Trial · NCT01074047
Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)
NCT01074047, Phase 3, Completed, Celgene; results first posted 2015-02-26
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML). Trials, record NCT01074047. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01074047
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Vidaza Versus Conventional Care Regimens for the Treatment of Acute Myeloid Leukemia (AML)
- NCT number
- NCT01074047
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 488 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Acute Myeloid Leukemia
- Interventions
- Azacitidine, Conventional Care Regimen
- First posted
- 2010-02-24
- Last updated
- 2017-08-29
- Results first posted
- 2015-02-26
Source ClinicalTrials.gov
Registered outcomes33
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Kaplan-Meier Estimates for Overall Survival | Day 1 (randomization) to 40 months | 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. |
| Secondary | One-year Overall Survival Rate | From Day 1 (randomization) to 40 months | 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. |
| Secondary | Event-free Survival (EFS) | 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 | 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. |
| Secondary | Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | 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 | 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. |
| Secondary | 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) | Day 1 (randomization) to 40 months | 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. |
| Secondary | Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates | Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse. | 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. |
| Secondary | Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC. | Day 1 (randomization) to 40 months | 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 |
| Secondary | Number of Participants With Adverse Events (AEs) | Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017 | 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 |
| Secondary | 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 | Baseline to Cycle 3; at approximately 3 months | 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). |
| Secondary | 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 | Baseline to Cycle 5, at approximately 5 months | 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). |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 registered outcomesOutcomes33
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Kaplan-Meier Estimates for Overall Survival | Day 1 (randomization) to 40 months | The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored | months | MEDIAN |
| SECONDARY | One-year Overall Survival Rate | From Day 1 (randomization) to 40 months | The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored | percentage of participants | NUMBER |
| SECONDARY | Event-free Survival (EFS) | 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 | The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored | months | MEDIAN |
| SECONDARY | Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | 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 | Participants who achieved a CR or CRi | months | MEDIAN |
| SECONDARY | 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) | Day 1 (randomization) to 40 months | The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored | percentage of participants | NUMBER |
| SECONDARY | Duration of Remission Assessed by the IRC Based on Kaplan-Meier Estimates | Day 1 (randomization) to 40 months; date of the first documented CR or CRi until date of first documented relapse. | Includes those who achieved a CR or CRi and assessed by the IRC; numbers of ITT participants in each treatment group | months | MEDIAN |
| SECONDARY | Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC. | Day 1 (randomization) to 40 months | The intent-to-treat (ITT) population was defined as all participants who were randomized, independent of whether they received study treatment or not. Includes participants who died and participants who were censored | participants | NUMBER |
| SECONDARY | Number of Participants With Adverse Events (AEs) | Day 1 (randomization) up to last visit completed; final data cut off of 28 Feb 2017 | Safety population = all randomized participants who received at least 1 dose of study drug and had 1 post-dose safety assessment. Because the BSC only regimen consisted of blood products or antibiotics given as needed, those assigned to BSC only were included in the safety population if they had at least 1 post-randomization safety assessment. | participants | NUMBER |
| SECONDARY | 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 | Baseline to Cycle 3; at approximately 3 months | The HRQoL Evaluable population included only participants with a baseline QoL assessment and at least 1 follow-up assessment. Time windows were applied post-hoc to increase the size of the analyzable population. | units on a scale | MEAN |
| SECONDARY | 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 | Baseline to Cycle 5, at approximately 5 months | The HRQoL Evaluable population included only participants with a baseline QoL assessment and at least 1 follow-up assessment. Time windows were applied post-hoc to increase the size of the analyzable population. | units on a scale | MEAN |
1 to 10 of 33Source ClinicalTrials.gov
See all 33 outcomesResults by group128
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Kaplan-Meier Estimates for Overall Survival | Azacitidine (AZA) | 10.4 | 95% Confidence Interval 8.0 to 12.7 | months | |
| Kaplan-Meier Estimates for Overall Survival | Conventional Care Regimens (CCR) | 6.5 | 95% Confidence Interval 5.0 to 8.6 | months | |
| One-year Overall Survival Rate | Azacitidine (AZA) | 46.5 | 95% Confidence Interval 40.1 to 52.7 | percentage of participants | |
| One-year Overall Survival Rate | Conventional Care Regimen | 34.3 | 95% Confidence Interval 28.3 to 40.3 | percentage of participants | |
| Event-free Survival (EFS) | Azacitidine (AZA) | 6.7 | 95% Confidence Interval 5.0 to 8.8 | months | |
| Event-free Survival (EFS) | Conventional Care Regimens (CCR) | 4.8 | 95% Confidence Interval 3.8 to 6.0 | months | |
| Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | Azacitidine (AZA) | 9.3 | 95% Confidence Interval 6.7 to 12.4 | months | |
| Relapse-Free Survival (RFS) for Participants Who Achieved a Complete Remission (CR) or Complete Remission With Incomplete Blood Count Recovery (CRi) | Conventional Care Regimen | 10.5 | 95% Confidence Interval 7.3 to 12.3 | months | |
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) | Azacitidine (AZA) | 27.8 | percentage of participants | ||
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) | Conventional Care Regimens (CCR) | 25.1 | percentage of participants |
1 to 10 of 128Source ClinicalTrials.gov
See all 128 results by groupParticipant flow26
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Phase | STARTED | Azacitidine (AZA) | 241 | |
| Treatment Phase | STARTED | Conventional Care Regimens (CCR) | 247 | |
| Treatment Phase | Treated Population | Azacitidine (AZA) | 236 | |
| Treatment Phase | Treated Population | Conventional Care Regimens (CCR) | 240 | |
| Treatment Phase | Safety Population | Azacitidine (AZA) | 236 | |
| Treatment Phase | Safety Population | Conventional Care Regimens (CCR) | 235 | |
| Treatment Phase | Evaluable Population | Azacitidine (AZA) | 179 | |
| Treatment Phase | Evaluable Population | Conventional Care Regimens (CCR) | 191 | |
| Treatment Phase | COMPLETED | Azacitidine (AZA) | 24 | |
| Treatment Phase | COMPLETED | Conventional Care Regimens (CCR) | 13 |
1 to 10 of 26Source ClinicalTrials.gov
See all 26 participant flowAdverse events1,755
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | ANAEMIA | Blood and lymphatic system disorders | Azacitidine | 41 | 236 | |
| other | ANAEMIA | Blood and lymphatic system disorders | BSC Only | 3 | 40 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Low-dose Cytarabine | 32 | 153 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Intensive Chemotherapy | 7 | 42 | |
| other | ANAEMIA | Blood and lymphatic system disorders | Azacitidine-extension | 4 | 22 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Azacitidine | 28 | 236 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | BSC Only | 2 | 40 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Low-dose Cytarabine | 21 | 153 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Intensive Chemotherapy | 12 | 42 | |
| other | FEBRILE NEUTROPENIA | Blood and lymphatic system disorders | Azacitidine-extension | 0 | 22 |
1 to 10 of 1,755Source ClinicalTrials.gov
See all 1,755 adverse eventsAdverse event totals15
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Azacitidine | ||
| Total, all-cause mortality | BSC Only | ||
| Total, all-cause mortality | Low-dose Cytarabine | ||
| Total, all-cause mortality | Intensive Chemotherapy | ||
| Total, all-cause mortality | Azacitidine-extension | ||
| Total, other adverse events | Azacitidine | 226 | 236 |
| Total, other adverse events | BSC Only | 33 | 40 |
| Total, other adverse events | Low-dose Cytarabine | 151 | 153 |
| Total, other adverse events | Intensive Chemotherapy | 42 | 42 |
| Total, other adverse events | Azacitidine-extension | 18 | 22 |
1 to 10 of 15Source ClinicalTrials.gov
See all 15 adverse event totalsPapers2
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons3
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Azacitidine (AZA) vs Conventional Care Regimens (CCR): Healthcare Resource Utilization (HRU): Number of Inpatient Hospitalizations | Trial comparisons |
| Azacitidine (AZA) vs Conventional Care Regimens (CCR): Number of Participants Who Achieved a Cytogenetic Complete Response (CRc-10) as Determined by the IRC. | Trial comparisons |
| Azacitidine (AZA) vs Conventional Care Regimen: HRU: Number of Participants Receiving Transfusions | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
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Read this trial from software.
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