Clinical Trial Readiness to Solve Barriers to Drug Development in FSHD

Summary

The primary cause of facioscapulohumeral muscular dystrophy (FSHD), a common adult-onset dystrophy, was recently discovered identifying targets for therapy. As multiple drug companies pursue treatments for FSHD, there is an urgent need to define the clinical trial strategies which will hasten drug development, including creating disease-relevant outcome measures and optimizing inclusion criteria. This proposal will develop two new outcome measures (FSHD-COM and EIM) and optimize eligibility criteria by testing 320 patients across 14 international sites over a period of 24 months.

Conditions

• Facioscapulohumeral Muscular Dystrophy

Interventions

DIAGNOSTIC_TEST

FSHD-specific functional rating scale

The FSHD-COM is composed of disease-relevant functional tasks such as leg function; shoulder and arm function; trunk function, hand function, and balance.

DEVICE

Electrical Impedance Myography

EIM is a non-invasive, painless, and fast technique for obtaining information on how a patient's muscle structure is changing. EIM uses a small electrical current to measure the health of the underlying muscle. The patient will be asked to lie down and a trained clinical evaluator will perform testing on 16 total muscles (8 on each side) on your arms and legs.

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS)

  collaborator NIH

• FSHD Society

  collaborator OTHER

• Friends Research Institute, Inc.

  collaborator OTHER

• Muscular Dystrophy Association

  collaborator OTHER

• AFM Telethon

  collaborator UNKNOWN

• University of Rochester

  collaborator OTHER

• Leiden University Medical Center

  collaborator OTHER

• Dyne Therapeutics

  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

Min Age

18 Years

Max Age

75 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2018-03-05

Primary Completion

2027-12-31

Completion

2027-12-31

FDA Device

Yes

Countries

• United States
• France
• Germany
• Italy
• Netherlands
• United Kingdom

Study Locations