Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age5-17
SponsorUniversity of Kansas Medical Center
About this trial
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
Eligibility criteria
Qualifiers
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%)
Disqualifiers
Unwilling or unable to provide informed consent or assent. Any other medical condition which in the opinion of the investigator would interfere with study participation.
Malignancy with ongoing treatment with chemotherapeutic agents or anabolic agents
Use of immunosuppressants including prednisone or performance enhancing drugs including testosterone within 6 months
Pregnancy
Trial design
Treatments tested in this trial
- Not listed
Trial groups
80 Participants
are grouped into 2 trial groupsSponsors and collaborators
University of Kansas Medical Center
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator
University of Rochester
Collaborator
Stanford University
Collaborator
Duke University
Collaborator
University of California, Irvine
Collaborator
Kennedy Krieger Institute, Baltimore, MD
Collaborator
University of Utah
Collaborator
Seattle Children's Hospital
Collaborator
University of Iowa
Collaborator
Coriell Institute
Collaborator
Leiden University Medical Center
Collaborator
Murdoch Childrens Research Institute
Collaborator
Source ClinicalTrials.gov