Central Nervous System Tumor Treatment with PD-0332991 – Phase I

A Study of CDK 4-6 Inhibitor PD-0332991 in Children with Recurrent, Progressive or Refractory Central Nervous System Tumors

Protocol Description

This trial by the Pediatric Brain Tumor Research Consortium studies the side effects and best dose of CDK 4-6 Inhibitor PD-0332991, also known as palbociclib isethionate, in treating younger patients with central nervous system tumors that have grown, come back, or not responded to treatment. Palbociclib isethionate may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Study Level: Phase I

Eligibility Criteria

Subject to certain exclusion criteria, this study is open to males and females, ages 4 to 21 years, who have histologically confirmed Rb1 positive primary recurrent, progressive, or refractory central nervous system tumors.
Males and Females: Ages 4 to 21 years


Following an initial screening exam, study participants will receive palbociclib isethionate orally once daily for 21 days followed by a 7-day break. This 4-week course will extend to 26 courses in the absence of disease progression or unacceptable toxicity. Blood draws, EKGs and brain scans will be conducted, among other tests, at periodic intervals.
Visits: Weekly during first month of therapy, followed by monthly visits and periodic lab testing in subsequent months
Duration: Approximately 2 years

Status: Open to Enrollment

Source(s) of Support

Pediatric Brain Tumor Consortium (Study PBTC-042)
Pfizer, Inc.

Primary Investigator

A. Kim Ritchey, MD

Contact Information

For more information about the study or enrollment, please contact:
Research Coordinator Angela Connelly, RN, BSN