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Through this study researchers are evaluating the effects of adding the investigational drug idursulfase-IT to therapy with Elaprase® on the neurodevelopment of pediatric patients with mucopolysaccharidosis type II (MPS II), also called Hunter syndrome, over an extended period. Approximately two thirds of patients with Hunter syndrome develop problems associated with accumulation of mucopolysaccharides in the central nervous system. Patients with this severe form of Hunter syndrome typically do not develop normal abilities with language, learning and other normal tasks. Idursulfase-IT is a new formulation of the enzyme replacement therapy Elaprase developed for delivery directly into cerebrospinal fluid rather than intravenously.
The study accepts male patients up to age 18, diagnosed with MPS II with evidence of illness related cognitive impairment and who completed the week-52 assessment of the study HGT-HIT-094 by the study sponsor.
Males: Up to age 18
Participants will continue to receive intrathecal (IT) idursulfase once every 28 days in addition to their regular IV Elaprase therapy, which will continue to be administered weekly as part of their standard care at a site chosen by the family and treating doctor. At intervals defined by the study protocol, imaging and lab work will required, and doctors will complete neurodevelopmental and other assessments.
Visits: Up to 60
Duration: Up to 5 years
Shire Human Genetic Therapies, Inc.
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:
Sandra Braden, RN, BSN
Children's Hospital's main campus is located in the Lawrenceville neighborhood. Our main hospital address is:
UPMC Children’s Hospital of Pittsburgh
One Children’s Hospital Way
4401 Penn Ave.
Pittsburgh, PA 15224
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