Temozolomide Plus Peripheral Stem Cell Transplantation in Treating Children With Newly Diagnosed Malignant Glioma or Recurrent CNS or Other Solid Tumors

This study has been completed.
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Duke University
ClinicalTrials.gov Identifier:
First received: July 5, 2000
Last updated: June 19, 2013
Last verified: February 2013

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: This phase I/II trial is studying the side effects and best dose of temozolomide when given with peripheral stem cell transplantation and to see how well they work in treating children with newly diagnosed malignant glioma or recurrent CNS tumors or other solid tumors.

Condition Intervention Phase
Brain and Central Nervous System Tumors
Childhood Germ Cell Tumor
Head and Neck Cancer
Kidney Cancer
Ovarian Cancer
Testicular Germ Cell Tumor
Biological: filgrastim
Drug: temozolomide
Procedure: peripheral blood stem cell transplantation
Phase 1
Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: A Phase I/II Trial of Temodar in Pediatric Patients and Young Adults With High-Risk or Recurrent Solid Tumors

Resource links provided by NLM:

Further study details as provided by Duke University:

Primary Outcome Measures:
  • Overall response at 12 months [ Designated as safety issue: No ]
  • Disease-free survival at 12 months [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Toxicity by NCI Common Toxicity Criteria v. 3.0 at 12 months [ Designated as safety issue: Yes ]
  • Engraftment related to autologous marrow or peripheral blood stem cell transplantation at 12 months [ Designated as safety issue: No ]

Estimated Enrollment: 30
Study Start Date: August 2000
Study Completion Date: November 2005
Primary Completion Date: November 2005 (Final data collection date for primary outcome measure)
Detailed Description:


  • Determine the maximum tolerated dose of temozolomide in children with newly diagnosed malignant glioma or recurrent CNS or other solid tumors.
  • Evaluate the toxicity of this treatment in these patients.
  • Determine the activity of this treatment in these patients.

OUTLINE: This is a dose escalation study of temozolomide.

Patients receive filgrastim (G-CSF) subcutaneously (SQ) or IV beginning on day -5 and continuing through at least day 3. Peripheral blood stem cells (PBSC) are collected on days 0, 2, and 4. Patients then receive oral temozolomide daily for 5 consecutive days. PBSC collections are reinfused 1 day after the last dose of temozolomide. Patients also receive G-CSF beginning at the time of transplant and continuing until blood counts recover. Treatment continues in the absence of disease progression or unacceptable toxicity.

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

Patients are followed every 3 months for 1-3 years, then annually thereafter.

PROJECTED ACCRUAL: A total of 30 patients will be accrued for this study over 12 months.


Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No


  • Histologically confirmed newly diagnosed malignant glioma or recurrent malignant CNS tumor of any pathology OR
  • Histologically confirmed non-CNS tumor

    • Recurrent soft tissue sarcomas (e.g., rhabdomyosarcoma)
    • Recurrent or resistant neuroblastoma
    • Recurrent Wilm's tumor
    • Recurrent Ewing's sarcoma
    • Recurrent primitive neuroectodermal tumors
    • Recurrent nasopharyngeal carcinoma
    • Recurrent germ cell tumor
  • Expected cure rate less than 10% with standard therapy
  • Measurable and/or active disease
  • History of bone marrow tumor infiltration with or without mass lesions or isolated abnormal CSF cytology as only evidence of recurrent disease allowed if complete response was first achieved with primary conventional therapy



  • 18 and under

Performance status:

  • Karnofsky 70-100% OR
  • Lansky 70-100%

Life expectancy:

  • Greater than 8 weeks


  • Reasonably cellular bone marrow (greater than 15% cellularity on biopsy)
  • Absolute neutrophil count greater than 1,000/mm^3
  • Platelet count greater than 75,000/mm^3


  • Bilirubin less than 2.0 mg/dL
  • SGPT less than 120 U/L


  • Creatinine less than 1.5 mg/dL


  • Systolic fraction or ejection fraction at least 80% predicted for age by echocardiogram


  • CVC or DLCO at least 60% predicted for age OR clearance from pulmonologist


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • HIV negative
  • No active infection
  • Able to tolerate vigorous hydration schedule


Biologic therapy:

  • No concurrent white blood cell transfusion
  • No other concurrent hematopoietic growth factors


  • See Disease Characteristics
  • At least 4 weeks since prior chemotherapy
  • No other concurrent cytotoxic drugs (systemic or intrathecal)

Endocrine therapy:

  • Concurrent corticosteroids allowed


  • See Disease Characteristics
  • At least 1 week since prior radiotherapy


  • At least 1 week since prior surgery


  • No other concurrent investigational agents
  Contacts and Locations
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00005952

United States, North Carolina
Duke Comprehensive Cancer Center
Durham, North Carolina, United States, 27710
Sponsors and Collaborators
Duke University
National Cancer Institute (NCI)
Study Chair: Henry S. Friedman, MD Duke Cancer Institute
  More Information

Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT00005952     History of Changes
Other Study ID Numbers: 1735  DUMC-1735-04-9R5  DUMC-1735-02-9R3  DUMC-1735-01-9R2  DUMC-1833-99-10  NCI-G00-1796  CDR0000067932 
Study First Received: July 5, 2000
Last Updated: June 19, 2013
Health Authority: United States: Federal Government
Duke University Health System Institutional Review Board

Keywords provided by Duke University:
childhood low-grade cerebral astrocytoma
recurrent childhood rhabdomyosarcoma
childhood craniopharyngioma
disseminated neuroblastoma
stage 4S neuroblastoma
recurrent neuroblastoma
stage IV Wilms tumor
stage V Wilms tumor
recurrent Wilms tumor and other childhood kidney tumors
childhood central nervous system germ cell tumor
stage III malignant testicular germ cell tumor
recurrent malignant testicular germ cell tumor
stage IV nasopharyngeal cancer
recurrent nasopharyngeal cancer
childhood germ cell tumor
metastatic childhood soft tissue sarcoma
recurrent childhood soft tissue sarcoma
stage IV ovarian germ cell tumor
recurrent ovarian germ cell tumor
childhood high-grade cerebral astrocytoma
childhood oligodendroglioma
childhood choroid plexus tumor
untreated childhood brain stem glioma
recurrent childhood brain stem glioma
untreated childhood supratentorial primitive neuroectodermal tumor
recurrent childhood supratentorial primitive neuroectodermal tumor
untreated childhood cerebellar astrocytoma
recurrent childhood cerebellar astrocytoma
recurrent childhood cerebral astrocytoma
untreated childhood medulloblastoma

Additional relevant MeSH terms:
Central Nervous System Neoplasms
Head and Neck Neoplasms
Neoplasms, Germ Cell and Embryonal
Nervous System Neoplasms
Testicular Neoplasms
Endocrine Gland Neoplasms
Endocrine System Diseases
Genital Diseases, Male
Genital Neoplasms, Male
Gonadal Disorders
Neoplasms by Histologic Type
Neoplasms by Site
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Neoplasms, Neuroepithelial
Nervous System Diseases
Neuroectodermal Tumors
Neuroectodermal Tumors, Primitive
Neuroectodermal Tumors, Primitive, Peripheral
Testicular Diseases
Urogenital Neoplasms
Alkylating Agents
Antineoplastic Agents
Antineoplastic Agents, Alkylating
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Therapeutic Uses

ClinicalTrials.gov processed this record on April 27, 2016