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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 and was developed for delivery directly into cerebrospinal fluid rather than intravenously.
The study accepts male patients, 3 to 18 years of age, diagnosed with MPS II with evidence of illness related cognitive impairment who were enrolled for 12 months in a prior related program (protocol HGT-HIT-045) by the study sponsor.
Males: Ages 3 years to 18
Participants will receive monthly intrathecal (IT) administration of idursulfase in addition to weekly intravenous infusions of Elaprase. IT delivers the study drug directly to the fluid surrounding the spinal cord and is done through an access port and tube study doctors place under the skin of participants.
Visits: Weekly for Elaprase regimen, with idursulfase-IT infusions once a month
Duration: 30 months
Shire Human Genetic Therapies
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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