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Topotecan in Treating Children With Refractory Leukemia

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: NCT00002705
Recruitment Status : Completed
First Posted : July 1, 2004
Last Update Posted : February 1, 2013
Information provided by (Responsible Party):
National Cancer Institute (NCI)

Brief Summary:
Phase I trial to study the effectiveness of topotecan in treating children with refractory leukemia. Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die.

Condition or disease Intervention/treatment Phase
Leukemia Drug: topotecan hydrochloride Phase 1

Detailed Description:


I. Describe the qualitative and quantitative toxic effects, including acute and chronic dose-limiting toxicity, cumulative toxicity, and time to recovery, in pediatric patients with refractory leukemia who are treated with a 30-minute daily infusion of topotecan (TOPO) for up to 12 consecutive days every 3 weeks.

II. Estimate the maximum tolerated dose of TOPO that results in tolerable, predictable, and reversible toxicity.

III. Determine the precautions and supportive therapy that should be used and the clinical and laboratory studies needed to monitor or alter therapy to prevent unacceptable toxicity in these patients.

IV. Observe any antileukemic effects that may occur during this phase I study in which duration of treatment is increased from 7 to 9 and then 12 days and TOPO doses are escalated.

V. Determine the recommended phase II pediatric dose of TOPO. VI. Characterize the pharmacokinetic parameters of TOPO and evaluate changes in these parameters with the first and last doses to determine whether there is drug accumulation.

VII. Correlate, if possible, these pharmacokinetic parameters with clinical response and toxicity.


Single-Agent Chemotherapy. Topotecan, TOPO, NSC-609699.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Study Start Date : April 1996
Actual Primary Completion Date : January 2001

Arm Intervention/treatment
Experimental: Arm I
Single-Agent Chemotherapy. Topotecan, TOPO, NSC-609699.
Drug: topotecan hydrochloride

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


-Histologically or cytologically confirmed leukemia refractory to conventional therapy or for which no effective curative therapy exists


  • Age: Under 21
  • Performance status: ECOG 0-2
  • Life expectancy: At least 8 weeks
  • Adequate platelet count and hemoglobin required (transfusion allowed)
  • Bilirubin no greater than 1.5 mg/dL
  • AST or ALT no greater than 2 times normal
  • Creatinine less than 1.5 mg/dL
  • Adequate nutritional status, e.g. higher than third percentile weight for height
  • Albumin at least 3 g/dL
  • No severe uncontrolled infection
  • No pregnant women
  • Effective contraception required of fertile women


  • At least 3 weeks since systemic chemotherapy (6 weeks since nitrosoureas)
  • Recovered at least 3 months since bone marrow transplant (at least 6 months since total-body irradiation)
  • No concurrent anticancer therapy
  • No concurrent treatment studies

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): NCT00002705

  Show 31 Study Locations
Sponsors and Collaborators
National Cancer Institute (NCI)
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Study Chair: Wayne Lee Furman, MD St. Jude Children's Research Hospital

Publications of Results:
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Responsible Party: National Cancer Institute (NCI) Identifier: NCT00002705     History of Changes
Other Study ID Numbers: NCI-2012-01833
CDR0000064511 ( Registry Identifier: PDQ (Physician Data Query) )
First Posted: July 1, 2004    Key Record Dates
Last Update Posted: February 1, 2013
Last Verified: May 2001
Keywords provided by National Cancer Institute (NCI):
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia
relapsing chronic myelogenous leukemia
Additional relevant MeSH terms:
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Neoplasms by Histologic Type
Topoisomerase I Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents