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Among other factors, this study evaluates biological activity of the investigational drug AGT-182, for adults with mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. People with MPS II lack normal amounts of a certain enzyme. Replacement enzymes can be blocked by a kind of wall between the brain and blood system. The antibody AGT-182 carries a version of the replacement enzyme Elaprase® and tricks the body, enabling it to carry the enzyme to the brain and elsewhere. Researchers believe that once delivered, the drug may be able to treat MPS II symptoms present in the brain and the rest of the body.
Subject to certain exclusion criteria, the study is accepting men who are 18 years of age and older, who have been diagnosed with MPS II.
Males: Ages 18 and older
Following the initial screening visit and exam, participants will receive 8 weeks of weekly infusions of the study drug at one of two dosing levels, 1 mg/kg or 3 mg/kg. During certain visits, additional testing will be done. Participants who have had prior enzyme therapy will be required to wait 6 weeks for all prior medications to clear. The waiting period is called the washout. There will be one final visit with study doctors one week after the last infusion.
Duration: 15 weeks, inclusive of the 6-week washout period
Study Description at National Institutes of Health
Hunter Syndrome (MPS-II)
Medical Genetics Research
Gerard Vockley, MD, PhD
For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
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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