Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: FSHD. Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Brazil, Canada, United Kingdom
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Overview
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).
Primary outcome measures
- 10m walk/run [Time frame: Baseline - 5 years]
- Shoulder and Arm Range of Motion [Time frame: Baseline - 5 years]
- Shoulder and Arm Function [Time frame: Baseline - 5 years]
- Spirometry (FVC, FEV1, PCF) [Time frame: Baseline - 5 years]
Secondary outcome measures (12)
- 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]
Eligibility criteria
Inclusion criteria
- Genetically confirmed FSHD (types 1 or 2) or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring.
Exclusion criteria
- Unwilling or unable to provide informed consent.
- Any other medical condition which in the opinion of the investigator would interfere with study participation.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 16 centers
- David Geffen School of Medicine at UCLA — Los Angeles
- Neuromuscular Disorders Program at Stanford University School of Medicine — Stanford
- University of Colorado Anschutz Medical Campus — Aurora
- Univeristy of Florida Gainesville — Gainesville
- University of Iowa — Iowa City
- Univeristy of Kansas Medical Center — Kansas City
- Kennedy Krieger Institute — Baltimore
- University of Rochester Medical Center — Rochester
- … and 8 more centers
Canada · 3 centers
- University of Calgary — Calgary
- Ottawa Hospital Research Institute — Ottawa
- University of McGill — Montreal
Brazil · 1 center
- University of Sao Paulo — São Paulo
United Kingdom · 1 center
- Sheffield Teaching Hospital — Sheffield
Identifiers
NCT: NCT04635891 · STUDY00145405