Muscular Dystrophy, FacioscapulohumeralMOVE PedsNCT06847282
Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
An observational study of Muscular Dystrophy, Facioscapulohumeral, run by University of Kansas Medical Center at 7 sites in 2 countries.
Trial summary
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD Read more about this study
- Who is it for?
- Ages 5 to 17 years
- Conditions the study is for
- Muscular Dystrophy, Facioscapulohumeral
- How many people
- 80 participants
- Where
- 7 sitesin 2 countries
- Study dates
- 22 May 2025to May 2028
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: 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
Who may be able to join
- 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%)
Who cannot join
- 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
Trial locations and contacts
7 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 2 countries
United States6 sites, 6 recruiting
- Recruiting
Duke University
Durham, North Carolina, 27708
Randy Smith, MPH, 919-684-1586, randy.s.smith@duke.edu
Stephanie Thera, stephanie.thera@duke.edu
- Not yet recruiting
University of Iowa
Iowa City, Iowa, 52242
Carrie Stephan, MA, BSN, RN, 319-356-2673, carrie-stephan@uiowa.edu
Catherine Buescher, MS, BHS, 319-353-6269, catherine-buescher@uiowa.edu
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Michaela Walker, MPH, 913-945-9920, mwalker20@kumc.edu
Rebecca Clay, 913-945-9936, rclay@kumc.edu
- Not yet recruiting
Stanford University
Palo Alto, California, 94304
Lin Karman, B.S., 650-384-9417, lkarman@stanford.edu
Sabrina Salvatore, MA, 650-387-5336, ssalvato@stanford.edu
- Recruiting
University of Rochester
Rochester, New York, 14642
Leann Lewis, MsGC, 585-275-7680, Leann_Lewis@URMC.Rochester.edu
Marisa Severino, 585-274-0271, Marisa_Severino@URMC.Rochester.edu
- Not yet recruiting
University of Utah
Salt Lake City, Utah, 84112
Ryan Kennington, 801-587-0833, ryan.kennington@hsc.utah.edu
Caroline Flood, Caroline.Flood@hsc.utah.edu
Australia1 site, 1 recruiting
- Recruiting
Murdoch Children's Research Institute
Melbourne
Katy deValle, BApp Sc Physiotherapy, PhD, +61 03 9345 4287, Katy.DeValle@rch.org.au
Ian R Woodcock, MBBS PhD MSc MRCPCH(UK) FRACP
About this study
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
Read the detailed description
MOVE Peds is a prospective 2-year study recruiting eighty pediatric participants to accelerate therapeutic development for pediatric-onset FSHD. The study aims to validate outcomes and refine clinical trial strategies. Previous cross-sectional studies suggest that younger age of onset is linked to greater clinical severity and that having 1-3 D4Z4 repeats is associated with extra-muscular complications in pediatric FSHD.
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:
- It results in a more significant disease burden than in adults.
- Treating FSHD at earlier ages may have a more lasting and profound effect.
- Genetic, molecular, and clinical factors may differ between pediatric and adult-onset FSHD.
- 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
- Muscular Dystrophy, Facioscapulohumeral
- Keywords
- FSHD, Pediatric
- Medical subject headings
- Muscular Dystrophy, Facioscapulohumeral
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Cohort One | Cohort One will be individuals who are able to complete the 10 meter walk/run test in less than twelve seconds | |
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pediatric FSHD-COM
Investigators will collect the FSH-Composite outcome measure for children and adolescents. This measure assesses multiple body regions identified as important by patients, including leg function, shoulder and arm function, trunk function, hand function, and functional balance. The FSHD-COM Peds has been modified to include lighter weights for shoulder abduction and flexion, as well as elbow flexion, to accommodate the motor development and lower strength levels seen in children. Although the FSHD-COM Peds involves different measurements for each body region, these measurements are combined to produce a single composite score. The total score for the FSHD-COM Peds is out of 84, representing one comprehensive outcome measure
Measured: Baseline-2 years
Reachable Work Space
Subjects are seated in front of a stereo-camera and perform a standardized upper extremity movement protocol under the supervision of a study clinical evaluator. Five-hundred-gram wrist weights will be added. The standardized simple set of movements consist of lifting the arm from the resting position to above the head while keeping the elbow extended, performing the same movement in vertical planes at around 0, 45, 90, 135 degrees. The second set of movements consists of horizontal sweeps at the level of the umbilicus and shoulder.
Measured: Baseline-2 years
Secondary outcomes (4)
Quantitative MRI (qMRI) Fat Fraction %
quantitative MRI measurements are considered gold standard for lean muscle volume measurement and disease biomarkers (fat volumes, fat faction, and STIR+ presence) that may be modulated by treatment. Muscle fat replacement and free-water hyperintensity indicate early disease changes before functional performance is affected. Muscles with an intermediate fat fraction (10-55%) predict disease progression over 1-2 years.
Investigators will use a whole-body MRI and will find the fat fraction % in pediatric subjects and compare these measurements with other factors to create a composite score over a two-year period
Measured: Baseline-2 years
Quantitative MRI (qMRI) Lean Muscle Volume (mL)
Quantitative MRI measurements are considered the gold standard for assessing lean muscle volume and disease biomarkers (fat volumes vs. lean muscle volumes and STIR+ presence) that may be modulated by treatment. Muscle fat replacement and free-water hyperintensity indicate early disease changes before functional performance is affected. Muscles with low lean muscle volume and an intermediate fat fraction (10-55%) predict disease progression over 1-2 years.
Investigators will use whole-body MRI to measure lean muscle volume (mL) in pediatric subjects and compare these measurements with other factors to create a composite score over a two-year period.
Measured: Baseline-2 years
Quantitative MRI (qMRI) Total Volume
Quantitative MRI measurements are considered the gold standard for assessing lean muscle volume and disease biomarkers (fat volumes, fat fraction and STIR+ presence) that may be modulated by treatment. The use of MRI has shown as a technique to predict disease progression.
Investigators will use whole-body MRI to measure total volume (mL) in pediatric subjects and compare these measurements with other factors to create a composite score over a two-year period. This refers to the overall volume of a specific tissue or organ measured in milliliters (mL) using MRI.
Measured: Baseline-2 years
Quantitative MRI (qMRI) STIR+ ( %)
Quantitative MRI measurements are considered the gold standard for assessing lean muscle volume and disease biomarkers, such as the percentage of lean muscle volume with Short Tau Inversion Recovery (STIR) enhancement. STIR MRI helps predict disease progression by highlighting areas of inflammation and edema within muscle tissue. This provides valuable insights into underlying disease processes, aiding in the prediction of progression and guiding treatment decisions.
Investigators will use whole-body MRI to measure STIR positivity, indicating inflammation and edema, in pediatric subjects. These measurements will be compared with other factors to create a composite score over a two-year period.
Measured: Baseline-2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Feb 2025
- Study start22 May 2025
- Primary completionMay 2027 (estimated)
- Study completionMay 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, FacioscapulohumeralStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester, Stanford University, Duke University, University of California, Irvine, Kennedy Krieger Institute, Baltimore, MD, University of Utah, Seattle Children's Hospital, University of Iowa, Coriell Institute, Leiden University Medical Center, Murdoch Childrens Research Institute
- Responsible party
- Principal investigator, Jeffrey Statland, University of Kansas Medical Center
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06847282
- Sponsor's protocol ID
- STUDY00160879
- Other IDs
- 1U01NS137702-01
- Acronym
- MOVE Peds
- Status last verified
- Feb 2026
- First submitted
- 23 Jan 2025
- Last updated
- 25 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds)
- ClinicalTrials.gov ID
- NCT06847282
- Acronym / sponsor ID
- MOVE Peds / STUDY00160879
- Conditions
- Muscular Dystrophy, Facioscapulohumeral
- Keywords
- FSHD, Pediatric
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS), University of Rochester, Stanford University, Duke University, University of California, Irvine, Kennedy Krieger Institute, Baltimore, MD, University of Utah, Seattle Children's Hospital, University of Iowa, Coriell Institute, Leiden University Medical Center, Murdoch Childrens Research Institute
- Study start
- 22 May 2025 (actual)
- Primary completion
- May 2027 (estimated)
- Study completion
- May 2028 (estimated)
- First posted
- 26 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary goal of this study is to validate motor and functional outcomes and refine clinical trial strategies for pediatric-onset FSHD
Detailed description
MOVE Peds is a prospective 2-year study recruiting eighty pediatric participants to accelerate therapeutic development for pediatric-onset FSHD. The study aims to validate outcomes and refine clinical trial strategies. Previous cross-sectional studies suggest that younger age of onset is linked to greater clinical severity and that having 1-3 D4Z4 repeats is associated with extra-muscular complications in pediatric FSHD.
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:
- It results in a more significant disease burden than in adults.
- Treating FSHD at earlier ages may have a more lasting and profound effect.
- Genetic, molecular, and clinical factors may differ between pediatric and adult-onset FSHD.
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort One | – | –Cohort One will be individuals who are able to complete the 10 meter walk/run test in less than twelve seconds |
| 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. |
Who may qualify
- Ages
- 5 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- 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
Key inclusion criteria (4 listed)
- 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%)
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Pediatric FSHD-COM
Time frame: Baseline-2 years
Investigators will collect the FSH-Composite outcome measure for children and adolescents. This measure assesses multiple body regions identified as important by patients, including leg function, shoulder and arm function, trunk function, hand function, and functional balance. The FSHD-COM Peds has been modified to include lighter weights for shoulder abduction and flexion, as well as elbow flexion, to accommodate the motor development and lower strength levels seen in children. Although the FSHD-COM Peds involves different measurements for each body region, these measurements are combined to produce a single composite score. The total score for the FSHD-COM Peds is out of 84, representing one comprehensive outcome measure
Reachable Work Space
Time frame: Baseline-2 years
Subjects are seated in front of a stereo-camera and perform a standardized upper extremity movement protocol under the supervision of a study clinical evaluator. Five-hundred-gram wrist weights will be added. The standardized simple set of movements consist of lifting the arm from the resting position to above the head while keeping the elbow extended, performing the same movement in vertical planes at around 0, 45, 90, 135 degrees. The second set of movements consists of horizontal sweeps at the level of the umbilicus and shoulder.
4 secondary outcomes
Quantitative MRI (qMRI) Fat Fraction %
Time frame: Baseline-2 years
Quantitative MRI (qMRI) Lean Muscle Volume (mL)
Time frame: Baseline-2 years
Quantitative MRI (qMRI) Total Volume
Time frame: Baseline-2 years
Quantitative MRI (qMRI) STIR+ ( %)
Time frame: Baseline-2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeffrey Statland, MD principal investigator
University of Kansas Medical Center
7 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Dystrophy, Facioscapulohumeral, run by University of Kansas Medical Center at 7 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 25 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Michaela Walker, MPH (913-945-9920, mwalker20@kumc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pediatric FSHD-COM (time frame: Baseline-2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2026.