Better motor outcome measures for children with FSHD
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
['FUNDING_U01'] · UNIVERSITY OF KANSAS MEDICAL CENTER · NIH-11172427
This project checks which motor tests and trial plans work best for children with early-onset facioscapulohumeral muscular dystrophy (FSHD) so treatments can move faster.
Quick facts
| Phase | ['FUNDING_U01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIVERSITY OF KANSAS MEDICAL CENTER (nih funded) |
| Locations | 1 site (KANSAS CITY, UNITED STATES) |
| Trial ID | NIH-11172427 on ClinicalTrials.gov |
What this research studies
You would be followed at experienced FSHD Clinical Trials Research Network (CTRN) centers where doctors use standardized motor tests, genetic information (including D4Z4 repeat status), and other measures over time to see which outcomes change reliably in childhood FSHD. The team will collect clinical exams, functional motor measures, and possibly imaging and biosamples across multiple U.S. sites to build trial-ready outcome tools. The work is designed to refine when and how to enroll kids in future gene- or RNA-based therapies, including AAV-delivered approaches under development. Participation may include scheduled clinic visits, simple physical tests, and sharing medical records.
Who could benefit from this research
Good fit: Children and adolescents with genetically or clinically confirmed early-onset FSHD (especially those with 1–3 D4Z4 repeats or symptom onset in childhood) are the ideal candidates.
Not a fit: People without FSHD, those with adult-onset FSHD not enrolled in pediatric-focused protocols, or patients with unrelated neuromuscular conditions are unlikely to benefit directly.
Why it matters
Potential benefit: If successful, this work could make pediatric FSHD trials faster and more reliable and help bring gene- or RNA-based treatments to children sooner.
How similar studies have performed: Cross-sectional studies and emerging adult FSHD trials support the overall approach, but prospective, multi-site pediatric outcome validation is relatively new.
Where this research is happening
KANSAS CITY, UNITED STATES
- UNIVERSITY OF KANSAS MEDICAL CENTER — KANSAS CITY, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: STATLAND, JEFFREY — UNIVERSITY OF KANSAS MEDICAL CENTER
- Study coordinator: STATLAND, JEFFREY
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.