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The purpose of this multi-center global clinical research study is to see if the new compound DNL310, an enzyme replacement therapy (ERT) that is thought to penetrate the central nervous system (CNS), is safe and tolerable when given to patients with mucopolysaccharidosis type II (MPS II), also called Hunter syndrome. In this inherited genetic condition, a defective iduronate-2-sulfatase gene (IDS) prevents production of the enzyme iduronate-2-sulfatase (I2S). The lack of circulating I2S enzyme can lead to progressive damage to the brain, heart, and other organs. The study drug has been designed to deliver I2S to both the body and brain.
Subject to exclusion criteria, this study is enrolling male participants ages 2 through 18 years with a confirmed diagnosis of MPS II.
Males: Ages 2 through 18 years
During the treatment period, participants will receive weekly intravenous infusions of DNL310 at dose levels specified by the study protocol. Examinations and other assessments will also be done during study visits.
Visits: 17, including screening
Duration: 24 weeks
Denali Therapeutics Inc.
Study Description at National Institutes of Health
Hunter Syndrome MPS II
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
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One Children’s Hospital Way
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Pittsburgh, PA 15224
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