Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
University of Kansas Medical Center
Summary
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
Description
MOVE Peds is a prospective 2-year study recruiting eighty pediatric participants to accelerate therapeutic development for pediatric-onset FSHD. The study aims to validate outcomes and refine clinical trial strategies. Previous cross-sectional studies suggest that younger age of onset is linked to greater clinical severity and that having 1-3 D4Z4 repeats is associated with extra-muscular complications in pediatric FSHD. Prospective studies in early-onset FSHD have been limited by the small number of sites and low recruitment and follow-up rates. Early-onset pediatric FSHD is of high interest…
Eligibility
- Age range
- 5–17 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Age 5-17 years. * Genetically confirmed FSHD (types 1 or 2). * Symptomatic weakness (facial, shoulder, core, or limb weakness) * Able to complete a 10-meter walk without the support of another person in less than 12 seconds (canes, walking sticks, and braces allowed; no walker). In order to include early onset participants up to 8 individuals will be entered with baseline 10MWR \> 12 seconds or who are no longer ambulatory (≤10%) Exclusion Criteria: * Unwilling or unable to provide informed consent or assent. Any other medical condition which in the opinion of the inve…
Locations (7)
- Stanford UniversityPalo Alto, California
- University of IowaIowa City, Iowa
- University of Kansas Medical CenterKansas City, Kansas
- University of RochesterRochester, New York
- Duke UniversityDurham, North Carolina
- University of UtahSalt Lake City, Utah