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

Phase 1
21 Years
Not Enrolling
Childhood Atypical Teratoid/Rhabdoid Tumor, Childhood Central Nervous System Germ Cell Tumor, Childhood Choroid Plexus Tumor, Childhood Craniopharyngioma, Childhood Ependymoblastoma, Childhood Grade I Meningioma, Childhood Grade II Meningioma, Childhood Grade III Meningioma, Childhood High-grade Cerebellar Astrocytoma, Childhood High-grade Cerebral Astrocytoma, Childhood Infratentorial Ependymoma, Childhood Low-grade Cerebellar Astrocytoma, Childhood Low-grade Cerebral Astrocytoma, Childhood Medulloepithelioma, Childhood Mixed Glioma, Childhood Oligodendroglioma, Childhood Supratentorial Ependymoma, Recurrent Childhood Brain Tumor, Recurrent Childhood Cerebellar Astrocytoma, Recurrent Childhood Cerebral Astrocytoma, Recurrent Childhood Ependymoma, Recurrent Childhood Medulloblastoma, Recurrent Childhood Pineoblastoma, Recurrent Childhood Subependymal Giant Cell Astrocytoma, Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor, Recurrent Childhood Visual Pathway and Hypothalamic Glioma

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Trial Information

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.

Inclusion Criteria:

- Patients with a histological diagnosis of a primary CNS tumor (including
histologically benign brain tumors (e.g. low-grade glioma) that is recurrent,
progressive, or refractory to standard therapy; patients with intrinsic brain stem or
diffuse optic pathway tumors do not require histological confirmation of disease but
should have clinical and/or radiographic evidence of progression

- Karnofsky Performance Scale (KPS for >= 16 yrs of age) or Lansky Performance Score
(LPS for ≤ 16 years of age) ≥ 60 assessed within two weeks prior to registration

- Patient must be able to swallow capsules

- Patients must have recovered from any significant acute toxicity associated with
prior therapy; patients must have no known curative therapy available; patients will
be eligible regardless of the number of prior therapies, as long as other eligibility
criteria are met

- Chemo: Prior use of thalidomide is acceptable; patients must have:

- Received their last dose of known myelosuppressive anticancer chemotherapy or
biological therapy at least three (3) weeks prior to study registration

- Received their last dose of nitrosourea or mitomycin-C at least six (6) weeks
prior to study registration

- Received their last dose of other investigational agent or an anticancer drug
known to not be myelosuppressive at least seven (7) days prior to study

- XRT: Patients must have had their last fraction of craniospinal irradiation ≥ 3
months prior to registration and their last fraction of local irradiation to primary
tumor ≥ 4 weeks prior to registration

- Bone Marrow Transplant: ≥ 6 months since allogeneic bone marrow transplant and ≥ 3
months since autologous bone marrow/stem cell prior to registration

- Growth factors: Off all colony forming growth factor(s) > 2 weeks prior to
registration (filgrastim, sargramostim, erythropoietin)

- The following laboratory values must be assessed within two (2) weeks prior to
registration and again within seven (7) days prior to the start of therapy;
laboratory tests should be repeated within 48 hours of beginning therapy if there has
been a significant clinical change

- Absolute neutrophil count ≥ 1000/μl (unsupported)

- Platelets ≥ 100,000/μl (unsupported)

- Hemoglobin ≥ 8.0 g/dL (may be supported)

- Serum creatinine within upper limit of institutional normal for age

- Or GFR ≥ 70 ml/min/1.73m^2

- Bilirubin ≤ 1.5 times upper limit of normal for age

- SGPT (ALT) ≤ 2.5x institutional upper limit of normal for age

- Albumin ≥ 2 g/dL

- No overt renal, hepatic, cardiac or pulmonary disease

- Female patients of childbearing potential must have negative serum or urine pregnancy
test (sensitivity of at least 50mIU/ml); patients must not be pregnant or

- All sexually active females must begin 2 methods of birth control, including 1 highly
effective method, and 1 additional method (at the same time) at least 4 weeks prior
to the first dose of CC-5013; this applies to all sexually active females of
childbearing potential unless they have not had a menstrual period in 2 years or have
undergone a hysterectomy

- Patients of child fathering potential must agree to use latex condoms during
intercourse with a woman while taking CC-5013 and for 4 weeks thereafter

- Signed informed consent according to institutional guidelines must be obtained

Exclusion Criteria:

- Patients with a body surface area (BSA) ≤ 0.4 m^2 are excluded

- Patients with a first-degree relative with a history of venous thrombosis before age
50 yrs or an arterial thrombosis before age 40 yrs are excluded

- Patients who have had a thromboembolic event that is not line-related are excluded

- Patients with any significant medical illnesses that, in the investigator's opinion,
cannot be adequately controlled with appropriate therapy or would compromise a
patient's ability to tolerate this therapy

- Patients with any disease that would obscure toxicity or dangerously alter drug

- Patients receiving any other chemotherapeutics or investigational agents

- Patients with uncontrolled infection

- Patients unable to swallow capsules

- Patients with known hypersensitivity to anhydrous lactose, microcrystalline
cellulose, croscarmellose sodium, and magnesium stearate

Type of Study:


Study Design:

Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

MTD, estimated using the modified Continual Reassessment Method (CRM)

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

Katherine Warren

Investigator Role:

Principal Investigator

Investigator Affiliation:

Pediatric Brain Tumor Consortium


United States: Food and Drug Administration

Study ID:




Start Date:

November 2004

Completion Date:

Related Keywords:

  • Childhood Atypical Teratoid/Rhabdoid Tumor
  • Childhood Central Nervous System Germ Cell Tumor
  • Childhood Choroid Plexus Tumor
  • Childhood Craniopharyngioma
  • Childhood Ependymoblastoma
  • Childhood Grade I Meningioma
  • Childhood Grade II Meningioma
  • Childhood Grade III Meningioma
  • Childhood High-grade Cerebellar Astrocytoma
  • Childhood High-grade Cerebral Astrocytoma
  • Childhood Infratentorial Ependymoma
  • Childhood Low-grade Cerebellar Astrocytoma
  • Childhood Low-grade Cerebral Astrocytoma
  • Childhood Medulloepithelioma
  • Childhood Mixed Glioma
  • Childhood Oligodendroglioma
  • Childhood Supratentorial Ependymoma
  • Recurrent Childhood Brain Tumor
  • Recurrent Childhood Cerebellar Astrocytoma
  • Recurrent Childhood Cerebral Astrocytoma
  • Recurrent Childhood Ependymoma
  • Recurrent Childhood Medulloblastoma
  • Recurrent Childhood Pineoblastoma
  • Recurrent Childhood Subependymal Giant Cell Astrocytoma
  • Recurrent Childhood Supratentorial Primitive Neuroectodermal Tumor
  • Recurrent Childhood Visual Pathway and Hypothalamic Glioma
  • Astrocytoma
  • Brain Neoplasms
  • Craniopharyngioma
  • Adamantinoma
  • Ependymoma
  • Glioma
  • Medulloblastoma
  • Meningioma
  • Oligodendroglioma
  • Central Nervous System Neoplasms
  • Choroid Plexus Neoplasms
  • Neoplasms, Germ Cell and Embryonal
  • Pinealoma
  • Neuroectodermal Tumors
  • Neuroectodermal Tumors, Primitive
  • Rhabdoid Tumor
  • Optic Nerve Glioma



Pediatric Brain Tumor Consortium Memphis, Tennessee  38105