Biomarker Development in LGMD2i

Completed

Conditions studied: Muscular Dystrophies, Limb Girdle Muscular Dystrophy

In brief

The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments (COAs), and to validate a muscle biomarker for LGMD2i to support therapeutic development.

Key facts

Study ID

NCT04202627

Run by

ML Bio Solutions, Inc.

People needed

101

Starts

2019-12-01

Expected to finish

2022-10-10

Last updated by the study team

2023-03-29

Who can join

Age: 10 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Age between 10-65 at enrollment
• Clinically affected (defined as weakness on bedside evaluation in either a limb-girdle pattern, or in a distal extremity)
• A genetically confirmed mutation in FKRP (LGMD2i)
• Willing and able to give informed consent and follow all procedures and requirements

You may not qualify if…

• Any other illness that would interfere with the ability to undergo safe testing or would interfere with interpretation of the results in the opinion of the site investigator.
• History of a bleeding disorder, platelet count <50,000, current use of an anticoagulant.
• Positive pregnancy test
• A 10-meter walk time of <4 seconds

Where it is running

• University of California Irvine — Irvine, California, United States
• University of Colorado Anschutz Medical Campus — Aurora, Colorado, United States
• University of Florida — Gainesville, Florida, 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
• Washington University School of Medicine — St Louis, Missouri, United States
• Atrium Health — Charlotte, North Carolina, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Virginia Commonwealth University — Richmond, Virginia, United States
• Copenhagen Neuromuscular Center — Copenhagen, Denmark

Full record on ClinicalTrials.gov

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