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Topotecan in Treating Children With Refractory Leukemia

This study has been completed.
Information provided by (Responsible Party):
National Cancer Institute (NCI) Identifier:
First received: November 1, 1999
Last updated: January 31, 2013
Last verified: May 2001
November 1, 1999
January 31, 2013
April 1996
January 2001   (Final data collection date for primary outcome measure)
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Complete list of historical versions of study NCT00002705 on Archive Site
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Topotecan in Treating Children With Refractory Leukemia
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.


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.

Phase 1
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Drug: topotecan hydrochloride
Experimental: Arm I
Single-Agent Chemotherapy. Topotecan, TOPO, NSC-609699.
Intervention: Drug: topotecan hydrochloride
Furman WL, Stewart CF, Kirstein M, Kepner JL, Bernstein ML, Kung F, Vietti TJ, Steuber CP, Becton DL, Baruchel S, Pratt C. Protracted intermittent schedule of topotecan in children with refractory acute leukemia: a pediatric oncology group study. J Clin Oncol. 2002 Mar 15;20(6):1617-24.

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Not Provided
January 2001   (Final data collection date for primary outcome measure)


-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
Sexes Eligible for Study: All
up to 20 Years   (Child, Adult)
Contact information is only displayed when the study is recruiting subjects
Canada,   Puerto Rico,   Switzerland,   United States
CDR0000064511 ( Registry Identifier: PDQ (Physician Data Query) )
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National Cancer Institute (NCI)
National Cancer Institute (NCI)
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Study Chair: Wayne Lee Furman, MD St. Jude Children's Research Hospital
National Cancer Institute (NCI)
May 2001

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