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

NCT01539772 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 85

Last updated 2018-06-15

No results posted yet for this study

Summary

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.

Conditions

  • Becker Muscular Dystrophy

Sponsors & Collaborators

  • Cooperative International Neuromuscular Research Group

    lead NETWORK

Principal Investigators

  • Paula R Clemens, MD · University of Pittsburgh

Eligibility

Min Age
4 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2012-04-30
Primary Completion
2018-08-31
Completion
2018-08-31

Countries

  • United States
  • Canada
  • Italy
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01539772 on ClinicalTrials.gov