Pomalidomide in Treating Younger Patients With Recurrent, Progressive, or Refractory Central Nervous System Tumors
Condition: 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
MeSH terms: Neoplasms; Glioma; Neurofibromatoses; Neurofibromatosis 1; Nervous System Neoplasms; Central Nervous System Neoplasms; Recurrence
Study type: Interventional
Phase: Phase 1

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.
    

Intervention name(s): Laboratory Biomarker Analysis; Pharmacological Study; Pomalidomide
Intervention type(s): Other; Other; Drug
Intervention description(s): Optional correlative studies; Correlative studies; Given PO
Other intervention name(s): 4-Aminothalidomide; Actimid; CC-4047; Imnovid; Pomalyst
Intervention MeSH terms: Pomalidomide
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment