Trial · NCT01093573
Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia
NCT01093573, Phase 1, Phase 2, Completed, Brenda Cooper, MD; results first posted 2020-05-01
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia. Trials, record NCT01093573. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01093573
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Midostaurin and Azacitidine in Treating Elderly Patients With Acute Myelogenous Leukemia
- NCT number
- NCT01093573
- Status
- Completed
- Phases
- Phase 1, Phase 2
- Enrollment
- 34 participants, actual
- Lead sponsor
- Brenda Cooper, MD
- Conditions
- Untreated Adult Acute Myeloid Leukemia
- Interventions
- midostaurin, azacitidine, bone marrow aspiration, mutation analysis, Pharmacokinetic study
- First posted
- 2010-03-26
- Last updated
- 2022-06-30
- Results first posted
- 2020-05-01
Source ClinicalTrials.gov
Registered outcomes9
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Maximum Tolerated Dose of Midostaurin in Combination With Azacitidine in Patients With Acute Myelogenous Leukemia (Phase I) | Day 28 | Patients received azacitidine 75 mg/m intravenous over 30 minutes daily for 7 consecutive days followed by escalating doses of oral midostaurin (25 mg bid, 50 mg bid, and 75 mg bid) days 8-21. Determination of the maximum tolerated dose (MTD) was based on dose-limiting toxicities (DLT) observed during the first cycle of treatment |
| Primary | Number of Participants With Hematologic Improvement (Phase I) | After 2 cycles of therapy | Number of participants with HI. Hematologic improvement (HI): must last at least 2 months in the absence of ongoing cytotoxic therapy and will be another endpoint of interest (although it will not be considered in the final statistical analysis) and will be defined according to IWG criteria . |
| Primary | Overall Response Rate (Phase II) | after 4 months of treatment | Number of participants with CR, CRi, PR and Hematologic improvement (HI). Response will be assessed using the standard morphologic criteria for acute leukemia as follows: Complete remission (CR): ANC ≥ 1000/ uL and platelets of \> 100,000/ uL without circulating blasts and bone marrow with \< 5% blasts and no Auer rods; Morphologic complete remission with incomplete blood recovery (CRi): Patients fulfills all of the criteria for remission except for residual neutropenia (ANC \< 1000/ uL) or thrombocytopenia (platelet count \< 100,000/uL). Partial remission (PR): This designation requires at least a 50% decrease in the bone marrow blasts to 5-25%. |
| Primary | Toxicity Profile (Phase II) | during treatment up to 10 cycles | Number of patients experiencing at least one instance of specific treatment emergent adverse events |
| Secondary | Duration of Response | Up to 3 years | Time to progression after confirmed response |
| Secondary | Overall Survival (Phase II) | Up to 3 years | Median time of overall survival of participants from initiation of midostaurin-azacitidine |
| Secondary | Correlate Treatment Response With FLT3 Mutational Status in a Descriptive Fashion.(Phase I) | Baseline to 4 cycles (16 weeks) | Number of participants with FLT3 mutation that had a response to treatment |
| Other | Pharmacokinetic Profile of Midostaurin Given With Azacitidine (Phase I) | after 2 cycles | Change in Midostaurin trough levels and active metabolite levels between cycles one and two |
| Other | Changes of Phosphorylation Status of FLT3 in Blood and Bone Marrow Samples (Phase I/II) | Baseline to 4 cycles (16 weeks) |
Source ClinicalTrials.gov
Outcomes9
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Maximum Tolerated Dose of Midostaurin in Combination With Azacitidine in Patients With Acute Myelogenous Leukemia (Phase I) | Day 28 | All participants who received treatment for the Phase I part of the study | mg/bid | NUMBER |
| PRIMARY | Number of Participants With Hematologic Improvement (Phase I) | After 2 cycles of therapy | Participants that received treatment on the phase I portion of the study. | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Overall Response Rate (Phase II) | after 4 months of treatment | All participants that received at least 2 cycles of treatment at the MTD (Dose Level 3) | Participants | COUNT_OF_PARTICIPANTS |
| PRIMARY | Toxicity Profile (Phase II) | during treatment up to 10 cycles | Participants that received at least one dose of midostaurin at the MTD (Dose Level 3) | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Duration of Response | Up to 3 years | All participants on phase II (dose level 3) that were evaluable for response. | days | MEDIAN |
| SECONDARY | Overall Survival (Phase II) | Up to 3 years | All participants that received treatment at the MTD (dose level 3) | days | MEDIAN |
| SECONDARY | Correlate Treatment Response With FLT3 Mutational Status in a Descriptive Fashion.(Phase I) | Baseline to 4 cycles (16 weeks) | There were no subjects with FLT3 mutation, there for this outcome measure has no data. | ||
| OTHER_PRE_SPECIFIED | Pharmacokinetic Profile of Midostaurin Given With Azacitidine (Phase I) | after 2 cycles | |||
| OTHER_PRE_SPECIFIED | Changes of Phosphorylation Status of FLT3 in Blood and Bone Marrow Samples (Phase I/II) | Baseline to 4 cycles (16 weeks) |
Source ClinicalTrials.gov
Results by group12
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Maximum Tolerated Dose of Midostaurin in Combination With Azacitidine in Patients With Acute Myelogenous Leukemia (Phase I) | Midostaurin Dose Escalation | 75 | mg/bid | ||
| Number of Participants With Hematologic Improvement (Phase I) | Dose Level 1 | 0 | Participants | ||
| Number of Participants With Hematologic Improvement (Phase I) | Dose Level 2 | 0 | Participants | ||
| Number of Participants With Hematologic Improvement (Phase I) | Dose Level 3 | 2 | Participants | ||
| Overall Response Rate (Phase II) | Dose Level 3 | Complete Remission (CR) | 4 | Participants | |
| Overall Response Rate (Phase II) | Dose Level 3 | Complete Remission with incomplete recovery (CRi) | 1 | Participants | |
| Overall Response Rate (Phase II) | Dose Level 3 | Partial Remission | 2 | Participants | |
| Overall Response Rate (Phase II) | Dose Level 3 | No remission/response | 17 | Participants | |
| Overall Response Rate (Phase II) | Dose Level 3 | Hematologic improvement without CR/PR | 0 | Participants | |
| Toxicity Profile (Phase II) | Dose Level 3 | 24 | Participants |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 results by groupParticipant flow9
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Dose Level 1 | 3 | |
| Overall Study | STARTED | Dose Level 2 | 3 | |
| Overall Study | STARTED | Dose Level 3 | 28 | |
| Overall Study | COMPLETED | Dose Level 1 | 3 | |
| Overall Study | COMPLETED | Dose Level 2 | 3 | |
| Overall Study | COMPLETED | Dose Level 3 | 26 | |
| Overall Study | NOT COMPLETED | Dose Level 1 | 0 | |
| Overall Study | NOT COMPLETED | Dose Level 2 | 0 | |
| Overall Study | NOT COMPLETED | Dose Level 3 | 2 |
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 | Abdominal distension | Gastrointestinal disorders | Dose Level 1 | 0 | 3 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | Dose Level 2 | 0 | 3 | 0 |
| other | Abdominal distension | Gastrointestinal disorders | Dose Level 3 | 1 | 28 | 1 |
| other | Abdominal pain | Gastrointestinal disorders | Dose Level 1 | 0 | 3 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Dose Level 2 | 0 | 3 | 0 |
| other | Abdominal pain | Gastrointestinal disorders | Dose Level 3 | 1 | 28 | 3 |
| other | Acidosis | Metabolism and nutrition disorders | Dose Level 1 | 0 | 3 | 0 |
| other | Acidosis | Metabolism and nutrition disorders | Dose Level 2 | 0 | 3 | 0 |
| other | Acidosis | Metabolism and nutrition disorders | Dose Level 3 | 1 | 28 | 1 |
| other | Activated partial thromboplastin time prolonged | Investigations | Dose Level 1 | 1 | 3 | 1 |
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 | Dose Level 1 | 1 | 3 |
| Total, all-cause mortality | Dose Level 2 | 1 | 3 |
| Total, all-cause mortality | Dose Level 3 | 3 | 28 |
| Total, other adverse events | Dose Level 1 | 3 | 3 |
| Total, other adverse events | Dose Level 2 | 3 | 3 |
| Total, other adverse events | Dose Level 3 | 26 | 28 |
| Total, serious adverse events | Dose Level 1 | 3 | 3 |
| Total, serious adverse events | Dose Level 2 | 1 | 3 |
| Total, serious adverse events | Dose Level 3 | 22 | 28 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 32085993 | BACKGROUND |
Source PubMed
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 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.
GET https://conductscience.org/api/trials/v1/NCT01093573
GET https://conductscience.org/api/trials/v1/NCT01093573/events?page=2