High-Dose Topotecan and Peripheral Stem Cell Transplantation in Treating Patients With Refractory 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: NCT00002948
Recruitment Status : Terminated
First Posted : January 27, 2003
Last Update Posted : July 2, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Yale University

November 1, 1999
January 27, 2003
July 2, 2014
October 1996
January 2004   (Final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00002948 on Archive Site
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High-Dose Topotecan and Peripheral Stem Cell Transplantation in Treating Patients With Refractory Cancer
A Phase I Study of High Dose Topotecan With Filgrastim and Peripheral Blood Stem Cell Support for Patients With Refractory Malignancies

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 drugs and kill more tumor cells.

PURPOSE: Phase I trial to study the effectiveness of high-dose topotecan and peripheral stem cell transplantation in treating patients with refractory cancer.


  • Determine the maximum tolerated dose, toxicities, and preliminary antitumor activity of escalating doses of topotecan, with filgrastim (G-CSF) and peripheral blood stem cell support, when administered to patients with refractory malignancies for which no effective therapy exists.

OUTLINE: This is a dose-escalation study.

Prior to stem cell harvesting, patients receive 1-2 courses of mobilizing salvage chemotherapy.

After stem cell harvest, high-dose topotecan is administered according to an escalating dosage scale. Topotecan is given over 30 minutes daily for three days. A minimum of 3 patients are entered at each dose level. The MTD is defined as the dose immediately below that at which 2 patients experience dose limiting toxicity.

Peripheral blood stem cells (PBSC) are infused at least 24 hours after treatment with topotecan is complete. Filgrastim (G-CSF) is administered subcutaneously beginning on the day PBSC are infused and continuing until blood counts recover.

Patients are followed every 3 months for 1 year and then every 6 months thereafter.


Phase 1
Primary Purpose: Treatment
Unspecified Adult Solid Tumor, Protocol Specific
  • Biological: filgrastim
  • Drug: topotecan hydrochloride
  • Procedure: bone marrow ablation with stem cell support
  • Procedure: peripheral blood stem cell transplantation
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*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
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January 2004
January 2004   (Final data collection date for primary outcome measure)


  • Histologically confirmed refractory malignancies for which no effective therapy is currently available



  • Over 18

Performance status:

  • ECOG 0-2

Life expectancy:

  • At least 3 months


  • Platelet count at least 100,000/mm^3
  • Absolute neutrophil count at least 1,500/mm^3


  • Total bilirubin no greater than 2.0 mg/dL
  • No chronic active hepatitis


  • Creatinine clearance at least 60 mL/min


  • Left ventricular ejection fraction greater than 45%


  • DLCO greater than 60% of predicted


  • HIV negative
  • No active infection
  • No concurrent medical condition that would preclude therapy
  • Not pregnant or nursing
  • Fertile patients must use effective contraception


Biologic therapy:

  • Not specified


  • At least 4 weeks since prior chemotherapy (6 weeks since prior nitrosoureas or mitomycin)

Endocrine therapy:

  • Not specified


  • No radiotherapy to greater than 30% of bone marrow
  • No wide field radiotherapy


  • Recovered from any prior surgery
Sexes Eligible for Study: All
18 Years and older   (Adult, Senior)
Contact information is only displayed when the study is recruiting subjects
United States
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Yale University
Yale University
National Cancer Institute (NCI)
Study Chair: Mario Sznol, MD Yale University
Yale University
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP