Children's Hospital is part of the UPMC family.
Be safe anytime, anywhere.
To find a pediatrician or pediatric specialist, please call 412-692-7337 or search our directory.
A resource for our network of referring physicians.
For more information about research, please call our main office at 412-692-6438.
Children's Hospital is ranked One of America's Best Children's Hospitals.
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:
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.
With myCHP, you can request appointments, review test results, and more.
For questions about a hospital bill call:
To pay your bill online, please visit UPMC's online bill payment system.
Interested in giving to Children's Hospital? Visit Children's Hospital of Pittsburgh Foundation's website to: