This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML (NCT01883362), a randomized phase 2 study. The primary outcome is Proportion of Participants With Relapse Free Survival (RFS) up to 18 Months Post Transplant (Full Analysis Set) by Kaplan-Meier Analysis.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01883362 |
| Phase | Phase 2 |
| Condition | Acute Myeloid Leukemia |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 60 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
Patients with FLT3-ITD mutated acute myeloid leukemia who received an allogeneic hematopoietic stem cell transplant. The registry record lists acute myeloid leukemia as the condition and describes the intended population in relation to transplantation and FLT3-ITD mutation status.
Intervention
Standard of care after transplant plus midostaurin. The arm description specifies midostaurin 50mg twice a day for 12 months (cycles), in addition to standard care.
Comparator
Standard of care alone in the post-transplant setting. The record does not describe additional details about the components of standard care.
Primary question
What relapse-free survival was observed up to 18 months after transplant for participants assigned to each treatment arm? The stated endpoint is assessed by Kaplan-Meier analysis in the Full Analysis Set. Arm-level estimates and a between-arm comparison are not reported in the registry record.
The clinical question concerns relapse-free survival after transplant, rather than relapse alone. The endpoint definition treats relapse or death due to the disease as an event. That means the measure is intended to capture remaining free from either of those outcomes over follow-up, as defined in the record. It does not, by itself, describe other dimensions of health or treatment experience.
The registry describes the study as randomized and phase II. It enrolled 60 participants. The available record identifies the treatment arms and the primary outcome, but it does not give the primary outcome estimates needed to assess the observed separation between arms, its precision, or a statistical comparison. The question can therefore be described from the trial design, but the clinical result cannot be established from the posted outcome information.
3. Trial Design
This was a randomized, parallel-group phase II trial with no masking. One arm received standard of care with midostaurin after stem cell transplant; the other received standard of care alone. The registry describes midostaurin as 50mg twice a day for 12 months (cycles). Enrollment was 60, and the study status is completed.
The trial’s stated purpose was to determine whether adding midostaurin to standard of care reduces relapse in FLT3-ITD mutated acute myeloid leukemia patients receiving an allogeneic hematopoietic stem cell transplant. The primary outcome was relapse-free survival up to 18 months post transplant, assessed by Kaplan-Meier analysis in the Full Analysis Set. The registry defines relapse-free survival from transplant to relapse or death due to the disease.
The record lists additional outcomes related to relapse-free survival at other time points, overall survival, non-relapse mortality, mutation status in archived material when available, and plasma pharmacokinetics. These outcome descriptions establish the range of questions the trial planned to address, but their numerical results are not reported in the registry record available for this analysis.
The registry lists a study start date of 2014-02-06 and a primary completion date of 2018-04-30. These dates describe the registered study timeline; they do not supply details about participant follow-up, data cut-off, or the timing of any particular analysis. The method used to generate the randomization sequence, any allocation concealment procedure, and the process for assigning participants are not reported in the registry record.
The study is described as open-label through the masking entry of none. In general, an unmasked design means participants and study personnel may know the assigned treatment. The record does not describe whether endpoint assessment was independently adjudicated or whether any safeguards were used to reduce the influence of treatment knowledge on outcome assessment.
The registry record does not report the planned sample-size justification or a detailed statistical analysis plan. It also does not report whether interim analyses were planned or conducted. Consequently, the design features that can be stated are limited to the registered phase, randomization, parallel groups, masking status, interventions, enrollment, and outcome descriptions.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Treatment
| Stage | Standard of Care With Midostaurin | Standard of Care |
|---|---|---|
| STARTED | 30 | 30 |
| COMPLETED | 14 | 16 |
| NOT COMPLETED | 16 | 14 |
Reasons for not completing this period:
| Reason | Standard of Care With Midostaurin | Standard of Care |
|---|---|---|
| Adverse Event | 1 | 8 |
| Withdrawal by Subject | 6 | 2 |
| Administrative problems | 4 | 1 |
| Relapse | 4 | 2 |
| Randomized, not treated | 1 | 1 |
Post Treatment Follow-up
| Stage | Standard of Care With Midostaurin | Standard of Care |
|---|---|---|
| STARTED | 26 | 27 |
| COMPLETED | 10 | 13 |
| NOT COMPLETED | 16 | 14 |
Reasons for not completing this period:
| Reason | Standard of Care With Midostaurin | Standard of Care |
|---|---|---|
| Protocol Violation | 0 | 1 |
| Withdrawal by Subject | 4 | 3 |
| Lost to Follow-up | 1 | 4 |
| Administrative problems | 2 | 0 |
| Death | 8 | 4 |
| Relapse | 1 | 2 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Standard of Care With Midostaurin | Standard of Care | Total |
|---|---|---|---|---|
| Participants analysed | 30 | 30 | 60 | |
| Age, Continuous | years | 50.8 (SD 13.68) | 46.0 (SD 11.08) | 48.4 (SD 12.58) |
| Sex: Female, Male: Female | Participants | 14 | 12 | 26 |
| Sex: Female, Male: Male | Participants | 16 | 18 | 34 |
| Race/Ethnicity, Customized: Caucasian | participants | 27 | 27 | 54 |
| Race/Ethnicity, Customized: Black | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Asian | participants | 1 | 1 | 2 |
| Race/Ethnicity, Customized: Other | participants | 1 | 2 | 3 |
| Body Mass Index | kg/m^2 | 26.9934 (SD 7.11368) | 26.6400 (SD 5.27475) | 26.8101 (SD 6.17055) |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Participants With Relapse Free Survival (RFS) up to 18 Months Post Transplant (Full Analysis Set) by Kaplan-Meier Analysis, measured over date of transplant up to 18 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | proportion of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the proportion of participants with relapse-free survival up to 18 months post transplant, assessed by Kaplan-Meier analysis. The record defines relapse-free survival as the time from transplant to relapse or death due to the disease. Relapse after complete response is defined as reappearance of leukemic blasts in peripheral blood or finding more than 5% blasts in bone marrow.
This endpoint is used to represent the duration of remaining free from the specified events. It includes relapse and disease-related death, so it addresses a broader event definition than relapse considered on its own. The endpoint description does not say that deaths from other causes count as events; the definition specifically refers to death due to the disease. Non-relapse mortality is listed separately among the secondary outcomes.
In general, Kaplan-Meier analysis estimates the probability of remaining event-free over time while accounting for participants whose event status is not observed for the entire follow-up period. Participants without a recorded event by their last available observation can be censored, meaning their observed follow-up contributes up to that point. How censoring was defined or applied in this trial is not reported in the registry record.
The endpoint name and method are recorded, but the registry record does not report the arm-specific relapse-free survival estimates, confidence intervals, or a between-arm test result. Without those results, the magnitude and precision of the primary outcome cannot be characterized.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinically focused primary outcome: relapse-free survival after transplant, with relapse or death due to the disease counted as an event. It also identifies the treatment contrast of interest: standard of care with midostaurin versus standard of care alone. However, it does not report the primary endpoint values for either arm. As a result, there is no posted estimate from which to describe the observed difference, its direction, or its size.
The endpoint is specified as a Kaplan-Meier analysis up to 18 months, but the registry record does not give the corresponding survival estimates or confidence intervals. A confidence interval communicates the statistical uncertainty around an estimate under the method used. Without the estimates and their intervals, readers cannot assess how precise the results were or which values remain compatible with the data. The record also does not report a p-value or another formal between-arm comparison for the primary outcome.
The outcome listing indicates that relapse-free survival at additional time points, overall survival, and non-relapse mortality were also considered. Their descriptions do not establish what results were observed. Numerical results for these secondary outcomes are not reported in the registry record, so they cannot be used to corroborate or qualify the primary outcome. Mutation-status and pharmacokinetic outcomes are also listed, without numerical results in the record.
The adverse-event summary contains counts for participants at risk and affected, as well as a list of serious adverse-event terms with percentages. These data are descriptive. The summary does not supply a treatment-effect estimate for safety or specify enough detail to infer how event rates compare after accounting for exposure duration, event recurrence, severity grading, or attribution. Counts alone do not show whether a treatment caused an event. The record’s overall death counts are also not interchangeable with the primary endpoint, which specifies death due to the disease.
In general, a randomized comparison can support an estimate of the effect of assignment to the study arms when the analysis and follow-up are appropriate. That general principle does not substitute for the missing results. The registry describes randomization, but does not report the randomization procedure, the primary analysis details, or the outcome estimates required to evaluate the comparison. It is therefore possible to characterize the question and design, but not to draw a conclusion about the observed primary outcome from this record.
The registry’s study status is completed, but completion does not itself provide evidence about the size, precision, or statistical significance of an outcome. The central statistical limitation is the absence of numerical primary and secondary efficacy results in the registry record. Conclusions about the clinical meaning of the assigned treatments would require results beyond the information recorded.
9. Statistical Methods Explained
The registered primary analysis uses Kaplan-Meier methods for relapse-free survival through 18 months after transplant. This is a time-to-event outcome: the analysis considers both whether an event occurred and how long participants were followed before the event or the end of their observed follow-up. The event definition is relapse or death due to the disease, with relapse following complete response described by the specified findings in blood or bone marrow.
What does Kaplan-Meier analysis do?
In general, Kaplan-Meier analysis estimates event-free probability over time while incorporating participants with different observed follow-up lengths. A participant who has no recorded event by the end of observation is not treated as if an event occurred. Instead, that participant is censored at the last point at which event status is known. This approach relies on assumptions about censoring, including that censoring is not related to the future event process in a way that would bias the estimate. The registry record does not report the censoring rules or diagnostics for this trial.
Kaplan-Meier curves can be summarized at a specified time point, such as the registered 18-month endpoint. Such an estimate depends on the number and timing of events and on how many participants remain under observation over time. The record states the time point and method but does not report the arm-level estimates, confidence intervals, numbers at risk over time, or the details needed to reproduce the analysis.
What does the confidence interval communicate?
The primary outcome description specifies a 95% confidence interval. In general, a confidence interval expresses the uncertainty around an estimated quantity under a statistical method and its assumptions. A narrower interval indicates greater precision than a wider interval, while the interval’s location indicates the range of values compatible with the estimate and method. It is not a guarantee that the true value lies within a particular interval. The confidence-interval values for the primary endpoint are not reported in the registry record.
A confidence interval for each arm would describe the precision of each arm’s estimated relapse-free survival. To evaluate a treatment contrast, readers also need an appropriate comparison estimate and its uncertainty, or a clearly reported test. Separate arm intervals are not a substitute for an interval on the between-arm contrast. The record does not report a primary comparison estimate or test result.
Why is the event definition important?
In general, a composite time-to-event endpoint combines more than one event type into a single outcome. Here, relapse and death due to the disease are included in the relapse-free survival definition. The resulting measure reflects time until either specified event, rather than giving a separate estimate for each component. Component-specific results can help show whether the composite is driven mainly by one type of event, but those primary endpoint component results are not reported in the registry record.
Relapse-free survival is distinct from overall survival and non-relapse mortality, which are listed as secondary outcomes. Overall survival concerns death, while non-relapse mortality is a separately named outcome. The registry record does not give the results or detailed event definitions for those secondary analyses. Their presence in the outcome list should not be taken as evidence of a particular result or as a replacement for the primary endpoint findings.
What can be inferred from the safety summary?
In general, safety summaries may report the number of participants experiencing an event, the number of events, or both. Those quantities are not interchangeable: a participant can experience more than one event, and a participant-level count does not indicate the total number of event occurrences. The registry’s adverse-event summary identifies participants at risk and participants affected for serious and other adverse events. It also lists selected serious-event terms as percentages. The exact event-counting conventions, exposure-adjusted rates, severity criteria, and attribution rules are not reported in the registry record.
Risk differences or ratios can be used in general to compare event occurrence between groups, but interpreting such contrasts requires clear definitions of the denominator, follow-up period, event unit, and analysis. No new safety comparison is calculated. The posted summary does not provide enough context to infer causation from the event counts or percentages alone.
What statistical planning details are absent?
The registry record does not report the sample-size justification, the planned power for the primary endpoint, the prespecified treatment-effect target, or a detailed statistical analysis plan. It also does not report whether interim analyses or multiplicity adjustments were planned. These omissions prevent an assessment of how the trial’s size and analysis strategy were intended to address the primary and secondary questions.
In general, a study’s planned sample size reflects assumptions about the outcome, target effect, variability or event frequency, significance threshold, and desired power. A planning calculation based on observed results would be illustrative rather than an account of the trial’s original design. No numerical planning results are available in the registry record for this analysis, and none can be inferred from the enrollment alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary relapse-free survival outcome and several secondary outcomes, including relapse-free survival at other time points, overall survival, non-relapse mortality, mutation status, and pharmacokinetic measures. Considering multiple outcomes or time points raises the general issue of multiplicity: when many statistical tests are performed, the chance of at least one apparently unusual result can increase. An analysis plan may address this through prespecified prioritization, adjustment, or other methods. Whether such a strategy was used is not reported in the registry record.
The record identifies the Full Analysis Set for the primary endpoint and several secondary outcomes. It does not define which randomized participants were included in that set, how eligibility for analysis was determined, or how participants with missing outcome information were handled. The handling of withdrawals, incomplete follow-up, protocol deviations, and missing covariates is not reported in the registry record. No assumption about an intention-to-treat analysis can be made from the Full Analysis Set label alone.
In general, missing data can affect both the amount of information available and the validity of an estimate. For time-to-event analyses, censoring rules describe how follow-up is used when event status is not observed through the full period. For other outcomes, methods may include complete-case analysis, imputation, or sensitivity analyses. The registry record does not specify which methods, if any, were used for missing data or whether sensitivity analyses were conducted.
The registry also does not report a detailed statistical analysis plan, interim analysis procedures, or a multiplicity strategy. As a result, the extent to which outcome definitions, analysis populations, and inferential decisions were prespecified cannot be evaluated from the record.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Standard of Care With Midostaurin | 9 | 30 | 30.0% |
| Standard of Care | 15 | 30 | 50.0% |
| ALL Patients | 24 | 60 | 40.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Standard of Care With Midostaurin | 1 of 30 |
| Standard of Care | 0 of 30 |
| ALL Patients | 1 of 60 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Standard of Care With Midostaurin | 28 of 30 |
| Standard of Care | 26 of 30 |
| ALL Patients | 54 of 60 |
Most frequent serious adverse event terms
| Event term | Organ system | Standard of Care With Midostaurin | Standard of Care | ALL Patients |
|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 4 of 30 | 2 of 30 | 6 of 60 |
| Nausea | Gastrointestinal disorders | 1 of 30 | 3 of 30 | 4 of 60 |
| Pyrexia | General disorders | 2 of 30 | 2 of 30 | 4 of 60 |
| Anaemia | Blood and lymphatic system disorders | 2 of 30 | 1 of 30 | 3 of 60 |
| Febrile neutropenia | Blood and lymphatic system disorders | 1 of 30 | 2 of 30 | 3 of 60 |
| Vomiting | Gastrointestinal disorders | 1 of 30 | 2 of 30 | 3 of 60 |
| Abdominal pain | Gastrointestinal disorders | 1 of 30 | 1 of 30 | 2 of 60 |
| Device related infection | Infections and infestations | 1 of 30 | 1 of 30 | 2 of 60 |
| Parainfluenzae virus infection | Infections and infestations | 1 of 30 | 1 of 30 | 2 of 60 |
| Pneumonia | Infections and infestations | 1 of 30 | 1 of 30 | 2 of 60 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Standard of Care With Midostaurin | Standard of Care | ALL Patients |
|---|---|---|---|---|
| Vomiting | Gastrointestinal disorders | 20 of 30 | 5 of 30 | 25 of 60 |
| Nausea | Gastrointestinal disorders | 19 of 30 | 5 of 30 | 24 of 60 |
| Oedema peripheral | General disorders | 6 of 30 | 10 of 30 | 16 of 60 |
| Fatigue | General disorders | 7 of 30 | 7 of 30 | 14 of 60 |
| Cough | Respiratory, thoracic and mediastinal disorders | 8 of 30 | 6 of 30 | 14 of 60 |
| Diarrhoea | Gastrointestinal disorders | 9 of 30 | 4 of 30 | 13 of 60 |
| Headache | Nervous system disorders | 8 of 30 | 5 of 30 | 13 of 60 |
| Aspartate aminotransferase increased | Investigations | 5 of 30 | 7 of 30 | 12 of 60 |
| Alanine aminotransferase increased | Investigations | 5 of 30 | 6 of 30 | 11 of 60 |
| Rash | Skin and subcutaneous tissue disorders | 5 of 30 | 6 of 30 | 11 of 60 |
Serious events with the highest arm-level rate: diarrhoea (13.3%), nausea (10.0%), anaemia (6.7%), febrile neutropenia (6.7%), vomiting (6.7%).
The adverse-event summary reports serious-event participants at risk and affected separately for the two arms. For standard of care with midostaurin, the summary lists 30 at risk and 9 affected; for standard of care alone, it lists 30 at risk and 15 affected. The all-patients entry lists 60 at risk and 24 affected. For other adverse events, the corresponding affected counts are 28, 26, and 54; the listed at-risk counts are 30, 30, and 60. The death counts are 1, 0, and 1, respectively.
The serious-event terms listed with percentages are diarrhoea, 13.3%; nausea, 10.0%; anaemia, 6.7%; febrile neutropenia, 6.7%; vomiting, 6.7%; and pyrexia, 6.7%. The registry record does not provide arm-specific percentages for these terms or identify the denominator used for each percentage in the summary.
These figures should be read as descriptive registry counts, not as proof that treatment caused or prevented an event. The record does not state whether affected participants could have experienced multiple events, how long participants were exposed to treatment, or the event attribution and grading rules. The death counts are not described as disease-related, so they cannot be treated as the relapse-free survival event counts.
12. What the Result Does, and Does Not, Mean
The registry record establishes the question the trial was designed to address: whether adding midostaurin to standard care after transplant was associated with relapse-free survival through 18 months in patients with FLT3-ITD mutated acute myeloid leukemia. It describes a randomized, phase II, parallel-group comparison and defines the primary endpoint. These details clarify the intended comparison and the outcome being measured.
The record does not report the primary outcome estimates for either arm, their confidence intervals, or a between-arm statistical comparison. It therefore does not establish the observed magnitude or precision of any difference in relapse-free survival. The absence of posted estimates is not evidence that the arms were the same, and it is not evidence that one arm had a better outcome. No conclusion about benefit or lack of benefit can be drawn from the record’s outcome descriptions alone.
The same limitation applies to the secondary outcomes. Their inclusion in the registry indicates that they were listed for assessment, but numerical findings are not reported in the record. The adverse-event summary offers counts and selected percentages, yet it does not establish causation or provide a complete account of exposure, recurrence, severity, or attribution. Descriptive safety counts should not be interpreted as a causal comparison without those details and an appropriate analysis.
In general, a randomized trial’s findings are interpreted by considering the estimated treatment contrast, its uncertainty, the analysis population, outcome definitions, missing data, and the design’s limitations. Here, several of those elements are not reported. The trial’s completed status and enrollment do not fill the gap left by absent efficacy results. The registry record is useful for describing the trial’s design and registered questions, but it does not provide enough outcome information to judge the primary comparison.
Any assessment of the clinical meaning of the study would require access to numerical outcome results and the accompanying analysis methods. Until those are available in the registry record or another source, the sound statistical interpretation is limited to describing what was planned and noting which results are not reported.
13. Why This Trial Matters Statistically
This trial is statistically notable because it frames a randomized comparison around relapse-free survival following allogeneic hematopoietic stem cell transplantation. The endpoint combines relapse and death due to the disease, and the registry specifies a time origin at transplant and follow-up through 18 months. Those choices define what the primary analysis is intended to estimate and what events count toward it.
The design also illustrates why the outcome definition and analysis method need to be read together. Kaplan-Meier analysis is suited in general to time-to-event data with differing follow-up, while the composite endpoint summarizes more than one event type. Interpretation would require the event-free estimates, uncertainty intervals, follow-up information, and details about censoring. The registry record does not report those numerical results or methodological details.
The listed secondary outcomes broaden the scientific questions to survival, non-relapse mortality, mutation status, and pharmacokinetics. They also raise general issues about multiple outcomes, analysis populations, and missing information. The record does not report how these statistical issues were handled. Consequently, the trial is useful for illustrating the difference between a registered analysis question and a fully reported result: design and endpoint information alone do not establish an estimated treatment effect.
The safety summary further demonstrates the distinction between event counts and comparative evidence. Counts and percentages can describe what was recorded, but interpretation depends on definitions, exposure, follow-up, and attribution. Without those details and appropriate comparative estimates, they should not be treated as causal evidence.
For biostatistical reading, the key point is that an endpoint, a design, and an analysis label are not a substitute for results. The registry record identifies the planned primary question but does not report the findings needed to quantify or judge the randomized contrast.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The principal limitation is that numerical results for the primary relapse-free survival outcome are not reported in the registry record. Arm-specific estimates, confidence intervals, and a between-arm comparison are absent. Numerical results for the listed secondary outcomes are also not reported, so the stated outcomes cannot be used to assess consistency or provide additional context for the primary question.
The record does not report how randomization was generated or concealed, nor does it give details on endpoint adjudication or the implementation of the Full Analysis Set. Although the endpoint and analysis population are named, the specific analysis rules are not reported. Censoring definitions, missing-data procedures, follow-up completeness, and sensitivity analyses are also not reported in the registry record.
The record does not include a detailed statistical analysis plan, sample-size justification, interim-analysis information, or a multiplicity strategy. This limits assessment of how the study was planned to control statistical uncertainty across its primary and secondary questions. The registry’s completed status does not resolve these omissions.
The safety information is limited to participant counts at risk and affected, selected serious-event percentages, and death counts. The registry record does not report event attribution, grading criteria, exposure-adjusted rates, event recurrence, or the denominator details for the listed percentages. These limitations restrict interpretation of comparative safety patterns and prevent causal conclusions from the descriptive summary.
The registry lists a phase II randomized design and enrollment of 60, but those details alone do not establish the precision of an outcome estimate or the study’s ability to detect a particular effect. The sample-size justification and planned statistical power are not reported. Accordingly, the most important trial-specific limitation for interpreting efficacy is the lack of posted numerical outcome findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Standard of Care +/- Midostaurin to Prevent Relapse Post Stem Cell Transplant in Patients With FLT3-ITD Mutated AML — NCT01883362.
- Registry record last updated 2021-03-17; status last verified 2021-03.
- Results first posted 2021-03-17.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT01883362 is a completed, randomized phase II, parallel-group study with no masking. It enrolled 60 participants with acute myeloid leukemia and examined standard of care with or without midostaurin after allogeneic hematopoietic stem cell transplantation for FLT3-ITD mutated disease. The study start date is 2014-02-06, and the primary completion date is 2018-04-30.
The primary outcome is relapse-free survival up to 18 months post transplant, assessed by Kaplan-Meier analysis in the Full Analysis Set. The endpoint is defined as time from transplant to relapse or death due to the disease. The registry also lists secondary survival, non-relapse mortality, mutation-status, and pharmacokinetic outcomes.
The posted record does not report numerical primary or secondary efficacy results, a between-arm primary comparison, or detailed statistical methods for missing data, multiplicity, and analysis populations. Its adverse-event summary provides descriptive counts and selected serious-event percentages, but not enough detail for causal interpretation.