Becker Muscular Dystrophy - A Natural History Study to Predict Efficacy of Exon Skipping

Status unconfirmed

Conditions studied: Becker Muscular Dystrophy

In brief

This is a multi-center natural history study that will be conducted at participating centers in the Cooperative International Neuromuscular Research Group (CINRG). Following a baseline evaluation, participants will have three follow-up visits over a three-year period. The investigators will characterize the Becker muscular dystrophy phenotype, and correlate specific abnormal dystrophin proteins with the range of clinical outcomes.

Key facts

Study ID

NCT01539772

Run by

Cooperative International Neuromuscular Research Group

People needed

85

Starts

2012-04-01

Expected to finish

2018-08-01

Last updated by the study team

2018-06-15

Who can join

Age: 4 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Male
• Age 4 or older
• Diagnosis of BMD with an in-frame deletion in the dystrophin gene, where the boundaries of the mutations are confirmed.

You may not qualify if…

• Investigator assessment of inability to comply with protocol

Where it is running

• University of California Davis — Sacramento, California, United States
• Children's National Health System — Washington D.C., District of Columbia, United States
• University of Florida — Gainesville, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois, United States
• University of Minnesota — Minneapolis, Minnesota, United States
• Washington University — St Louis, Missouri, United States
• Carolinas Medical Center — Charlotte, North Carolina, United States
• Duke Children's Hospital and Health Center — Durham, North Carolina, United States
• Penn State Hershey Medical Center — Hershey, Pennsylvania, United States
• University of Pittsburgh — Pittsburgh, Pennsylvania, United States
• University of Tennessee — Memphis, Tennessee, United States
• UT Southwestern Medical Center — Dallas, Texas, United States
• Alberta Children's Hospital — Calgary, Alberta, Canada
• Centro Clinico Nemo — Milan, Italy
• Institute of Genetic Medicine - Newcastle University — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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