FSHDMOVE FSHDNCT04635891
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
An observational study of FSHD, run by University of Kansas Medical Center at 21 sites in 4 countries.
Trial summary
The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- FSHD
- How many people
- 450 participants
- Where
- 21 sitesin 4 countries
- Study dates
- 15 Dec 2020to Jan 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants with FSHD that are seen in the researchers clinic. This study is meant to be 'inclusive' and cover the full range of clinical FSHD: including children and adults, all ranges of abilities (ambulatory and non-ambulatory), and both genetic types of FSHD.
Who may be able to join
- Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring.
Who cannot join
- Unwilling or unable to provide informed consent.
- Any other medical condition which in the opinion of the investigator would interfere with study participation.
Trial locations and contacts
21 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
21 sites in 4 countries
United States16 sites, 16 recruiting
- Recruiting
University of Colorado Anschutz Medical Campus
Aurora, Colorado, 80045
303-724-4644, neurologyresearchpartners@cuanschutz.edu
Stacy Dixon, MD
- Recruiting
Austin Neuromuscular Center
Austin, Texas, 78759
Hina Patel, 512-920-0140 ext. 212, hina@austinneuromuscle.com
Emil Hussain, 512-920-0140 ext. 212, emil@austinneuromuscle.com
- Recruiting
Kennedy Krieger Institute
Baltimore, Maryland, 21205
Mary Yep, 443-923-7318, Yep@kennedykrieger.org
Geni Bibat, 423-923-2629, bibat@kennedykrieger.org
- Recruiting
The Ohio State University Medical Center
Columbus, Ohio, 43221
Alison Arter, 614-685-8661, Alison.Arter@osumc.edu
Marco Tellez, 614-688-7837, Marco.Tellez@osumc.edu
- Recruiting
Univeristy of Texas Southwestern Medical Center
Dallas, Texas, 75390
Tyler Arrington, 214-645-2759, Tyler.Arrington@UTSouthwestern.edu
Steve Hopkins, 214-648-9380, Steve.Hopkins@UTSouthwestern.edu
- Recruiting
Duke University - Lenox Baker Children's Hospital
Durham, North Carolina, 27705
Randy Smith, MPH, 919-684-1586, randy.s.smith@duke.edu
Stepanie Thera, stephanie.thera@duke.edu
- Recruiting
Univeristy of Florida Gainesville
Gainesville, Florida, 32608
Julie Segura, 352-733-2412, Julie.segura@neurology.ufl.edu
Subramony H Subramony, MD
- Recruiting
University of Iowa
Iowa City, Iowa, 52242
Catherine Buescher, MS, BHS, 319-353-6269, catherine-buescher@uiowa.edu
Carrie Stephan, MA, BSN, RN, 319-356-2673, carrie-stephan@uiowa.edu
- Recruiting
Univeristy of Kansas Medical Center
Kansas City, Kansas, 66160
Andrea Klempnauer, 913-574-0008, atenney@kumc.edu
Rebecca Clay, 913-945-9936, rclay@kumc.edu
- Recruiting
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
Dova Levin, 310-825-3264, dvlevin@mednet.ucla.edu
Merve Kaleli, 310-825-3264, MKaleli@mednet.ucla.edu
- Recruiting
Virginia Commonwealth University
Richmond, Virginia, 23219
Resheed Reid, 646-804-2367, resheed.reid1@vcuhealth.org
Levi Headrick, 804-828-6110, levi.headrick@vcuhealth.org
- Recruiting
University of Rochester Medical Center
Rochester, New York, 14642
Leann Lewis, MS, 585-275-7680, Leann_Lewis@URMC.Rochester.edu
Marisa Rosenberg, 585-274-0271, Marisa_Rosenberg@URMC.Rochester.edu
- Recruiting
University of Utah
Salt Lake City, Utah, 84132
Ryan Kennington, 801-587-0833, ryan.kennington@hsc.utah.edu
Caroline Flood, Caroline.Flood@hsc.utah.edu
- Not yet recruiting
University of Texas Health San Antonio
San Antonio, Texas, 78229
Marlon Humbert Tamayo Muradas, 210-450-7370, tamayomurada@uthscsa.edu
Edgar Morales, 210-567-8222, moralese6@uthscsa.edu
- Recruiting
University of Washington Medical Center
Seattle, Washington, 98195
Lilly Young, eyoung@uw.edu
Leo Wang, MD, PhD
- Recruiting
Neuromuscular Disorders Program at Stanford University School of Medicine
Stanford, California, 94306
650-725-4341, NeuromuscularResearch@stanford.edu
John Day, MD, PhD
Canada3 sites, 3 recruiting
- Not yet recruiting
University of Calgary
Calgary, Alberta, T2N1N4
Janet Petrillo, 403-210-7006, japetril@ucalgary.ca
Carissa Wong, 403-210-3835, carissa.wong@ucalgary.ca
- Not yet recruiting
University of McGill
Montreal, Quebec, H3A 2B4
Jessica Cuerquis, 514-396-1023, jessica.cuerquis@mcgill.ca
Audrey Trottier, audrey.trottier@mail.mcgill.ca
- Recruiting
Ottawa Hospital Research Institute
Ottawa, Ontario, K1y 4e9
Jessica Belanger, 613-798-5555 ext. #19627, jemacgregor@ohri.ca
Kathryn Beaudry, 617-798-5555 ext. #19864, kabeaudry@ohri.ca
Brazil1 site, 1 recruiting
- Recruiting
University of Sao Paulo
São Paulo
Eliene Dutra Campos, +55 11 3061-8553, eliene.dcampos@hc.fm.usp.br
Cristiane Moreno, MD
United Kingdom1 site, 1 recruiting
- Recruiting
Sheffield Teaching Hospital
Sheffield, South Yorkshire, S10 2JF
Jon Street, 4-411-4271-1626, j.street1@nhs.net
sth.neuromuscularresearch@nhs.net
About this study
The primary goal of this proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and reachable workspace, as well as optional muscle biopsy and wearable device (US participants only).
- Conditions
- FSHD
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 |
|---|---|---|
| MOVE FSHD Study Visits | Patients will receive standard of care as determined by their treating physician. Study visits will occur per standard of care and are anticipated to occur at least once a year. |
What is being measured
The primary outcome is the main question the study is designed to answer.
10m walk/run
The 10-meter walk/run (previously the 30 foot go) or gait speed task will be performed during study visits. This task tests a range of different abilities, from power, to endurance, and balance. Also, the 10 meter walk/run is a predictor of loss of ambulation in Duchenne Muscular Dystrophy.
Measured: Baseline - 5 years
Shoulder and Arm Range of Motion
Range of motion tasks mimicking lifting or reaching up will be performed.
Measured: Baseline - 5 years
Shoulder and Arm Function
Participants will be timed on stacking cans.
Measured: Baseline - 5 years
Spirometry (FVC, FEV1, PCF)
Investigators will obtain forced vital capacity and forced expiratory volume in 1 second, both standardized outcomes used commonly in clinic and clinical trials. Also, for sites who routinely collect Peak Cough Flow this will also be obtained.
Measured: Baseline - 5 years
Secondary outcomes (13)
Trunk Function
There are no specific functional tasks designed to measure trunk function in FSHD so Investigators have chosen a practical task that will reflect changes in core truncal muscle groups. The ability to sit up and the timed supine to sitting test reflects core muscle strength and coordination.
Measured: Baseline - 5 years
Hand Function
Hand function is captured by examining hand grip strength.
Measured: Baseline - 5 years
Timed Up and Go (TUG)
Balance and mobility are assessed by using the Timed Up and Go (TUG), a standard outcome measure for the elderly that is also increasingly being used in neuromuscular disorders. Participants are asked to rise from a chair, walk 3 meters, turn 180 degrees and return to a seated position in the chair.
Measured: Baseline - 5 years
Saliva Methylation
Investigators will isolate DNA from blood cells in saliva - and will determine the methylation levels in the 4q region on chromosome 4 using this new technique. Investigators will compare methylation levels between participants and compare to other clinical information collected in this study.
Measured: Baseline (collected only once)
FSHD Clinical Severity Scores
A limited physical exam and strength testing will be used to derive two FSHD clinical severity scores. These severity scores both rank weakness in the face, shoulders, arms, distal, and proximal lower extremities on either a 10 or 15 point scale. The higher the severity score the more affected the individual.
Measured: Baseline - 5 years
Patient-Reported Outcomes Measurement Information System-57 (PROMIS57)
The PROMIS57 is an instrument developed by the NIH which generates scores for physical function, and the impact of physical limitations on daily life. 57 questions are summed into a total score, which is transformed into a normalized t-score with 50 representing normal, and lower scores representing increasing disability.
Measured: Baseline - 5 years
The Upper Extremity Functional Index
This index measures upper extremity dysfunction. 20 questions are combined into a total score, the score is transformed into a normalized score with 80 representing normal, and lower scores representing increasing disability.
Measured: Baseline - 5 years
Exercise and Pain Assessment
An exercise and pain assessment questionnaire was comprised specifically for this study and will be completed at every visit.
Measured: Baseline - 5 years
The Facial Disability Index (FDI)
The FDI is a short 5 item questionnaire. The five questions are summed into total score which transformed onto a percentage scale, with 100 representing normal, and lower scores representing increasing disability.
Measured: Baseline - 5 years
FSHD Rasch-built overall disability scales (FSHD-RODS)
The FSHD-RODS is a questionnaire about the relationship between daily activities and health. 32 questions are are constructed using a modern clinimetric technique, the Rasch analysis, which by ordering the items in increasing difficulty, allows for the calculation of the sum of item scores to achieve a total score. Answers provide information about how FSHD affects daily and social activities and to what degree participants are able to perform usual activities.
Measured: Baseline - 5 years
MIP/MEP and SNIP
For sites who routinely obtain negative inspiratory or expiratory force (MIP/MEP) will be collected.
Measured: Baseline - 5 years
4 Stair Climb
Time to ascend and descend 4 standard stairs.
Measured: Baseline - 5 years
Manual Muscle Testing (MMT)
For MMT a modified Medical Research Council 13-point scale will be used - with standardized positions for each muscle.
Measured: Baseline - 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2020
- Study start15 Dec 2020
- Primary completionDec 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for FSHDStudies 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
- 450 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche, Springbok Analytics, Novartis Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Jeffrey Statland, MD, Principal investigator, University of Kansas Medical CenterRabi Tawil, MD, Principal investigator, University of Rochester
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04635891
- Sponsor's protocol ID
- STUDY00145405
- Acronym
- MOVE FSHD
- Status last verified
- May 2026
- First submitted
- 29 Oct 2020
- Last updated
- 7 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
- ClinicalTrials.gov ID
- NCT04635891
- Acronym / sponsor ID
- MOVE FSHD / STUDY00145405
- Conditions
- FSHD
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 450 participants (estimated)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Collaborators
- FSHD Society, Inc., Friends Research Institute, Inc., University of Rochester, University of Nevada, Reno, FSHD Canada, Avidity Biosciences, Inc., AMRA Medical, Seattle Children's Hospital, Dyne Therapeutics, Hoffmann-La Roche, Springbok Analytics, Novartis Pharmaceuticals
- Study start
- 15 Dec 2020 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 19 Nov 2020 (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 proposal is to collect motor and functional outcomes specific to FSHD over time. By collecting measures specific to FSHD, this will help ensure the best level of clinical care is being provided. Also, the hope is to speed up drug development by gaining a better understanding of how having FSHD impacts motor function and other health outcomes (i.e. breathing, wheelchair use, etc.) and how big a change in motor function would be clinically meaningful to those with FSHD.
Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD) will have approximately 450 FSHD participants followed for a minimum of 3 years. A subset of MOVE FSHD participants, approximately 200, will participate in the MOVE+ sub-study which includes whole body MRI and reachable workspace, as well as optional muscle biopsy and wearable device (US participants only).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MOVE FSHD Study Visits | – | –Patients will receive standard of care as determined by their treating physician. Study visits will occur per standard of care and are anticipated to occur at least once a year. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants with FSHD that are seen in the researchers clinic. This study is meant to be 'inclusive' and cover the full range of clinical FSHD: including children and adults, all ranges of abilities (ambulatory and non-ambulatory), and both genetic types of FSHD.
Key inclusion criteria (1 listed)
- Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring.
Key exclusion criteria (2 listed)
- Unwilling or unable to provide informed consent.
- Any other medical condition which in the opinion of the investigator would interfere with study participation.
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
10m walk/run
Time frame: Baseline - 5 years
The 10-meter walk/run (previously the 30 foot go) or gait speed task will be performed during study visits. This task tests a range of different abilities, from power, to endurance, and balance. Also, the 10 meter walk/run is a predictor of loss of ambulation in Duchenne Muscular Dystrophy.
Shoulder and Arm Range of Motion
Time frame: Baseline - 5 years
Range of motion tasks mimicking lifting or reaching up will be performed.
Shoulder and Arm Function
Time frame: Baseline - 5 years
Participants will be timed on stacking cans.
Spirometry (FVC, FEV1, PCF)
Time frame: Baseline - 5 years
Investigators will obtain forced vital capacity and forced expiratory volume in 1 second, both standardized outcomes used commonly in clinic and clinical trials. Also, for sites who routinely collect Peak Cough Flow this will also be obtained.
13 secondary outcomes
Trunk Function
Time frame: Baseline - 5 years
Hand Function
Time frame: Baseline - 5 years
Timed Up and Go (TUG)
Time frame: Baseline - 5 years
Saliva Methylation
Time frame: Baseline (collected only once)
FSHD Clinical Severity Scores
Time frame: Baseline - 5 years
Patient-Reported Outcomes Measurement Information System-57 (PROMIS57)
Time frame: Baseline - 5 years
The Upper Extremity Functional Index
Time frame: Baseline - 5 years
Exercise and Pain Assessment
Time frame: Baseline - 5 years
The Facial Disability Index (FDI)
Time frame: Baseline - 5 years
FSHD Rasch-built overall disability scales (FSHD-RODS)
Time frame: Baseline - 5 years
MIP/MEP and SNIP
Time frame: Baseline - 5 years
4 Stair Climb
Time frame: Baseline - 5 years
Manual Muscle Testing (MMT)
Time frame: Baseline - 5 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 7 May 2026.
Related trials for FSHD
Active, not recruiting
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
RecruitingPhase 1/2
A Trial to Assess Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of SFL-0821 in Adults With FSHD
Facioscapulohumeral Muscular Dystrophy (FSHD)
RecruitingObservational study
Biomarker Development for Muscular Dystrophies
Myotonic Dystrophy, Duchenne Muscular Dystrophy
RecruitingPhase 1
Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
FSHD - Facioscapulohumeral Muscular Dystrophy
Not yet recruitingPhase 2
A Study of SAT-3247 to Evaluate the Safety, Tolerability, and Efficacy in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular Dystrophy (FSHD)
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
Rabi Tawil, MD principal investigator
University of Rochester
21 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of FSHD, run by University of Kansas Medical Center at 21 sites in 4 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 1 inclusion and 2 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 7 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 21 sites in United States, Canada, Brazil, United Kingdom. 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?
10m walk/run (time frame: Baseline - 5 years). The study lists 4 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 7 May 2026.