Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy (LGMD)

Completed

Conditions studied: Limb Girdle Muscular Dystrophy, Muscular Dystrophies

In brief

Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles. While the individual genetic mutations are rare, as a cohort, LGMDs are one of the four most common muscular dystrophies. The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments (COAs) for limb girdle muscular dystrophies (LGMD) to hasten therapeutic development.

Key facts

Study ID

NCT03981289

Run by

Virginia Commonwealth University

People needed

116

Starts

2019-06-14

Expected to finish

2025-06-30

Last updated by the study team

2026-02-27

Who can join

Age: 4 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

Where it is running

• University of California Irvine — Irvine, California, United States
• The University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• University of Iowa — Iowa City, Iowa, United States
• University of Kansas Medical Center — Kansas City, Kansas, United States
• Kennedy Krieger Institute — Baltimore, Maryland, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University School of Medicine — St Louis, Missouri, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• John Walton Muscular Dystrophy Research Centre (Newcastle Upon Tyne) — Newcastle, United Kingdom

Full record on ClinicalTrials.gov

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