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Phase II Study of Clofarabine in Pediatric Acute Myelogenous Leukemia (AML) Patients

This study has been completed.
Information provided by:
Sanofi Identifier:
First received: July 26, 2002
Last updated: March 4, 2015
Last verified: March 2015

Clofarabine (injection) is approved by the Food and Drug Administration (FDA) for the treatment of pediatric patients 1 to 21 years old with relapsed acute lymphoblastic leukemia (ALL) who have had at least 2 prior treatment regimens.

The purpose of this study is to determine whether Clofarabine is safe and effective in the treatment of Acute Myelogenous Leukemia (AML.)

Condition Intervention Phase
Leukemia, Myelocytic, Acute, Pediatric Drug: clofarabine (IV formulation) Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase II, Open Label Study of Clofarabine in Pediatric Patients With Refractory or Relapsed Acute Myelogenous Leukemia

Resource links provided by NLM:

Further study details as provided by Sanofi:

Estimated Enrollment: 40
Study Start Date: May 2002
Study Completion Date: August 2004
Detailed Description:
This is a non-randomized, open label, Phase II study of Clofarabine in pediatric patients with refractory or relapsed acute myelogenous leukemia (AML). Eligible patients must be in first or subsequent relapse or be refractory. Forty eligible patients will be enrolled in a Fleming 2-stage sequential study design in order to better assess the efficacy and safety of clofarabine in this patient population.

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

Inclusion Criteria:

  • Have a diagnosis of AML according to FAB classification with greater than or equal to 25% blasts in the bone marrow.
  • Be less than or equal to 21 years old at time of initial diagnosis.
  • Not be eligible for therapy of higher curative potential, and must be in first or subsequent relapse and/or refractory. Where an alternative therapy has been shown to prolong survival in an analogous population, this should be offered to the patient prior to discussing this study.
  • Patients with acute promyelocytic leukemia (M3) must have been treated with at least 2 regimens-a retinoic acid-containing regimen and an arsenic trioxide-containing regimen before being considered for this study.
  • Have a Karnofsky Performance Status (KPS) of greater than or equal to 70.
  • Provide signed, written informed consent from parent or guardian and assent from patients greater than or equal to 7 years old according to local IRB and institutional requirements.
  • Have adequate organ function as indicated by the following laboratory values, obtained within 2 weeks prior to registration: Serum bilirubin less than or equal to 1.5 x ULN; AST and ALT less than or equal to 5 x ULN; Serum Creatinine less than 2 x ULN for age. ULN= Institutional Upper Limit of Normal

Exclusion Criteria:

  • Received previous treatment with Clofarabine.
  • Have an active, uncontrolled systemic infection considered opportunistic, life threatening, or clinically significant at the time of treatment.
  • Are pregnant or lactating. Male and female patients who are fertile must agree to use an effective means of birth control (i.e., latex condom, diaphragm, cervical cap, etc) to avoid pregnancy.
  • Have psychiatric disorders that would interfere with consent, study participation, or follow up.
  • Are receiving any other chemotherapy. Patients must have been off previous therapy for at least 2 weeks (with the exception of intrathecal therapy, which is allowed up to 24hrs prior to 1st dose of study drug) and must have recovered from acute toxicity of all previous therapy prior to enrollment. Treatment may start earlier, following consultation with the ILEX Medical Monitor, if there is evidence of disease relapse prior to that time.
  • Have any other severe concurrent disease, which, in the judgment of the investigator, would make the patient inappropriate for entry into this study.
  • Have symptomatic CNS involvement.
  • Febrile neutropenia at time of study entry.
  • Known or suspected fungal infection (ie. patients on parenteral antifungal therapy).
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00042354

United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, California
Children's Hospital
Los Angeles, California, United States, 90027
Children's Hospital
Orange County, California, United States
Children's Hospital
San Diego, California, United States
United States, Colorado
Children's Hospital
Denver, Colorado, United States
United States, Connecticut
University of Connecticut Health Center
Hartford, Connecticut, United States, 06106
United States, Illinois
Children's Memorial Hospital
Chicago, Illinois, United States, 60614
United States, Maryland
Johns Hopkins Children's Center
Baltimore, Maryland, United States, 21231
United States, Missouri
Children's Hospital
St. Louis, Missouri, United States
United States, Nebraska
University of Nebraska Medical Center
Omaha, Nebraska, United States, 67198
United States, New York
Memorial Sloan-Kettering
New York, New York, United States
United States, Pennsylvania
Children's Hospital
Philadelphia, Pennsylvania, United States
Children's Hospital
Pittsburgh, Pennsylvania, United States, 15213
United States, Tennessee
St. Jude Children's Research Hospital
Memphis, Tennessee, United States
United States, Texas
Children's Medical Center
Dallas, Texas, United States
Cook's Children's Medical Center
Fort Worth, Texas, United States, 76104
Texas Children's Cancer Center
Houston, Texas, United States
The University of Texas M.D. Anderson Cancer Center
Houston, Texas, United States
Sponsors and Collaborators
Genzyme, a Sanofi Company
Study Director: Medical Monitor Genzyme, a Sanofi Company
  More Information

Additional Information:
Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00042354     History of Changes
Other Study ID Numbers: CLO222
Study First Received: July 26, 2002
Last Updated: March 4, 2015

Keywords provided by Sanofi:
Pediatric Acute Myelogenous Leukemia

Additional relevant MeSH terms:
Leukemia, Myeloid
Leukemia, Myeloid, Acute
Neoplasms by Histologic Type
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents processed this record on September 21, 2017