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The objective of this multicenter study is to confirm effectiveness of eteplirsen (AVI-4658) in improving muscle function in boys who have a certain type of Duchenne muscular dystrophy (DMD). Additional objectives include evaluation of safety, biomarkers, and the long-term effects of eteplirsen use.
Subject to certain exclusion criteria, the study is accepting male participants ages 7 to 16, who have a confirmed diagnosis of DMD with a type of genetic code error that may be treatable with eteplirsen using a principle called exon skipping – specifically exon 51 skipping. They will be included in the arm of the study that receives the investigational drug. A second study arm will enroll boys with DMD who have a genetic error that investigators believe is not treatable through exon 51 skipping. This group will not receive eteplirsen, but will be observed as a comparative control group.
Boys: Ages 7 to 16
Participants in the treatment arm of this study will receive weekly infusions of eteplirsen for 96 weeks. After 52 weeks, infusions may optionally be provided at the patient’s home by a visiting nurse, but monthly visits will still be required for assessments. Physical testing, health assessments, and biological sample collection will be done at specific, less-frequent intervals. Participants in the control arm will receive the same assessments, without the weekly infusions.
Visits: Up to 100; fewer if infusions are done at patient’s home
Duration: About 2 years and 2 months
Study Description at National Institutes of Health
Muscular Dystrophy Program at Children’s Hospital of Pittsburgh of UPMC
Hoda Abdel-Hamid, MD, MS
For more information about the study or enrollment, please contact:
Jennifer Monahan, Clinical Research Coordinator
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Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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