A Phase I Trial of CC-5013 (Lenalidomide) in Pediatric Patients With Recurrent or Refractory Primary CNS Tumors
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.
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.
All patients are followed for at least 30 days after the last dose of lenalidomide. Patients
with treatment-related toxicity are followed for up to 3 months.
Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
MTD, estimated using the modified Continual Reassessment Method (CRM)
Pediatric Brain Tumor Consortium
United States: Food and Drug Administration
|Pediatric Brain Tumor Consortium||Memphis, Tennessee 38105|