Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

Condition(s) studied

Study 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. 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).

Eligibility

Primary outcome measure(s)

• 10m walk/run — 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 — Baseline - 5 years
  Range of motion tasks mimicking lifting or reaching up will be performed.
• Shoulder and Arm Function — Baseline - 5 years
  Participants will be timed on stacking cans.
• Spirometry (FVC, FEV1, PCF) — 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.

Trial sites (21)

Other trials for the same condition