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The main objective of this multicenter study is to evaluate SRP-4045 and SRP-4053 in treating muscle function in boys who have a certain type of Duchenne muscular dystrophy (DMD). Additional objectives include evaluation of safety, pharmacokinetics, and biomarkers related to treatment with these drugs.
Subject to certain exclusion criteria, the study is accepting male participants ages 7 to 13, who have a confirmed diagnosis of DMD with a type of genetic code error, called a deletion, that may be treatable with SRP-4045 or SRP-4053, using a principle called exon skipping – specifically exon 45 or exon 53 skipping.
Boys: Ages 7 to 13
Participants will be randomly divided into groups that will receive 96 weekly infusions of either a placebo or the investigational drug appropriate to their gene deletion. After 96 weeks, members of the placebo group will receive weekly infusions of whichever study drug is appropriate for their gene deletion for another 96 weeks, and the two other groups will continue with weekly infusions of the drug they had been receiving. Other physical examinations, including biological sample collection, will be done at prescribed intervals, and two muscle biopsies will be required.
Visits: 192 for weekly infusions
Duration: About 3 years and 9 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
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
Children’s Hospital of Pittsburgh of UPMC
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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