Omacetaxine Mepesuccinate, Cytarabine, and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Omacetaxine Mepesuccinate, Cytarabine, and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia (NCT02029417), a na phase 2 study. The primary outcome is Proportion of the Evaluable Population of Interest Who Experience a Complete Response in the Poor and Good Prognosis Groups.

2. Clinical Question

3. Trial Design

This was a phase II, single-group study sponsored by Roswell Park Cancer Institute. The registry lists allocation as not applicable and masking as none. These features mean the design did not assign participants between randomized treatment groups and did not use masking. There is no concurrent control group against which to estimate a treatment contrast.

The study addressed older patients with newly diagnosed acute myeloid leukemia. Its official title specifies patients at least 65 years of age, while the listed conditions include multiple AML subtypes and secondary AML. The registry lists enrollment as 2. It records a start in 2014-07, primary completion in 2015-11, and a terminated status. The stated reason relevant to the primary endpoint is early termination and low accrual; further reasons for termination are not reported in the registry record.

The named interventions include cytarabine, omacetaxine mepesuccinate, decitabine, and laboratory biomarker analysis. The treatment arm description gives induction details for cytarabine and omacetaxine: both were administered subcutaneously twice daily on days 1–14. Induction was to repeat every 28 days for up to 4 courses or until complete response in the absence of progression or unacceptable toxicity. The arm description ends at a consolidation-therapy heading without describing a consolidation regimen. Although decitabine is listed among the interventions and in the study title, the available arm text does not specify its dosing or schedule. The protocol’s full treatment details are not reported in the registry record.

The primary endpoint timeframe was up to 4 years. The registry describes exact binomial probabilities in a two-stage design for the response assessment, but it does not report whether or how that design was implemented before termination. The detailed protocol, rationale for the enrollment target, and statistical analysis plan are not reported in the registry record.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Due to the study's early termination and low accrual, data were not collected for this assessment.

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: All treated and eligible patients

6. Primary Endpoint

The registry lists the primary outcome as Proportion of the Evaluable Population of Interest Who Experience a Complete Response in the Poor and Good Prognosis Groups, measured over Up to 4 years.

The primary endpoint was the proportion of the evaluable population of interest in the poor- and good-prognosis groups who experienced complete response. The registry defines complete response using recovery of morphologically normal bone marrow, with fewer than 5% blasts, and recovery of blood counts: an absolute neutrophil count of at least 1x10^9/L and platelet counts of at least 100x10^9/LO. The definition also requires rare circulating leukemic blasts or no evidence of extramedullary disease. The platelet unit appears as recorded in the registry.

A complete-response endpoint combines disease assessment in the marrow with evidence of blood-count recovery. In general, this kind of endpoint is used to describe whether a participant meets a prespecified clinical and laboratory response definition, rather than to describe a change in only one measurement. The endpoint definition therefore matters: a response proportion can be interpreted only in relation to the criteria and the population assessed.

The record specifies an assessment timeframe of up to 4 years and says responses would be tabulated using exact binomial probabilities in a two-stage design. It does not provide the number of evaluable participants, the number of responses, or an estimated response proportion. It explicitly states that data were not collected for the primary assessment because the study terminated early and accrual was low. The registry therefore records the endpoint definition and intended analysis, but not an observed primary endpoint result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies a single-group model, with allocation not applicable and no masking. In general, a single-group study can describe outcomes within the treated group, but it does not create a randomized comparison between treatments. A randomized contrast helps separate treatment differences from other differences between groups; this record has no such concurrent contrast. Therefore, no relative treatment effect against a control can be estimated from the design described in the registry.

The interventions include cytarabine, omacetaxine mepesuccinate, decitabine, and laboratory biomarker analysis. The arm text describes induction with subcutaneous cytarabine and omacetaxine twice daily on days 1–14, repeating every 28 days for up to 4 courses or until specified clinical stopping conditions. It does not provide the consolidation regimen, and it does not specify how decitabine was administered. Statistical interpretation depends on knowing what treatment participants actually received, but those additional details are not reported in the registry record.

How should the primary endpoint be understood?

The primary endpoint is a binary response outcome: a participant either meets the complete-response definition or does not. The planned summary was the proportion of the evaluable population of interest experiencing complete response, considered across the poor- and good-prognosis groups. In general, a proportion describes the frequency of an outcome within a defined population. Its interpretation depends on the response criteria, which participants count in the denominator, and whether outcome data are available for that population.

The stated definition requires morphologically normal bone marrow with fewer than 5% blasts, specified blood-count recovery, and rare circulating leukemic blasts or no evidence of extramedullary disease. The registry gives a timeframe of up to 4 years. These criteria help define what qualifies as a response; they do not indicate that any participant met them. The record explicitly says that primary endpoint data were not collected because the study terminated early and accrual was low.

What does the exact binomial description mean?

The registry says the response assessment was to use exact binomial probabilities in a two-stage design, with the number of responses tabulated. In general, a binomial model represents a binary outcome across a group, such as response or no response, and exact methods calculate probabilities without relying on a large-sample approximation for that outcome. This can be useful when data are sparse. A confidence interval, when calculated, communicates uncertainty around an estimated quantity; wider intervals generally indicate less precision. No response estimate or interval is reported for this endpoint.

A two-stage design can, in general, define an assessment at an initial stage and a later stage, with a decision rule that may determine whether the study continues. The registry’s description does not report the stage-specific thresholds, decision rules, or whether any such interim assessment occurred. The existence of a two-stage design description should not be interpreted as evidence that interim analyses were performed. Details of the statistical plan are not reported in the registry record.

How should planning calculations be separated from trial results?

Any sample-size illustrations associated with the posted material are planning calculations, not outcomes observed in this study. Their stated assumptions include a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect defined as a stated fraction of an observed effect. For time-to-event illustrations, the planning quantity is the total number of events; for other illustrations, it is participants per arm. These assumptions do not describe the actual single-group design, and the registry does not report that they were the trial’s design assumptions.

In general, a sample-size calculation asks how much information a hypothetical study might need under specified assumptions. It does not measure the power achieved by a completed study. Any sample sizes for participants per arm for 80% power are planning illustrations, not observed power. Here, the absence of a reported primary response effect also prevents treating an observed response result as the basis for a clinically grounded planning interpretation. The illustrations cannot replace the missing endpoint assessment.

What can be learned from the adverse-event summaries?

The adverse-event entries are counts and a listed serious-event term for the single treatment group. In general, adverse-event summaries are descriptive: they report events under the registry’s recording and grouping conventions. They do not by themselves establish that treatment caused an event or quantify risk relative to an untreated or alternative-treatment group. Comparisons of event rates require comparable groups and clear definitions of the reporting period and population.

The record gives the serious- and other-event group entries, and lists sepsis as the top serious term at 100.0%. It does not report event-level timing, repeat events, attribution, or the relationship between the serious and other categories. These limitations constrain interpretation of the safety entries. No deaths are recorded in the group’s death field; the reason that field is empty is not reported in the registry record.

10. Multiplicity, Missing Data and Analysis Populations

The registry record does not report a multiplicity adjustment or a plan for controlling error across multiple endpoints, subgroups, or analyses. It describes the primary response endpoint for poor- and good-prognosis groups and lists 2 outcome measures, but the details and results of the other measure are not reported in the registry record. The absence of a reported multiplicity plan does not show whether one was present in the protocol; it means the record does not allow that assessment.

For the primary endpoint, the stated analysis was to tabulate the number of responses using exact binomial probabilities in a two-stage design. The registry does not report stage-specific rules, an analysis population definition beyond the phrase evaluable population of interest, or any completed analysis. The planned timeframe was up to 4 years. The record states that data were not collected for the primary assessment because the study terminated early and accrual was low.

Missing outcome data and data that were never collected for an assessment are not interchangeable with a recorded nonresponse. In general, an analysis must specify how participants with unavailable endpoint measurements are handled, because different choices can change the interpretation of an estimated proportion. The registry does not report missing-data procedures, exclusions from the evaluable population, or the number assessed for the primary endpoint. It would therefore be inappropriate to infer that participants without recorded assessment failed to respond, or to reconstruct a denominator or response rate from enrollment alone.

The record also does not describe interim analyses beyond the design’s reference to two stages, nor does it explain whether any stage-based decision was reached. A sample-size illustration is not an analysis of missingness or a substitute for an analysis population definition. These gaps leave the intended inferential procedure and the disposition of endpoint data unresolved in the public record.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: sepsis (100.0%).

The registry lists one treatment group for safety. Its fields record 2 participants at risk and 2 affected for serious adverse events, and the same entries for other adverse events. Sepsis is listed as the top serious adverse-event term at 100.0%. These are descriptive entries from a small, single-group record, not a comparison with another treatment or a causal estimate.

The percentage attached to sepsis should be interpreted only in the context of the registry’s serious-event listing. The record does not explain the denominator definition for that term, provide event-level details, or state whether participants could contribute more than one event. It also does not report timing, duration, attribution, severity beyond the serious-event classification, or recurrence. The entries do not establish whether an event was caused by the study treatment.

The death field is null; deaths are therefore not reported in the registry record. A null entry should not be read as confirmation that no deaths occurred. Since the study was single-group and terminated early, the safety summary cannot support a comparative risk assessment or a broad estimate for other populations. It records what was entered for this group, with limited detail for interpretation.

12. What the Result Does, and Does Not, Mean

The registry record is informative about the study’s design and its incomplete primary assessment. It identifies a phase II, single-group investigation of a regimen including cytarabine, omacetaxine mepesuccinate, and decitabine in older patients with newly diagnosed AML. It also records that the study was terminated, enrollment was 2, and primary endpoint data were not collected because of early termination and low accrual. Those details describe the status of the evidence in the record.

The record does not provide an observed complete-response proportion. It does not report a response count, a denominator for the evaluable population, or a confidence interval. Without those data, the primary endpoint cannot be estimated from this registry entry. Enrollment alone is not a response outcome, and absence of an assessment should not be recast as response or nonresponse.

Nor does the record offer a randomized comparison. The single-group design cannot show how outcomes would have differed under another treatment. Even a reported outcome in the treatment group would need cautious interpretation because the design lacks a concurrent control. The registry also leaves aspects of the regimen unclear: the listed interventions include decitabine, but its schedule is not specified in the available arm description, and the consolidation heading has no accompanying details.

The serious and other adverse-event figures are descriptive and come from the same treatment group. They do not provide a comparative safety estimate, and the record lacks detail on event timing, attribution, recurrence, and the meaning of the null death field. The listed sepsis percentage should not be interpreted as a treatment effect.

In general, statistical precision depends on the amount and completeness of outcome information. Here, the main limitation is more fundamental than imprecision: the registry states that the primary endpoint data were not collected. Any planning illustration is not an observed result or observed power. The record therefore documents a study question, treatment description, design, and limited safety entries, but it does not answer whether the planned primary response outcome occurred.

13. Why This Trial Matters Statistically

This study matters statistically because it illustrates how a clearly stated endpoint can remain unanswered when the outcome assessment is not collected. The registry specifies a complete-response definition and an intended exact binomial, two-stage approach. Yet it also states that early termination and low accrual meant the primary assessment data were not collected. A prespecified outcome and method cannot yield an estimate without the observations needed to apply them.

The single-group structure is another important feature. A one-group study can describe outcomes among participants receiving a regimen if those outcomes are recorded, but it does not provide a randomized comparator. That distinction matters when readers encounter treatment descriptions and safety counts: descriptive observations are not equivalent to estimates of comparative benefit or harm.

The record also demonstrates why analysis populations and missing-data handling need explicit reporting. The primary endpoint is framed around an evaluable population, but the registry does not define that population in detail or report its size. Because no primary assessment data were collected, the record cannot establish how many participants would have entered the endpoint denominator or how unavailable measurements would have been handled.

Finally, planning calculations and study evidence answer different questions. Planning illustrations can explore hypothetical sample-size requirements under stated assumptions; they cannot recover missing trial outcomes or show achieved power. In this record, the registry’s information supports a careful account of design and data availability, while the main response question remains unresolved. That distinction is essential for reading small, early-terminated studies without overstating what their records establish.

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 most consequential limitation is that the registry explicitly reports no collected data for the primary complete-response assessment because the study ended early and accrual was low. The record lists enrollment as 2, but does not report the number evaluable for the endpoint, any response count, or an estimate of the response proportion. The primary question therefore cannot be answered from the recorded outcome information.

The study was single-group, with no masking and allocation listed as not applicable. There is no concurrent control group, so the record cannot support a randomized comparison. Its terminated status further limits the available evidence. The reason for termination beyond the stated early termination and low accrual is not reported in the registry record.

The treatment description is incomplete. It gives induction details for cytarabine and omacetaxine, while decitabine is listed as an intervention without a schedule in the available arm text. The arm description ends at a consolidation heading without describing consolidation treatment. The record also does not provide the detailed protocol, the enrollment rationale, or a full statistical analysis plan.

Important endpoint-analysis details are absent, including a detailed definition of the evaluable population, missing-data procedures, stage-specific rules for the two-stage design, and whether any interim assessment occurred. The safety summary has limited event-level detail, and the death field is null. These gaps prevent a fuller assessment of the endpoint analysis, treatment exposure, and safety experience. They are limitations of what the registry record reports, not evidence that a particular unreported procedure or event did or did not occur.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Omacetaxine Mepesuccinate, Cytarabine, and Decitabine in Treating Older Patients With Newly Diagnosed Acute Myeloid Leukemia — NCT02029417.
• Registry record last updated 2016-05-09; status last verified 2016-04.
• Results first posted 2016-05-09.
• 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.

18. Record Summary

The registry describes a phase II, single-group study sponsored by Roswell Park Cancer Institute, investigating omacetaxine mepesuccinate, cytarabine, and decitabine for older patients with newly diagnosed AML. The official title specifies patients at least 65 years of age. The record lists enrollment as 2, a start in 2014-07, primary completion in 2015-11, and terminated status.

The primary endpoint was the proportion of the evaluable population of interest in the poor- and good-prognosis groups experiencing complete response, with a timeframe of up to 4 years. The record says the primary assessment data were not collected because of early termination and low accrual. No primary response estimate is reported.

The treatment arm description specifies induction with subcutaneous cytarabine and omacetaxine twice daily on days 1–14, repeated every 28 days for up to 4 courses or until stated stopping conditions. The record lists 2 at risk and 2 affected in both serious and other adverse-event categories and identifies sepsis at 100.0% among the top serious terms. It does not report deaths in the group field.