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Temozolomide in Treating Young Patients With Refractory or Recurrent 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: NCT00083070
Recruitment Status : Completed
First Posted : May 17, 2004
Last Update Posted : February 20, 2014
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Children's Oncology Group

Brief Summary:

RATIONALE: Drugs used in chemotherapy, such as temozolomide, work in different ways to stop cancer cells from dividing so they stop growing or die.

PURPOSE: This phase I trial is studying the side effects and best dose of temozolomide in treating young patients with refractory or recurrent leukemia.

Condition or disease Intervention/treatment Phase
Leukemia Drug: temozolomide Phase 1

Detailed Description:



  • Determine the maximum tolerated dose and recommended phase II dose of temozolomide in pediatric patients with refractory or recurrent leukemia.
  • Determine the toxic effects of this drug in these patients.
  • Determine the pharmacokinetics of this drug in these patients.


  • Determine the antitumor activity of this drug in these patients.
  • Determine the biologic activity and mechanism(s) of resistance to this drug in these patients.

OUTLINE: This is an open-label, dose-escalation, multicenter study.

Patients receive oral temozolomide once daily on days 1-5. Treatment repeats every 28 days for up to 12 courses in the absence of disease progression or unacceptable toxicity.

Cohorts of 3-6 patients receive escalating doses of temozolomide until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which 2 of 6 patients experience dose-limiting toxicity.

PROJECTED ACCRUAL: A total of 3-36 patients will be accrued for this study within 18-24 months.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 16 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Trial Of Temozolomide In Pediatric Patients With Refractory/Recurrent Leukemias
Study Start Date : March 2004
Actual Primary Completion Date : September 2004
Actual Study Completion Date : June 2008

Arm Intervention/treatment
Experimental: Temozolomide Therapy Drug: temozolomide
Other Names:
  • Temodar
  • NSC # 362856

Primary Outcome Measures :
  1. Maximum tolerated dose and recommended phase II dose
  2. Toxicity as assessed by CTCAE 3.0
  3. Pharmacokinetics as assessed by CI, area under the curve (AUC), and half-life (T ½)

Secondary Outcome Measures :
  1. Antitumor activity
  2. Biologic activity and mechanisms of resistance

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   1 Year to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • Histologically confirmed leukemia of any of the following types:

    • Acute lymphoblastic leukemia
    • Acute myeloid leukemia
    • Chronic myelogenous leukemia in blast crisis
  • Refractory or recurrent disease
  • Immunophenotypic confirmation of disease at initial diagnosis or recurrence
  • More than 25% blasts in the bone marrow (M3)
  • Active extramedullary disease allowed except for leptomeningeal disease
  • No known curative therapy or therapy proven to prolong survival with an acceptable quality of life
  • No active CNS disease



  • 1 to 21

Performance status

  • Karnofsky 50-100% (for patients > 10 years of age)
  • Lansky 50-100% (for patients ≤ 10 years of age)

Life expectancy

  • Not specified


  • WBC < 30,000/mm^3 (hydroxyurea or leukapheresis allowed at the discretion of the principal investigator)
  • Platelet count ≥ 20,000/mm^3 (platelet transfusions allowed)
  • Hemoglobin ≥ 8.0 g/dL (red blood cell transfusions allowed)


  • ALT ≤ 5 times upper limit of normal (ULN)
  • Albumin ≥ 2 g/dL
  • Bilirubin ≤ 1.5 times ULN


  • Creatinine normal for age OR
  • Creatinine clearance OR radioisotope glomerular filtration rate ≥ 70 mL/min/1.73 m^2


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No uncontrolled infection


Biologic therapy

  • At least 7 days since prior biologic therapy, including immunotherapy
  • At least 3 months since prior stem cell transplantation

    • No evidence of active graft-vs-host disease
  • No concurrent biologic therapy
  • No concurrent immunotherapy


  • Recovered from prior chemotherapy
  • At least 6 weeks since prior nitrosoureas
  • Prior therapy with hydroxyurea allowed for up to 24 hours before initiation of study drug
  • No other concurrent chemotherapy

Endocrine therapy

  • Concurrent hydrocortisone or other corticosteroids allowed as premedications prior to blood product transfusions in patients with prior severe allergic reactions


  • Recovered from prior radiotherapy
  • No concurrent radiotherapy


  • Not specified


  • No other concurrent anticancer agents
  • No other concurrent investigational drugs

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): NCT00083070

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United States, California
Stanford Cancer Center at Stanford University Medical Center
Stanford, California, United States, 94305
United States, Indiana
Indiana University Cancer Center
Indianapolis, Indiana, United States, 46202-5289
United States, Minnesota
Fairview University Medical Center - University Campus
Minneapolis, Minnesota, United States, 55455
Mayo Clinic Cancer Center
Rochester, Minnesota, United States, 55905
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216-4505
United States, New York
Herbert Irving Comprehensive Cancer Center at Columbia University
New York, New York, United States, 10032
SUNY Upstate Medical University Hospital
Syracuse, New York, United States, 13210
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-3039
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104-9786
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105
United States, Texas
Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75390
Baylor University Medical Center - Houston
Houston, Texas, United States, 77030-2399
United States, Washington
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, United States, 98105
Canada, Ontario
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Canada, Quebec
Hopital Sainte Justine
Montreal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
Children's Oncology Group
National Cancer Institute (NCI)
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Study Chair: Terzah M. Horton, MD, PhD Texas Children's Cancer Center
Publications of Results:
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Responsible Party: Children's Oncology Group Identifier: NCT00083070    
Other Study ID Numbers: ADVL0411
CDR0000362059 ( Other Identifier: Clinical )
COG-ADVL0411 ( Other Identifier: Children's Oncology Group )
First Posted: May 17, 2004    Key Record Dates
Last Update Posted: February 20, 2014
Last Verified: February 2014
Keywords provided by Children's Oncology Group:
recurrent childhood acute lymphoblastic leukemia
recurrent childhood acute myeloid leukemia
relapsing chronic myelogenous leukemia
childhood acute promyelocytic leukemia (M3)
blastic phase chronic myelogenous leukemia
Additional relevant MeSH terms:
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Neoplasms by Histologic Type
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents