Motor Outcomes to Validate Evaluations in FSHD (MOVE FSHD)

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).

Conditions

• FSHD

Sponsors & Collaborators

• FSHD Society, Inc.

  collaborator UNKNOWN

• Friends Research Institute, Inc.

  collaborator OTHER

• University of Rochester

  collaborator OTHER

• University of Nevada, Reno

  collaborator OTHER

• FSHD Canada

  collaborator UNKNOWN

• Avidity Biosciences, Inc.

  collaborator INDUSTRY

• AMRA Medical

  collaborator UNKNOWN

• Seattle Children's Hospital

  collaborator OTHER

• Dyne Therapeutics

  collaborator INDUSTRY

• Hoffmann-La Roche

  collaborator INDUSTRY

• Springbok Analytics

  collaborator UNKNOWN

• Novartis Pharmaceuticals

  collaborator INDUSTRY

• University of Kansas Medical Center

  lead OTHER

Principal Investigators

• Jeffrey Statland, MD · University of Kansas Medical Center

• Rabi Tawil, MD · University of Rochester

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2020-12-15

Primary Completion

2027-12-31

Completion

2028-01-31

Countries

• United States
• Brazil
• Canada
• United Kingdom

Study Locations