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Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission
Trial Status: complete
This study enrolled 472 participants, aged 55 or older, with a diagnosis of de novo acute
myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or chronic
myelomonocytic leukemia (CMML), and who have achieved first complete remission (CR)/
complete remission with incomplete blood count recovery (CRi) following induction with or
without consolidation chemotherapy.
The study is amended to include an extension phase (EP). The EP allows participants who
are currently receiving oral azacitidine and who are demonstrating clinical benefit as
assessed by the investigator, to continue receiving oral azacitidine after unblinding by
sponsor until the participant meets the criteria for study discontinuation or until oral
azacitidine becomes commercially available and reimbursed. In addition, all participants
in the placebo arm and participants who had been discontinued from the treatment phase
(irrespective of randomization arm) and continuing in the follow-up phase will be
followed for survival in the EP.
Inclusion Criteria
Male or female participants ≥ 55 years of age
Newly diagnosed, histologically confirmed de novo acute myeloid leukemia (AML) or AML secondary to prior myelodysplastic disease or CMML (Chronic myelomonocytic leukemia)
First complete remission (CR)/ complete remission with incomplete blood count recovery (CRi) with induction therapy with intensive chemotherapy with or without consolidation therapy within 4 months (+/- 7 days of achieving CR or CRi)
Eastern Cooperative Oncology Group (ECOG) performance status - 0, 1, 2, 3 Key Inclusion Criteria in the Extended Phase of the study: At the Investigator's discretion and with approval of the sponsor, participants meeting
all of the following eligibility criteria are eligible to enter the extension phase:
All participants randomized into the oral azacitidine or placebo arm and are continuing in either the treatment phase or follow-up phase of the CC-486-AML-001 study;
Participants randomized to oral azacitidine treatment arm and continuing in the treatment phase demonstrating clinical benefit as assessed by the investigator are eligible to receive oral azacitidine in the extension phase (EP);
Participants randomized into placebo arm of the study will not receive oral azacitidine in the EP, but will be followed for survival in the EP;
Participants currently in the follow-up phase will continue to be followed for survival in the EP;
Participants who have signed the informed consent for the EP of the study;
Participants who do not meet any of the criteria for study discontinuation Key
Exclusion Criteria
AML with inversion (inv)(16), translocation = t(8;21), t(16;16), t(15;17), or t(9;22) or molecular evidence of such translocations
Prior bone marrow or stem cell transplantation
Have achieved CR/CRi following therapy with hypomethylating agents
Diagnosis of malignant disease within the previous 12 months
Proven central nervous system (CNS) leukemia
Additional locations may be listed on ClinicalTrials.gov for NCT01757535.
