Clinical Trial: Lenalidomide in Treating Young Patients With Recurrent, Progressive, or Refractory CNS Tumors

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: A Phase I Trial of CC-5013 (Lenalidomide) in Pediatric Patients With Recurrent or Refractory Primary CNS Tumors

Brief Summary: This phase I trial is studying the side effects and best dose of lenalidomide in treating young patients with recurrent, progressive, or refractory CNS tumors. Lenalidomide may stop the growth of CNS tumors by blocking blood flow to the tumor. It may also stimulate the immune system in different ways and stop tumor cells from growing.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To estimate the MTD of oral CC-5013 administered to children with recurrent or refractory primary CNS tumors once daily for 21 days of a 28 day course.

II. To describe the toxicity profile and define the dose-limiting toxicity of CC-5013 in children with recurrent or refractory primary CNS tumors.

SECONDARY OBJECTIVES:

I. To characterize the pharmacokinetics of CC-5013 in children and adolescents. II. To characterize the pharmacogenetics of CC-5013 in children and adolescents.

III. To evaluate changes in circulating endothelial cells (CECs) and circulating endothelial cell precursors (CEPs) in patients treated with CC-5013, and to investigate the correlation between changes in CECs and CEPs, plasma, serum and urine levels of proteins associated with angiogenesis including thrombospondin, b-FGF, TNF-α, IL-12, IL-8 and VEGF, and correlate these changes with changes in MR perfusion and clinical outcome.

IV. To evaluate changes in MR perfusion and diffusion during treatment.

OUTLINE: This is a dose-escalation, multicenter study.

Patients receive oral lenalidomide once daily on days 1-21. Treatment repeats every 28 days for 24 courses in the absence of disease progression or unacceptable toxicity.

Cohorts of 2-3 patients receive escalating doses of lenalidomide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose at which an estimated 25% of patients experience dose-limiting toxicity.

Presented in tabular and graphical form and determined using compartmental methods. Dose proportionality in pharmacokinetic parameters will be determined by performing one-way analysis of variance (ANOVA) on dose-normalized parameters.