Phase 1/2 study of Nationwide Micro-Dystrophin Gene Delivery in people with Duchenne

Questions and Answers

What stage is this research?

This Phase 1/2a clinical trial is actively recruiting participants.

What is the goal or purpose of this study?

The goal of this study is evaluate safety and efficacy in 12 boys with Duchenne following a single infusion of rAAVrh74.MHCK7.micro-dystrophin.

Who is funding this study?

This study is sponsored by Nationwide Children’s Hospital and was funded by Parent Project Muscular Dystrophy.

Who is eligible to participate in this study?

To participate in this study you must be a boy with Duchenne, between 3 months and 7 years of age, ambulatory, and have a Duchenne gene mutation between exons 18-58. For a complete list of all the inclusion and exclusion criteria, please visit ClinicalTrials.gov.

What do I have to do if I decide to participate in this study?

This study involves a single infusion in the arm of the rAAVrh74.MHCK7.micro-dystrophin. This will take place at Nationwide Children’s Hospital. There will be close follow-up for 3 years, including several muscle biopsies, laboratory tests, physical therapy assessments and physical exams.

Where does this study take place?

This study takes place at Nationwide Children’s Hospital in Columbus, Ohio, and Washington University School of Medicine in St. Louis, MO.

Where can I learn more about this study?

You can learn more about this study at www.ClinicalTrials.org (NCT03375164). You can also contact the study coordinator Alana Mahley at Alana.Mahley@nationwidechildrens.org or 614-355-2606.

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