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This multicenter international study will help researchers learn about the safety and effects of PF-06252616 on male children with Duchenne muscular dystrophy (DMD), a disease that damages the muscles causing muscle weakness and loss of the ability to walk. PF-06252616 is an engineered protein, called a monoclonal antibody, designed to inhibit a naturally occurring protein called myostatin that has a role in regulating muscle growth. Researchers believe that PF-06252616 may prevent myostatin from adversely limiting the size of muscles in people with DMD and may provide improved function.
Subject to certain exclusion criteria, the study is accepting boys, ages 6 through 15, who have DMD and are ambulatory (can walk).
Boys: 6 through 15 years of age
Participants in this study will receive a comprehensive initial exam, including imaging, physical testing and biological samples, and then will be randomly assigned to one of three study groups. In this study, participants will receive intravenous dosages of the study drug, provided at escalating dose levels up to a maximum. Some participants will receive a placebo instead of the study drug during portions of the study.
Visits: About 28 (every 4 weeks)
Duration: 26 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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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