DIPG Treatment with Panobinostat – Phase I

PBTC-047: Panobinostat in Children with Diffuse Intrinsic Pontine Glioma

Protocol Description

Through this multi-center Pediatric Brain Tumor Consortium investigation, researchers are studying the effects of panobinostat in young patients with diffuse intrinsic pontine glioma (DIPG), a type of inoperable brain stem tumor. Investigators will determine how the body processes the study drug and optimal dosage levels for future studies. Lab tests and other studies have demonstrated that panobinostat can inhibit certain cellular activities that promote tumor growth.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is enrolling pediatric patients who have been diagnosed with progressive DIPG.
Males and Females: Ages 2 to 21


Participants will take the study drug in capsule form once a day, 3 times a week for 3 weeks followed by a week of not taking the drug. This 4-week period is called a cycle. Barring adverse events, participants may remain 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: About 40
Duration: Up to 2 years

Status: Open to Enrollment

Source(s) of Support

National Cancer Institute
Pediatric Brain Tumor Consortium (PBTC-047)

Primary Investigator

James Felker, MD

Contact Information

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