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rAAVrh74.MCK.GALGT2 Gene Therapy in Duchenne Muscular Dystrophy
NCT03333590Phase 2Completed
- Sponsor
- Kevin Flanigan (Nationwide Children's Hospital)
- Start
- Nov 2017
- Primary completion
- Nov 2020
- Modality
- Gene Editing, Gene Silencing, Viral Vectors
- Countries
- 1
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Overview
Phase 2, open-label trial of rAAVrh74.MCK.GALGT2 in Duchenne muscular dystrophy (DMD) (2 cohorts), sponsored by Kevin Flanigan (Nationwide Children's Hospital). Primary endpoint: Number of Unanticipated Grade III or Higher Treatment-Related Toxicities at 2 years. Started Nov 2017; status: completed.
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Drugs
rAAVrh74.MCK.GALGT2
03
Conditions
Duchenne muscular dystrophy (DMD)
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