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Trial details imported from ClinicalTrials.gov

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

AZA-AML-001

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Acute Myeloid Leukemia

Condition category

Condition code

Cancer

Leukaemia - Acute leukaemia

Cancer

Leukaemia - Chronic leukaemia

Cancer

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]

Treatment: Drugs

Comparator / control treatment

Control group

Outcomes

Primary outcome [1]

Kaplan-Meier Estimates for Overall Survival

Assessment method [1]

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]

Day 1 (randomization) to 40 months

Secondary outcome [1]

One-year Overall Survival Rate

Assessment method [1]

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]

From Day 1 (randomization) to 40 months

Secondary outcome [2]

Event-free Survival (EFS)

Assessment method [2]

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]

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]

Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi)

Assessment method [3]

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]

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]

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)

Assessment method [4]

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]

Day 1 (randomization) to 40 months

Secondary outcome [5]

Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates

Assessment method [5]

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]

Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse.

Secondary outcome [6]

Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC.

Assessment method [6]

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]

Day 1 (randomization) to 40 months

Secondary outcome [7]

Number of Participants With Adverse Events (AEs)

Assessment method [7]

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]

Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017

Secondary outcome [8]

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

Assessment method [8]

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]

Baseline to Cycle 3; at approximately 3 months

Secondary outcome [9]

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

Assessment method [9]

Timepoint [9]

Baseline to Cycle 5, at approximately 5 months

Secondary outcome [10]

Assessment method [10]

Timepoint [10]

Baseline to Cycle 7, at approximately 7 months

Secondary outcome [11]

Assessment method [11]

Timepoint [11]

Baseline to Cycle 9, at approximately 9 months

Secondary outcome [12]

Assessment method [12]

Timepoint [12]

Baseline to End of Study; at approximately 11-12 months

Secondary outcome [13]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

Assessment method [13]

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]

Baseline to Cycle 3, at approximately 3 months

Secondary outcome [14]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

Assessment method [14]

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]

Baseline to Cycle 5, at approximately 5 months

Secondary outcome [15]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

Assessment method [15]

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]

Baseline to Cycle 7, at approximately 7 months

Secondary outcome [16]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

Assessment method [16]

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]

Baseline to Cycle 9, at approximately 9 months

Secondary outcome [17]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea

Assessment method [17]

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]

Baseline to end of study, at approximately 11-12 months

Secondary outcome [18]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

Assessment method [18]

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]

Baseline to Cycle 3, at approximately 3 months

Secondary outcome [19]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

Assessment method [19]

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]

Baseline to Cycle 5, at approximately 5 months

Secondary outcome [20]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

Assessment method [20]

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]

Baseline to Cycle 7, at approximately 7 months

Secondary outcome [21]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

Assessment method [21]

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]

Baseline to Cycle 9, at approximately 9 months

Secondary outcome [22]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain

Assessment method [22]

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]

Baseline to end of study, at approximately 11-12 months

Secondary outcome [23]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

Assessment method [23]

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]

Baseline to Cycle 3, at approximately 3 months

Secondary outcome [24]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

Assessment method [24]

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]

Baseline to Cycle 5, at approximately 5 months

Secondary outcome [25]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

Assessment method [25]

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]

Baseline to Cycle 7, at approximately 7 months

Secondary outcome [26]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

Assessment method [26]

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]

Baseline to Cycle 9, at approximately 9 months

Secondary outcome [27]

HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain

Assessment method [27]

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]

Baseline to end of study, at approximately 11-12 months

Secondary outcome [28]

Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations

Assessment method [28]

Timepoint [28]

Day 1 (randomization) to 40 months

Secondary outcome [29]

Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Per Year

Assessment method [29]

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]

Day 1 (randomization) to 40 months

Secondary outcome [30]

HRU: Number of Participants Receiving Transfusions

Assessment method [30]

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]

Day 1 (randomization) to 40 months

Secondary outcome [31]

HRU: Rate of Transfusions Per Patient Year

Assessment method [31]

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]

Day 1 (randomization) to 40 months

Secondary outcome [32]

Number of Participants in the Extension Phase With Treatment Emergent Adverse Events (TEAEs)

Assessment method [32]

Timepoint [32]

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

Sex

Both males and females

Can healthy volunteers participate?

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

1/06/2010

Date of last participant enrolment

Anticipated

Actual

Date of last data collection

Anticipated

Actual

25/07/2016

Sample size

Target

Accrual to date

Final

488

Recruitment in Australia

Recruitment state(s)

NSW,SA,VIC

Recruitment hospital [1]

Prince of Wales Hospital - Randwick

Recruitment hospital [2]

Royal Adelaide Hospital - Adelaide

Recruitment hospital [3]

Peter MacCallum Cancer Centre - East Melbourne

Recruitment hospital [4]

Western Hospital - Footscray

Recruitment hospital [5]

Royal Melbourne Hospital - Melbourne

Recruitment hospital [6]

St Vincent's Hospital - Fitzroy

Recruitment postcode(s) [1]

2031 - Randwick

Recruitment postcode(s) [2]

5000 - Adelaide

Recruitment postcode(s) [3]

3002 - East Melbourne

Recruitment postcode(s) [4]

3011 - Footscray

Recruitment postcode(s) [5]

3050 - Melbourne

Recruitment postcode(s) [6]

3065 - Fitzroy

Recruitment outside Australia

Country [1]

United States of America

State/province [1]

Massachusetts

Country [2]

United States of America

State/province [2]

Texas

Country [3]

Austria

State/province [3]

Upper Austria

Country [4]

Austria

State/province [4]

Vienna

Country [5]

Austria

State/province [5]

Salzburg

Country [6]

Belgium

State/province [6]

Hainaut

Country [7]

Belgium

State/province [7]

Namur

Country [8]

Belgium

State/province [8]

Oost-vlaanderen

Country [9]

Belgium

State/province [9]

West-vlaanderen

Country [10]

Belgium

State/province [10]

La Louvière

Country [11]

Canada

State/province [11]

Alberta

Country [12]

Canada

State/province [12]

Manitoba

Country [13]

Canada

State/province [13]

Nova Scotia

Country [14]

Canada

State/province [14]

Ontario

Country [15]

Canada

State/province [15]

Quebec

Country [16]

Canada

State/province [16]

Calgary

Country [17]

China

State/province [17]

Beijing

Country [18]

China

State/province [18]

Jiangsu

Country [19]

China

State/province [19]

Shanghai

Country [20]

China

State/province [20]

Sichuan

Country [21]

China

State/province [21]

Tianjin

Country [22]

Czechia

State/province [22]

Jihormoravsky Kraj

Country [23]

Czechia

State/province [23]

Olomoucký Kraj

Country [24]

Czechia

State/province [24]

Praha

Country [25]

France

State/province [25]

Alsace

Country [26]

France

State/province [26]

ILE-DE-France

Country [27]

France

State/province [27]

Ile-de-france

Country [28]

France

State/province [28]

Limousin Lorraine

Country [29]

France

State/province [29]

Midi-pyrénées

Country [30]

France

State/province [30]

Nice

Country [31]

France

State/province [31]

Pays de La Loire

Country [32]

France

State/province [32]

Picardie

Country [33]

France

State/province [33]

Provence Alpes Cote D'azur

Country [34]

France

State/province [34]

Aquitaine

Country [35]

France

State/province [35]

Lyon Cedex 03

Country [36]

Germany

State/province [36]

Baden-wuerttemberg

Country [37]

Germany

State/province [37]

Mecklenburg-vorpommern

Country [38]

Germany

State/province [38]

Nordrhein-westfalen

Country [39]

Germany

State/province [39]

Nordrhein-Westfallen

Country [40]

Germany

State/province [40]

Sachsen

Country [41]

Germany

State/province [41]

Thueringen

Country [42]

Israel

State/province [42]

Beersheva

Country [43]

Israel

State/province [43]

Beer Yaakov

Country [44]

Israel

State/province [44]

Jerusalem

Country [45]

Israel

State/province [45]

Petach Tikva

Country [46]

Israel

State/province [46]

Tel Aviv

Country [47]

Israel

State/province [47]

Tel Hashomer

Country [48]

Italy

State/province [48]

Potenza

Country [49]

Italy

State/province [49]

Turin

Country [50]

Italy

State/province [50]

Alessandria

Country [51]

Italy

State/province [51]

Ancona

Country [52]

Italy

State/province [52]

Bari

Country [53]

Italy

State/province [53]

Bologna

Country [54]

Italy

State/province [54]

Firenze

Country [55]

Italy

State/province [55]

Reggio Calabria

Country [56]

Italy

State/province [56]

Roma

Country [57]

Italy

State/province [57]

Udine

Country [58]

Italy

State/province [58]

Varese

Country [59]

Korea, Republic of

State/province [59]

Seoul

Country [60]

Korea, Republic of

State/province [60]

Daegu

Country [61]

Netherlands

State/province [61]

Groningen

Country [62]

Poland

State/province [62]

Dolnoslaskie

Country [63]

Poland

State/province [63]

Lodzkie

Country [64]

Poland

State/province [64]

Mazowieckie

Country [65]

Poland

State/province [65]

Slaskie

Country [66]

Russian Federation

State/province [66]

Ekaterinburg

Country [67]

Russian Federation

State/province [67]

Moscow

Country [68]

Russian Federation

State/province [68]

Nizhniy Novgorod

Country [69]

Russian Federation

State/province [69]

Saint Petersburg

Country [70]

Russian Federation

State/province [70]

Saratov

Country [71]

Spain

State/province [71]

Asturias

Country [72]

Spain

State/province [72]

Baleares

Country [73]

Spain

State/province [73]

Barcelona

Country [74]

Spain

State/province [74]

Madrid

Country [75]

Spain

State/province [75]

Salamanca

Country [76]

Spain

State/province [76]

Sevilla

Country [77]

Spain

State/province [77]

Valencia

Country [78]

Taiwan

State/province [78]

Kaohsiung

Country [79]

Taiwan

State/province [79]

Taipei

Country [80]

United Kingdom

State/province [80]

Surrey

Country [81]

United Kingdom

State/province [81]

Bournemouth

Country [82]

United Kingdom

State/province [82]

London

Country [83]

United Kingdom

State/province [83]

Manchester

Country [84]

United Kingdom

State/province [84]

Oxford

Country [85]

United Kingdom

State/province [85]

Wolverhampton

Funding & Sponsors

Primary sponsor type

Commercial sector/industry

Name

Celgene

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/study/NCT01074047

Trial related presentations / publications

Seymour JF, Dohner H, Butrym A, Wierzbowska A, Selleslag D, Jang JH, Kumar R, Cavenagh J, Schuh AC, Candoni A, Recher C, Sandhu I, Del Castillo TB, Al-Ali HK, Falantes J, Stone RM, Minden MD, Weaver J, Songer S, Beach CL, Dombret H. Azacitidine improves clinical outcomes in older patients with acute myeloid leukaemia with myelodysplasia-related changes compared with conventional care regimens. BMC Cancer. 2017 Dec 14;17(1):852. doi: 10.1186/s12885-017-3803-6.
Dombret H, Seymour JF, Butrym A, Wierzbowska A, Selleslag D, Jang JH, Kumar R, Cavenagh J, Schuh AC, Candoni A, Recher C, Sandhu I, Bernal del Castillo T, Al-Ali HK, Martinelli G, Falantes J, Noppeney R, Stone RM, Minden MD, McIntyre H, Songer S, Lucy LM, Beach CL, Dohner H. International phase 3 study of azacitidine vs conventional care regimens in older patients with newly diagnosed AML with >30% blasts. Blood. 2015 Jul 16;126(3):291-9. doi: 10.1182/blood-2015-01-621664. Epub 2015 May 18.

Public notes

Contacts

Principal investigator

Name

C L Beach, PharmD

Address

Celgene Corporation

Country

Phone

Contact person for public queries

Name

Address

Country

Phone

Contact person for scientific queries

No information has been provided regarding IPD availability

What supporting documents are/will be available?

No Supporting Document Provided

Results publications and other study-related documents

No documents have been uploaded by study researchers.

Results are available at https://clinicaltrials.gov/study/NCT01074047

Trial Review

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