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Arsenic Trioxide in Treating Young Patients With Refractory Leukemia or Lymphoma

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: NCT00020111
Recruitment Status : Completed
First Posted : January 27, 2003
Last Update Posted : April 29, 2015
Information provided by:
National Cancer Institute (NCI)

Tracking Information
First Submitted Date  ICMJE July 11, 2001
First Posted Date  ICMJE January 27, 2003
Last Update Posted Date April 29, 2015
Study Start Date  ICMJE March 2000
Primary Completion Date Not Provided
Current Primary Outcome Measures  ICMJE Not Provided
Original Primary Outcome Measures  ICMJE Not Provided
Change History Complete list of historical versions of study NCT00020111 on Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
Descriptive Information
Brief Title  ICMJE Arsenic Trioxide in Treating Young Patients With Refractory Leukemia or Lymphoma
Official Title  ICMJE Phase I Trial and Pharmacokinetic Study of Arsenic Trioxide in Pediatric Patients With Refractory Leukemia or Lymphoma
Brief Summary

RATIONALE: Drugs used in chemotherapy 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 arsenic trioxide in treating young patients with leukemia or lymphoma.

Detailed Description


  • Determine the toxic effects of arsenic trioxide in pediatric patients with refractory leukemia or lymphoma.
  • Determine the maximum tolerated dose of this drug in this patient population.
  • Determine the plasma pharmacokinetics of this drug in these patients.

OUTLINE: This is a dose-escalation, multicenter study. Patients are stratified according to disease (acute promyelocytic leukemia [APL] vs non-APL).

  • Stratum I (APL patients): Patients receive standard-dose arsenic trioxide IV over 2 hours daily 5 days a week for 4 weeks. Treatment continues every 6 weeks for up to 3 courses in the absence of disease progression or unacceptable toxicity.
  • Stratum II (Non-APL patients): Cohorts of 3-6 patients receive escalating doses of arsenic trioxide (according to the stratum 1 schedule above) until the maximum tolerated dose (MTD) is determined. The MTD is defined as the dose preceding that at which at least 2 of 3 or 2 of 6 patients experience dose-limiting toxicity. Once the MTD is determined, additional patients are accrued to receive treatment with arsenic trioxide at the recommended phase II dose.

Leukemia patients in both strata without progressive disease who have not achieved complete remission after the first 20 doses may continue to receive arsenic trioxide for 2 additional weeks.

Patients are followed every 3 months for 1 year.

PROJECTED ACCRUAL: A maximum of 18 patients will be accrued for stratum I of this study within 2-3 years. A total of 3-30 patients will be accrued for stratum II of this study within 1-2 years.

Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Primary Purpose: Treatment
Condition  ICMJE
  • Leukemia
  • Lymphoma
Intervention  ICMJE Drug: arsenic trioxide
Study Arms  ICMJE Not Provided
Publications *

*   Includes publications given by the data provider as well as publications identified by Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status  ICMJE Completed
Enrollment  ICMJE Not Provided
Original Enrollment  ICMJE Not Provided
Study Completion Date  ICMJE Not Provided
Primary Completion Date Not Provided
Eligibility Criteria  ICMJE


  • Histologically confirmed leukemia or lymphoma refractory to standard curative treatment regimens
  • Measurable or evaluable disease
  • No meningeal leukemia or lymphoma
  • No HIV-related lymphoma
  • No lymphoproliferative diseases



  • 2 to 21

    • Acute promyelocytic leukemia (APL) patients (stratum I) must be age 2 to 12

Performance status:

  • ECOG 0-2

Life expectancy:

  • Not specified


  • Not specified


  • Bilirubin normal
  • SGPT less than 2 times upper limit of normal
  • No significant hepatic dysfunction that would preclude study therapy


  • Creatinine normal (age adjusted) OR
  • Creatinine clearance at least 60 mL/min
  • Potassium, magnesium, and calcium at least lower limit of normal (oral or IV supplementation allowed)
  • No significant renal dysfunction that would preclude study therapy


  • Rate corrected QTc interval no greater than 0.48 on EKG
  • No significant cardiac dysfunction that would preclude study therapy
  • No cardiac disease, including dysrhythmias


  • No significant pulmonary dysfunction that would preclude study therapy


  • Not pregnant or nursing
  • Negative pregnancy test
  • Fertile patients must use effective contraception
  • No persistent grade 3 or greater sensory or motor neuropathy
  • No prior grand mal seizures (grade 3 or greater) within the past 2 years other than febrile seizures (except for patients with APL at discretion of investigator)
  • No clinically significant unrelated systemic illness that would preclude study therapy (e.g., serious infection)
  • HIV negative


Biologic therapy:

  • At least 1 week since prior colony-stimulating factors (e.g., filgrastim [G-CSF], sargramostim [GM-CSF], and epoetin alfa)
  • No concurrent immunotherapy


  • No prior arsenic trioxide
  • At least 2 weeks since prior chemotherapy (4 weeks for nitrosoureas) and recovered
  • No concurrent intrathecal chemotherapy except for acute promyelocytic leukemia (APL) patients experiencing progressive meningeal leukemia and demonstrating benefit from arsenic trioxide for systemic disease
  • No other concurrent anticancer chemotherapy

Endocrine therapy:

  • No concurrent steroids (except corticosteroids for retinoic acid syndrome)


  • At least 4 weeks since prior radiotherapy and recovered
  • No concurrent radiotherapy


  • Not specified


  • At least 6 months since prior anticonvulsants
  • At least 1 week since prior retinoid therapy
  • No concurrent retinoids
  • No other concurrent investigational agents
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 2 Years to 21 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Canada,   United States
Removed Location Countries  
Administrative Information
NCT Number  ICMJE NCT00020111
Other Study ID Numbers  ICMJE CDR0000067717
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Not Provided
Study Sponsor  ICMJE National Cancer Institute (NCI)
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Chair: Frank M. Balis, MD National Cancer Institute (NCI)
PRS Account National Cancer Institute (NCI)
Verification Date February 2005

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