Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
NCT ID: NCT06847282
Last Updated: 2025-06-04
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
80 participants
OBSERVATIONAL
2025-05-22
2028-05-31
Brief Summary
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Detailed Description
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Prospective studies in early-onset FSHD have been limited by the small number of sites and low recruitment and follow-up rates. Early-onset pediatric FSHD is of high interest to drug companies because:
1. It results in a more significant disease burden than in adults.
2. Treating FSHD at earlier ages may have a more lasting and profound effect.
3. Genetic, molecular, and clinical factors may differ between pediatric and adult-onset FSHD.
4. Smaller body size and faster progression rates may make AAV-delivered gene therapies more feasible.
The FSHD CTRN's previous research showed that the FSHD composite functional measure (FSHD-COM), reachable workspace (RWS), and quantitative MRI measures (qMRI) are responsive to disease progression or treatment in adults with FSHD and correlate with performance. Investigators hypothesize that early changes in qMRI in pediatric subjects will predict 2-year changes in FSHD-COM Peds or RWS.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Cohort One
Cohort One will be individuals who are able to complete the 10 meter walk/run test in less than twelve seconds
No interventions assigned to this group
Cohort Two
cohort two will be individuals who complete the 10 meter walk/run test in more than 12 seconds or is no longer able to complete.
No interventions assigned to this group
Eligibility Criteria
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Inclusion Criteria
* 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
* 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
5 Years
17 Years
ALL
No
Sponsors
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National Institute of Neurological Disorders and Stroke (NINDS)
NIH
University of Rochester
OTHER
Stanford University
OTHER
Duke University
OTHER
University of California, Irvine
OTHER
Kennedy Krieger Institute, Baltimore, MD
UNKNOWN
University of Utah
OTHER
Seattle Children's Hospital
OTHER
University of Iowa
OTHER
Coriell Institute
UNKNOWN
Leiden University Medical Center
OTHER
Murdoch Childrens Research Institute
OTHER
University of Kansas Medical Center
OTHER
Responsible Party
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Jeffrey Statland
Professor
Principal Investigators
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Jeffrey Statland, MD
Role: PRINCIPAL_INVESTIGATOR
University of Kansas Medical Center
Locations
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Stanford University
Palo Alto, California, United States
University of Iowa
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
University of Rochester
Rochester, New York, United States
Duke University
Durham, North Carolina, United States
University of Utah
Salt Lake City, Utah, United States
Murdoch Children's Research Institute
Melbourne, , Australia
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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STUDY00160879
Identifier Type: -
Identifier Source: org_study_id
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