MOVE Peds: Moving Toward Better Outcomes for Pediatric FSHD
Official title: Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
Funded at University of Kansas Medical Center · NIH-11599383 · Data updated 2026-11-12
A study in kids with a rare muscle disease (FSHD) will test how well certain outcomes reflect change and help guide future clinical trials.
Quick facts
| Grant type | U01 cooperative agreement |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Kansas Medical Center NIH-funded |
| Lab location | 1 site (Kansas City, United States) |
| Project start | August 20, 2024 |
| Project end | July 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $124,199 |
| NIH project number | 3U01NS137702-03S1 |
| Project ID | NIH-11599383 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are children and adolescents with FSHD, particularly those with early-onset disease and relevant genetic features (e.g., 1-3 D4Z4 repeats).
✕Less likely to help
Some patients without early-onset disease or without participation at participating sites may not directly benefit.
What this research studies
This project focuses on children with pediatric-onset FSHD to speed up drug development by validating what doctors should measure in trials and refining trial strategies. It leverages the FSHD Clinical Trials Research Network to enroll participants across sites and to track outcomes that matter most to patients and families. The work includes exploring how early age of onset and genetic repeats relate to disease features and risk of extra-muscular problems, with an eye toward therapies that silence the DUX4 gene using approaches like antisense or RNA interference. By coordinating across multiple sites and shortening recruitment and follow-up hurdles, the study aims to provide reliable measures and practical trial designs for future therapies. Results could help manufacturers and researchers design effective pediatric trials sooner and more efficiently.
Why it matters
Potential benefit: If successful, this work could enable faster development of treatments for children with FSHD and clarify which outcomes best show benefit.
How similar studies have gone: Prior cross-sectional data suggest links between early onset and severity, but prospective pediatric-wide studies are limited; this project aims to build on limited prior work with broader recruitment.
Where this research is happening
-
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.