Clinical Trial: CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

Official Title: A Phase II Study of CPX-351 for Treatment of AML or Higher Risk MDS Relapsed or Refractory to Prior Therapy With Hypomethylating (HMA) Agent

Brief Summary: This phase II clinical trial studies how well liposomal cytarabine-daunorubicin CPX-351 (CPX-351) works in treating patients with relapsed or refractory acute myeloid leukemia or myelodysplastic syndrome. Drugs used in chemotherapy, such as liposomal cytarabine-daunorubicin CPX-35, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.

Detailed Summary:

PRIMARY OBJECTIVES:

I. Determine the efficacy and safety profile of the use of CPX-351 in older patients (age 60 and older) with: higher risk of myelodysplastic syndrome (MDS) who are refractory/relapsed after prior hypomethylating (HMA) therapy; subjects greater than 75 years old with higher risk MDS who are HMA relapsed/refractory who have progressed to acute myeloid leukemia (AML)); AML with refractory/relapsed disease after prior HMA therapy for AML.

SECONDARY OBJECTIVES:

I. Determine the duration of remission following induction therapy with CPX-351.

II. Determine overall survival at 12 months. III. Determine the early induction mortality (at 60 days) following CPX-351 in this cohort following induction therapy.

OUTLINE:

INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 intravenously (IV) over 90 minutes on days 1, 3, and 5. Patients with reduced blast count not achieving a morphological leukemia free state (< 5% blasts) receive a second course of induction therapy. Patients achieving a complete remission (CR) or a CR with incomplete blood count recovery (CRi) at day 14 or after a second course of induction therapy proceed to consolidation therapy.

SECOND INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3.

CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV over 90 minutes on days 1 and 3. Patients may receive a second course after 28-75 days in the absence of disease progression or unacceptable toxicit
Sponsor: Bruno C. Medeiros

Current Primary Outcome:

  • Incidence of mortality assessed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 [ Time Frame: At day 30 ]
    To analyze the safety data, adverse events recorded using CTCAE 4.0 for each patient will reviewed and totaled, for the total number of adverse events for each adverse event compared to total group of patients (rate of outcome of each adverse event in study population).
  • Incidence of mortality assessed using the CTCAE version 4.0 [ Time Frame: At day 60 ]
    To analyze the safety data, adverse events recorded using CTCAE 4.0 for each patient will reviewed and totaled, for the total number of adverse events for each adverse event compared to total group of patients (rate of outcome of each adverse event in study population).
  • Incidence of serious adverse events as assessed by CTCAE version 4.0 [ Time Frame: Up to 4 weeks after completion of treatment ]
    To analyze the safety data, adverse events recorded using CTCAE 4.0 for each patient will reviewed and totaled, for the total number of adverse events for each adverse event compared to total group of patients (rate of outcome of each adverse event in study population).
  • Frequency of grade 3-5 adverse events as assessed by CTCAE version 4.0 [ Time Frame: Up to 4 weeks after completion of treatment ]
    To analyze the safety data, adverse events recorded using CTCAE 4.0 for each patient will reviewed and totaled, for the total number of adverse events for each adverse event compared to total group of patients (rate of outcome of each adverse event in stud

    Original Primary Outcome: Same as current

    Current Secondary Outcome:

    • Duration of remission following induction with CPX-351 [ Time Frame: From the start of response until disease relapse or death, assessed up to 1 year ]
      Calculated by counting the number of days from the date of remission until date of disease relapse for patients who achieve a CR (and CRi for AML). The average days of remission will be calculated to determine the median duration of remission.
    • Overall survival [ Time Frame: From the date of entry into trial to death from any cause, assessed at 12 months ]
      Determined by the number of patients who are alive at 12 months compared to the number of patients who initiated into the study.
    • Early induction mortality after first induction [ Time Frame: Day 60 ]
      Calculated by determining the number of deaths within the first 60 days, using day 1 of the first induction therapy as the first day, compared to the total number of patients who have received any dose of CPX-351.


    Original Secondary Outcome: Same as current

    Information By: Stanford University

    Dates:
    Date Received: November 25, 2013
    Date Started: February 2014
    Date Completion: December 2017
    Last Updated: April 25, 2016
    Last Verified: April 2016