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Through this multi-center Pediatric Brain Tumor Consortium study, researchers are investigating the effects of APX005M in young patients with a recurrent or refractory primary malignant central nervous system (CNS) tumor or newly diagnosed diffuse intrinsic pontine glioma (DIPG), a type of inoperable brain stem tumor. Researchers will determine how the body processes the study drug and optimal dosage levels for future studies. Lab tests and other studies have demonstrated that APX005M can stimulate cellular activity that can inhibit tumor cell growth or kill tumor cells.
Subject to certain exclusion criteria, this study is enrolling young patients who have been diagnosed with a recurrent or refractory brain tumor or a newly diagnosed DIPG.
Males and Females: Ages 1 to 21
Participants will receive the study drug via intravenous infusion on an outpatient basis once every 21 days. Each 21-day period is a cycle. Barring disease progression or unacceptable toxicity, participants may remain in the study for up to 36 cycles. Physical examinations will be given throughout the study, with imaging, blood work, and other tests done at prescribed intervals.
Duration: Up to 2 years
National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-051)
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
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