BECKER MUSCULAR DYSTROPHY - A Natural History Study to Predict Efficacy of Exon Skipping

 

Frequently Asked Questions (FAQs) about this study:

 

What stage is this research?

  • This study is closed to new enrollment currently, but actively following previously enrolled participants. We are very pleased to have reached and even exceeded our enrollment goals with 85 participants enrolled. The study enrollment officially closed on March 31, 2016.

 

What is the goal or purpose of this study?

  • This is a natural history study to characterize the Becker muscular dystrophy clinical presentation and also to provide information regarding the possible effectiveness of a therapy currently in development for Duchenne muscular dystrophy (exon skipping). Researchers will correlate specific genetic changes with a range of clinical outcomes including physical development, mental development, and quality of life in patients with Becker. The observed variability between individuals with Becker will be studied to deepen our understanding of molecular mechanisms relevant to the optimization of exon skipping therapeutic approaches, as well as to optimize study designs and outcome measures for future clinical trials in Becker.  

 

Who is funding this study?

  • This study is funded by the National Institutes of Health (NIH).

 

Who is eligible to participate in this study?

  • Enrolled participants were male with Becker, age 4 and above, and have an in-frame dystrophin Glossary Link gene deletion where the boundaries of the mutations are confirmed.  

 

Where does this study take place?

  • This study will be run at select Cooperative International Neuromuscular Research Group (CINRG) network sites and affiliates. The participating centers include:
    • Children’s National Medical Center in Washington DC
    • University of Pittsburgh in Pittsburgh, PA
    • Penn State Hershey Medical Center in Hershey, PA
    • Carolinas Medical Center in Charlotte, NC
    • Duke University in Durham, NC
    • University of Minnesota in Minneapolis, MN
    • Ann & Robert H. Lurie Children's Hospital of Chicago in Chicago, IL
    • University of Tennessee in Memphis, TN.
    • University of Florida in Gainesville, FL
    •  Children's Healthcare of Atlanta/Emory University in Atlanta, GA
    • University of California, Davis, in Sacramento, CA
    • Alberta Children's Hospital in Calgary, Canada
    • Newcastle University in Newcastle upon Tyne, United Kingdom
    • Centro Clinico Nemo in Milan, Italy 
    • UT Southwestern Medical Center in Dallas, TX

 

Is there any funding to help pay for travel?

  • The study has received a grant from the Muscular Dystrophy Association (MDA) to assist participants with their travel for follow-up visits. Participants should contact their study sites to obtain this support for their next visits.

 

Where can I learn more about this study?

 

(Updated 4/19/2017)