Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or

Status Completed

Clinical Trial 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.

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

Central Nervous System Neoplasms
Glioma
Neoplasms
Nervous System Neoplasms
Neurofibromatoses
Neurofibromatosis 1
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

Clinical Trial Details

NCT number	NCT02415153
Study type	Interventional
Source	National Cancer Institute (NCI)
Contact
Status	Completed
Phase	Phase 1
Start date	July 14, 2015
Completion date	May 27, 2020

View Details

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Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

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

Clinical Trial Summary

Clinical Trial Description

Study Design

Related Conditions & MeSH terms