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 Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation (NCT02634827), a na phase 2 study. The primary outcome is Proportion of Complete Responses to Therapy, Where a Success is Defined as a CR or CRi as the Objective Status.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02634827 |
| Phase | Phase 2 |
| Condition | Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, FLT3 Tyrosine Kinase Domain Point Mutation |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Mayo Clinic |
| Results posted | Yes |
2. Clinical Question
The study asked whether a combination of midostaurin and decitabine could produce complete response or complete response with incomplete recovery in older patients newly diagnosed with acute myeloid leukemia and a FLT3 mutation. Its primary endpoint was the proportion of evaluable patients whose objective status was a complete response or complete response with incomplete recovery, assessed up to 2 years.
Population
Older patients newly diagnosed with acute myeloid leukemia and FLT3 mutation. The record also lists several related conditions, including FLT3/ITD mutation, FLT3 tyrosine kinase domain point mutation, secondary acute myeloid leukemia, and untreated adult acute myeloid leukemia. Only one patient was accrued, and patient-level details are withheld.
Intervention
Decitabine and midostaurin were given as a combination. The arm description specifies intravenous decitabine over 1 hour on days 1-5 and oral midostaurin twice daily on days 8-21 of courses 1 and 2, then on days 1-28 of each subsequent course.
Comparator
There was no separate comparison group. The study is listed as single-group and was not randomized, so it does not compare the combination with a control treatment.
Primary question
Among evaluable patients, what proportion achieved complete response or complete response with incomplete recovery? The registry describes estimating this proportion and calculating exact binomial confidence intervals, but the result for the accrued patient is not reported in the registry record.
3. Trial Design
This was a phase II, single-group study sponsored by Mayo Clinic. The registry lists the study as open-label, with no masking, and no randomized allocation. As a result, the design describes treatment with the combination rather than a randomized comparison between treatments. The study status is terminated.
The registry lists enrollment of 1. Its recorded start date is 2015-12-30, and the primary completion date is 2017-03-18. The primary endpoint timeframe is up to 2 years. The record does not state why the study was terminated, so the reason should not be inferred from the status or the enrollment figure.
The treatment arm combined decitabine and midostaurin. Decitabine was administered intravenously over 1 hour on days 1-5. Midostaurin was administered orally twice daily on days 8-21 of courses 1 and 2, and on days 1-28 of each subsequent course. The arm description is incomplete after mentioning patients who fail to achieve specified response states; further treatment details are not reported in the registry record.
Listed secondary outcomes included duration of complete response, adverse events, overall survival, overall response rate, and progression-free survival. The registry lists 8 outcome measures, but the posted results contain no analyses. Thus, the design information establishes the intended treatment setting and response question, while it does not provide a comparative estimate of treatment effects or a reported clinical outcome.
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.
Overall Study
| Stage | Treatment (Decitabine, Midostaurin) |
|---|---|
| STARTED | 1 |
| COMPLETED | 1 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.
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.
Population: Since only one patient was accrued, patient confidentiality prevents the reporting of this patient.
| Characteristic | Unit | Treatment (Decitabine, Midostaurin) |
|---|---|---|
| Participants analysed | 0 | |
| Sex: Female, Male: Female | — | |
| Sex: Female, Male: Male | — | |
| Race (NIH/OMB): American Indian or Alaska Native | — | |
| Race (NIH/OMB): Asian | — | |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | — | |
| Race (NIH/OMB): Black or African American | — | |
| Race (NIH/OMB): White | — | |
| Race (NIH/OMB): More than one race | — | |
| Race (NIH/OMB): Unknown or Not Reported | — |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Complete Responses to Therapy, Where a Success is Defined as a CR or CRi as the Objective Status, measured over Up to 2 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the proportion of evaluable patients whose objective status is complete response or complete response with incomplete recovery. A success is defined as either of those response categories. The registry describes estimating the proportion by dividing the number of successes by the total number of evaluable patients, with exact binomial confidence intervals for the true success proportion. The assessment timeframe is up to 2 years.
This endpoint expresses response as a proportion rather than as a time-to-event measure. In general, a response proportion is useful for describing how often a prespecified response state is reached within the study’s assessment framework. The confidence interval is intended to show the uncertainty around that estimate, not just its central value.
In this record, the endpoint cannot be evaluated from the posted results. Although enrollment is listed as 1, the primary outcome description states that the patient’s information is withheld for confidentiality. The response count, evaluable-patient denominator, estimated proportion, and confidence interval are therefore not reported in the registry record. No response rate can be inferred from enrollment alone.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a phase II study of a midostaurin and decitabine combination in older patients newly diagnosed with acute myeloid leukemia and FLT3 mutation. It was a single-group, unmasked study, not a randomized comparison. Its primary question concerned the proportion of evaluable patients achieving complete response or complete response with incomplete recovery.
The key interpretive constraint is that only one patient was accrued and the primary outcome description says that the patient is not reported because of confidentiality. The posted results therefore do not give the number of successes, the number of evaluable patients, a response proportion, or a confidence interval. The study record does not support a quantitative conclusion about the primary endpoint. In particular, enrollment is not itself a response result, and the absence of an outcome estimate must not be treated as either a response or a nonresponse.
The registry lists 8 outcome measures and 0 analyses. Secondary outcomes include duration of complete response, adverse events, overall survival, overall response rate, and progression-free survival. Numerical results for these measures are not reported in the registry record. The adverse-event record lists zero patients at risk and zero affected in its group counts, with no serious-event terms. Given the confidentiality statement and the lack of reported analyses, those entries do not establish that no adverse events occurred; the posted record does not give an interpretable event estimate.
The study is marked terminated, but a reason for termination is not reported in the registry record. The enrollment count and status do not establish why recruitment stopped or what treatment experience occurred. The record also does not report an analysis comparing the regimen with another treatment, so no relative effect can be estimated from it.
Overall, the record documents the study design, treatment schedule, endpoint definition, and the confidentiality-related withholding of the only patient’s primary outcome. It does not provide response or safety estimates that can be interpreted as observed results. Any conclusions about the magnitude or precision of the response proportion, or about secondary outcomes, would go beyond the posted data.
9. Statistical Methods Explained
The registry describes the primary outcome as a proportion: the number of evaluable patients with complete response or complete response with incomplete recovery divided by the total number of evaluable patients. The success categories are prespecified in the endpoint definition. The analysis population is therefore described in terms of evaluability, rather than simply all enrolled patients. The registry does not provide the operational criteria for evaluability or a detailed account of how response was assessed.
What does an exact binomial confidence interval describe?
General statistical explanation: A proportion is calculated from a count of successes and a denominator. A confidence interval expresses the uncertainty associated with estimating an underlying response proportion from observed participants. An exact binomial interval uses the binomial sampling model and an exact procedure rather than relying on a large-sample approximation. The registry says this type of interval would be calculated for the primary success proportion. However, the success count and evaluable denominator are not reported, so neither the estimate nor its interval can be recovered from this record.
The distinction between a point estimate and its interval matters. A point estimate alone would describe the observed response fraction in the evaluable group; the interval would indicate how imprecisely that fraction estimates an underlying proportion. With the only patient’s primary outcome withheld, neither component is available. The planned method does not substitute for an observed estimate.
How should the design shape interpretation?
The study is listed as single-group, with no randomized allocation and no masking. In general, a single-group study can describe outcomes among participants receiving the study regimen, but it does not supply a concurrent randomized control group for estimating a comparative treatment effect. This record has no reported primary outcome estimate to describe even on that single-group basis. The absence of a comparator also means that any outcome, if reported, would need to be interpreted as an observed experience in the study group rather than as a randomized contrast.
The study is described as phase II. The registry does not report a formal sample-size justification, a stopping rule, interim analyses, or a detailed statistical analysis plan. Those items should not be inferred from the phase label or the outcome description.
What do the planning assumptions mean?
The planning illustration is distinct from the trial’s actual design. Its stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is defined as a stated fraction of the observed effect. The record explicitly characterizes this as a planning illustration computed from the observed effect, not as the trial’s own design. It is therefore not evidence that this single-group study enrolled two arms or used those assumptions to set its enrollment target.
General statistical explanation: Power planning asks how many participants would be needed, under specified assumptions, to detect an effect of a stated size with a chosen probability. Such a calculation is conditional on those assumptions. If the effect used as a reference is estimated from data, planning around a fraction of that effect illustrates hypothetical future-study requirements; it does not show the probability that the completed study found a real effect. These quantities are not observed power, and they cannot replace an estimate and uncertainty interval for the study’s actual primary endpoint.
The planning description distinguishes time-to-event outcomes from other outcomes: time-to-event planning gives a total number of events using the Schoenfeld approach, whereas other planning results are expressed as participants per arm. This is a general distinction in the stated planning method, not a description of this trial’s enrollment or analysis. No numerical planning outputs or row-specific estimates are reported in the registry record.
What can be said about secondary outcomes?
The registry lists duration of complete response, adverse events graded according to NCI CTCAE Version 4.0, overall survival, overall response rate, and progression-free survival among the secondary outcomes. General statistical explanation: A duration endpoint concerns the time between a defined response state and a subsequent event, while overall survival and progression-free survival are time-to-event outcomes. Their analysis ordinarily depends on information about event times and follow-up. Those data and analyses are not reported in the registry record, so no secondary endpoint estimate can be interpreted.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 8 outcome measures, including a primary response proportion and several secondary outcomes. It does not report a plan for controlling multiplicity across outcomes, a hierarchy for testing them, or any other multiplicity procedure. The number of analyses is listed as 0. Therefore, the record does not establish whether any statistical tests were performed or how multiple outcome questions would have been handled.
The primary outcome is defined for evaluable patients, but the criteria for inclusion in that analysis and the handling of missing response assessments are not reported in the registry record. The only patient’s primary outcome is withheld for confidentiality. Consequently, the number of evaluable patients, the amount or pattern of missing endpoint information, and any missing-data method cannot be determined from the posted results.
The registry does not report an intention-to-treat analysis population, a per-protocol population, or another analysis set beyond its description of evaluable patients for the primary endpoint. It also does not describe imputation, sensitivity analyses, or procedures for incomplete follow-up on the secondary outcomes. These are not details that can be assumed from the endpoint definition.
General statistical explanation: When multiple outcomes are tested, the chance of at least one false-positive finding can depend on how many questions are examined and how the tests are conducted. Missing outcome data can also affect an estimate if the observed participants differ from those whose outcomes are unavailable. These are reasons to look for prespecified analysis and missing-data methods. In this record, the relevant methods are not reported, and the primary result itself is unavailable, so no multiplicity-adjusted or missing-data-sensitive conclusion can be drawn.
11. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of midostaurin and decitabine enrolled 1 patient and defined a primary endpoint based on complete response or complete response with incomplete recovery. It also specifies an intended treatment schedule and states that the patient’s primary outcome is withheld for confidentiality.
It does not establish the observed response proportion, the response count, or the evaluable-patient denominator. The exact binomial confidence interval described for the endpoint is not reported as a result. Thus, the record cannot quantify either the observed response experience or the precision of an estimate. It does not show that the regimen did or did not produce a response.
The study’s single-group design does not provide a randomized comparison. Even if an outcome estimate were available, the design would not by itself estimate how outcomes differed from those under a separate control treatment. Here, the primary outcome is not available, and no secondary outcome analyses are posted.
The adverse-event entries should be read cautiously. The registry lists zero at risk and affected counts for the group and no serious-event terms, but it does not provide an interpretable adverse-event analysis alongside the confidentiality statement and the absent analyses. These entries should not be converted into a conclusion that no adverse events occurred.
General statistical explanation: A registry record is a structured account of a study and its posted results, not a substitute for outcome data that are not reported. A defined endpoint and a stated method describe what was intended to be measured; they do not reveal the result. Likewise, a planning illustration is about hypothetical future sample sizes under stated assumptions, not a finding about the treatment experience in the enrolled patient. The evidence available in this record is therefore descriptive of study conduct and reporting limits, not an estimate of efficacy or safety.
12. Why This Trial Matters Statistically
This study illustrates why the distinction between a planned endpoint and a reported result matters in clinical-trial interpretation. The registry defines a clear response-based primary question and specifies an exact binomial confidence interval, but the response information for the only accrued patient is withheld. Without the numerator and denominator, there is no response proportion to interpret and no way to assess its precision.
The single-group design is also important statistically. It can frame a question about outcomes among treated participants, but it does not create a randomized comparison with another treatment. This affects what kind of inference the study could support even if results were available. In this record, the absence of a reported endpoint estimate further limits inference: neither an absolute response estimate nor a comparative effect is available.
The study’s listed secondary measures show that the intended assessment included response duration, adverse events, overall survival, overall response rate, and progression-free survival. Yet the registry reports no analyses. A list of outcomes does not establish that results were observed, analyzed, or reported. This distinction is especially relevant when a record contains numerical entries such as zero event counts that cannot be interpreted as a complete safety result in isolation.
The sample-size material has a separate statistical role. It is explicitly described as a planning illustration rather than the trial’s own design, with assumptions that include two arms even though this study is single-group. Keeping those purposes separate prevents hypothetical planning outputs from being mistaken for trial enrollment, observed power, or clinical findings.
The statistical value of this record is therefore primarily methodological: it documents the study question and illustrates the limits imposed by a very small accrued group and confidential outcome reporting. It cannot support a quantitative conclusion about the response endpoint or the secondary outcomes.
13. 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 the only accrued patient’s primary outcome is withheld for confidentiality. Although the registry gives an enrollment of 1, it does not report whether that patient achieved a complete response or complete response with incomplete recovery. The primary estimate, its denominator, and its confidence interval are unavailable, leaving the central study question unanswered by the posted results.
The study was single-group and not randomized. There is no separate control group in the record, and no comparative treatment estimate can be derived. The unmasked design is also stated in the registry. The record does not report whether or how outcome assessment was protected from knowledge of treatment.
Secondary outcome results and analyses are not reported in the registry record. This includes numerical findings for response duration, adverse events, overall survival, overall response rate, and progression-free survival. The adverse-event count entries cannot be taken as evidence that no events occurred, particularly because the record does not provide an interpretable analysis for the sole accrued patient.
Important methodological details are not reported in the registry record, including a sample-size justification for the trial, criteria for evaluability, methods for handling missing outcomes, multiplicity procedures, and a detailed statistical analysis plan. The reason for termination is also not reported. These absences limit assessment of how the study was planned, conducted, and analyzed; none should be filled in by assumption.
14. Related Tutorials
15. Related Statistical Calculators
16. Sources
- ClinicalTrials.gov: Midostaurin and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia and FLT3 Mutation — NCT02634827.
- Registry record last updated 2019-08-07; status last verified 2018-06.
- Results first posted 2019-08-07.
- 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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Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
17. Record Summary
NCT02634827 is a phase II, single-group study sponsored by Mayo Clinic, titled as a study of midostaurin and decitabine in older patients newly diagnosed with acute myeloid leukemia and FLT3 mutation. The record lists no masking, no randomized allocation, enrollment of 1, and study status as terminated. The recorded start date is 2015-12-30, and the primary completion date is 2017-03-18.
The primary endpoint is the proportion of evaluable patients achieving complete response or complete response with incomplete recovery, with a timeframe of up to 2 years. The registry describes exact binomial confidence intervals, but the accrued patient’s outcome is withheld for confidentiality. The record lists 8 outcome measures and 0 analyses; numerical results for the primary and secondary outcomes are not reported in the registry record.
The posted information documents the study design, treatment schedule, and endpoint definition. It does not provide an estimable response proportion, a treatment comparison, or interpretable outcome estimates.