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Phase III Study of CPX-351 Versus 7+3 in Patients 60-75 Years Old With Untreated High Risk (Secondary) Acute Myeloid Leukemia

Phase 3
Completed
Conditions
High Risk Acute Myeloid Leukemia
Interventions
Drug: 7+3 (cytarabine and daunorubicin)
Registration Number
NCT01696084
Lead Sponsor
Jazz Pharmaceuticals
Brief Summary

To confirm the efficacy of CPX-351 compared to 7+3 as first line therapy in elderly patients (60-75 yrs) with high risk (secondary) Acute Myeloid Leukemia. The primary efficacy endpoint will be overall survival.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
309
Inclusion Criteria
  • Ability to understand and voluntarily give informed consent

  • Age 60-75 years at the time of diagnosis of AML

  • Pathological diagnosis of AML according to WHO criteria (with at least 20% blasts in the peripheral blood or bone marrow)

  • Confirmation of:

    • Therapy related AML: t-AML must have a documented history of prior cytotoxic therapy or ionizing radiotherapy for an unrelated disease
    • AML with a history of myelodysplasia: MDSAML must have bone marrow documentation of prior MDS
    • AML with a history of CMMoL: CMMoLAML must have bone marrow documentation of prior CMMoL
    • De novo AML with karyotypic abnormalities characteristic of MDS: de novoAML must have cytogenetics with abnormalities per WHO.
  • Eastern Cooperative Oncology Group (ECOG) performance status 0-2

  • Able to adhere to the study visit schedule and other protocol requirements

  • Laboratory values fulfilling the following:

    • Serum creatinine < 2.0 mg/dL
    • Serum total bilirubin < 2.0 mg/dL, patients with Gilbert's Syndrome should contact the medical monitor
    • Serum alanine aminotransferase or aspartate aminotransferase < 3 times the ULN Note: If elevated liver enzymes, above the ULN, are related to disease; contact medical monitor to discuss.
  • Cardiac ejection fraction ≥ 50% by echocardiography or MUGA

  • Patients with second malignancies in remission may be eligible if there is clinical evidence of disease stability for a period of greater than 6 months off cytotoxic chemotherapy, documented by imaging, tumor marker studies, etc., at screening. Patients maintained on long-term non-chemotherapy treatment, e.g., hormonal therapy, are eligible.

Exclusion Criteria
  • Except for CMMoL, patients with history of myeloproliferative neoplasms (MPN) (defined as a history of essential thrombocytosis or polycythemia vera, or idiopathic myelofibrosis prior to the diagnosis of AML) or combined MDS/MPN are not eligible.
  • Acute promyelocytic leukemia [t(15;17)] or favorable cytogenetics, including t(8;21) or inv16 if known at the time of randomization.
  • Clinical evidence of active CNS leukemia
  • Patients with active (uncontrolled, metastatic) second malignancies are excluded.
  • Prior treatment intended for induction therapy of AML; only hydroxyurea is permitted for control of blood counts. For example, a patient with MDS that changes HMA dose and schedule after the diagnosis of AML is excluded. AML-type therapy, such as cytarabine alone (>1g/m2/day) or cytarabine plus an anthracycline as well as prior HSCT are also excluded.
  • Administration of any therapy for MDS (conventional or investigational) must be completed by 2 weeks prior to of the first dose of study drug; in the event of rapidly proliferative disease use of hydroxyurea is permitted until 24 hours before the start of study treatment. Toxicities associated with prior MDS therapy must have recovered to grade 1 or less prior to start of treatment.
  • Any major surgery or radiation therapy within four weeks.
  • Patients with prior cumulative anthracycline exposure of greater than 368 mg/m2 daunorubicin (or equivalent).
  • Any serious medical condition, laboratory abnormality or psychiatric illness that would prevent obtaining informed consent
  • Patients with myocardial impairment of any cause (e.g. cardiomyopathy, ischemic heart disease, significant valvular dysfunction, hypertensive heart disease, and congestive heart failure) resulting in heart failure by New York Heart Association Criteria (Class III or IV staging)
  • Active or uncontrolled infection. Patients with an infection receiving treatment (antibiotic, antifungal or antiviral treatment) may be entered into the study but must be afebrile and hemodynamically stable for ≥72 hrs.
  • Current evidence of invasive fungal infection (blood or tissue culture); patients with recent fungal infection must have a subsequent negative cultures to be eligible; known HIV (new testing not required) or evidence of active hepatitis B or C infection (with rising transaminase values)
  • Hypersensitivity to cytarabine, daunorubicin or liposomal products
  • History of Wilson's disease or other copper-metabolism disorder

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Arm B (7+3)7+3 (cytarabine and daunorubicin)Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with cytarabine and daunorubicin given as a 7 + 3, or 5 days of continuous infusion of cytarabine and 2 days of daunorubicin (5+2, second induction, consolidation courses) therapy. The number of inductions and consolidations a subject received depended on response.
Arm A (CPX-351)CPX-351Subjects are eligible to receive up to 2 inductions and up to 2 consolidations with CPX-351. The number of inductions and consolidations a subject received depended on response.
Primary Outcome Measures
NameTimeMethod
Overall SurvivalFrom the date of randomization to death from any cause

Overall survival was measured from the date of randomization to death from any cause, subjects not known to have died by the last follow-up were censored on the date they were last known to be alive.

Secondary Outcome Measures
NameTimeMethod
Proportion of Subjects With a ResponsePost Induction

Complete Remission (CR)

Event-free SurvivalFrom the date of randomization to the date that persistent disease was documented or the date of relapse after CR or death, whichever came first

All randomized subjects were assessed for event-free survival (EFS). EFS was defined as the time from study randomization to the date of induction treatment failure (persistent disease), relapse from CR or CRi or death from any cause, whichever came first. Subjects alive and not known to have any of these events were censored on thee date they were last examined on study.

Remission DurationFrom the date of achievement of a remission until the date of relapse or death from any cause

Only subjects achieving CR or CRi were assessed for remission duration.

Rate of Achieving Morphologic Leukemia-free StateDay 14

All randomized subjects with at least 1 evaluable postrandomization bone marrow assessment performed on or after Day 14 after the last induction were assessed for MLFS.

Proportion of Subjects Receiving a Stem Cell TransplantPost Induction

The number and percentage of subjects transferred for HSCT after induction treatment was recorded.

Trial Locations

Locations (43)

Hackensack University Medical Center

🇺🇸

Hackensack, New Jersey, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

Dana-Farber Cancer Institute

🇺🇸

Boston, Massachusetts, United States

Northside Hospital

🇺🇸

Atlanta, Georgia, United States

UPMC

🇺🇸

Pittsburgh, Pennsylvania, United States

Dartmouth Hitchcock Medical Center

🇺🇸

Lebanon, New Hampshire, United States

University of Chicago

🇺🇸

Chicago, Illinois, United States

Rush University Medical Center

🇺🇸

Chicago, Illinois, United States

Columbia University

🇺🇸

New York, New York, United States

University of Florida

🇺🇸

Gainesville, Florida, United States

Washington University

🇺🇸

Saint Louis, Missouri, United States

Baylor Research Insitute

🇺🇸

Dallas, Texas, United States

Hopital Maisonneuve-Rosemont

🇨🇦

Montreal, Quebec, Canada

North Shore LIJ Health System

🇺🇸

Long Island City, New York, United States

Cornell U, Weill Medical College

🇺🇸

New York, New York, United States

M.D. Anderson Cancer Center

🇺🇸

Houston, Texas, United States

British Columbia Cancer Center

🇨🇦

Vancouver, British Columbia, Canada

Franciscan St. Francis Health

🇺🇸

Indianapolis, Indiana, United States

University of Minnesota

🇺🇸

Minneapolis, Minnesota, United States

Duke University Medical Center

🇺🇸

Durham, North Carolina, United States

Sarah Cannon Research Institute

🇺🇸

Nashville, Tennessee, United States

UCLA

🇺🇸

Los Angeles, California, United States

University of CA San Diego

🇺🇸

San Diego, California, United States

University of Missouri

🇺🇸

Columbia, Missouri, United States

New York Medical College

🇺🇸

Valhalla, New York, United States

Fox Chase Cancer Center

🇺🇸

Philadelphia, Pennsylvania, United States

Vanderbilt University

🇺🇸

Nashville, Tennessee, United States

Fred Hutchinson Cancer Research Center

🇺🇸

Seattle, Washington, United States

University of Alberta Hospital

🇨🇦

Edmonton, Alberta, Canada

Princess Margaret Hospital

🇨🇦

Toronto, Ontario, Canada

Stanford University

🇺🇸

Stanford, California, United States

University of Alabama at Birmingham

🇺🇸

Birmingham, Alabama, United States

Yale University

🇺🇸

New Haven, Connecticut, United States

H. Lee Moffitt Cancer Center

🇺🇸

Tampa, Florida, United States

University of Michigan

🇺🇸

Ann Arbor, Michigan, United States

Medical College of Wisconsin

🇺🇸

Milwaukee, Wisconsin, United States

Wake Forest University Health Services

🇺🇸

Winston-Salem, North Carolina, United States

Providence Portland Medical Center

🇺🇸

Portland, Oregon, United States

Oregon Health and Science University

🇺🇸

Portland, Oregon, United States

University of North Carolina at Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Medical University of South Carolina

🇺🇸

Charleston, South Carolina, United States

Montefiore Medical Center

🇺🇸

Bronx, New York, United States

University of Kansas Medical Center

🇺🇸

Kansas City, Kansas, United States

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