Trial · NCT01757535
Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission
NCT01757535, Phase 3, Completed, Celgene; results first posted 2020-11-06
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission. Trials, record NCT01757535. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01757535
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission
- NCT number
- NCT01757535
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 472 participants, actual
- Lead sponsor
- Celgene
- Conditions
- Leukemia, Myeloid, Acute
- Interventions
- Oral Azacitidine, Placebo
- First posted
- 2012-12-31
- Last updated
- 2025-07-08
- Other registries' IDs
- eudract:2012-003457-28
- Results first posted
- 2020-11-06
Source ClinicalTrials.gov
Registered outcomes10
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Kaplan-Meier (K-M) Estimate for Overall Survival (OS) | Day 1 (randomization) up to data cut off date of 15 July 2019; median follow-up for OS estimated by the reverse K-M method was 41.2 months for all participants. | Overall survival was defined as time from randomization to death from any cause; participants surviving at the end of the follow-up period, or who withdraw consent, or who were lost to follow up were censored at the date last known alive. |
| Secondary | Kaplan-Meier Estimate of Relapse Free Survival (RFS) | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | RFS was defined as the time from the date of randomization to the date of documented relapse or death from any cause, whichever occurred first. Participants who were still alive without documented relapse, or who were lost to follow-up or withdrew consent without documented relapse, were censored at the date of their last bone marrow assessment, prior to receiving any other therapy for AML. Documented relapse was defined as the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of \> 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast \[myeloblasts\] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days. |
| Secondary | Kaplan-Meier Estimate of Time to Relapse | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | Time to relapse was defined as the interval (in months) from the date of randomization to the date of documented relapse. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from complete remission (CR)/ complete remission with incomplete blood count recovery (CRi). Documented relapse was defined as, the earliest date of the following: • ≥ 5% bone marrow blasts (myeloblasts) from Central Pathology report, or • appearance of \> 0% blasts in the peripheral blood with a later bone marrow confirmation (bone marrow blast \[myeloblasts\] ≥ 5%) within 100 days, or • at least 2 peripheral blasts ≥ 5% within 30 days. |
| Secondary | Kaplan-Meier Estimates of Time to Discontinuation From Treatment | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | Time to discontinuation from treatment was assessed and defined as the interval from the date of randomization to the date of discontinuation from study drug. Participants who were receiving treatment at the time of study closure were censored at the date of last visit. Estimates of relapse rate were based on the cumulative incidence function from a competing risk analysis with death as a competing risk for relapse from CR/ CRi. |
| Secondary | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | TEAEs include AEs that started between first dose date and 28 days after the last dose of study drug. A serious adverse event (SAE) is: • Death • Life-threatening event • Inpatient hospitalization or prolongation of existing hospitalization • Persistent or significant disability or incapacity • Congenital anomaly or birth defect • Other important medical event The severity of AEs were assessed by the investigator according to the Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0: Grade 1 (Mild): asymptomatic/mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 (Moderate): minimal, local or noninvasive intervention indicated; limiting age-appropriate activities of daily living. Grade 3: Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care Grade 4: Life-threatening; urgent intervention indicated. Grade 5: Death due to AE. |
| Secondary | Mean Change in the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0) Score From Baseline | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The functional assessment of chronic illness therapy (FACIT-Fatigue Scale V 4.0) is a subscale of the FACIT-F and has been validated in the oncology setting. The FACIT-Fatigue Scale is a short, 13-item, self-administered tool that measures the level of fatigue in an individual during usually daily activities over the past week. The level of fatigue is measured on a 5-point Likert scale (0 = not at all; 4 = very much. The scores range from 0 to 52, with higher scores indicating less fatigue. If there were missing items, but the participant answered at least 50% of the items, then subscores were prorated. |
| Secondary | Mean Change in the European Quality of Life-Five Dimensions-Three Levels (EQ-5D-3L) Score From Baseline | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The instrument is scored using the United Kingdom (UK) index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'. |
| Secondary | Time to Definitive Clinically Meaningful Deterioration for ≥ 2 Consecutive Visits as Measured Using the EQ-5D HRQoL Scale | From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months | Clinically meaningful deterioration was defined at least 0.10 point of deterioration from baseline for at least 2 consecutive visits for the EQ-5D Health Utility Index. The EQ-5D-3L is a self-administered questionnaire consisting of 5 questions, pertaining to specific health dimensions (ie, mobility, self-care, pain, usual activities, and anxiety/depression) and a health status scale. Each question has 3 levels of severity, corresponding to no problems, moderate problems and severe problems. Canadian population sample weights were used to derive health utility scores. A higher utility score represents a better health state. A clinically meaningful improvement or worsening was defined as at least 0.08 points of improvement or 0.10 points of worsening from baseline, respectively, for the EQ-5D-3L Health Utility Index. The EQ-5D-3L is scored using the UK index ranges from -0.594 to 1, where 0 equates to death and 1 equates to full health; -0.594 is considered 'worse than death'. |
| Secondary | Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective. |
| Secondary | Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | HRU is defined as any consumption of healthcare resources directly or indirectly related to the treatment of the patient. HRU is a key component to understand treatment costs and budget impact of new treatments from a provider perspective. |
Source ClinicalTrials.gov
Outcomes10
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Kaplan-Meier (K-M) Estimate for Overall Survival (OS) | Day 1 (randomization) up to data cut off date of 15 July 2019; median follow-up for OS estimated by the reverse K-M method was 41.2 months for all participants. | The intent to treat (ITT) population includes participants who were randomized, regardless of whether they received treatment or not. | Months | MEDIAN |
| SECONDARY | Kaplan-Meier Estimate of Relapse Free Survival (RFS) | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not. | Months | MEDIAN |
| SECONDARY | Kaplan-Meier Estimate of Time to Relapse | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not. | months | MEDIAN |
| SECONDARY | Kaplan-Meier Estimates of Time to Discontinuation From Treatment | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The intent to treat population includes participants who were randomized, regardless of whether they received treatment or not. | months | MEDIAN |
| SECONDARY | Number of Participants With Treatment Emergent Adverse Events (TEAEs) | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | The safety population included all randomized participants who received at least 1 dose of study drug. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Mean Change in the Functional Assessment of Chronic Illness Therapy (FACIT-Fatigue Scale V 4.0) Score From Baseline | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | All treated participants with FACIT-Fatigue measurement by EOT | units on a scale | MEAN |
| SECONDARY | Mean Change in the European Quality of Life-Five Dimensions-Three Levels (EQ-5D-3L) Score From Baseline | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | All treated participants with a EQ-5D-3L measurement at the end of treatment | Units on a scale | MEAN |
| SECONDARY | Time to Definitive Clinically Meaningful Deterioration for ≥ 2 Consecutive Visits as Measured Using the EQ-5D HRQoL Scale | From day 1 (randomization) up to data cut off date of 15 July 2019; approximately 74 months | The Health Related Quality of Life (HRQoL) evaluable population was defined as the ITT participants with a non-missing EQ-5D-3L score at baseline (C1D1) and at least one post-baseline visit. Results are presented for each post-baseline visit with sample size ≥ 25 in both arms. Death was censored at the date of the last HRQoL assessment visit. | Weeks | MEDIAN |
| SECONDARY | Healthcare Resource Utilization (HRU): Rate of Hospital Events Per Person Year | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | Safety population includes all participants who received at least 1 dose of study drug. | Hospitalizations per person-years | NUMBER |
| SECONDARY | Healthcare Resource Utilization (HRU): Number of Days Hospitalized Per Person-Year | From day 1 (randomization) up to data cut off date of 06 August 2024; approximately 135.5 months | Safety population includes all participants who received at least 1 dose of study drug. | Days per person-years | NUMBER |
Source ClinicalTrials.gov
Results by group40
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Kaplan-Meier (K-M) Estimate for Overall Survival (OS) | Oral Azacitidine Plus Best Supportive Care | 24.7 | 95% Confidence Interval 18.7 to 30.5 | Months | |
| Kaplan-Meier (K-M) Estimate for Overall Survival (OS) | Placebo Plus Best Supportive Care | 14.8 | 95% Confidence Interval 11.7 to 17.6 | Months | |
| Kaplan-Meier Estimate of Relapse Free Survival (RFS) | Oral Azacitidine Plus Best Supportive Care | 10.2 | 95% Confidence Interval 7.9 to 12.9 | Months | |
| Kaplan-Meier Estimate of Relapse Free Survival (RFS) | Placebo Plus Best Supportive Care | 4.8 | 95% Confidence Interval 4.6 to 6.4 | Months | |
| Kaplan-Meier Estimate of Time to Relapse | Oral Azacitidine Plus Best Supportive Care | 10.2 | 95% Confidence Interval 8.3 to 13.4 | months | |
| Kaplan-Meier Estimate of Time to Relapse | Placebo Plus Best Supportive Care | 4.9 | 95% Confidence Interval 4.6 to 6.4 | months | |
| Kaplan-Meier Estimates of Time to Discontinuation From Treatment | Oral Azacitidine Plus Best Supportive Care | 14.6 | 95% Confidence Interval 11.3 to 20.1 | months | |
| Kaplan-Meier Estimates of Time to Discontinuation From Treatment | Placebo Plus Best Supportive Care | 6.9 | 95% Confidence Interval 5.3 to 7.9 | months | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Oral Azacitidine Plus Best Supportive Care | ≥ 1 TEAE | 235 | Participants | |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) | Placebo Plus Best Supportive Care | ≥ 1 TEAE | 233 | Participants |
1 to 10 of 40Source ClinicalTrials.gov
See all 40 results by groupParticipant flow8
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Oral Azacitidine Plus Best Supportive Care | 238 | |
| Overall Study | STARTED | Placebo Plus Best Supportive Care | 234 | |
| Overall Study | Received Treatment | Oral Azacitidine Plus Best Supportive Care | 236 | |
| Overall Study | Received Treatment | Placebo Plus Best Supportive Care | 233 | |
| Overall Study | COMPLETED | Oral Azacitidine Plus Best Supportive Care | 0 | |
| Overall Study | COMPLETED | Placebo Plus Best Supportive Care | 0 | |
| Overall Study | NOT COMPLETED | Oral Azacitidine Plus Best Supportive Care | 238 | |
| Overall Study | NOT COMPLETED | Placebo Plus Best Supportive Care | 234 |
Source ClinicalTrials.gov
Adverse events414
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 | Oral Azacitidine Plus Best Supportive Care | 51 | 236 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Plus Best Supportive Care | 42 | 233 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Oral Azacitidine Plus Best Supportive Care | 14 | 236 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Placebo Plus Best Supportive Care | 10 | 233 | |
| other | Leukopenia | Blood and lymphatic system disorders | Oral Azacitidine Plus Best Supportive Care | 27 | 236 | |
| other | Leukopenia | Blood and lymphatic system disorders | Placebo Plus Best Supportive Care | 19 | 233 | |
| other | Neutropenia | Blood and lymphatic system disorders | Oral Azacitidine Plus Best Supportive Care | 104 | 236 | |
| other | Neutropenia | Blood and lymphatic system disorders | Placebo Plus Best Supportive Care | 61 | 233 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Oral Azacitidine Plus Best Supportive Care | 81 | 236 | |
| other | Thrombocytopenia | Blood and lymphatic system disorders | Placebo Plus Best Supportive Care | 62 | 233 |
1 to 10 of 414Source ClinicalTrials.gov
See all 414 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Oral Azacitidine Plus Best Supportive Care | 172 | 238 |
| Total, all-cause mortality | Placebo Plus Best Supportive Care | 176 | 234 |
| Total, other adverse events | Oral Azacitidine Plus Best Supportive Care | 235 | 236 |
| Total, other adverse events | Placebo Plus Best Supportive Care | 224 | 233 |
| Total, serious adverse events | Oral Azacitidine Plus Best Supportive Care | 110 | 236 |
| Total, serious adverse events | Placebo Plus Best Supportive Care | 109 | 233 |
Source ClinicalTrials.gov
Papers8
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 comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Oral Azacitidine Plus Best Supportive Care vs Placebo Plus Best Supportive Care: deaths from any cause | Trial comparisons |
| Oral Azacitidine Plus Best Supportive Care vs Placebo Plus Best Supportive Care: serious adverse events | 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.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT01757535
GET https://conductscience.org/api/trials/v1/NCT01757535/results?page=2