Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

University of Kansas

Status: Enrolling

Conditions: FSHD

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT04635891

STUDY00145405

Details and patient eligibility

About

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).

Enrollment: 450 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

450 participants in 1 patient group

MOVE FSHD Study Visits

Description:

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.