New hope for duchenne kids: testing next treatment after gene therapy
NCT ID NCT06100887
Summary
This study is testing whether a daily oral medication called EDG-5506 is safe and can help control muscle damage in children and teenagers with Duchenne muscular dystrophy who have already received gene therapy. About 43 participants aged 6-17 will take either the medication or a placebo for 12 weeks, then all can continue taking the medication for up to 3 years. The research aims to see if this treatment can provide additional benefit to those who have already undergone gene therapy.
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Contacts and locations
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Locations
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rare Disease Research
Hillsborough, North Carolina, 27278, United States
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UC Davis Medical Center
Sacramento, California, 95817, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Massachusetts Memorial Medical Center
Worcester, Massachusetts, 01605, United States
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Washington University School of Medicine
St Louis, Missouri, 63110, United States
Conditions
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