One-Shot gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT05693142
Summary
This study is testing a one-time intravenous gene therapy called RGX-202 for boys with Duchenne muscular dystrophy (DMD). The goal is to see if delivering a new, functional gene to muscles is safe and can help slow or stop the progression of the disease. The study will enroll about 65 boys, aged 1 year and older, who can still walk independently.
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Contacts and locations
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Study contacts
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Contact
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Locations
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Alberta Children's Hospital
NOT_YET_RECRUITINGCalgary, Alberta, T3B 6A, Canada
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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BC Children's Hospital
RECRUITINGVancouver, British Columbia, V65 3N1, Canada
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Children's Hospital Colorado
RECRUITINGAurora, Colorado, 80045, United States
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Children's Hospital London Health Science Centre
RECRUITINGLondon, Ontario, Canada
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Children's Hospital of Eastern Ontario
RECRUITINGOttawa, Ontario, K1H 8L1, Canada
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Children's Hospital of Richmond at Virginia Commonwealth University
RECRUITINGRichmond, Virginia, 23298, United States
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Cincinnati Children's
RECRUITINGCincinnati, Ohio, 45229, United States
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Oregon Health & Science University
RECRUITINGPortland, Oregon, 97239, United States
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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Stanford School of Medicine /Division of Neuromuscular Medicine
RECRUITINGPalo Alto, California, 94304, United States
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The University of Texas Southwestern Medical Center
RECRUITINGDallas, Texas, 75390, United States
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University of Florida
RECRUITINGGainesville, Florida, 32610, United States
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University of Iowa
RECRUITINGIowa City, Iowa, 52242, United States
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01608, United States
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Conditions
Explore the condition pages connected to this study.