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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

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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



Memorial Sloan-Kettering Cancer Center New York, New York  10021
Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Mayo Clinic Cancer Center Rochester, Minnesota  55905
University of Chicago Cancer Research Center Chicago, Illinois  60637
CCOP - Kalamazoo Kalamazoo, Michigan  49007-3731
Presbyterian - St. Luke's Medical Center Denver, Colorado  80218
Carole and Ray Neag Comprehensive Cancer Center at the University of Connecticut Health Center Farmington, Connecticut  06360-2875
CCOP - MeritCare Hospital Fargo, North Dakota  58122
Sanford Cancer Center at Sanford USD Medical Center Sioux Falls, South Dakota  57117-5039
Avera Cancer Institute Sioux Falls, South Dakota  57105
Deaconess Medical Center Spokane, Washington  99210-0248
Holden Comprehensive Cancer Center at University of Iowa Iowa City, Iowa  52242-1002
Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill Chapel Hill, North Carolina  27599-7570
Cleveland Clinic Taussig Cancer Center Cleveland, Ohio  44195
Children's Hospital of Orange County Orange, California  92668
Children's National Medical Center Washington, District of Columbia  20010-2970
Children's Mercy Hospital Kansas City, Missouri  64108
St. Joseph's Hospital and Medical Center Paterson, New Jersey  07503
Children's Hospital and Regional Medical Center - Seattle Seattle, Washington  98105
Cook Children's Medical Center - Fort Worth Fort Worth, Texas  76104
City of Hope Comprehensive Cancer Center Duarte, California  91010
Children's Hospital Central California Madera, California  93638-8762
Kosair Children's Hospital Louisville, Kentucky  40202-3830
Covenant Children's Hospital Lubbock, Texas  79410
Children's Hospital of the King's Daughters Norfolk, Virginia  23507
Overlook Hospital Summit, New Jersey  07902-0220
Carol G. Simon Cancer Center at Morristown Memorial Hospital Morristown, New Jersey  07962
Winthrop University Hospital Mineola, New York  11501
Kaiser Permanente Medical Center - Oakland Sacramento, California  95825
Albert Einstein Cancer Center at Albert Einstein College of Medicine Bronx, New York  10461
NYU Cancer Institute at New York University Medical Center New York, New York  10016
Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039
Rainbow Babies and Children's Hospital Cleveland, Ohio  44106-5000
Medical University of Ohio Cancer Center Toledo, Ohio  43614
Texas Tech University Health Sciences Center School of Medicine - Amarillo Amarillo, Texas  79106
Methodist Children's Hospital of South Texas San Antonio, Texas  78229-3993
Primary Children's Medical Center Salt Lake City, Utah  84113-1100
Group Health Central Hospital Seattle, Washington  98104
Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai Medical Center Los Angeles, California  90048-1865
Jonsson Comprehensive Cancer Center at UCLA Los Angeles, California  90095-1781
Childrens Hospital Los Angeles Los Angeles, California  90027
Children's Hospital and Research Center Oakland Oakland, California  94609
UCSF Helen Diller Family Comprehensive Cancer Center San Francisco, California  94115
Children's Hospital Center for Cancer and Blood Disorders Aurora, Colorado  80045
Alfred I. duPont Hospital for Children Wilmington, Delaware  19803
Lombardi Comprehensive Cancer Center at Georgetown University Medical Center Washington, District of Columbia  20007
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus Atlanta, Georgia  30322
MBCCOP - Medical College of Georgia Cancer Center Augusta, Georgia  30912-3730
Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical Center Savannah, Georgia  31403-3089
Indiana University Melvin and Bren Simon Cancer Center Indianapolis, Indiana  46202-5289
Lucille P. Markey Cancer Center at University of Kentucky Lexington, Kentucky  40536-0093
C.S. Mott Children's Hospital at University of Michigan Medical Center Ann Arbor, Michigan  48109-0286
Butterworth Hospital at Spectrum Health Grand Rapids, Michigan  49503-2560
Masonic Cancer Center at University of Minnesota Minneapolis, Minnesota  55455
Children's Hospitals and Clinics of Minnesota - Minneapolis Minneapolis, Minnesota  55404
Children's Hospitals and Clinics of Minnesota - St. Paul St. Paul, Minnesota  55102-2392
CCOP - Nevada Cancer Research Foundation Las Vegas, Nevada  89109-2306
Beth Israel Medical Center - Petrie Division New York, New York  10003-3803
Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center New York, New York  10032
Akron Children's Hospital Akron, Ohio  44308-1062
Nationwide Children's Hospital Columbus, Ohio  43205-2696
Dayton Children's - Dayton Dayton, Ohio  45404-1815
Knight Cancer Institute at Oregon Health and Science University Portland, Oregon  97239-3098
Penn State Children's Hospital Hershey, Pennsylvania  17033-0850
Children's Hospital of Pittsburgh of UPMC Pittsburgh, Pennsylvania  15213
Mary Bridge Children's Hospital and Health Center - Tacoma Tacoma, Washington  98405