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Investigator Initiated Trial of CPX-351 for Untreated Acute Myeloid Leukemia

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ClinicalTrials.gov Identifier: NCT03335267
Recruitment Status : Recruiting
First Posted : November 7, 2017
Last Update Posted : November 7, 2017
Sponsor:
Information provided by (Responsible Party):
Weill Medical College of Cornell University

Brief Summary:
This is an open label study to assess the suitability of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML. Patients may have received prior AML treatment with non-intensive regimens, e.g. hypomethylating agents, low dose Ara C or lenolidomide, but may not have received intensive AML treatment with anthracyclines and/or cytarabine prior to enrollment on this trial. The outcome of elderly patients following intensive treatment with CPX-351 will be measured by clinical endpoints for efficacy and safety and by biological/functional response.

Condition or disease Intervention/treatment Phase
Leukemia, Myeloid, Acute Drug: CPX-351 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Phase II Trial of CPX (Cytarabine:Daunorubicin) Liposome Injection in Patients >/=60 Years of Age With AML Previously Untreated By Intensive Chemotherapy
Actual Study Start Date : October 19, 2017
Estimated Primary Completion Date : October 2019
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: CPX-351 (Cytarabine:Daunorubicin) Injection

Dosing for first induction: CPX-351

• CPX-351 at 100u/m2 will be administered on study days 1, 3 and 5

Dosing for second induction:

• CPX-351 at 100 u/m2 will be administered on days 1 and 3

Dosing for consolidation:

• CPX-351 at 65 u/m2 will be administered on days 1 and 3

Drug: CPX-351
Cytarabine:Daunorubicin Liposome Injection




Primary Outcome Measures :
  1. Assessment of the feasibility of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML based on safety and efficacy. [ Time Frame: 5 years ]
    CPX-351 alone will be declared feasible if one or two induction courses (3 doses or 5 doses) can be delivered to patients with acceptable 30-day mortality.


Secondary Outcome Measures :
  1. Efficacy of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML by bone marrow biopsy determination of treatment response rate. [ Time Frame: Up to 5 years, as needed. ]
    The efficacy of CPX-351 in this population will be assessed by treatment response rate, as determined by bone marrow analysis at Day 14 of each induction and consolidation, and as needed thereafter, up to 5 years.

  2. Safety of CPX-351 as first intensive therapy in elderly (age ≥60 years) patients with AML by evaluation of the frequency and severity of adverse events. [ Time Frame: 5 years ]
    The safety of CPX-351 in this population will be assessed by evaluation of the frequency and severity of adverse events.

  3. Quality of Life Assessment using the Functional Assessment of Cancer Therapy-Leukemia (FACT-LEU). [ Time Frame: 5 years ]

    The higher the score, the better the QOL. Physical Well-Being (PWB): Range:0-28. To derive: Subtract the answer from "4" for each of 7 questions. Add scores, multiply by 7 and divide by # of questions answered. Social/Family Well-Being (SWB): Range:0-28. To derive: Add answers of 7 questions, multiply by 7 and divide by # of questions answered.

    Emotional Well-Being (EWB): Range:0-24. To derive: Subtract the answers from "4" for each of the 6 questions, except #2 (added without modification). Add values, multiply by 6 and divide by # of questions answered. Functional Well-Being (FWB): Range:0-28. To derive: Add answers of 7 questions, multiply by 7 and divide by # of questions answered.

    Leukemia Subscale (LeuS): Range:0-68. To derive: Subtract the answers from "4" for each of the 17 questions, except #s 11 and 12 (these are added without modification). Add values, multiply by 17 and divide by # of questions answered. FACT-Leu total =PWB+SWB+EWB+FWB+LeuS. Range: 0-176.


  4. Cognitive function [ Time Frame: 5 years ]
    The relationship of cognitive function to outcome will be assessed using the Blessed Orientation-Memory-Concentration Test

  5. Cognitive function [ Time Frame: 5 years ]
    The relationship of cognitive function to outcome will be assessed using the Montreal Cognitive Assessment.

  6. Rate of morphologic leukemia-free state (MLFS) [ Time Frame: 5 years ]
    The rate of morphologic leukemia-free state (MLFS) will be determined to supplement the efficacy assessment of CPX-351.



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Ages Eligible for Study:   60 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Ability to understand and voluntarily give informed consent
  • Age≥60 years at the time of study treatment
  • Pathological diagnosis of AML according to WHO criteria (with >20% blasts in the peripheral blood or bone marrow) including:
  • De novo AML with normal karyotype or adverse karyotypes (including patients with karyotypic abnormalities characteristic of MDS)
  • Secondary AML: transformed from prior MDS or MPN, confirmed by bone marrow documentation of prior antecedent hematologic disorder
  • Therapy-related AML: t-AML, requires documented history of prior cytotoxic therapy or ionizing radiotherapy for an unrelated disease
  • Performance status >50% KPS, ECOG 0-2
  • Laboratory values fulfilling the following:
  • Serum creatinine < 2.5 mg/dL
  • Serum total bilirubin < 2.5 mg/dL,
  • Serum alanine aminotransferase or aspartate aminotransferase < 3 times the ULN
  • Patients with elevated liver enzymes and serum creatinine values secondary to AML are eligible after discussion with PI
  • Cardiac ejection fraction ≥ 50% by echocardiography or MUGA
  • Patients with history of second malignancies in remission may be eligible if there is clinical evidence of disease stability 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:

  • Acute promyelocytic leukemia [t(15;17)]
  • Clinical evidence of active CNS leukemia
  • Prior intensive chemotherapy for AML with anthracycline/cytarabine-based regimens and/ or prior HSCT. Patients may have been treated with commercially available or investigational hypomethylating agents (e.g. decitabine, azacitidine, SGI-110), lenalidomide, or low-dose cytarabine (not to exceed 20 mg/m2 daily for 14 days for ≤ 6 cycles)
  • Prior treatment including HMA, systemic chemotherapy, surgery, or radiation therapy must have been completed at least 7 days before start of study treatment or after discussion with PI. Treatment with investigational agents must have been completed at least 14 days prior to study drug treatment. Hydroxyurea is permitted for control of blood counts before the start of study treatment. Toxicities associated with prior therapies must have recovered to grade 1 or less prior to start of study treatment.
  • 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.
  • Patients with current or recent 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
  • History of prior bone marrow or solid organ transplantation

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 ClinicalTrials.gov identifier (NCT number): NCT03335267


Contacts
Contact: Ellen K Ritchie, MD 646-962-2700 ritchie@med.cornell.edu
Contact: Elena Lascu, MA 212-746-0974 ell2028@med.cornell.edu

Locations
United States, New York
Weill Cornell Medical College Recruiting
New York, New York, United States, 10021
Contact: Ellen K Ritchie, MD    646-962-2700    ritchie@med.cornell.edu   
Contact: Elena Lascu, MA    212-746-0974    ell2028@med.cornell.edu   
Sponsors and Collaborators
Weill Medical College of Cornell University
Investigators
Principal Investigator: Ellen K Ritchie, MD Weill Medical College of Cornell University

Responsible Party: Weill Medical College of Cornell University
ClinicalTrials.gov Identifier: NCT03335267     History of Changes
Other Study ID Numbers: 1510016710
First Posted: November 7, 2017    Key Record Dates
Last Update Posted: November 7, 2017
Last Verified: November 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Leukemia, Myeloid, Acute
Leukemia
Leukemia, Myeloid
Neoplasms by Histologic Type
Neoplasms
Cytarabine
Daunorubicin
Antimetabolites, Antineoplastic
Antimetabolites
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