NCT02415153

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

A Phase I Trial of Pomalidomide for Children With Recurrent, Progressive, or Refractory CNS Tumors

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 29 (actual)
• Sponsor: National Cancer Institute (NCI) · NIH
• Sex: All
• Age: 3 Years – 20 Years
• Healthy volunteers: Not accepted

Summary

This phase I trial studies the side effects and best dose of pomalidomide in treating younger patients with tumors of the brain or spine (central nervous system) that have come back or are continuing to grow. Pomalidomide may interfere with the ability of tumor cells to grow and spread and may also stimulate the immune system to kill tumor cells.

Detailed description

PRIMARY OBJECTIVES: I. To determine the maximum tolerated dose (MTD) and/or recommended phase II dose (RP2D) of pomalidomide, in children from \>= 3 years to \< 21 years of age with recurrent, progressive or refractory central nervous system (CNS) tumors when given once daily for 21 consecutive days of a 28-day course. II. To describe the toxicity profile and dose-limiting toxicities of pomalidomide in children from \>= 3 years to \< 21 years of age with recurrent, progressive or refractory CNS tumors. III. To characterize the pharmacokinetics of pomalidomide when administered orally in children from \>= 3 years old to \< 21 years of age with recurrent, progressive or refractory CNS tumors and study the association of pharmacokinetic (PK) parameters with age and steroid use. SECONDARY OBJECTIVES: I. To explore the preliminary efficacy of pomalidomide in this patient population as defined by radiographic response rate, duration of response, and event-free survival (EFS) within the confines of a Phase 1 study. \*For the purposes of this study, long-term stable disease will be considered a response (defined as stable disease for \>= 6 courses). II. To investigate a relationship between pomalidomide dose and exposure with radiographic response and changes in immune function (for example, T-cell subsets, natural killer \[NK\] cell activity, granzyme B and circulating levels of IL-12, IL-2, IL-15, GM-CSF). OUTLINE: This is a dose-escalation study. Patients receive pomalidomide orally (PO) once daily (QD) on days 1-21. Treatment repeats every 28 days for up to 26 courses in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 2 years.

Conditions

• Neurofibromatosis Type 1
• Recurrent Childhood Brain Stem Glioma
• Recurrent Childhood Visual Pathway Glioma
• Recurrent Primary Central Nervous System Neoplasm
• Refractory Primary Central Nervous System Neoplasm

Interventions

Type	Name	Description
OTHER	Laboratory Biomarker Analysis	Optional correlative studies
OTHER	Pharmacological Study	Correlative studies
DRUG	Pomalidomide	Given PO

Timeline

• Start date: 2015-07-14
• Primary completion: 2019-07-01
• Completion: 2020-05-27
• First posted: 2015-04-14
• Last updated: 2022-04-25

Locations

12 sites across 1 country: United States