Trial · NCT03280030
A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML
NCT03280030, Phase 2, Completed, Novartis Pharmaceuticals; results first posted 2025-02-11
3 sourcestrials-2026-11-12-0.4.0
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ConductScience Library. A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML. Trials, record NCT03280030. Release trials-2026-11-12-0.4.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03280030
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Midostaurin Efficacy and Safety in Newly Diagnosed Patients With FLT3-mutated AML
- NCT number
- NCT03280030
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 67 participants, actual
- Lead sponsor
- Novartis Pharmaceuticals
- Conditions
- Acute Myeloid Leukemia
- Interventions
- Midostaurin, Placebo
- First posted
- 2017-09-12
- Last updated
- 2025-03-20
- Results first posted
- 2025-02-11
Source ClinicalTrials.gov
Registered outcomes13
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Safety Events (Part 1, Japan Only) | up to Day 21 of the first Consolidation cycle; cycle = 28 days | Percentage of Safety Events, defined as death or serious adverse event leading to treatment discontinuation that occurs on or before Day 21 of the first Consolidation cycle. This was determined by the Independent Safety Committee (ISC) to be definitely or probably related to midostaurin. Percentage was calculated based on the percentage of subjects with safety event out of 3 evaluable subjects in Part 1. |
| Primary | Event Free Survival (EFS) (Part 2 - Randomized, Controlled) | up to 3 years after last patient started treatment | Event Free survival is defined as the time from the date of randomization until an EFS event is observed. An EFS event is defined as a failure to obtain a complete remission (CR) within an induction 2, relapse after CR, or death due to any cause, whichever occurs first. The objective was to evaluate the efficacy based on EFS of midostaurin versus placebo in combination with daunorubicin/cytarabine induction, with high-dose cytarabine consolidation, and with midostaurin single agent continuation therapy in newly diagnosed patients with FLT3-mutated AML. |
| Secondary | Overall Survival | up to 3 years after last patient started treatment | Overall survival defined as the time from the date of randomization to date of death due to any cause |
| Secondary | Percentage of Participants With Complete Remission (CR) | up to 3 years after last patient started treatment | Complete Remission is defined as the percentage of participants with a CR according to Chelson Criteria, at various timepoints |
| Secondary | Percentage of Participants With Cumulative Incidence of Relapse (CIR) | up to 3 years after last patient started treatment | CIR (only for patients who achieved CR after study treatment initiation), is measured from the date of first CR to relapse or death due to AML, whichever occurs first. |
| Secondary | Pharmakinetics (PK) for Midostaurin: AUClast & AUC0-t | Induction Phase: Pre-dose and 1, 3, 6 and 12 hours post dose in Cycle 1 Day 8 | Evaluate AUClast \& AUC0-t PK parameters for midostaurin. AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast) (mass x time x volume-1) AUC0-t: The area under the curve (AUC) from time zero to a measurable concentration sampling time (t) (mass x time x volume-1). |
| Secondary | Pharmakinetics (PK) for Midostaurin: Cmax | Induction Phase: Pre-dose and 1, 3, 6 and 12 hours post dose in Cycle 1 Day 8 | Evaluate Cmax parameter for midostaurin. Cmax: The maximum (peak) observed plasma drug concentration after the first dose administration of midostaurin (mass x volume-1). |
| Secondary | Metabolite CGP52421: PK Parameters AUClast, AUC0-t | Induction Phase: Cycle 1 Day 8 | Evaluate the pharmacokinetic of major metabolite of midostaurin CGP52421. AUClast: The AUC from time zero to the last measurable concentration sampling time (Tlast) (mass x time x volume-1) AUC0-t: The area under the curve (AUC) from time zero to a measurable concentration sampling time (t) (mass x time x volume-1). |
| Secondary | Metabolite CGP52421: PK Parameter Cmax | Induction Phase: Cycle 1 Day 8 | Evaluate the pharmacokinetic of major metabolite of midostaurin CGP52421: Cmax. Cmax: The maximum (peak) observed plasma drug concentration after the first dose administration of midostaurin (mass x volume-1). |
| Secondary | Metabolite CGP62221: PK Parameters: AUClast, AUC0-t | Induction Phase: Cycle 1 Day 8 | Evaluate the pharmacokinetic of major metabolite of Midostaurin CGP62221 PK parameters AUClast, AUC0-t |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 registered outcomesOutcomes14
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Safety Events (Part 1, Japan Only) | up to Day 21 of the first Consolidation cycle; cycle = 28 days | Patients enrolled in Part 1: 3 patients out of 5 patients in Part 1 were evaluable for safety evaluation as determined by Independent Safety Committee (ISC). | Participants | NUMBER |
| PRIMARY | Event Free Survival (EFS) (Part 2 - Randomized, Controlled) | up to 3 years after last patient started treatment | Full Analysis Set (FAS): The FAS comprised of all subjects to whom study drug (midostaurin/placebo) had been assigned by randomization. Therefore, all Japanese subjects treated during Part 1 were not eligible for the FAS. | Months | MEDIAN |
| SECONDARY | Overall Survival | up to 3 years after last patient started treatment | FAS: The FAS comprised of all subjects to whom study drug (midostaurin/placebo) had been assigned by randomization. Therefore, all Japanese subjects treated during Part 1 were not eligible for the FAS. | Months | MEDIAN |
| SECONDARY | Percentage of Participants With Complete Remission (CR) | up to 3 years after last patient started treatment | FAS: The FAS comprised of all subjects to whom study drug (midostaurin/placebo) had been assigned by randomization. Therefore, all Japanese subjects treated during Part 1 were not eligible for the FAS. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Cumulative Incidence of Relapse (CIR) | up to 3 years after last patient started treatment | FAS: The FAS comprised of all subjects to whom study drug (midostaurin/placebo) had been assigned by randomization. Therefore, all Japanese subjects treated during Part 1 were not eligible for the FAS. This analysis was on FAS but only on participants who had achieved a CR. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Pharmakinetics (PK) for Midostaurin: AUClast & AUC0-t | Induction Phase: Pre-dose and 1, 3, 6 and 12 hours post dose in Cycle 1 Day 8 | PK analysis set for full PK (PAS-full): The PAS-full included all subjects in the PAS-all, who provide an evaluable PK profile. A profile was considered evaluable if all of the following conditions are satisfied: Subject received the planned dose of midostaurin on C1D8 of induction therapy; Subject did not vomit within 4 hours of the dosing of midostaurin on C1D8 of induction therapy; Subject provided at least one primary PK parameter. | ng*hr/mL | GEOMETRIC_MEAN |
| SECONDARY | Pharmakinetics (PK) for Midostaurin: Cmax | Induction Phase: Pre-dose and 1, 3, 6 and 12 hours post dose in Cycle 1 Day 8 | PAS-full: PK analysis set for full PK (PAS-full): The PAS-full included all subjects in the PAS-all, who provide an evaluable PK profile. A profile was considered evaluable if all of the following conditions are satisfied: Subject received the planned dose of midostaurin on C1D8 of induction therapy; Subject did not vomit within 4 hours of the dosing of midostaurin on C1D8 of induction therapy; Subject provided at least one primary PK parameter. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Metabolite CGP52421: PK Parameters AUClast, AUC0-t | Induction Phase: Cycle 1 Day 8 | PAS-full: PK analysis set for full PK (PAS-full): The PAS-full included all subjects in the PAS-all, who provide an evaluable PK profile. A profile was considered evaluable if all of the following conditions are satisfied: Subject received the planned dose of midostaurin on C1D8 of induction therapy; Subject did not vomit within 4 hours of the dosing of midostaurin on C1D8 of induction therapy; Subject provided at least one primary PK parameter. | ng*hr/mL | GEOMETRIC_MEAN |
| SECONDARY | Metabolite CGP52421: PK Parameter Cmax | Induction Phase: Cycle 1 Day 8 | PAS-full: PK analysis set for full PK (PAS-full): The PAS-full included all subjects in the PAS-all, who provide an evaluable PK profile. A profile was considered evaluable if all of the following conditions are satisfied: Subject received the planned dose of midostaurin on C1D8 of induction therapy; Subject did not vomit within 4 hours of the dosing of midostaurin on C1D8 of induction therapy; Subject provided at least one primary PK parameter. | ng/mL | GEOMETRIC_MEAN |
| SECONDARY | Metabolite CGP62221: PK Parameters: AUClast, AUC0-t | Induction Phase: Cycle 1 Day 8 | PAS-full: PK analysis set for full PK (PAS-full): The PAS-full included all subjects in the PAS-all, who provide an evaluable PK profile. A profile was considered evaluable if all of the following conditions are satisfied: Subject received the planned dose of midostaurin on C1D8 of induction therapy; Subject did not vomit within 4 hours of the dosing of midostaurin on C1D8 of induction therapy; Subject provided at least one primary PK parameter. | ng*hr/mL | GEOMETRIC_MEAN |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 outcomesResults by group103
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Safety Events (Part 1, Japan Only) | Midostaurin: Part 1 (Japan Only) | Experienced at least 1 adverse event (AE) | 5 | Participants | |
| Percentage of Safety Events (Part 1, Japan Only) | Midostaurin: Part 1 (Japan Only) | Safety events determined by ISC related to midostaurin (n = 3, 0, 0) | 0 | Participants | |
| Event Free Survival (EFS) (Part 2 - Randomized, Controlled) | Midostaurin: Part 2 | 7.6 | 95% Confidence Interval 3.6 to NA | Months | |
| Event Free Survival (EFS) (Part 2 - Randomized, Controlled) | Placebo: Part 2 | NA | 95% Confidence Interval 8.2 to NA | Months | |
| Overall Survival | Midostaurin: Part 2 | NA | 95% Confidence Interval 16.8 to NA | Months | |
| Overall Survival | Placebo: Part 2 | NA | 95% Confidence Interval 18.8 to NA | Months | |
| Percentage of Participants With Complete Remission (CR) | Midostaurin: Part 2 | Overall CR | 70.0 | 95% Confidence Interval 50.60 to 85.27 | Percentage of participants |
| Percentage of Participants With Complete Remission (CR) | Placebo: Part 2 | Overall CR | 78.1 | 95% Confidence Interval 60.03 to 90.72 | Percentage of participants |
| Percentage of Participants With Complete Remission (CR) | Midostaurin: Part 2 | Morphologic CR with incomplete platelet count recovery (CRp) | 6.7 | 95% Confidence Interval 0.86 to 22.07 | Percentage of participants |
| Percentage of Participants With Complete Remission (CR) | Placebo: Part 2 | Morphologic CR with incomplete platelet count recovery (CRp) | 0.0 | 95% Confidence Interval 0.00 to 10.89 | Percentage of participants |
1 to 10 of 103Source ClinicalTrials.gov
See all 103 results by groupParticipant flow21
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Part 1 - Safety Evaluation | STARTED | Midostaurin: Part 1 (Japan Only) | 5 | |
| Part 1 - Safety Evaluation | STARTED | Midostaurin: Part 2 | 0 | |
| Part 1 - Safety Evaluation | STARTED | Placebo: Part 2 | 0 | |
| Part 1 - Safety Evaluation | COMPLETED | Midostaurin: Part 1 (Japan Only) | 0 | |
| Part 1 - Safety Evaluation | COMPLETED | Midostaurin: Part 2 | 0 | |
| Part 1 - Safety Evaluation | COMPLETED | Placebo: Part 2 | 0 | |
| Part 1 - Safety Evaluation | NOT COMPLETED | Midostaurin: Part 1 (Japan Only) | 5 | |
| Part 1 - Safety Evaluation | NOT COMPLETED | Midostaurin: Part 2 | 0 | |
| Part 1 - Safety Evaluation | NOT COMPLETED | Placebo: Part 2 | 0 | |
| Part 2 - Efficacy | STARTED | Midostaurin: Part 1 (Japan Only) | 0 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 participant flowAdverse events1,183
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 | Midostaurin: Part 1 (Japan Only) (On-treatment) | 3 | 5 | |
| other | Anaemia | Blood and lymphatic system disorders | Midostaurin: Part 2 (On-treatment) | 17 | 30 | |
| other | Anaemia | Blood and lymphatic system disorders | Placebo Part 2 (On-treatment) | 15 | 31 | |
| other | Anaemia | Blood and lymphatic system disorders | Midostaurin: Part 1 (Japan Only) (Post-treatment Survival Follow-up) | 0 | ||
| other | Anaemia | Blood and lymphatic system disorders | Midostaurin: Part 2 (Post-treatment Survival Follow-up) | 0 | ||
| other | Anaemia | Blood and lymphatic system disorders | Placebo: Part 2 (Post-treatment Survival Follow-up) | 0 | ||
| other | Anaemia | Blood and lymphatic system disorders | Other Participants | 0 | ||
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Midostaurin: Part 1 (Japan Only) (On-treatment) | 0 | 5 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Midostaurin: Part 2 (On-treatment) | 1 | 30 | |
| other | Disseminated intravascular coagulation | Blood and lymphatic system disorders | Placebo Part 2 (On-treatment) | 2 | 31 |
1 to 10 of 1,183Source ClinicalTrials.gov
See all 1,183 adverse eventsAdverse event totals21
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Midostaurin: Part 1 (Japan Only) (On-treatment) | 1 | 5 |
| Total, all-cause mortality | Midostaurin: Part 2 (On-treatment) | 3 | 30 |
| Total, all-cause mortality | Placebo Part 2 (On-treatment) | 1 | 31 |
| Total, all-cause mortality | Midostaurin: Part 1 (Japan Only) (Post-treatment Survival Follow-up) | 2 | 4 |
| Total, all-cause mortality | Midostaurin: Part 2 (Post-treatment Survival Follow-up) | 7 | 27 |
| Total, all-cause mortality | Placebo: Part 2 (Post-treatment Survival Follow-up) | 6 | 30 |
| Total, all-cause mortality | Other Participants | 7 | 7 |
| Total, other adverse events | Midostaurin: Part 1 (Japan Only) (On-treatment) | 5 | 5 |
| Total, other adverse events | Midostaurin: Part 2 (On-treatment) | 30 | 30 |
| Total, other adverse events | Placebo Part 2 (On-treatment) | 31 | 31 |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 adverse event totalsPapers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 comparisons1
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison |
|---|
| Midostaurin: Part 2 vs Placebo: Part 2: Percentage of Participants With Cumulative Incidence of Relapse (CIR) |
Source 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/NCT03280030
GET https://conductscience.org/api/trials/v1/NCT03280030/results?page=2