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Chemotherapy and Stem Cell Transplantation in Treating Children With Central Nervous System Cancer

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00053118
Recruitment Status : Completed
First Posted : January 28, 2003
Last Update Posted : March 1, 2011
Information provided by:
Roswell Park Cancer Institute

Brief Summary:

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells.

PURPOSE: Phase I trial to study the effectiveness of combining chemotherapy with peripheral stem cell transplantation in treating children who have central nervous system cancer.

Condition or disease Intervention/treatment Phase
Brain and Central Nervous System Tumors Lymphoma Neuroblastoma Retinoblastoma Biological: filgrastim Drug: carboplatin Drug: etoposide Procedure: bone marrow ablation with stem cell support Procedure: peripheral blood stem cell transplantation Phase 1

Detailed Description:


  • Determine the feasibility of administering an outpatient protocol comprising high-dose carboplatin with autologous stem cell support and etoposide in pediatric patients with primary central nervous system malignancies.
  • Determine the maximum tolerated dose of carboplatin when administered in this regimen in these patients.
  • Determine the toxicity of this regimen in these patients.

OUTLINE: This is dose-escalation study of carboplatin.

Patients receive filgrastim (G-CSF) IV once daily for 6 days followed by a maximum of 5 apheresis sessions. If the target number of peripheral blood stem cells is not achieved, some patients receive G-CSF and undergo apheresis as above after a 2-week rest.

At least 3 days after completion of G-CSF, patients receive high-dose carboplatin IV over 1 hour on day 1, stem cell reinfusion on day 3, G-CSF subcutaneously on days 4-18 and 43-61, and oral etoposide 3 times daily on days 21-42. Treatment continues for a maximum of 4 courses in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of carboplatin until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 3 or 2 of 6 patients experience dose-limiting toxicity.

Patients are followed monthly for 1 year and then annually thereafter.

PROJECTED ACCRUAL: A total of 3-15 patients will be accrued for this study.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 1 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: High Dose Carboplatin Combined With Oral VP-16 In The Treatment Of Pediatric CNS Malignancies
Study Start Date : March 2002
Actual Primary Completion Date : July 2004
Actual Study Completion Date : July 2004

Intervention Details:
  • Biological: filgrastim
  • Drug: carboplatin
  • Drug: etoposide
  • Procedure: bone marrow ablation with stem cell support
  • Procedure: peripheral blood stem cell transplantation

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed primary central nervous system malignancy
  • Recurrent, persistent, or progressive disease after at least 1 prior first-line treatment regimen



  • 18 and under at initial diagnosis

Performance status

  • ECOG 0-2

Life expectancy

  • At least 8 weeks


  • Absolute neutrophil count greater than 750/mm^3
  • WBC greater than 2,500/mm^3
  • Platelet count greater than 100,000/mm^3
  • No underlying myelodysplasia, stem cell disorder, or other inherent hematologic synthetic defect


  • Liver function tests less than 2 times normal OR
  • Absence of active hepatitis by liver biopsy
  • Bilirubin less than 1.5 mg/dL


  • Glomerular filtration rate greater than 60 mL/min by radionucleotide assay


  • Ejection fraction at least 45%


  • Clinically normal pulmonary function (patients 5 years of age and under)
  • FEV_1 and FVC at least 50% (patients over 5 years of age) OR
  • Arterial blood gas normal and DLCO greater than 50%


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No mucositis or mucosal infection
  • HIV negative


Biologic therapy

  • Not specified


  • At least 3 weeks since prior systemic cytotoxic chemotherapy

Endocrine therapy

  • Not specified


  • At least 6 months since prior radiotherapy to the pelvis or spine


  • Not specified

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00053118

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United States, Missouri
St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, New York
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263-0001
United States, Texas
University of Texas - MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Roswell Park Cancer Institute
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Study Chair: Barbara Jean Bambach, MD Roswell Park Cancer Institute

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Responsible Party: Barbara Bambach, MD, Roswell Park Cancer Institute Identifier: NCT00053118     History of Changes
Other Study ID Numbers: CDR0000269284
First Posted: January 28, 2003    Key Record Dates
Last Update Posted: March 1, 2011
Last Verified: February 2011
Keywords provided by Roswell Park Cancer Institute:
childhood central nervous system germ cell tumor
childhood choroid plexus tumor
childhood craniopharyngioma
childhood grade I meningioma
childhood grade II meningioma
childhood grade III meningioma
recurrent childhood brain stem glioma
recurrent childhood cerebellar astrocytoma
recurrent childhood cerebral astrocytoma
recurrent childhood ependymoma
recurrent childhood medulloblastoma
recurrent childhood supratentorial primitive neuroectodermal tumor
recurrent neuroblastoma
recurrent retinoblastoma
childhood visual pathway and hypothalamic glioma
childhood atypical teratoid/rhabdoid tumor
primary central nervous system non-Hodgkin lymphoma
Additional relevant MeSH terms:
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Nervous System Neoplasms
Central Nervous System Neoplasms
Neoplasms by Histologic Type
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms by Site
Nervous System Diseases
Retinal Neoplasms
Eye Neoplasms
Eye Diseases, Hereditary
Eye Diseases
Retinal Diseases
Antineoplastic Agents
Antineoplastic Agents, Phytogenic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action