COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC:

Get the latest research information from NIH: Menu

Clofarabine and Ara-C for the Treatment of Relapsed AML and Untreated MDS

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: NCT00334074
Recruitment Status : Completed
First Posted : June 6, 2006
Results First Posted : July 17, 2013
Last Update Posted : July 17, 2013
Information provided by (Responsible Party):
Baylor Research Institute

Brief Summary:
The purpose of this trial is to to determine the safety and effectiveness of therapeutic combination - Clofarabine and Cytarabine for the treatment of AML and MDS.

Condition or disease Intervention/treatment Phase
Acute Myelogenous Leukemia Myelodysplastic Syndromes Chronic Myelogenous Leukemia Drug: Clofarabine and Cytarabine Phase 2

Detailed Description:

Previous studies of Clofarabine and Cytarabine combination treatment in adult AML and MDS patients showed promising results.

This study is done to confirm the findings from previous studies. Primary objective is to determine the overall response rate (complete response [CR] plus partial response [PR]); secondary objective of this study is to characterize and quantify the toxicity profile associated with clofarabine plus cytarabine treatment.

A maximum of 35 patients will be treated on this study. They will receive 5 consecutive days of clofarabine intra venous infusion (IVI) followed 4 hours later by cytarabine IVI.Patients will receive up to a maximum of 4 cycles of study treatment. Next cycle will start approximately 4 weeks after Day 1 of previous cycle.No other investigational or commercial agents including chemotherapy, radiotherapy, or immunotherapy may be administered to patients enrolled in this study with the intention of treating the underlying malignancy

Patients will remain on study, and be monitored until 4 months have elapsed from the beginning date of their last cycle of treatment.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 30 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of Clofarabine and Cytarabine in Relapsed Standard-Risk AML and Untreated High-Risk MDS in Adult Patients, and Untreated AML in Selected Elderly Patients at High Risk of Anthracycline Toxicity
Study Start Date : August 2005
Actual Primary Completion Date : February 2007
Actual Study Completion Date : February 2008

Arm Intervention/treatment
Experimental: Clofarabine and Cytarabine
Five consecutive days of clofarabine 40 mg/m^2 IVI over 1 hour followed 4 hours later by cytarabine 1000 mg/m^2 IVI over 2 hours
Drug: Clofarabine and Cytarabine
Phase II Trial of Clofarabine and Cytarabine in Relapsed Standard-Risk AML and Untreated High-Risk MDS in Adult Patients, and Untreated AML in selected Elderly Patients at high risk of anthracycline toxicity
Other Names:
  • Clofarabine: CAFdA; Cl-F-ara-A
  • Cytarabine: Ara-C

Primary Outcome Measures :
  1. Response Rate (Complete Response [CR] Plus Partial Response [PR]) of Clofarabine Plus Cytarabine in Patients With Relapsed/Refractory AML, Untreated MDS, CML in Blast Phase, or in Selected Untreated Patients With High Risk of Anthracycline Toxicity [ Time Frame: Proportion of confirmed responses was estimated by the number of patients who achieved a CR or PR, defined as two consecutive evaluations at least 4 weeks apart, divided by the number of eligible participants in the study. ]

    Based on International working group for diagnosis, standardization of response criteria, and treatment outcomes for reporting standards for therapeutic trials in Acute myeloid Leukemia:

    Complete Response (CR) was defined as normalization of marrow blasts (< 5%), recovery of normal heamtopoiesis (absolute neutrophil count >1 X 10^9/l, platelet count ≥100 X10^9/l, and absence of peripheral blood blasts, independent of transfusions and growth factor support.

    Partial response was defined as blood count recovery as for complete response with the exception of leukemic marrow blasts in the range of 6%-25% or a ≥50% decrease in bone marrow blasts.

    Treatment failure was defined as a <25% change in marrow blasts within 30 days of starting therapy

Secondary Outcome Measures :
  1. Number of Participants Who Had an Adverse Event While on Treatment With Clofarabine Plus Cytarabine [ Time Frame: Up to five months (includes follow up period of 30 days) from the day patient received their first dose of study drug ]
    Patients will be monitored clinically and diagnostically using measures including blood test, bone marrow aspiration and MUGA. Toxicity assessment every week using the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 3.0 will be performed.

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:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Adult patients who are at least 18 years old with histologically confirmed disease as follows:

  • Standard or poor cytogenetic risk acute myelogenous leukemia (AML) according to the Southwestern Oncology Group (SWOG) criteria in first relapse or primary refractory status
  • Untreated high-risk myelodysplastic syndrome (MDS) defined as >10% blasts
  • Chronic myelogenous leukemia (CML) in accelerated phase or blast crisis failing imatinib therapy.
  • Selected elderly patients with untreated AML who are at high risk of anthracycline toxicity.

Inclusion Criteria:

  • Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or
  • Laboratory values obtained less than or equal to 7 days prior to receiving study treatment:

    • Total bilirubin < 2.0 mg/dL unless elevated due to hemolysis
    • Aspartate transaminase (AST)/alanine transaminase (ALT) less than or equal to 5 × upper limit of normal (ULN)
    • Serum creatinine < 2.0 mg/dL
  • Capable of understanding the investigational nature, potential risks and benefits of the study, and able to provide valid informed consent.
  • Female patients of childbearing potential must have a negative serum pregnancy test within 2 weeks prior to enrollment.
  • Male and female patients must use an effective contraceptive method during the study and for a minimum of 6 months after study treatment.

Exclusion Criteria:

  • Patients with FAB M3 unless relapsed after treatment with ATRA and arsenic trioxide.
  • Patients eligible to receive curative allogeneic transplant as determined by performance status, organ function, availability of a matched donor, etc.
  • Current concomitant chemotherapy, radiation therapy, or immunotherapy.
  • Use of investigational agents within 30 days or any anticancer therapy within 3 weeks before study entry. The patient must have recovered from all acute toxicities from any previous therapy.
  • Active heart disease including myocardial infarction within the preceding 3 months.
  • History of severe coronary artery disease, arrhythmias other than atrial flutter or fibrillation requiring medication, or uncontrolled congestive heart failure
  • Dyspnea at rest or with minimal exertion.
  • Patients with an active, uncontrolled systemic infection considered to be opportunistic, life-threatening, or clinically significant at the time of treatment or with a known or suspected fungal infection (ie, patients on parenteral antifungal therapy).
  • Pregnant or lactating patients.
  • Prior enrollment in this trial.
  • Any significant concurrent disease, illness, or psychiatric disorder that would compromise patient safety or compliance, interfere with consent, study participation, follow up, or interpretation of study results.

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): NCT00334074

Layout table for location information
United States, Texas
Baylor University Medical Center
Dallas, Texas, United States, 75246
Sponsors and Collaborators
Baylor Research Institute
Layout table for investigator information
Principal Investigator: Edward Agura, MD Baylor University Medical Center - Director Blood and Marrow Transplantation Services
Layout table for additonal information
Responsible Party: Baylor Research Institute Identifier: NCT00334074    
Other Study ID Numbers: 004-145
004-145 ( Other Identifier: Baylor Internal Review Board )
First Posted: June 6, 2006    Key Record Dates
Results First Posted: July 17, 2013
Last Update Posted: July 17, 2013
Last Verified: July 2013
Keywords provided by Baylor Research Institute:
Chemo treatment
standard or poor cytogenetic risk AML
untreated high-risk (>10% blasts) MDS
CML in accelerated phase or blast crisis
Elderly AML patients with risk of anthracycline toxicity
Additional relevant MeSH terms:
Layout table for MeSH terms
Leukemia, Myeloid
Leukemia, Myelogenous, Chronic, BCR-ABL Positive
Leukemia, Myeloid, Acute
Myelodysplastic Syndromes
Neoplasms by Histologic Type
Bone Marrow Diseases
Hematologic Diseases
Myeloproliferative Disorders
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs