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A Pilot Study of Intensive Chemotherapy With Peripheral Stem Cell Support for Infants With Malignant Brain Tumors

Phase 1
2 Years
Not Enrolling
Brain Tumors, Central Nervous System Tumors, Neuroblastoma, Sarcoma

Thank you

Trial Information

A Pilot Study of Intensive Chemotherapy With Peripheral Stem Cell Support for Infants With Malignant Brain Tumors


- Determine the maximum tolerated dose of thiotepa in infants with malignant brain or
spinal cord tumors receiving intensive chemotherapy.

- Determine the feasibility and toxicity of intensive chemotherapy with peripheral blood
stem cell (PBSC) rescue in these patients.

- Assess the feasibility of harvesting PBSCs in these patients.

- Determine the complete response rate and overall event-free survival rate in patients
treated with this regimen.

OUTLINE: This is a pilot, multicenter study.

Patients undergo surgery for diagnosis and maximal tumor resection.

Within 6 weeks of surgery or when stable, patients begin induction chemotherapy comprising
cisplatin IV over 6 hours on day 0; vincristine IV on days 0, 7, and 14; cyclophosphamide IV
over 1 hour on days 1-2; and etoposide IV over 1 hour on days 0-2. Twenty four hours after
the last cyclophosphamide dose, patients receive filgrastim (G-CSF) subcutaneously (SC) and
undergo peripheral blood stem cell harvest 2 days later. Treatment repeats every 21 days for
up to 3 courses in the absence of disease progression or unacceptable toxicity.

Within 6 weeks after induction chemotherapy, patients receive consolidation chemotherapy
comprising carboplatin IV over 2 hours on days 0-1 followed immediately by escalating doses
of thiotepa IV over 2 hours. Patients then undergo peripheral blood stem cell
transplantation 48 hours after the last thiotepa dose. Patients receive G-CSF SC daily on
days 3 to 21. Treatment repeats every 21 days for up to 3 courses in the absence of disease
progression or unacceptable toxicity.

Patients experiencing dose-limiting toxicity due to thiotepa are removed from the study.

Patients are followed at 4 weeks, every 3 months for 1 year, every 6 months for 3 years, and
then annually for 3 years or until relapse.

PROJECTED ACCRUAL: A total of 83 patients will be accrued for this study within 1 year.

Inclusion Criteria


- Histologically proven malignant brain or spinal cord tumor, including the following:

- Primitive neuroectodermal tumor

- Ganglioneuroblastoma

- Medulloblastoma neuroblastoma

- Desmoplastic medulloblastoma

- Medulloepithelioma

- Ependymoma neuroepithelioma

- Anaplastic ependymoma germ cell tumor

- Astrocytoma germinoma

- Anaplastic astrocytoma

- Embryonal carcinoma

- Glioblastoma endodermal sinus tumor

- Gliosarcoma malignant teratoma

- Choroid plexus carcinoma

- Mixed germ cell tumor

- Cerebellar sarcoma

- Pineoblastoma

- Atypical teratoid/rhabdoid tumor

- Choriocarcinoma

- Teratoma (malignant or with malignant transformations)

- Diffusely involved brain stem tumors allowed if there is evidence of brain stem
glioma by CT scan or MRI



- 6 months to less than 3 years

Performance Status:

- Not specified

Life Expectancy:

- More than 8 weeks


- Absolute neutrophil count greater than 1,000/mm^3

- Platelet count greater than 100,000/mm^3


- Bilirubin less than 2.0 mg/dL


- Glomerular filtration rate or creatinine clearance greater than 70 mL/min


Biologic therapy:

- No prior biologic therapy


- No prior chemotherapy

Endocrine therapy:

- Prior corticosteroids allowed


- No prior radiotherapy


- No more than 6 weeks since prior surgery

- Recovered from prior surgery (stable)

Type of Study:


Study Design:

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

Outcome Measure:


Outcome Description:

Demonstrate the feasibility of administering this regimen, to select an acceptable Thiotepa dose for Consolidation therapy, and to document significant toxicities and estimate their overall rates

Outcome Time Frame:

Up to 4 weeks after completion of study treatment

Safety Issue:


Principal Investigator

Bruce H. Cohen, MD

Investigator Role:

Study Chair

Investigator Affiliation:

The Cleveland Clinic


United States: Federal Government

Study ID:




Start Date:

March 1998

Completion Date:

October 2011

Related Keywords:

  • Brain Tumors
  • Central Nervous System Tumors
  • Neuroblastoma
  • Sarcoma
  • childhood infratentorial ependymoma
  • childhood supratentorial ependymoma
  • disseminated neuroblastoma
  • stage 4S neuroblastoma
  • embryonal childhood rhabdomyosarcoma
  • childhood high-grade cerebral astrocytoma
  • childhood choroid plexus tumor
  • previously untreated childhood rhabdomyosarcoma
  • untreated childhood brain stem glioma
  • untreated childhood supratentorial primitive neuroectodermal tumor
  • untreated childhood cerebellar astrocytoma
  • untreated childhood medulloblastoma
  • newly diagnosed childhood ependymoma
  • localized resectable neuroblastoma
  • localized unresectable neuroblastoma
  • regional neuroblastoma
  • childhood spinal cord neoplasm
  • childhood atypical teratoid/rhabdoid tumor
  • childhood low-grade cerebral astrocytoma
  • childhood central nervous system choriocarcinoma
  • childhood central nervous system embryonal tumor
  • childhood central nervous system germinoma
  • childhood central nervous system mixed germ cell tumor
  • childhood central nervous system teratoma
  • childhood central nervous system yolk sac tumor
  • Brain Neoplasms
  • Nervous System Neoplasms
  • Neuroblastoma
  • Spinal Cord Neoplasms
  • Central Nervous System Neoplasms
  • Sarcoma



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Children's Hospital of Orange CountyOrange, California  92668
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St. Joseph's Hospital and Medical CenterPaterson, New Jersey  07503
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Cook Children's Medical Center - Fort WorthFort Worth, Texas  76104
City of Hope Comprehensive Cancer CenterDuarte, California  91010
Children's Hospital Central CaliforniaMadera, California  93638-8762
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Covenant Children's HospitalLubbock, Texas  79410
Children's Hospital of the King's DaughtersNorfolk, Virginia  23507
Overlook HospitalSummit, New Jersey  07902-0220
Carol G. Simon Cancer Center at Morristown Memorial HospitalMorristown, New Jersey  07962
Winthrop University HospitalMineola, New York  11501
Kaiser Permanente Medical Center - OaklandSacramento, California  95825
Albert Einstein Cancer Center at Albert Einstein College of MedicineBronx, New York  10461
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Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Rainbow Babies and Children's HospitalCleveland, Ohio  44106-5000
Medical University of Ohio Cancer CenterToledo, Ohio  43614
Texas Tech University Health Sciences Center School of Medicine - AmarilloAmarillo, Texas  79106
Methodist Children's Hospital of South TexasSan Antonio, Texas  78229-3993
Primary Children's Medical CenterSalt Lake City, Utah  84113-1100
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AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston CampusAtlanta, Georgia  30322
MBCCOP - Medical College of Georgia Cancer CenterAugusta, Georgia  30912-3730
Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical CenterSavannah, Georgia  31403-3089
Indiana University Melvin and Bren Simon Cancer CenterIndianapolis, Indiana  46202-5289
Lucille P. Markey Cancer Center at University of KentuckyLexington, Kentucky  40536-0093
C.S. Mott Children's Hospital at University of Michigan Medical CenterAnn Arbor, Michigan  48109-0286
Butterworth Hospital at Spectrum HealthGrand Rapids, Michigan  49503-2560
Masonic Cancer Center at University of MinnesotaMinneapolis, Minnesota  55455
Children's Hospitals and Clinics of Minnesota - MinneapolisMinneapolis, Minnesota  55404
Children's Hospitals and Clinics of Minnesota - St. PaulSt. Paul, Minnesota  55102-2392
CCOP - Nevada Cancer Research FoundationLas Vegas, Nevada  89109-2306
Beth Israel Medical Center - Petrie DivisionNew York, New York  10003-3803
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Nationwide Children's HospitalColumbus, Ohio  43205-2696
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Knight Cancer Institute at Oregon Health and Science UniversityPortland, Oregon  97239-3098
Penn State Children's HospitalHershey, Pennsylvania  17033-0850
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