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Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer

This study has been completed.
Information provided by:
Memorial Sloan Kettering Cancer Center Identifier:
First received: November 1, 1999
Last updated: June 20, 2013
Last verified: June 2013

RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Bone marrow transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells.

PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy with thiotepa, carboplatin, and topotecan followed by bone marrow transplantation in treating patients who have metastatic or progressive rare cancer.

Condition Intervention Phase
Childhood Germ Cell Tumor Extragonadal Germ Cell Tumor Head and Neck Cancer Kidney Cancer Liver Cancer Lymphoma Neuroblastoma Ovarian Cancer Retinoblastoma Sarcoma Testicular Germ Cell Tumor Biological: filgrastim Drug: carboplatin Drug: thiotepa Drug: topotecan hydrochloride Procedure: autologous bone marrow transplantation Procedure: bone marrow ablation with stem cell support Procedure: in vitro-treated bone marrow transplantation Phase 2

Study Type: Interventional
Study Design: Primary Purpose: Treatment
Official Title: Myeloablative Chemotherapy With Bone Marrow Rescue For Rare Poor-Prognosis Cancers

Resource links provided by NLM:

Further study details as provided by Memorial Sloan Kettering Cancer Center:

Study Start Date: October 1992
Study Completion Date: April 2005
Primary Completion Date: April 2005 (Final data collection date for primary outcome measure)
Detailed Description:


  • Improve the long term disease-free survival of patients with rare cancers at high risk for lethal relapse by using myeloablative chemotherapy with thiotepa, carboplatin, and topotecan followed by autologous bone marrow or peripheral blood stem cell rescue.

OUTLINE: Autologous bone marrow or peripheral blood stem cells (PBSC) are harvested. Patients receive high-dose thiotepa IV over 3 hours on days -8 to -6, carboplatin IV over 4 hours on days -5 to -3, and topotecan IV over 30 minutes on days -8 to -4. Autologous bone marrow or PBSC are reinfused on day 0. Patients receive filgrastim (G-CSF) IV twice daily beginning on day 1.

Patients are followed for 1 year.

PROJECTED ACCRUAL: Approximately 50 patients will be accrued for this study within 5 years.


Ages Eligible for Study:   up to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed malignancy of one of the following types:

    • Wilms' tumor
    • Liver cancer
    • Desmoplastic or other small round cell tumor
    • Nasopharyngeal carcinoma
    • Fibrosarcoma
  • Disease that has metastasized and has a cure rate of no greater than 25% with conventional treatment or disease that has progressed after prior chemotherapy, was not then surgically resectable, and has a salvage rate with nonmyeloablative therapies of no greater than 25% required
  • Maximal benefit from conventional (nonmyeloablative) doses of combination chemotherapy required prior to entry, and it is recommended that patients have received a minimum of one of the following:

    • 2 courses of high-dose cyclophosphamide (as per protocol MSKCC-90062)
    • 2 courses of high-dose ifosfamide/etoposide (as in the poor-risk sarcoma protocol MSKCC-90071A)
    • 1 course of high-dose cyclophosphamide plus 1 course of high-dose ifosfamide/etoposide
  • Within 3 weeks of initiation of protocol therapy, patients must be:

    • In CR or good PR OR
    • Tumor considered "chemosensitive", i.e., a 50% or greater decrease in at least 1 measurable tumor parameter attributable to prior chemotherapy without evidence of progressive disease by any other parameter
  • Ineligible for other IRB-approved myeloablative regimens
  • No evidence of current bone marrow involvement on bone marrow aspiration (x4) and biopsy (x2)



  • 21 and under

Performance status:

  • Not specified


  • Not specified


  • Bilirubin no greater than 1.5 times upper limit of normal (ULN)
  • SGOT no greater than 1.5 times ULN
  • Alkaline phosphatase no greater than 1.5 times ULN
  • 5'-Nucleotidase no greater than 1.5 times ULN


  • Creatinine normal
  • Creatinine clearance at least 60 mL/min


  • CPK normal
  • Echocardiogram (or RNCA) normal
  • EKG normal


Biologic therapy

  • Not specified


  • See Disease Characteristics

Endocrine therapy

  • Not specified


  • Not specified


  • See Disease Characteristics
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00002515

United States, New York
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
Sponsors and Collaborators
Memorial Sloan Kettering Cancer Center
Study Chair: Brian H. Kushner, MD Memorial Sloan Kettering Cancer Center
  More Information Identifier: NCT00002515     History of Changes
Other Study ID Numbers: 92-148
CDR0000078115 ( Registry Identifier: PDQ (Physician Data Query) )
Study First Received: November 1, 1999
Last Updated: June 20, 2013

Keywords provided by Memorial Sloan Kettering Cancer Center:
recurrent childhood rhabdomyosarcoma
stage IV childhood liver cancer
recurrent neuroblastoma
recurrent childhood liver cancer
recurrent Wilms tumor and other childhood kidney tumors
stage IV Wilms tumor
recurrent retinoblastoma
stage IV childhood lymphoblastic lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent osteosarcoma
stage IV ovarian germ cell tumor
recurrent malignant testicular germ cell tumor
childhood germ cell tumor
alveolar childhood rhabdomyosarcoma
recurrent childhood soft tissue sarcoma
recurrent ovarian germ cell tumor
childhood fibrosarcoma
extragonadal germ cell tumor
childhood desmoplastic small round cell tumor
recurrent childhood small noncleaved cell lymphoma
stage IV childhood small noncleaved cell lymphoma
recurrent childhood large cell lymphoma
stage IV childhood large cell lymphoma
recurrent Ewing sarcoma/peripheral primitive neuroectodermal tumor
stage IV lymphoepithelioma of the nasopharynx
stage IV squamous cell carcinoma of the nasopharynx
recurrent squamous cell carcinoma of the nasopharynx
recurrent lymphoepithelioma of the nasopharynx

Additional relevant MeSH terms:
Head and Neck Neoplasms
Liver Neoplasms
Neoplasms, Germ Cell and Embryonal
Kidney Neoplasms
Carcinoma, Renal Cell
Testicular Neoplasms
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Neoplasms, Connective and Soft Tissue
Neoplasms by Site
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Digestive System Neoplasms
Digestive System Diseases
Liver Diseases
Urologic Neoplasms
Urogenital Neoplasms
Kidney Diseases processed this record on September 21, 2017