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Multicenter Study of CPX-351(Cytarabine:Daunorubicin) Liposome Injection in Patients With Advanced Hematologic Cancer.

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: NCT00389428
Recruitment Status : Completed
First Posted : October 18, 2006
Last Update Posted : May 17, 2012
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Brief Summary:
The primary objective of this study is to determine the recommended dose of CPX-351 for use in a phase 2 efficacy study in patients with leukemia. Secondarily, the study will assess the safety, serious adverse effects and how the body handles CPX-351. Preliminary evidence of antitumor activity will also be determined.

Condition or disease Intervention/treatment Phase
Hematologic Neoplasms Drug: CPX-351 (Cytarabine:Daunorubicin) Liposome Injection Phase 1

Detailed Description:

CPX-351 is a liposomal formulation of a fixed combination of the antineoplastic drugs cytarabine and daunorubicin. The two drugs are present inside the liposome in a 5:1 molar ratio. The development of CPX-351 (cytarabine:daunorubicin) Liposome Injection was based on 1) defining a synergistic ratio of the two active moieties, cytarabine and daunorubicin, using cell-based screening assays and 2) designing a liposomal drug carrier to maintain this ratio after intravenous administration. CPX-351 was found to be more active in in vivo models of cancer than combinations of conventional cytarabine and daunorubicin. Both cytarabine and daunorubicin are active chemotherapeutic agents, each approved for clinical use in the United States for the treatment of hematological neoplasms.

CPX-351 is being developed with the hypothesis that it is superior to the currently used regimen of cytarabine and daunorubicin in the treatment of acute leukemia. This phase I study will determine the dose to carry forward into phase II trials.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase 1 Study of CPX-351(Cytarabine:Daunorubicin) Liposome Injection in Patients With Advanced Hematologic Malignancies.
Study Start Date : September 2006
Actual Primary Completion Date : December 2008
Actual Study Completion Date : December 2009

Resource links provided by the National Library of Medicine

Primary Outcome Measures :
  1. To determine the maximum tolerated dose (MTD) for use in phase 2

Secondary Outcome Measures :
  1. To evaluate the safety and dose-limiting toxicities (DLT) of CPX-351.
  2. To determine the pharmacokinetic parameters of CPX-351 administered in this schedule.
  3. To assess preliminary efficacy information of CPX-351 administered in this schedule in patients with advanced leukemias.

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

Inclusion Criteria:

  • Ability to understand and voluntarily sign an informed consent form
  • Age > 18 years at the time of signing the informed consent form
  • Pathological confirmation of leukemia or myelodysplastic syndrome.
  • AML according to WHO criteria; except for core-binding factor AMLs (t(8;21), inv(16) or t(16;16)) and APL
  • ALL
  • MDS
  • Patients with AML include the following:
  • Patients in 2nd or greater relapse
  • Patients in first relapse with initial CR duration lasting <6 months
  • Patients in first relapse refractory to induction therapy
  • Patients with primary refractory AML
  • Patients with ALL include the following
  • Patients with T-cell ALL refractory or in relapse following nelarabine
  • Patients with other ALL that is refractory or in relapse.
  • Patients with MDS include the following:
  • The subset of RAEB-2 patients with >10% blasts with at least 1 prior therapy that includes a hypomethylating agent.
  • Previously untreated chemotherapy induced AML
  • Eastern Cooperative Oncology Group (ECOG) performance status score of 0,1 or 2
  • Able to adhere to the study visit schedule and other protocol requirements
  • Life expectancy of at least 12 weeks
  • Laboratory values fulfilling the following:
  • Serum creatinine < 1.5 mg/dL
  • Serum total bilirubin < 1.5 mg/dL
  • Serum alanine aminotransferase or aspartate aminotransferase < 150 IU/liter Note: If elevated liver enzymes are related to disease; contact medical monitor to discuss.
  • Cardiac ejection fraction > 50% by MUGA scan or echocardiography
  • All men and women must agree to practice effective contraception during the study period if not otherwise documented to be infertile.

Exclusion Criteria:

  • Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent the patient from signing the informed consent form
  • Chemotherapy or other investigational anticancer therapeutic drugs in the two weeks prior to study entry; in the event of rapidly proliferative disease, however, the use of hydroxyurea is permitted up to 24 hours before study entry
  • Clinical evidence of active CNS leukemic involvement
  • Pregnant or lactating women
  • Clinically significant cardiac disease (New York Heart Association Class III or IV)
  • Severe debilitating pulmonary disease
  • Active and uncontrolled infection. Patients with an infection under active treatment with antibiotics and whose infection is controlled may be entered into the study
  • Current evidence of invasive fungal infection (blood or tissue culture); HIV or hepatitis C infection
  • Hypersensitivity to cytarabine, daunorubicin or liposomal products
  • History of Wilson's disease or other copper-related disorder

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): NCT00389428

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United States, District of Columbia
Georgetown University Medical Center
Washington, District of Columbia, United States, 20007
United States, Florida
H. Lee Moffit Cancer Center & Research Institute at the University of S. Florida
Tampa, Florida, United States, 33612-9497
United States, New York
North Shore University Hospital
Manhasset, New York, United States, 11030
New York Presbyterian Hospital Weill Medical College of Cornell University
New York, New York, United States, 10021
Sponsors and Collaborators
Jazz Pharmaceuticals
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Study Director: Arthur Louie, M.D. Jazz Pharmaceuticals
Principal Investigator: Jonathan Kolitz, M.D. New York School of Medicine at North Shore University Hospital
Publications automatically indexed to this study by Identifier (NCT Number):
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Responsible Party: Jazz Pharmaceuticals Identifier: NCT00389428    
Other Study ID Numbers: CLTR0305-101
First Posted: October 18, 2006    Key Record Dates
Last Update Posted: May 17, 2012
Last Verified: May 2012
Keywords provided by Jazz Pharmaceuticals:
Hematologic malignancies
Malignancies, hematologic
Hematopoietic malignancies
Additional relevant MeSH terms:
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Hematologic Neoplasms
Neoplasms by Site
Hematologic Diseases
Antimetabolites, Antineoplastic
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Antiviral Agents
Anti-Infective Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antibiotics, Antineoplastic
Topoisomerase II Inhibitors
Topoisomerase Inhibitors
Enzyme Inhibitors