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Among other factors, this study evaluates biological activity of the investigational drug AGT-181, for adults with mucopolysaccharidosis type I (MPS I), which includes Hurler, Hurler-Scheie and Scheie syndromes. People with MPS I lack normal amounts of a certain enzyme. Aldurazyme is an approved treatment to replace this enzyme, but is blocked by a kind of wall between the brain and blood system. The antibody AGT-181 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 help treat MPS I symptoms present in the brain and the rest of the body.
Subject to exclusion criteria, this study is accepting men and women ages 18 and older.
Men and Women: 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 replacement therapy can opt 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
Hurler Syndrome (MPS I)
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
In addition to the main hospital, Children's has many convenient locations in other neighborhoods throughout the greater Pittsburgh region.
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