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

Brief Summary:

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.

Condition or disease Intervention/treatment Phase
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 Phase 1

Detailed Description:


  • 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.


Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: A Phase I Study of High Dose Topotecan With Filgrastim and Peripheral Blood Stem Cell Support for Patients With Refractory Malignancies
Study Start Date : October 1996
Actual Primary Completion Date : January 2004
Actual Study Completion Date : January 2004

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00002948

United States, Connecticut
Yale Comprehensive Cancer Center
New Haven, Connecticut, United States, 06520-8032
Sponsors and Collaborators
Yale University
National Cancer Institute (NCI)
Study Chair: Mario Sznol, MD Yale University

Responsible Party: Yale University Identifier: NCT00002948     History of Changes
Other Study ID Numbers: CDR0000065416
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: July 2, 2014
Last Verified: July 2014

Keywords provided by Yale University:
unspecified adult solid tumor, protocol specific

Additional relevant MeSH terms:
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents