TriangleNCT01074047 · CITED IN SOURCE DOCUMENTS
Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)
An NCI or FDA source cites this study. A citation does not establish that it applies to an individual diagnosis.
- Phase
- PHASE3
- Status at capture
- COMPLETED
- Registry last update
- 2017-08-29
The purpose of this study is to compare the effect of azacitidine (Vidaza) to conventional care regimens on overall survival in elderly AML patients.
Open the original ClinicalTrials.gov record → · Download preserved record
What did the study report?
Outcomes, safety and baseline populations are separate source sections. Quality-of-life measures appear under their original outcome titles.
Participant flow
Baseline characteristics
Kaplan-Meier Estimates for Overall Survival
One-year Overall Survival Rate
Event-free Survival (EFS)
Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi)
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)
Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates
Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC.
Number of Participants With Adverse Events (AEs)
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
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
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
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
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
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
HRQoL: Change From Baseline in the EORTC QLQ-C30 Dyspnea
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Physical Functioning Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
HRQoL: Change From Baseline in the EORTC QLQ-C30 Global Health Status-/Quality of Life Domain
Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations
Healthcare Resource Utilization (HRU): Rate of Inpatient Hospitalizations Per Year
HRU: Number of Participants Receiving Transfusions
HRU: Rate of Transfusions Per Patient Year
Number of Participants in the Extension Phase With Treatment Emergent Adverse Events (TEAEs)
Adverse events
Limitations, agreements, and source contact
Who could take part
- eligibility Criteria
- 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 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
- healthy Volunteers
- false
- minimum Age
- 65 Years
- sex
- ALL
- std Ages
- OLDER_ADULT
Treatment arms and interventions
- arm Groups
- description
- Azacitidine daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity
- intervention Names
- Drug: Azacitidine
- label
- Azacitidine
- type
- EXPERIMENTAL
- description
- Conventional Care Regimen
- intervention Names
- Drug: Conventional Care Regimen
- label
- Conventional Care Regimen
- type
- ACTIVE_COMPARATOR
- interventions
- arm Group Labels
- Azacitidine
- description
- 75 mg/m\^2 subcutaneous (SC) daily for 7 days for 28 day cycles until disease progression or unacceptable toxicity
- name
- Azacitidine
- other Names
- Vidaza
- type
- DRUG
- arm Group Labels
- Conventional Care Regimen
- description
- 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
- name
- Conventional Care Regimen
- type
- DRUG
Study design
- allocation
- RANDOMIZED
- intervention Model
- PARALLEL
- masking Info
- masking
- NONE
- primary Purpose
- TREATMENT
Enrollment
- count
- 488
- type
- ACTUAL
Registered outcome plans (not posted results)
- primary Outcomes
- description
- 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.
- measure
- Kaplan-Meier Estimates for Overall Survival
- time Frame
- Day 1 (randomization) to 40 months
- secondary Outcomes
- description
- 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.
- measure
- One-year Overall Survival Rate
- time Frame
- From Day 1 (randomization) to 40 months
- description
- 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.
- measure
- Event-free Survival (EFS)
- time Frame
- 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
- description
- 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.
- measure
- Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi)
- time Frame
- 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
- description
- 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.
- measure
- 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)
- time Frame
- Day 1 (randomization) to 40 months
- description
- 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.
- measure
- Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates
- time Frame
- Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse.
- description
- 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
- measure
- Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC.
- time Frame
- Day 1 (randomization) to 40 months
- description
- 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
- measure
- Number of Participants With Adverse Events (AEs)
- time Frame
- Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017
- description
- 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).
- measure
- 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
- time Frame
- Baseline to Cycle 3; at approximately 3 months
- description
- 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).
- measure
- 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
- time Frame
- Baseline to Cycle 5, at approximately 5 months
- description
- 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).
- measure
- 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
- time Frame
- Baseline to Cycle 7, at approximately 7 months
- description
- 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).
- measure
- 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
- time Frame
- Baseline to Cycle 9, at approximately 9 months
- description
- 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).
- measure
- 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
- time Frame
- Baseline to End of Study; at approximately 11-12 months
Full study description
- 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.
Source references
- references
- citation
- 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.
- pmid
- 29241450
- type
- DERIVED
- citation
- 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.
- pmid
- 25987659
- type
- DERIVED
Source notices and limitations
Discovery and provenance
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- Acute Myeloid Leukemia Treatment (PDQ®)–Health Professional Version
- source update dates
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- Updated: March 14, 2025
- datetime
- 2025-03-14T12:00:00Z
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- March 14, 2025
Preserved source evidence · Not medical advice