ACUTE MYELOID LEUKEMIA

Primary: To compare disease-free survival in patients 60 years or older with acute myeloid leukemia (AML) who are randomly assigned to receive either OCV-501 monotherapy or placebo. Secondary: • To compare overall survival between both treatment arms • To compare patient quality of life (European Organization for Research and Treatment of Cancer [EORTC] QLQ-C30) and performance status (Eastern Cooperative Oncology Group performance status [ECOG PS]) between both treatment arms • To characterize and compare safety in the both treatment arms • To assess immunological responses (OCV-501-specific IFN-γ production, WT1-killer peptide specific IFN-γ production, anti-OCV-501 antibody level, anti-WT1 antibody level), immunoglobulin • To assess WT1 mRNA level

OCV-501

OCV-501

aqueous solution

0.6 mL

[Efficacy]
Primary endpoint:
Disease-free survival (DFS)
Secondary endpoints:
• Overall survival (OS)
• Quality of life (EORTC QLQ-C30)
• Performance status (ECOG PS)

[Safety]
Adverse events, hematology test, blood biochemistry tests, vital signs (blood pressure, pulse rate, body temperature), urinalysis, body weight, and electrocardiogram
[Exploratory]
OCV-501-specific IFN-γ production, WT1-killer peptide specific IFN-γ production, anti-OCV-501 antibody level, and anti-WT1 antibody level, immunoglobulin, and WT1 mRNA

Patients who meet all of the following criteria at the time of enrollment will be selected.
1) Patients with AML (WHO classification 2008) who achieved first complete remission within one or two courses of standard induction therapy*, and completed standard consolidation therapy* (more than one course).
• Applicable types of disease for this trial in WHO classification
− AML with recurrent genetic abnormalities
− AML, not otherwise specified
2) Patients who are 60 years or older at the time of providing informed consent
3) Sex: Male or female
4) Patients with an Eastern Cooperative Oncology Group (ECOG) performance status of 0 - 2 at the time of trial enrollment
5) Patients with no significant impairments in the functions of major organs (as indicated by laboratory values) meeting the following criteria within 3 weeks of investigational medicinal product (IMP) administration:
• Hematological functions
Patients must not require administration of a blood-derived product or blood transfusion, or granulocyte colony-stimulating factor (G-CSF), granulocyte macrophage colony-stimulating factor (GM-CSF).
− Neutrophil count: > 1,000/μL,
− Platelet count: > 50,000/μL
• Hepatic functions
− AST and ALT: < 5 × the upper limit of normal (ULN)
− Total bilirubin: < 3 × ULN
• Renal function
− Serum creatinine: < 3 × ULN
6) Patients with a life expectancy of at least 3 months
7) Patients who have provided written informed consent within 90 days from the last dose of consolidation therapy on an informed consent form that has been approved by an institutional review board or independent ethics committee

Patients who fall under any of the following exclusion criteria at the time of enrollment will be excluded from participation in the trial.
1) Patients who have acute promyelocytic leukemia (APL) with t(15;17)(q22;q12), (PML/RARA) karyotype abnormalities,
and other variant types.
2) Patients who are scheduled for hematopoietic stem cell transplantation
3) Patients who have undergone cancer immunotherapy (eg, cancer vaccine [including OCV-501], lymphocyte or dendric cell transfusional treatment)
4) Patients who have received drugs potentially affecting the immune system within 4 weeks before starting IMP administration or who may receive such drugs after start of the trial (eg, corticosteroid systemic administration, immunosuppressants, immunostimulants, and anti-cancer drugs)
5) Patients who have a history or complication of autoimmune diseases (eg, Hashimoto's disease, idiopathic thrombocytopenic purpura, autoimmune hepatitis, or connective tissue disease) or primary immune deficiency disease
6) Patients who have active infectious diseases
7) Patients who have a history or complication of interstitial pneumonia
8) Patients who have a severe concurrent disease or psychiatric illness (eg, cardiac failure [NYHA class III or IV], uncontrolled diabetes mellitus, uncontrolled hypertension) likely to interfere with participation in this trial
9) Patients who are HIV antibody positive, HBV-DNA positive or HCV antibody positive
10) Patients who have a history of hypersensitivity or serious adverse drug reaction to any of the components of IMP
11) Patients who have a concurrent second cancer (except carcinoma in situ, intramucosal cancers, or malignancies treated at least 5 years previously with no evidence of relapse)
12) Patients who have been administered investigational drugs or individually imported drugs within 12 weeks before starting IMP
13)Women with confirmed or suspected pregnancy, or breast-feeding women
14) Patients not agreeing to take adequate contraceptive measures during the trial period and until 180 days (for males) or 120 days (for females) after the last IMP administration
15) Patients judged to be ineligible by the investigator (or subinvestigator) for any other reasons