NCT02255461

Palbociclib Isethionate in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors

Phase I Study of CDK 4-6 Inhibitor PD-0332991 (Palbociclib; IBRANCE) in Children With Recurrent, Progressive or Refractory Central Nervous System Tumors

• Status: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 35 (actual)
• Sponsor: Pediatric Brain Tumor Consortium · Network
• Sex: All
• Age: 4 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

This phase I trial studies the side effects and best dose of 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.

Detailed description

PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD)/phase II recommended dose and describe toxicities related to PD-0332991 (palbociclib isethionate) in children with retinoblastoma protein 1 (Rb1) positive recurrent, progressive or refractory primary central nervous system (CNS) tumors. II. To determine plasma pharmacokinetics of PD-0332991 in children with Rb1positive recurrent, progressive or refractory primary CNS tumors. SECONDARY OBJECTIVES: I. To record preliminary evidence of efficacy of PD-0332991 in children with recurrent CNS tumors. II. To evaluate cyclin-dependent kinase (CDK)4/6, cyclin D1-3, Ink4a-ARF copy-number variations in available tumor tissue by array comparative, genomic hybridization (aCGH). III. To explore the potential relationships between the pharmacokinetics of PD-0332991 and pharmacodynamic response (e.g. percentage change in absolute neutrophil count \[ANC\], platelet counts). IV. To explore the pharmacogenetic polymorphisms in PD-0332991 metabolizing enzymes and transporters and relate these polymorphisms to PD-0332991 pharmacokinetics. OUTLINE: This is a dose-escalation study. Patients receive palbociclib isethionate orally (PO) once daily (QD) on days 1-21. Treatment repeats every 4 weeks for 26 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 30 days.

Conditions

• Childhood Choroid Plexus Tumor
• Childhood Ependymoblastoma
• Childhood Grade III Meningioma
• Childhood High-grade Cerebellar Astrocytoma
• Childhood High-grade Cerebral Astrocytoma
• Childhood Medulloepithelioma
• Recurrent Childhood Anaplastic Astrocytoma
• Recurrent Childhood Anaplastic Oligoastrocytoma
• Recurrent Childhood Anaplastic Oligodendroglioma
• Recurrent Childhood Brain Stem Glioma
• Recurrent Childhood Cerebellar Astrocytoma
• Recurrent Childhood Cerebral Astrocytoma
• Recurrent Childhood Giant Cell Glioblastoma
• Recurrent Childhood Glioblastoma
• Recurrent Childhood Gliomatosis Cerebri
• Recurrent Childhood Gliosarcoma
• Recurrent Childhood Medulloblastoma
• Recurrent Childhood Pineoblastoma
• Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor

Interventions

Type	Name	Description
DRUG	palbociclib isethionate	Given PO
OTHER	pharmacological study	Correlative studies
OTHER	laboratory biomarker analysis	Correlative studies

Timeline

• Start date: 2014-12-08
• Primary completion: 2019-02-25
• Completion: 2019-02-25
• First posted: 2014-10-02
• Last updated: 2021-03-02
• Results posted: 2020-04-08

Locations

11 sites across 1 country: United States

Regulatory

• FDA-regulated drug study