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.
Ranked #6 Nationally by U.S. News & World Report.
The purpose of this multi-center study is to assess the safety and effects of a gene transfer called RGX-121 to help patients with mucopolysaccharidosis type II (MPS II) also called Hunter syndrome. In this inherited genetic condition, a defective iduronate-2-sulfatase gene (IDS) prevents production of the enzyme iduronate-2-sulfatase (I2S). The lack of circulating I2S enzyme can lead to progressive damage to the brain, heart, and other organs. RGX-121 contains a healthy IDS gene that is thought to help cells to make the needed enzyme. This study is for boys whose disease progression has affected their neurocognitive abilities.
Subject to exclusion criteria, this study is enrolling male babies, toddlers, and very young boys who have been diagnosed with MPS II with neurocognitive involvement.
Boys: Ages 4 months to 5 years
Participants will receive a single infusion of RGX-121 delivered to the central nervous system. After that, participants will return to UPMC Children’s Hospital of Pittsburgh for examinations, testing, imaging, and blood draws in accordance with the protocol schedule over a 2-year period. By invitation, participants may be able to participate in a long-term follow-up study.
Duration: 104 weeks (about 2 years) after receiving RGX-121
Study Description at National Institutes of Health
Hunter Syndrome MPS II
Neurodevelopment in Rare Disorders Research
Deepa Soundara Rajan, MD
For more information about the study or enrollment, please contact:
Dawn Kolar, Clinical Research Coordinator
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
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? Support the hospital by making a donation online, joining our Heroes in Healing monthly donor program, or visiting our site to learn about the other ways you can give back.