Working… Menu

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)

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.

Condition or disease Intervention/treatment Phase
Leukemia Lymphoma Drug: arsenic trioxide Phase 1

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Primary Purpose: Treatment
Official Title: Phase I Trial and Pharmacokinetic Study of Arsenic Trioxide in Pediatric Patients With Refractory Leukemia or Lymphoma
Study Start Date : March 2000

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.

Layout table for eligibility information
Ages Eligible for Study:   2 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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

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): NCT00020111

  Hide Study Locations
Layout table for location information
United States, Arkansas
Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
Little Rock, Arkansas, United States, 72205
United States, California
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010-3000
Rebecca and John Moores UCSD Cancer Center
La Jolla, California, United States, 92103-8447
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027-0700
Jonsson Comprehensive Cancer Center, UCLA
Los Angeles, California, United States, 90095-1781
Children's Hospital of Orange County
Orange, California, United States, 92868
Lucile Packard Children's Hospital at Stanford University Medical Center
Palo Alto, California, United States, 94304
UCSF Comprehensive Cancer Center
San Francisco, California, United States, 94143
County of Los Angeles Harbor-UCLA Medical Center
Torrance, California, United States, 90509
United States, District of Columbia
Children's National Medical Center
Washington, District of Columbia, United States, 20010-2970
United States, Georgia
AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Scottish Rite Campus
Atlanta, Georgia, United States, 30342
United States, Illinois
Children's Memorial Hospital - Chicago
Chicago, Illinois, United States, 60614
United States, Indiana
Riley Children Cancer Center at Riley Hospital for Children
Indianapolis, Indiana, United States, 46202-5225
United States, Kansas
Kansas Masonic Cancer Research Institute at the University of Kansas Medical Center
Kansas City, Kansas, United States, 66160-7357
United States, Maryland
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Baltimore, Maryland, United States, 21231-2410
Warren Grant Magnuson Clinical Center - NCI Clinical Studies Support
Bethesda, Maryland, United States, 20892-1182
United States, Massachusetts
Floating Hospital for Children
Boston, Massachusetts, United States, 02111
Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02115
United States, Michigan
University of Michigan Comprehensive Cancer Center
Ann Arbor, Michigan, United States, 48109-0914
Children's Hospital of Michigan
Detroit, Michigan, United States, 48201
United States, Minnesota
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, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Cardinal Glennon Children's Hospital
Saint Louis, Missouri, United States, 63104
St. Louis Children's Hospital
Saint Louis, Missouri, United States, 63110
United States, New Jersey
Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, United States, 07601
United States, New York
NYU School of Medicine's Kaplan Comprehensive Cancer Center
New York, New York, United States, 10016
Memorial Sloan-Kettering Cancer Center
New York, New York, United States, 10021
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, North Carolina
Duke Comprehensive Cancer Center
Durham, North Carolina, United States, 27710
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229-2899
Ireland Cancer Center at University Hospitals of Cleveland and Case Western Reserve University
Cleveland, Ohio, United States, 44106-5065
United States, Oklahoma
Oklahoma University Medical Center
Oklahoma City, Oklahoma, United States, 73126
United States, Pennsylvania
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States, 38105-2794
Vanderbilt Children's Hospital
Nashville, Tennessee, United States, 37232-6310
United States, Texas
Simmons Cancer Center at University of Texas Southwestern Medical Center - Dallas
Dallas, Texas, United States, 75390-9063
Cook Children's Medical Center - Fort Worth
Fort Worth, Texas, United States, 76104
Texas Children's Cancer Center
Houston, Texas, United States, 77030-2399
MD Anderson Cancer Center at University of Texas
Houston, Texas, United States, 77030-4009
University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States, 78284-7811
United States, Utah
Huntsman Cancer Institute at University of Utah
Salt Lake City, Utah, United States, 84112
United States, Washington
Children's Hospital and Regional Medical Center - Seattle
Seattle, Washington, United States, 98105
United States, Wisconsin
University of Wisconsin Comprehensive Cancer Center
Madison, Wisconsin, United States, 53792-6164
Midwest Children's Cancer Center
Milwaukee, Wisconsin, United States, 53226
Australia, Victoria
Royal Children's Hospital
Parkville, Victoria, Australia, 3052
Australia, Western Australia
Princess Margaret Hospital for Children
Perth, Western Australia, Australia, 6001
Canada, Ontario
Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Canada, Quebec
Montreal Children's Hospital at McGill University Health Center
Montreal, Quebec, Canada, H3H 1P3
Hopital Sainte Justine
Montreal, Quebec, Canada, H3T 1C5
Sponsors and Collaborators
National Cancer Institute (NCI)
Layout table for investigator information
Study Chair: Frank M. Balis, MD National Cancer Institute (NCI)

Publications of Results:
Layout table for additonal information Identifier: NCT00020111     History of Changes
Obsolete Identifiers: NCT00004548
Other Study ID Numbers: CDR0000067717
First Posted: January 27, 2003    Key Record Dates
Last Update Posted: April 29, 2015
Last Verified: February 2005
Keywords provided by National Cancer Institute (NCI):
recurrent childhood acute myeloid leukemia
childhood acute promyelocytic leukemia (M3)
recurrent childhood acute lymphoblastic leukemia
recurrent childhood large cell lymphoma
recurrent childhood lymphoblastic lymphoma
recurrent childhood small noncleaved cell lymphoma
recurrent/refractory childhood Hodgkin lymphoma
childhood chronic myelogenous leukemia
atypical chronic myeloid leukemia, BCR-ABL1 negative
Additional relevant MeSH terms:
Layout table for MeSH terms
Neoplasms by Histologic Type
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Arsenic Trioxide
Antineoplastic Agents