Read the Latest
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.
Through this multi-center Pediatric Brain Tumor Consortium study, researchers are investigating the effects of APX005M in young patients with a recurrent or refractory SHH medulloblastoma, who may or may not be undergoing surgery for their condition. Researchers will establish safe dosing levels while investigating how the body processes the drug and observing anti-tumor activity demonstrated by the drug.
Subject to certain exclusion criteria, this study is enrolling patients who have been diagnosed with recurrent or refractory medulloblastoma and have adequate tissue for SHH subgrouping.
Males and Females: Ages 3 years and older
Study participants will take the study drug orally twice a day in 28-day (4-week) cycles. If their tumors are being removed surgically, dosing will start 5 to 7 days before. Participants may be in the study for up to 26 cycles. Physical examinations will be given throughout the study, with imaging, blood work, and other tests done at prescribed intervals.
Visits: 29, plus hospitalization if surgery is required
Duration: About 2 years
National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-053)
Study Description at the National Institutes of Health
Cancer and Blood Disorder Research
Alberto Broniscer, MD
For more information about the study or enrollment, please contact:
Research Coordinator Sharon Dibridge
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.