One-Shot gene therapy tested in boys with devastating muscle disease
NCT ID NCT07160634
Summary
This study is testing whether a single intravenous dose of an experimental gene therapy called SGT-003 can help boys with Duchenne muscular dystrophy (DMD) walk and move better. It will involve 80 boys who can still walk, comparing those who get the therapy to those who get a placebo. The main goal is to see if the treatment improves the speed at which a boy can stand up from lying down after 18 months, and participants will be monitored for safety for at least 5 years.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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BC Children's Hospital
RECRUITINGVancouver, British Columbia, Canada
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Children's Hospital of Westmead
RECRUITINGSydney, New South Wales, Australia
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
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