Central Nervous System Immunotherapy Using MK-3475 – Phase I

PBTC-045: Trial of MK-3475 in Children with Recurrent, Progressive, or Refractory Diffuse Intrinsic Pontine Glioma, Non-brainstem High-grade Gliomas, Ependymoma, Medulloblastoma, or Hypermutated Brain Tumors

Protocol Description

Through this Pediatric Brain Tumor Consortium investigation, researchers are studying the effects of pembrolizumab (MK-3475) in treating young patients with progressive, recurrent, or refractory high-grade gliomas, brain stem tumors (diffuse intrinsic pontine glioma, or DIPG), ependymoma, medulloblastoma, or brain tumors with a high number of genetic mutations. Researchers believe that immunotherapy with monoclonal antibodies, such as MK-3475, may induce changes in the body's immune system that may interfere with the ability of tumor cells to grow and spread.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling pediatric patients who have a progressive, recurrent, or refractory high-grade glioma, DIPG, enendymoma, medulloblastoma, or hypermutated brain tumors.
Males and Females: Ages 3 to 21


Participants will receive an intravenous infusion of MK-3475 on the first day of a 21-day cycle that repeats for 34 cycles, in the absence of disease progression or unacceptable toxicity. Physical examinations will be given prior to infusions, and more frequently during the participants’ first cycles of the study. Imaging, blood work, and other tests will be done at prescribed intervals.
Visits: About 42
Duration: Approximately 2 years

Status: Open to Enrollment

Source(s) of Support

National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-045)

Primary Investigator

James Felker, MD

Contact Information

For more information about the study or enrollment, please contact:
Research Coordinator Sharon Dibridge