Trial · NCT03092674
Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
NCT03092674, Phase 2, Phase 3, Completed, National Cancer Institute (NCI); results first posted 2024-07-23
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome. Trials, record NCT03092674. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03092674
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Azacitidine With or Without Nivolumab or Midostaurin, or Decitabine and Cytarabine Alone in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia or High-Risk Myelodysplastic Syndrome
- NCT number
- NCT03092674
- Status
- Completed
- Phases
- Phase 2, Phase 3
- Enrollment
- 76 participants, actual
- Lead sponsor
- National Cancer Institute (NCI)
- Conditions
- Acute Myeloid Leukemia, Myelodysplastic Syndrome, Myelodysplastic Syndrome/Acute Myeloid Leukemia
- Interventions
- Azacitidine, Cytarabine, Decitabine, Laboratory Biomarker Analysis, Midostaurin, Nivolumab
- First posted
- 2017-03-28
- Last updated
- 2025-01-16
- Results first posted
- 2024-07-23
Source ClinicalTrials.gov
Registered outcomes11
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Overall Survival (OS) (Phase II) | Day of registration on study until death from any cause, assessed for up to 5 years | Time from date of randomization on study until death from any cause with observations censored on the day of last contact for patients not known to have died. |
| Primary | OS (Phase III) | Day of registration on study until death from any cause, assessed for up to 5 years | Time from date of randomization on study until death from any cause with observations censored on the day of last contact for patients not known to have died. |
| Secondary | Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Duration of treatment and follow up until death or 5 years post randomization (registration to Step 2). | Only adverse events that are possibly, probably, or definitely related to study drugs are reported. CTCAE Version 5.0 was used for all AE reporting. |
| Other | Remission Rates | Up to 5 years | #participants(ps) w comp response(resp)(CR), comp remission w incomp bld ct recovery(CRp/CRi), morphol leukemia-free state(MLFS), erythroid\&neutrophil\&platelet resp(HI-E,HI-N,HI-P), HI-P, marrow comp resp(CRm), stable disease(SD), no resp(NR) CR:BM blasts\<5%; no circ blasts\&blasts w Auer rods(AR); no extramedullary disease(ED); ANC≥1x10\^9/L; plt ct≥100x10\^9/L CRp/CRi:CR criteria excl residual neutropenia\<1x10\^9/L or ITP\<100x10\^9/L MLFS:BM blasts\<5%; no blasts w AR, ED, hem recovery rqd HI-E:≥1.5g/dL↑Hb pretx;\&RBC trans-depen ps, achieve trans independ or relevant↓RBC trans rqd HI-N:ANC↑pretx≥100%,\&an absolute↑of\>500/mm3 pretx HI-P:Absolute↑≥30000/mm3 pretx for ps w plt ct\>20000/mm3 pretx. Ps w plt ct≤20000/mm3 pretx,↑≥100% pretx ct to plt ct\>20000/mm3. Plt trans-depend ps, plt trans independ rqd CRm:CR for BM exam≤5%myeloblasts\&marrow myeloblasts↓≥50%pretx SD:Not achieve≥PR, w no evidence prog NR:Not achieve CR,CRi,PR,MLFS,SD, excl ps w death in aplasia or due to indet cause |
| Other | OS | Day of registration on study until death from any cause, assessed for up to 5 years | Will be estimated using the Kaplan-Meier method or Cox regression models. Landmark analyses of different response categories will be evaluated based on dates at which 75% and 90% of patients have achieved a response (with other quantiles analyzed as needed). |
| Other | Relapse-free Survival (RFS) | Date of achievement of a remission until the date of relapse or death from any cause, assessed for up to 5 years | Time from the date of achievement of a remission (defined as patients achieving complete remission (CR), or CR with incomplete hematological recovery (CRi)) until the date of relapse or death from any cause; patients not known to have relapsed or died at last follow-up are censored on the date of last contact. |
| Other | Event-free Survival (EFS) | Date of randomization to the first of: date of primary refractory disease; date of progressive disease; date off protocol therapy without CR or CRi; date of relapse from CR or CRi, or death from any cause, assessed for up to 5 years | Time from from the date of randomization to the first of: date of primary refractory disease; date of progressive disease; date off protocol therapy without CR or CRi; date of relapse from CR or CRi, or death from any cause; patients not known to have any of these events are censored on the date of last contact. |
| Other | Cumulative Incidence of Relapse Defined as Achieving CR or CRi | Date of achievement of a remission until the date of relapse or death, assessed for up to 5 years | Cumulative incident endpoints and associations will be assessed using Cox regression models (for cause-specific hazards as appropriate). |
| Other | FLT3-ITD | Up to 5 years | The prognostic effect of FLT3-ITD (positive versus negative or non-evaluable) with respect to the outcome OS will be evaluated using Cox regression models. The predictive effect of FLT3-ITD (positive versus negative/non-evaluable) with respect to the outcome of OS and the treatments of azacitdine+midostaurin versus azacitidine will be evaluated using a Cox regression model with treatment arm and FLT3-ITD as covariates and including the interaction between the two covariates. |
| Other | Cytogenetic Abnormalities, Risk Categories, and Mutations in Bone Marrow | Up to 5 years | Univariate and multivariable regression models will be used to assess potential associations between outcomes (CR, OS, EFS, and RFS) and cytogenetic abnormalities and mutation status (including FLT3-ITD). Regression models including interaction terms with treatment arm will be fit. In addition to analyzing FLT3-ITD as categorical variable used in randomization stratification, we will analyze FLT3-TKD also and evaluate FLT3-ITD allelic ratio as a quantitative variable and as a binary variable using the threshold of 0.50. |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 registered outcomesOutcomes11
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Overall Survival (OS) (Phase II) | Day of registration on study until death from any cause, assessed for up to 5 years | The analysis population includes the 76 participants who were eligible and evaluable (26 in the Azacitidine arm, 25 in the Azacitidine+Nivolumab arm, and 25 in the Azacitidine+Midostaurin arm). | months | MEDIAN |
| PRIMARY | OS (Phase III) | Day of registration on study until death from any cause, assessed for up to 5 years | Due to unexpected toxicities on the Azacitidine + Nivolumab arm during the first two cycles of therapy, the trial was closed early, before the phase II portions was completed and before reaching the phase III portion. | ||
| SECONDARY | Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Duration of treatment and follow up until death or 5 years post randomization (registration to Step 2). | Participants who were eligible and received at least one dose of protocol treatment. | Participants | NUMBER |
| OTHER_PRE_SPECIFIED | Remission Rates | Up to 5 years | The analysis population includes the 76 participants who were eligible and evaluable (26 in the Azacitidine arm, 25 in the Azacitidine+Nivolumab arm, and 25 in the Azacitidine+Midostaurin arm). | Participants | COUNT_OF_PARTICIPANTS |
| OTHER_PRE_SPECIFIED | OS | Day of registration on study until death from any cause, assessed for up to 5 years | |||
| OTHER_PRE_SPECIFIED | Relapse-free Survival (RFS) | Date of achievement of a remission until the date of relapse or death from any cause, assessed for up to 5 years | The analysis population includes the 17 participants who were eligible and evaluable and had complete response or complete remission with incomplete blood count recovery (CRp/CRi) (6 in the Azacitidine arm, 5 in the Azacitidine+Nivolumab arm, and 6 in the Azacitidine+Midostaurin arm). | months | MEDIAN |
| OTHER_PRE_SPECIFIED | Event-free Survival (EFS) | Date of randomization to the first of: date of primary refractory disease; date of progressive disease; date off protocol therapy without CR or CRi; date of relapse from CR or CRi, or death from any cause, assessed for up to 5 years | The analysis population includes the 76 participants who were eligible and evaluable (26 in the Azacitidine arm, 25 in the Azacitidine+Nivolumab arm, and 25 in the Azacitidine+Midostaurin arm). | months | MEDIAN |
| OTHER_PRE_SPECIFIED | Cumulative Incidence of Relapse Defined as Achieving CR or CRi | Date of achievement of a remission until the date of relapse or death, assessed for up to 5 years | |||
| OTHER_PRE_SPECIFIED | FLT3-ITD | Up to 5 years | |||
| OTHER_PRE_SPECIFIED | Cytogenetic Abnormalities, Risk Categories, and Mutations in Bone Marrow | Up to 5 years |
1 to 10 of 11Source ClinicalTrials.gov
See all 11 outcomesResults by group198
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Overall Survival (OS) (Phase II) | Azacitidine | 6.2 | 95% Confidence Interval 3.9 to 10 | months | |
| Overall Survival (OS) (Phase II) | Azacitidine + Nivolumab | 5.2 | 95% Confidence Interval 1.1 to 13.1 | months | |
| Overall Survival (OS) (Phase II) | Azacitidine +Midostaurin | 7.4 | 95% Confidence Interval 2.9 to 16.4 | months | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine | Acidosis | 1 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine + Nivolumab | Acidosis | 0 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine +Midostaurin | Acidosis | 0 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine | Acute kidney injury | 0 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine + Nivolumab | Acute kidney injury | 1 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine +Midostaurin | Acute kidney injury | 0 | Participants | |
| Number of Participants With Gr 3 Through 5 Adverse Events That Are Related to Study Drugs | Azacitidine | Alanine aminotransferase increased | 0 | Participants |
1 to 10 of 198Source ClinicalTrials.gov
See all 198 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Azacitidine | 26 | |
| Overall Study | STARTED | Azacitidine + Nivolumab | 25 | |
| Overall Study | STARTED | Azacitidine + Midostaurin | 25 | |
| Overall Study | COMPLETED | Azacitidine | 0 | |
| Overall Study | COMPLETED | Azacitidine + Nivolumab | 0 | |
| Overall Study | COMPLETED | Azacitidine + Midostaurin | 0 | |
| Overall Study | NOT COMPLETED | Azacitidine | 26 | |
| Overall Study | NOT COMPLETED | Azacitidine + Nivolumab | 25 | |
| Overall Study | NOT COMPLETED | Azacitidine + Midostaurin | 25 |
Source ClinicalTrials.gov
Adverse events579
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 | Anemia | Blood and lymphatic system disorders | Azacitidine | 13 | 25 | |
| other | Anemia | Blood and lymphatic system disorders | Azacitidine + Nivolumab | 12 | 25 | |
| other | Anemia | Blood and lymphatic system disorders | Azacitidine +Midostaurin | 13 | 23 | |
| other | Blood and lymphatic system disorders-Other | Blood and lymphatic system disorders | Azacitidine | 1 | 25 | |
| other | Blood and lymphatic system disorders-Other | Blood and lymphatic system disorders | Azacitidine + Nivolumab | 2 | 25 | |
| other | Blood and lymphatic system disorders-Other | Blood and lymphatic system disorders | Azacitidine +Midostaurin | 2 | 23 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Azacitidine | 7 | 25 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Azacitidine + Nivolumab | 3 | 25 | |
| other | Febrile neutropenia | Blood and lymphatic system disorders | Azacitidine +Midostaurin | 6 | 23 | |
| other | Cardiac disorders-Other | Cardiac disorders | Azacitidine | 1 | 25 |
1 to 10 of 579Source ClinicalTrials.gov
See all 579 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Azacitidine | 24 | 26 |
| Total, all-cause mortality | Azacitidine + Nivolumab | 25 | 25 |
| Total, all-cause mortality | Azacitidine +Midostaurin | 23 | 25 |
| Total, other adverse events | Azacitidine | 23 | 25 |
| Total, other adverse events | Azacitidine + Nivolumab | 23 | 25 |
| Total, other adverse events | Azacitidine +Midostaurin | 22 | 23 |
| Total, serious adverse events | Azacitidine | 9 | 25 |
| Total, serious adverse events | Azacitidine + Nivolumab | 20 | 25 |
| Total, serious adverse events | Azacitidine +Midostaurin | 17 | 23 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants1
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.
| Project | Title | Principal investigators | Organization | Funder | Fiscal years |
|---|---|---|---|---|---|
| U10CA180888 | SWOG Network Group Operations Center of the NCTN | LARA, PRIMO N.; HERSHMAN, DAWN | UNIVERSITY OF CALIFORNIA AT DAVIS | NATIONAL CANCER INSTITUTE | 2014 to 2026 |
Source NIH RePORTER
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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