Validating motor outcomes in children with FSHD
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
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 |
|---|---|
| Enrollment | 80 (estimated) |
| Ages | 5 Years to 17 Years |
| Sex | All |
| Sponsor | University of Kansas Medical Center Academic / other |
| Drugs / interventions | prednisone |
| Locations | 7 sites (Palo Alto, California and 6 other locations) |
| Trial ID | NCT06847282 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are children aged 5-17 years with genetically confirmed FSHD and symptomatic weakness.
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.
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 performed: Previous studies have shown promise in validating functional measures in adult FSHD, but this specific approach in pediatric populations is relatively novel.
Eligibility criteria
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 this trial is running
Palo Alto, California and 6 other locations
- Stanford University — Palo Alto, California, United States (Not_yet_recruiting)
- University of Iowa — Iowa City, Iowa, United States (Not_yet_recruiting)
- 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)
- University of Utah — Salt Lake City, Utah, United States (Not_yet_recruiting)
- Murdoch Children's Research Institute — Melbourne, Australia (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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.