Getting treatments to young patients

MOVE Peds is essential to pave the way

by Natalie Katz, MD, and Katy de Valle, research physical therapist

More than 50% of people with FSHD develop symptoms as children or teens. When treatments to slow or stop FSHD are approved, common sense suggests that it will be best to treat patients as early as possible to save healthy muscles. Yet obstacles stand in the way of reaching this common-sense goal. Most importantly, data are scarce on how symptoms evolve over time in pediatric populations.

There have been few studies evaluating the sensitivity of “clinical trial outcome measures,” which are methods to measure whether children on treatment are benefiting compared to those who are untreated. To overcome this challenge, Jeffrey Statland, MD, of the University of Kansas (KUMC), and Ian Woodcock, MD, of Murdoch Children’s Research Institute, are co-leading an international effort to better understand disease progression in childhood-onset FSHD.

This prospective natural history study, MOVE Peds, will be similar to the ongoing MOVE (Motor Outcomes to Validate Evaluations) FSHD study, but specifically designed for children. Initial evaluations will be conducted at seven centers across the US and one in Australia. Additional sites may be added in the future.

What to expect if your child participates

Participants in the study will be seen at six-month intervals for a total of five visits over a 24-month period. At each visit, participants will complete a variety of assessments including: Strength testing, functional measures (mainly consisting of how quickly they are able to complete certain tasks), breathing tests, collection of biological samples for biomarker development, and surveys to capture different aspects of how FSHD affects their lives. One of the most exciting things about this study is that children will also be evaluated using Reachable Workspace and whole-body magnetic resonance imaging – the first time these assessments will be done specifically in children with FSHD.

We anticipate the study will begin enrolling toward the end of 2024. Our goal is to recruit at least 80 children with FSHD who are 5 to 18 years old at the time they enroll. Participants must have genetic confirmation of FSHD as well as clinical symptoms consistent with FSHD to enroll. Many companies are actively developing treatments for FSHD, and several are interested in making these available to children. We hope MOVE Peds will help make this vision a reality.

Where will the study be done?

IN THE US

To enroll, contact: Michaela Walker (mwalker20@kumc.edu) • Leann Lewis (Leann_Lewis@URMC.Rocester.edu) • Rebecca Clay (rclay@kumc.edu)

• University of Kansas, Kansas City, Kansas
• University of Rochester, Rochester, New York
• Duke University, Raleigh-Durham, North Carolina
• Nationwide Children’s Hospital, Columbus, Ohio
• Stanford University, Palo Alto, California
• University of Utah, Salt Lake City, Utah
• University of Iowa, Iowa City, Iowa

IN AUSTRALIA

To enroll, contact Katy de Valle (katy.devalle@rch.org.au)

• Murdoch Children’s Research Institute, Melbourne