Central Nervous System Tumor Treatment with Savolitinib – Phase I

PBTC-049: Study of Savolitinib in Recurrent, Progressive, or Refractory Medulloblastoma, High-Grade Glioma, or Diffuse Intrinsic Pontine Glioma

Protocol Description

Through this multi-center Pediatric Brain Tumor Consortium study, researchers are investigating the effects of savolitinib in young patients with recurrent, refractory, or progressive central nervous system (CNS) tumors, including medulloblastomas, high-grade gliomas, and diffuse intrinsic pontine gliomas (DIPG). Investigators will determine how the body processes the study drug and optimal dosage levels for future studies. In lab tests and other studies, savolitinib has demonstrated strong activity against c-MET kinases and downstream signaling targets, significantly decreasing tumor cell growth.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling pediatric patients who have been diagnosed with recurrent, progressive or refractory CNS tumors.
Males and Females: Ages 6 to 21


Study participants will take the study drug orally in tablet form once a day for each 28-day cycle, with dosing levels adjusted per the study protocol. Barring disease progression or unacceptable toxicity, participants may remain in the study for up to 39 cycles. Physical examinations will be given throughout the study, with imaging, blood work, and other tests done at prescribed intervals.
Visits: About 80
Duration: Up to 3 years

Status: Open to Enrollment

Source(s) of Support

National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-049)

Primary Investigator

James Felker, MD

Contact Information

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