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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT01074047




Registration number
NCT01074047
Ethics application status
Date submitted
16/02/2010
Date registered
24/02/2010
Date last updated
29/08/2017

Titles & IDs
Public title
Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)
Scientific title
A Phase 3, Multicenter, Randomized, Open-Label, Study of Azacitidine (Vidaza®) Versus Conventional Care Regimens for the Treatment of Older Subjects With Newly Diagnosed Acute Myeloid Leukemia
Secondary ID [1] 0 0
AZA-AML-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Acute Myeloid Leukemia 0 0
Condition category
Condition code
Cancer 0 0 0 0
Leukaemia - Acute leukaemia
Cancer 0 0 0 0
Leukaemia - Chronic leukaemia
Cancer 0 0 0 0
Children's - Leukaemia & Lymphoma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Azacitidine
Treatment: Drugs - Conventional Care Regimen

Experimental: Azacitidine - Azacitidine daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

Active Comparator: Conventional Care Regimen - Conventional Care Regimen


Treatment: Drugs: Azacitidine
75 mg/m^2 subcutaneous (SC) daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity

Treatment: Drugs: Conventional Care Regimen
Physician pre-selects prior to randomization from one of the following:
Intensive chemotherapy (cytarabine 100-200 mg/m^2 continuous intravenous infusion for 7 days + anthracycline IV x 3 days) + Best Supportive Care; induction with up to 2 consolidation cycles
Low-dose cytarabine 20 mg subcutaneous (SC) twice a day (BID) for 10 days, for 28 day cycles + BSC; until disease progression or unacceptable toxicity
Best Supportive Care only; until study end

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Kaplan-Meier Estimates for Overall Survival - Overall Survival was defined as the time from randomization to death from any cause. Overall survival was calculated by the formula: date of death - date of randomization + 1. Participants surviving at the end of the follow-up period or who withdrew consent to follow-up were censored at the date of last contact. Participants who were lost to follow-up were censored at the date last known alive.
Timepoint [1] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [1] 0 0
One-year Overall Survival Rate - Kaplan Meier methods were used to estimate the 1-year survival probabilities for time to death from any cause. Estimates of the 1-year (365 day) survival probabilities and corresponding 95% confidence intervals (CI) were presented by treatment group. The CI for the difference in the 1-year survival probabilities was derived using Greenwoods variance estimate.
Timepoint [1] 0 0
From Day 1 (randomization) to 40 months
Secondary outcome [2] 0 0
Event-free Survival (EFS) - Event-free survival was defined as the interval from the date of randomization to the date of treatment failure, progressive disease, relapse after complete remission (CR) or complete remission with incomplete blood count recovery (CRi), death from any cause, or lost to follow-up, whichever occurs first. Participants who were still alive without any of these events were censored at the date of their last response assessment.
Timepoint [2] 0 0
Day 1 (randomization) to date of treatment failure, progressive disease, relapse after Complete Remission (CR) or Complete remission with incomplete blood count recovery (CRi), death from any cause. Day 1 (randomization) to 40 months
Secondary outcome [3] 0 0
Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi) - Relapse-free survival was defined as the interval from the date of first documented CR or CRi to the date of relapse, death from any cause, or lost to follow-up, whichever occurred first. Participants who were still alive and in continuous CR or CRi were censored at the date of their last response assessment.
Timepoint [3] 0 0
Day 1 of first documented CR or CRi to the date of relapse, death from any cause, or lost to follow-up. Day 1 (randomization) to 40 months
Secondary outcome [4] 0 0
Percentage of Participants Who Achieved a Morphologic CR + CRi as Determined by the Independent Review Committee (IRC) Based on International Working Group (IWG) Response Criteria for Acute Myeloid Leukemia (AML) - A complete remission (CR) is defined as a leukemia-free state defined as less than 5% blasts in a BM aspirate with marrow spicules and with at least 200 nucleated cells (there should be no blasts with Auer rods), an absolute neutrophil count (ANC) of = 1 x 10^9/L, a platelet count = 100 x 10^9/L, and transfusion independence (no transfusions for 1 week prior to each assessment). No duration of these findings is required for confirmation of this response. A CR with incomplete blood count recovery (CRi) is defined as <5% BM blasts with the ANC count < 1 x 10^9/L and/or the platelet count may be < 100 x 10^9/L. Where the date of the hematology assessment used is the earliest on or following the date of the BM sample up to 8 days after the BM date.
Timepoint [4] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [5] 0 0
Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates - The time from the date CR or CRi was first documented until the date of documented relapse from CR/CRi. Duration of remission was defined only for those participants who achieved a CR or CRi, as determined by the IRC. Participants who were lost to follow-up without documented relapse, or were alive at last follow-up without documented relapse were censored at the date of their last response assessment.
Timepoint [5] 0 0
Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse.
Secondary outcome [6] 0 0
Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC. - The CRc is a normal karyotype defined as no clonal abnormalities after review of at least 10 metaphases using conventional cytogenetic techniques. Cytogenetic complete remission rate (CRc) is when the following criteria are met: 1) CR criteria met and 2) an abnormal karyotype is present at baseline and 3) there is reversion to normal karyotype at the time of CR (based on = 10 metaphases), where date of cytogenetic sample = date of BM sample used for the CR assessment
Timepoint [6] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [7] 0 0
Number of Participants With Adverse Events (AEs) - AEs = any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the participant's health, regardless of cause. Serious AE (SAE) = any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. The severity of AEs were graded based upon the participants symptoms according to the Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0); AEs were evaluated for severity according to the following scale: Grade 1 = Mild - transient or mild discomfort; no medical intervention required; Grade 2 = Moderate - mild to moderate limitation in activity; Grade 3 = Severe; Grade 4 = Life threatening; Grade 5 = Death
Timepoint [7] 0 0
Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017
Secondary outcome [8] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [8] 0 0
Baseline to Cycle 3; at approximately 3 months
Secondary outcome [9] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [9] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [10] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [10] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [11] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [11] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [12] 0 0
Health Related Quality of Life (HRQoL): Change From Baseline in the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire for Patients With Cancer (EORTC QLQ-C30) Fatigue Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Fatigue Scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate reduction in fatigue (i.e. improvement in symptom) and positive values indicate increases in fatigue (i.e. worsening of symptom).
Timepoint [12] 0 0
Baseline to End of Study; at approximately 11-12 months
Secondary outcome [13] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Timepoint [13] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [14] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Timepoint [14] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [15] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Timepoint [15] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [16] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Timepoint [16] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [17] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Dyspnea scale is scored between 0 and 100, with a high score indicating a higher level of symptoms. Negative change from Baseline values indicate decreased dyspnea (i.e. improvement in symptom) and positive values indicate increased dyspnea (i.e. worsening of symptom).
Timepoint [17] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [18] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [18] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [19] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [19] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [20] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [20] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [21] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [21] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [22] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 PhysicalFunctioning Scale is scored between 0 and 100, with a high score indicating better functioning. Negative change from Baseline values indicate deterioration in functioning and positive values indicate improvement.
Timepoint [22] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [23] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Timepoint [23] 0 0
Baseline to Cycle 3, at approximately 3 months
Secondary outcome [24] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Timepoint [24] 0 0
Baseline to Cycle 5, at approximately 5 months
Secondary outcome [25] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Timepoint [25] 0 0
Baseline to Cycle 7, at approximately 7 months
Secondary outcome [26] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Timepoint [26] 0 0
Baseline to Cycle 9, at approximately 9 months
Secondary outcome [27] 0 0
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain - The European Organization for Research and Treatment of Cancer (EORTC) Core Quality of Life (QOL) questionnaire (EORTC QLQ-C30) is a 30-question tool used to assess the overall quality of life in cancer patients. It consists of 15 domains: 1 global health status (GHS) scale, 5 functional scales (Physical, Role, Cognitive, Emotional, Social), and 9 symptom scales/items (Fatigue, Nausea and Vomiting, Pain, Dyspnea, Sleep Disturbance, Appetite Loss, Constipation, Diarrhea, Financial Impact). The EORTC QLQ-C30 Global Health Status/QOL scale is scored between 0 and 100, with a high score indicating better Global Health Status/QOL. Negative change from Baseline values indicate deterioration in Global Health Status/QOL and positive values indicate improvement.
Timepoint [27] 0 0
Baseline to end of study, at approximately 11-12 months
Secondary outcome [28] 0 0
Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations - HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective.
Timepoint [28] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [29] 0 0
Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Per Year - HRU was defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective. The rate of inpatient hospitalizations per patient year was calculated as the total number of hospitalizations divided by the total number of patient-years followed in the study period. Patient-years (PY) were calculated as the duration from baseline to last available HRQL assessment for each patient.
Timepoint [29] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [30] 0 0
HRU: Number of Participants Receiving Transfusions - Count of study participants who had transfusions during the treatment phase. HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective.
Timepoint [30] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [31] 0 0
HRU: Rate of Transfusions Per Patient Year - HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU Analysis may help in evaluating potential costs and budget impact of new treatments from a payer perspective. The rate of transfusions per patient year was calculated as the total number of transfusions divided by the total number of patient-years followed in the study period. Patient-years (PY) were calculated as the duration from baseline to last available HRQL assessment for each patient.
Timepoint [31] 0 0
Day 1 (randomization) to 40 months
Secondary outcome [32] 0 0
Number of Participants in the Extension Phase With Treatment Emergent Adverse Events (TEAEs) - AEs = any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. It may be a new intercurrent illness, a worsening concomitant illness, an injury, or any concomitant impairment of the participant's health, regardless of cause. Serious AE (SAE) = any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. The severity of AEs were graded based upon the participants symptoms according to the Common Terminology Criteria for Adverse Events (CTCAE, Version 4.0); AEs were evaluated for severity according to the following scale: Grade 1 = Mild - transient or mild discomfort; no medical intervention required; Grade 2 = Moderate - mild to moderate limitation in activity; Grade 3 = Severe; Grade 4 = Life threatening; Grade 5 = Death
Timepoint [32] 0 0
From the date of informed consent for the Extension Phase through to the date of last dose of study drug + 28 days up to last visit completed 24 July 2016; maximum duration of exposure to Azacitidine was 871 days

Eligibility
Key inclusion criteria
- Diagnosis of one of the following

- Newly diagnosed de novo acute myeloid leukemia (AML)

- AML secondary to myelodysplastic syndromes (MDS)

- AML secondary to exposure to leukemogenic therapy or agents with primary
malignancy in remission for at least 2 years

- Bone marrow blasts >30%

- Age = 65 years

- Easter Cooperative Oncology Group (ECOG) 0-2
Minimum age
65 Years
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
- Previous cytotoxic or biologic treatment for AML (except hydroxyurea)

- Previous treatment with azacitidine, decitabine or cytarabine

- Prior use of targeted therapy agents (e.g., FLT3 inhibitors, other kinase inhibitors)

- AML French American British subtype (FAB M3)

- AML associated with inv(16), t(8;21), t(16;16), t(15:17), or t(9;22) karyotypes

- Prior bone marrow or stem cell transplantation

- Candidate for allogeneic bone marrow or stem cell transplant

- Diagnosis of malignant disease within the previous 12 months (excluding base cell
carcinoma, "in-situ" carcinoma of the cervix or breast or other local malignancy
excised or irradiated with a high probability of cure)

- Malignant hepatic tumors

- Uncontrolled systemic infection

- Active viral infection with Human Immunodeficiency Virus (HIV) or Hepatitis type B or
C

- Use of any experimental drug or therapy within 28 days prior to Day 1

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,SA,VIC
Recruitment hospital [1] 0 0
Prince of Wales Hospital - Randwick
Recruitment hospital [2] 0 0
Royal Adelaide Hospital - Adelaide
Recruitment hospital [3] 0 0
Peter MacCallum Cancer Centre - East Melbourne
Recruitment hospital [4] 0 0
Western Hospital - Footscray
Recruitment hospital [5] 0 0
Royal Melbourne Hospital - Melbourne
Recruitment hospital [6] 0 0
St Vincent's Hospital - Fitzroy
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment postcode(s) [2] 0 0
5000 - Adelaide
Recruitment postcode(s) [3] 0 0
3002 - East Melbourne
Recruitment postcode(s) [4] 0 0
3011 - Footscray
Recruitment postcode(s) [5] 0 0
3050 - Melbourne
Recruitment postcode(s) [6] 0 0
3065 - Fitzroy
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Massachusetts
Country [2] 0 0
United States of America
State/province [2] 0 0
Texas
Country [3] 0 0
Austria
State/province [3] 0 0
Upper Austria
Country [4] 0 0
Austria
State/province [4] 0 0
Vienna
Country [5] 0 0
Austria
State/province [5] 0 0
Salzburg
Country [6] 0 0
Belgium
State/province [6] 0 0
Hainaut
Country [7] 0 0
Belgium
State/province [7] 0 0
Namur
Country [8] 0 0
Belgium
State/province [8] 0 0
Oost-vlaanderen
Country [9] 0 0
Belgium
State/province [9] 0 0
West-vlaanderen
Country [10] 0 0
Belgium
State/province [10] 0 0
La Louvière
Country [11] 0 0
Canada
State/province [11] 0 0
Alberta
Country [12] 0 0
Canada
State/province [12] 0 0
Manitoba
Country [13] 0 0
Canada
State/province [13] 0 0
Nova Scotia
Country [14] 0 0
Canada
State/province [14] 0 0
Ontario
Country [15] 0 0
Canada
State/province [15] 0 0
Quebec
Country [16] 0 0
Canada
State/province [16] 0 0
Calgary
Country [17] 0 0
China
State/province [17] 0 0
Beijing
Country [18] 0 0
China
State/province [18] 0 0
Jiangsu
Country [19] 0 0
China
State/province [19] 0 0
Shanghai
Country [20] 0 0
China
State/province [20] 0 0
Sichuan
Country [21] 0 0
China
State/province [21] 0 0
Tianjin
Country [22] 0 0
Czechia
State/province [22] 0 0
Jihormoravsky Kraj
Country [23] 0 0
Czechia
State/province [23] 0 0
Olomoucký Kraj
Country [24] 0 0
Czechia
State/province [24] 0 0
Praha
Country [25] 0 0
France
State/province [25] 0 0
Alsace
Country [26] 0 0
France
State/province [26] 0 0
ILE-DE-France
Country [27] 0 0
France
State/province [27] 0 0
Ile-de-france
Country [28] 0 0
France
State/province [28] 0 0
Limousin Lorraine
Country [29] 0 0
France
State/province [29] 0 0
Midi-pyrénées
Country [30] 0 0
France
State/province [30] 0 0
Nice
Country [31] 0 0
France
State/province [31] 0 0
Pays de La Loire
Country [32] 0 0
France
State/province [32] 0 0
Picardie
Country [33] 0 0
France
State/province [33] 0 0
Provence Alpes Cote D'azur
Country [34] 0 0
France
State/province [34] 0 0
Aquitaine
Country [35] 0 0
France
State/province [35] 0 0
Lyon Cedex 03
Country [36] 0 0
Germany
State/province [36] 0 0
Baden-wuerttemberg
Country [37] 0 0
Germany
State/province [37] 0 0
Mecklenburg-vorpommern
Country [38] 0 0
Germany
State/province [38] 0 0
Nordrhein-westfalen
Country [39] 0 0
Germany
State/province [39] 0 0
Nordrhein-Westfallen
Country [40] 0 0
Germany
State/province [40] 0 0
Sachsen
Country [41] 0 0
Germany
State/province [41] 0 0
Thueringen
Country [42] 0 0
Israel
State/province [42] 0 0
Beersheva
Country [43] 0 0
Israel
State/province [43] 0 0
Beer Yaakov
Country [44] 0 0
Israel
State/province [44] 0 0
Jerusalem
Country [45] 0 0
Israel
State/province [45] 0 0
Petach Tikva
Country [46] 0 0
Israel
State/province [46] 0 0
Tel Aviv
Country [47] 0 0
Israel
State/province [47] 0 0
Tel Hashomer
Country [48] 0 0
Italy
State/province [48] 0 0
Potenza
Country [49] 0 0
Italy
State/province [49] 0 0
Turin
Country [50] 0 0
Italy
State/province [50] 0 0
Alessandria
Country [51] 0 0
Italy
State/province [51] 0 0
Ancona
Country [52] 0 0
Italy
State/province [52] 0 0
Bari
Country [53] 0 0
Italy
State/province [53] 0 0
Bologna
Country [54] 0 0
Italy
State/province [54] 0 0
Firenze
Country [55] 0 0
Italy
State/province [55] 0 0
Reggio Calabria
Country [56] 0 0
Italy
State/province [56] 0 0
Roma
Country [57] 0 0
Italy
State/province [57] 0 0
Udine
Country [58] 0 0
Italy
State/province [58] 0 0
Varese
Country [59] 0 0
Korea, Republic of
State/province [59] 0 0
Seoul
Country [60] 0 0
Korea, Republic of
State/province [60] 0 0
Daegu
Country [61] 0 0
Netherlands
State/province [61] 0 0
Groningen
Country [62] 0 0
Poland
State/province [62] 0 0
Dolnoslaskie
Country [63] 0 0
Poland
State/province [63] 0 0
Lodzkie
Country [64] 0 0
Poland
State/province [64] 0 0
Mazowieckie
Country [65] 0 0
Poland
State/province [65] 0 0
Slaskie
Country [66] 0 0
Russian Federation
State/province [66] 0 0
Ekaterinburg
Country [67] 0 0
Russian Federation
State/province [67] 0 0
Moscow
Country [68] 0 0
Russian Federation
State/province [68] 0 0
Nizhniy Novgorod
Country [69] 0 0
Russian Federation
State/province [69] 0 0
Saint Petersburg
Country [70] 0 0
Russian Federation
State/province [70] 0 0
Saratov
Country [71] 0 0
Spain
State/province [71] 0 0
Asturias
Country [72] 0 0
Spain
State/province [72] 0 0
Baleares
Country [73] 0 0
Spain
State/province [73] 0 0
Barcelona
Country [74] 0 0
Spain
State/province [74] 0 0
Madrid
Country [75] 0 0
Spain
State/province [75] 0 0
Salamanca
Country [76] 0 0
Spain
State/province [76] 0 0
Sevilla
Country [77] 0 0
Spain
State/province [77] 0 0
Valencia
Country [78] 0 0
Taiwan
State/province [78] 0 0
Kaohsiung
Country [79] 0 0
Taiwan
State/province [79] 0 0
Taipei
Country [80] 0 0
United Kingdom
State/province [80] 0 0
Surrey
Country [81] 0 0
United Kingdom
State/province [81] 0 0
Bournemouth
Country [82] 0 0
United Kingdom
State/province [82] 0 0
London
Country [83] 0 0
United Kingdom
State/province [83] 0 0
Manchester
Country [84] 0 0
United Kingdom
State/province [84] 0 0
Oxford
Country [85] 0 0
United Kingdom
State/province [85] 0 0
Wolverhampton

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Celgene
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
The purpose of this study is to compare the effect of azacitidine (Vidaza) to conventional
care regimens on overall survival in elderly AML patients.
Trial website
https://clinicaltrials.gov/show/NCT01074047
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
C L Beach, PharmD
Address 0 0
Celgene Corporation
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Summary results
Other publications