Study of Elacytarabine Versus Investigator's Choice in Patients With Late Stage Acute Myeloid Leukaemia (AML)

Inclusion Criteria:

• 18 years of age or older

• Confirmed diagnosis of AML according to WHO classification (excluding acutepromyelocytic leukaemia) who have received two or three previousinduction/re-induction regimens or patients of age ≥ 65 with adverse cytogenetics whohave received 1-3 previous induction/re-induction regimens. One of the (re-)inductionregimens could be stem cell transplantation (SCT) for achievement of remission.Maintenance and consolidation (including SCT) may have been given, but are not countedas previous regimens.

• Bone marrow aspirates and/or biopsies must contain > 5 % leukaemic blast cells orpatient must have biopsy-proven extramedullary AML, or patient's peripheral bloodshows occurrence of leukaemic blast cells

• Patients must

• have never attained CR or CRi (primary refractory), or

• have failed initial induction therapy, and have attained CR or CRi after salvagetherapy(ies), and then relapsed within < 6 months, or

• have attained CR or CRi after initial induction therapy and relapsed within <12months, and failed to respond to salvage therapy(ies), or

• have relapsed after the latest CR or CRi within < 6 months

• Patients younger than 65 years should have received previous treatment with cytarabine

• Patients must have recovered from previous bone marrow and/or stem celltransplantation to a stage that the patient can tolerate the study treatment. There isno restriction on number of regimens or type of treatment administered for maintenanceor consolidation during previous stages of the disease

• ECOG performance status (PS) of 0 - 2

• Women of child-bearing potential must have a negative serum or urine pregnancy testwithin 2 weeks prior to treatment start

• Male and female patients must use acceptable contraceptive methods for the duration oftime on study, and males also for 3 months after the last elacytarabine dose

• Capable of understanding and complying with protocol requirements, and must be ableand willing to sign a written informed consent form

Exclusion Criteria:

• A history of allergic reactions to egg. A history of allergic reactions of CTCAE grade 3 or 4 to cytarabine

• Persistent clinically significant toxicities from previous chemotherapy

• A cancer history that, according to the investigator, might confound the assessment ofthe study endpoints

• Known positive status for human immunodeficiency virus (HIV)

• Pregnant and nursing patients

• Uncontrolled intercurrent illness including, but not limited to, uncontrolledinfection, or psychiatric illness/social situations that would limit compliance withstudy requirements

• Impairment of hepatic or renal function to such an extent that the patient, in theopinion of the investigator, will be exposed to an excessive risk if entered into thisclinical study

• Active heart disease including myocardial infarction within previous 3 months,symptomatic coronary artery disease, arrhythmias not controlled by medication, oruncontrolled congestive heart failure. Any New York Heart Association (NYHA)functional classification grade 3 or 4

• Applicable only for patients for whom an anthracycline is part of the selected controltreatment: Left ventricular ejection fraction (LVEF) must be ≥ 45 % as measured byMUGA scan or 2D ECHO within 14 days prior to start of therapy. Either method isacceptable for measuring LVEF

• Applicable only for patients for whom an anthracycline is part of the selected controltreatment: The patient should tolerate minimum one course of combination therapy

• Any anti-leukaemic agents within the last 3 weeks. Hydroxyurea,however, is allowed forup to 12 hours prior to study treatment

• Any investigational treatment within the last 14 days

• Any medical condition which in the opinion of the investigator places the patient atan unacceptably high risk for toxicities