New study aims to sharpen tools for testing kids with rare muscle disease
NCT ID NCT06847282
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study is testing different ways to measure muscle strength and movement in children with a rare muscle disease called FSHD. The goal is to find the best tests to use in future studies of new treatments. About 80 children aged 5 to 17 with confirmed FSHD will take part. The results will help design better clinical trials for this condition.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Participants
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About 80 people
The number the study aims to enrol. It can still change while the study runs.
- Started
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May 2025
- Expected to finish
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May 2028
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
Who is studied
This study is meant to target children most likely to be included in clinical trials: children who are clinically affected, old enough to perform functional measures, and still ambulatory. On outcomes proposed for MOVE Peds they would have room to show progression or improvement and be at a stage ideal to assess potential inclusion criteria: in particular whether symptom onset age or genetics separate early-onset from other childhood onset FSHD, or whether they exist on a spectrum of severities. At least half will qualify as early-onset (facial weakness before age 5 and shoulder weakness before age 10 or symptom onset before age 18 and 1-3 D4Z4 repeats). We will be also looking at a small cohort of early onset participants who's ambulation has been affected or is no longer able to ambulate. this will help us assess all child progression and understand the connection of early-onset and ambulation
- Ages
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5 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The places running it
7 sites in 2 countries. The list below names each one and where it is.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
Contacts and locations
Locations
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Duke University
RECRUITINGDurham, North Carolina, 27708, United States
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Murdoch Children's Research Institute
RECRUITINGMelbourne, Australia
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Stanford University
NOT_YET_RECRUITINGPalo Alto, California, 94304, United States
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University of Iowa
NOT_YET_RECRUITINGIowa City, Iowa, 52242, United States
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University of Kansas Medical Center
RECRUITINGKansas City, Kansas, 66160, United States
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University of Rochester
RECRUITINGRochester, New York, 14642, United States
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University of Utah
NOT_YET_RECRUITINGSalt Lake City, Utah, 84112, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New hope for FSHD: Long-Term drug safety trial underway
- New hope for FSHD: experimental drug AOC 1020 completes early testing
- FSHD patients unite: new registry aims to accelerate research and care
- Could a common asthma drug help with a rare muscle disease? new study launches.
- New hope for rare muscle disease: experimental drug enters human trials