  4. * Patients that have used an experimental drug in an FSHD clinical trial within the past 30 days
  5. * Patients that are pregnant

Contacts and Locations

Study Contact

Michaela Walker
CONTACT
913-945-9920
mwalker20@kumc.edu
Leann Lewis
CONTACT
585-275-7680
leann_lewis@urmc.rochester.edu

Principal Investigator

Jeffrey Statland, MD
PRINCIPAL_INVESTIGATOR
University of Kansas Medical Center
Rabi Tawil, MD
PRINCIPAL_INVESTIGATOR
University of Rochester

Study Locations (Sites)

University of California Los Angeles
Los Angeles, California, 90095
United States
University of Kansas Medical Center
Kansas City, Kansas, 66160
United States
Kennedy Krieger Institute
Baltimore, Maryland, 21205
United States
University of Rochester Medical Center
Rochester, New York, 14642
United States
The Ohio State University
Columbus, Ohio, 43210
United States
University of Utah
Salt Lake City, Utah, 84132
United States
Virginia Commonwealth University
Richmond, Virginia, 23298
United States
University of Washington
Seattle, Washington, 98195
United States

Collaborators and Investigators

Sponsor: 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

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-03-05
Study Completion Date2027-03

Study Record Updates

Study Start Date2018-03-05
Study Completion Date2027-03

Terms related to this study

Keywords Provided by Researchers

  • muscular dystrophy.
  • FSHD

Additional Relevant MeSH Terms

  • Facioscapulohumeral Muscular Dystrophy