New hope for boys with Muscle-Wasting disease: experimental drug enters advanced testing
NCT ID NCT05540860
Summary
This study is testing an oral medication called EDG-5506 (sevasemten) in children with Duchenne muscular dystrophy (DMD), a serious muscle-wasting condition. Researchers want to see if the drug is safe, how it moves through the body, and if it can reduce signs of muscle damage. The trial has two parts: first, some children get the drug and some get a placebo for 12 weeks, then all children can receive the drug in a long-term extension phase for up to four years.
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Contacts and locations
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Locations
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Cincinnati Children's Hospital
Cincinnati, Ohio, 45229, United States
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Cook Children's Medical Center
Fort Worth, Texas, 76104, United States
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Kennedy Krieger Institute
Baltimore, Maryland, 21205, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Rare Disease Research
Atlanta, Georgia, 30329, 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 Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 66160, 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
Explore the condition pages connected to this study.