Last updated on August 2019

Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy


Brief description of study

This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.

Detailed Study Description

This study is a randomized, double-blind, placebo-controlled, 72-week study, followed by a 72-week open-label period. The purpose is to characterize the long-term effects of ataluren-mediated dystrophin restoration on disease progression. Participants will be randomized in a 1:1 ratio to ataluren or placebo. Participants will receive blinded study drug three times daily (TID) at morning, midday, and evening for 72 weeks, after which all participants will receive open-label ataluren for an additional 72 weeks (144 weeks in total). Study assessments will be performed at clinic visits every 12 weeks during the double-blind period and every 24 weeks during the open-label period. The total sample size of ~250 subjects will include ~160 subjects who meet the criteria for inclusion in the primary analysis population (age 7 to 16 years old, baseline six minute walk distance (6MWD) greater than or equal to (>=) 300 meters, supine to stand >= 5 seconds). The study will be conducted in the United States and other countries around the world.

Clinical Study Identifier: NCT03179631

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Yale New Haven Hospital

New Haven, CT United States
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Queen Mary Hospital

Hong Kong, Hong Kong
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University Of Utah

Salt Lake City, UT United States
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Texas Children's Hospital

Houston, TX United States
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Seattle Children's Hospital

Seattle, WA United States
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University of Minnesota

Minneapolis, MN United States
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Prevea Clinic, Inc

Green Bay, WI United States
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Phoenix Childrens Hospital

Phoenix, AZ United States
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Cook Childrens Medical Center

Fort Worth, TX United States
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Samsung Medical Center

Seoul, Korea, Republic of
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