Validating motor outcomes in children with FSHD
Official title: Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
Run by University of Kansas Medical Center · NCT06847282 · Data updated 2026-11-12
This study is trying to see how children with facioscapulohumeral muscular dystrophy (FSHD) move and function to help improve future treatments for them.
Quick facts
| Study type | Observational |
|---|---|
| Ages | 5 Years to 17 Years |
| Sex | All |
| Enrollment | 80 (estimated) |
| Sponsor | University of Kansas Medical Center Academic / other |
| Drugs / interventions | prednisone |
| Locations | 7 sites (Palo Alto, California and 6 other locations) |
| Status | Recruiting |
| Started | May 22, 2025 |
| Main results expected | May 2027 |
| Study ends | May 2028 |
| Registry record updated | February 25, 2026 |
| Trial ID | NCT06847282 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged 5-17 years with genetically confirmed FSHD and symptomatic weakness.
✕Probably not a fit
Patients with other medical conditions that interfere with study participation or those currently undergoing treatment for malignancy may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
MOVE Peds is a prospective observational study aimed at validating motor and functional outcomes in pediatric patients with facioscapulohumeral muscular dystrophy (FSHD). Over two years, the study will recruit eighty participants aged 5-17 years to refine clinical trial strategies and accelerate therapeutic development. It focuses on understanding the impact of early-onset FSHD, which is associated with greater clinical severity and unique genetic factors. The study will utilize measures such as the FSHD composite functional measure and quantitative MRI to track disease progression and treatment response.
Why it matters
Potential benefit: If successful, this study could lead to improved therapeutic strategies and outcomes for children with FSHD.
How similar studies have gone: Previous studies have shown promise in validating functional measures in adult FSHD, but this specific approach in pediatric populations is relatively novel.
Who can join: the full rules
Show full inclusion / exclusion criteria
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 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 * Recent or ongoing infection * Presence of contraindication to performance of MRI: pacemaker, metallic foreign body in eye, brain aneurysm clip (unless documented as MRI compatible) * In the opinion of the investigator unable to follow directions for standardized testing * Note: Not being able to complete MRI will not result in a screen failure. If subject is not able to complete the procedure due to fear or anxiety, they will have the opportunity to try again at later visits. However, Subject must be willing to attempt to perform the MRI to meet inclusion/exclusion criteria
Where it's running
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University of Kansas Medical Center
Kansas City, Kansas, United States Recruiting -
University of Rochester
Rochester, New York, United States Recruiting -
Duke University
Durham, North Carolina, United States Recruiting -
Murdoch Children's Research Institute
Melbourne, Australia Recruiting -
Stanford University
Palo Alto, California, United States Not yet recruiting -
University of Iowa
Iowa City, Iowa, United States Not yet recruiting
Show 1 more location
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University of Utah
Salt Lake City, Utah, United States Not yet recruiting
Study contacts
- Principal investigator: Jeffrey Statland, MD — University of Kansas Medical Center
- Study coordinator: Michaela Walker, MPH
- Email: mwalker20@kumc.edu
- Phone: 913-945-9920
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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BetterLife FSHD: a patient-driven health platform
This project tries to connect people with FSHD to personalized resources and collect their health experiences so they can be matched to relevant studies and help improve care.
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Clenbuterol treatment targeting DUX4 in FSHD
Try clenbuterol at three dose levels in adults with FSHD to see if it is safe and tolerable.
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Comparing MRI and Ultrasound for Evaluating Muscle Function in Facioscapulohumeral Dystrophy
This study is testing whether ultrasound and MRI can help us understand how muscle function is affected in people with Facioscapulohumeral Dystrophy.
How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.