SHH Medulloblastoma Treatment with CX-4945 – Phase I/II

PBTC-053: Surgical Study of CX-4945 in Patients with Recurrent SHH Medulloblastoma

Protocol Description

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.

Study Level: Phase I/II

Eligibility Criteria

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

Status: Open to Enrollment

Source(s) of Support

National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-053)

Primary Investigator

James Felker, MD

Contact Information

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