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 AML-02: Omacetaxine With Standard-of-Care Induction With Cytarabine & Idarubicin in Newly-Diagnosed AML Patients (NCT02440568), a non-randomized phase 1, phase 2 study. The primary outcome is Optimally Tolerated Dose.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02440568 |
| Phase | Phase 1, Phase 2 |
| Condition | Acute Myeloid Leukemia |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Illinois Chicago |
| Results posted | Yes |
2. Clinical Question
Population
Patients with newly diagnosed acute myeloid leukemia (AML) were eligible for the study. The registry lists an enrollment of 22. The posted safety information is organized by four dose cohorts; it does not provide further participant characteristics in the record.
Intervention
Omacetaxine mepesuccinate was added to standard induction with cytarabine and idarubicin. The record describes a dose-escalation study, with omacetaxine dose levels of 0.625mg/m^2, 1.25mg/m^2, 2.0mg/m^2, and 3.0mg/m^2 across the cohorts.
Comparator
There was no randomized control group. The registry identifies the allocation as non-randomized and the study model as sequential. The cohorts represent different omacetaxine dose levels, not a like-for-like comparison against induction without omacetaxine.
Primary question
The primary question was which omacetaxine dose level would meet the registry’s criteria for an optimally active and safe dose when added to induction chemotherapy. The definition required fewer than 30% of patients to experience hematologic toxicity and greater than 50% to achieve complete remission (CR). The posted primary endpoint result is not reported in the registry record.
This is a dose-finding question rather than a randomized test of whether adding omacetaxine improves outcomes. The criteria combine a safety threshold with a response threshold. The registry also lists toxicity assessed using CTCAE grading, time to hematologic recovery, overall participant survival, event-free survival, and progression-free survival as secondary outcomes. Their results are not reported in the registry record.
3. Trial Design
The registry identifies AML-02 as a phase 1 and phase 2 study sponsored by the University of Illinois Chicago. It was non-randomized, had no masking, and used a sequential model. Its stated purpose was to evaluate omacetaxine in combination with a standard induction regimen in patients with newly diagnosed AML. The registry lists enrollment of 22 and a status of terminated. The record gives a start date of 2015-06-05 and a primary completion date of 2018-11-30; it does not report why the study was terminated.
The induction regimen is described as “7+3”: cytarabine for Days 1-7 and idarubicin for Days 1-3. Omacetaxine was added at dose levels of 0.625mg/m^2, 1.25mg/m^2, 2.0mg/m^2, and 3.0mg/m^2 in four sequential cohorts. Because allocation was not randomized, differences between cohorts cannot be interpreted as if participants had been assigned to comparable treatment groups. The registry does not report a concurrent control arm receiving the induction regimen without omacetaxine.
The arm descriptions say participants would be followed in outpatient clinic at least every 2 months for a total of 6 months, with a final study visit 6 months (+/-1 week) after the last omacetaxine dose. These are descriptions of the planned follow-up, not evidence that every participant completed it. Details about adherence, actual treatment received, and the number followed for each outcome are not reported in the registry record. The study’s sequential dose-escalation structure is relevant to interpreting the cohort counts: cohort membership is tied to dose level, not to random assignment intended to balance prognostic factors.
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 | Patients With Newly Diagnosed AML, Cohort 1 | Patients With Newly Diagnosed AML, Cohort 2 | Patients With Newly Diagnosed AML, Cohort 3 | Patients With Newly Diagnosed AML, Cohort 4 |
|---|---|---|---|---|
| STARTED | 5 | 4 | 10 | 3 |
| COMPLETED | 1 | 1 | 5 | 1 |
| NOT COMPLETED | 4 | 3 | 5 | 2 |
Reasons for not completing this period:
| Reason | Patients With Newly Diagnosed AML, Cohort 1 | Patients With Newly Diagnosed AML, Cohort 2 | Patients With Newly Diagnosed AML, Cohort 3 | Patients With Newly Diagnosed AML, Cohort 4 |
|---|---|---|---|---|
| Death | 1 | 3 | 4 | 2 |
| Lack of Efficacy | 2 | 0 | 1 | 0 |
| Lost to Follow-up | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Number of participants in each cohort
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 | Patients With Newly Diagnosed AML Cohort 1 | Patients With Newly Diagnosed AML Cohort 2 | Patients With Newly Diagnosed AML Cohort 3 | Patients With Newly Diagnosed AML Cohort 4 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 5 | 4 | 10 | 3 | 22 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 5 | 4 | 7 | 2 | 18 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 3 | 1 | 4 |
| Sex: Female, Male: Female | Participants | 2 | 2 | 7 | 2 | 13 |
| Sex: Female, Male: Male | Participants | 3 | 2 | 3 | 1 | 9 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 1 | 4 | 0 | 6 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 4 | 3 | 6 | 3 | 16 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 2 | 0 | 0 | 0 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 1 | 3 | 1 | 6 |
| Race (NIH/OMB): White | Participants | 2 | 3 | 7 | 2 | 14 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | Participants | 5 | 4 | 10 | 3 | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Optimally Tolerated Dose, measured over Within 50 days (duration of hematologic recovery).
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants treated with OD |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is titled “Optimally Tolerated Dose.” Its unit is participants treated with OD, and its stated population is the number of participants in each cohort. The description frames the objective as identifying an optimally active and safe omacetaxine dose when added to standard induction chemotherapy, while estimating efficacy and response rate. The registry defines OD using both hematologic toxicity and complete remission: fewer than 30% of patients should experience hematologic toxicity, while greater than 50% should achieve CR. The timeframe is within 50 days, described as the duration of hematologic recovery.
Using a combined safety-and-response criterion reflects the dose-finding purpose: the selected dose is intended to satisfy both parts of the stated definition, rather than to maximize response without regard to toxicity. In general, a threshold-based endpoint can support a decision about dose selection, but it does not by itself establish a comparative treatment effect. The endpoint also depends on how hematologic toxicity and CR were assessed and on which participants were included in each evaluation; those analysis details are not reported in the registry record.
The posted record does not report the number meeting the OD criteria, the selected dose, or a primary endpoint estimate. The endpoint definition therefore describes what the study set out to determine, not the result of that determination.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a small, non-randomized, sequential dose-escalation study, with enrollment of 22 across four omacetaxine dose cohorts. The study’s intended contribution was to identify a dose meeting its combined safety and response criteria. However, the posted primary outcome does not give the cohort-level response results or state which dose, if any, met the definition. The registry record therefore does not support a conclusion about whether the dose-finding objective was achieved.
The safety information reports counts by cohort. For serious adverse events, the record lists 2 affected participants among 5 at risk in cohort 1, 3 among 4 in cohort 2, 4 among 10 in cohort 3, and 2 among 3 in cohort 4. For other adverse events, it lists 5 among 5, 4 among 4, 10 among 10, and 3 among 3, respectively. The record also lists deaths by cohort: 2, 3, 4, and 2. These are descriptive counts. They should not be treated as evidence that omacetaxine caused the events or deaths, and the record does not state whether deaths were attributed to treatment.
The cohort figures do not establish a dose-response pattern. Cohorts were sequential rather than randomized, and the sizes differ. Differences in the counts may reflect the varying cohort sizes, participant characteristics, observation, or other factors; those explanations cannot be separated using the reported information. No adjusted comparisons, confidence intervals, or hypothesis tests are reported in the registry record.
The record lists Death as the top serious-event term at 75.0%. That percentage is not enough to determine a participant-level probability or to compare risk across dose cohorts, because its denominator and calculation basis are not reported. The cohort death counts are separately listed, but the record does not link individual deaths to adverse-event terms or explain their attribution.
The posted information is therefore most informative as a description of trial structure, endpoint definitions, and reported safety counts. The primary endpoint result, secondary outcome results, and statistical analysis results are not reported in the registry record. Without those results and details of the analysis populations and follow-up, the efficacy and precision of the study findings cannot be assessed from the registry record.
9. Statistical Methods Explained
The study design determines what can be inferred from its results. AML-02 is identified as non-randomized and sequential, with four cohorts assigned to increasing omacetaxine dose levels. In general, sequential dose escalation is used to examine tolerability across planned dose levels and inform dose selection. It does not create the balance that random allocation is intended to achieve. Participants in later cohorts may differ from those in earlier cohorts for reasons unrelated to dose, so direct comparisons between the cohort counts are descriptive rather than causal.
What does non-randomized allocation mean?
In a randomized trial, treatment assignment is determined by a random process, which helps balance known and unknown participant characteristics on average. This study is explicitly listed as non-randomized, and its cohorts are not a randomized comparison. The record does not report a concurrent group receiving cytarabine and idarubicin without omacetaxine. As a result, the posted information cannot isolate an effect of adding omacetaxine from the effects of induction chemotherapy, participant characteristics, or other influences.
How should the dose criteria be understood?
The primary endpoint uses a joint criterion: fewer than 30% of patients with hematologic toxicity and greater than 50% achieving CR. Both conditions are part of the registry’s definition. General statistical point: a joint rule is more demanding than considering either component alone, because a dose must meet both criteria to qualify under the stated definition. The registry does not report the observed toxicity and CR counts needed to determine whether any cohort met that rule, nor does it report the selected OD.
What do the adverse-event counts measure?
The safety record separates participants at risk from participants affected for serious and other adverse events, and also reports deaths by cohort. Counts describe what was recorded for participants in each cohort. General statistical point: a count is easier to interpret when its denominator, observation period, event definition, and relationship between repeated events and affected participants are clear. The registry gives at-risk counts for the listed event categories, but does not report the detailed event-level data, timing, exposure duration, or attribution needed for a fuller assessment. The posted counts should not be converted into causal conclusions.
Why do confidence intervals matter?
General statistical explanation: a confidence interval expresses the uncertainty around an estimated quantity under a specified statistical model. It helps readers judge the range of values reasonably compatible with the observed data, rather than focusing only on a point estimate. No confidence intervals or other uncertainty estimates for the primary outcome or the cohort safety counts are reported in the registry record. Precision therefore cannot be evaluated from the posted results.
What is the role of over-dispersion?
General statistical explanation: over-dispersion occurs when observed variability in count data is greater than a simple model assumes. It can make standard errors too small if ignored, leading to uncertainty estimates that are too narrow. Whether over-dispersion is relevant depends on the outcome, data structure, and model. The registry record does not report a statistical model for the outcomes, so it is not possible to assess whether over-dispersion was considered or whether any particular count model was used.
Were formal statistical analyses reported?
The registry reports no analyses for the outcome measures. It does not report a statistical analysis plan, adjusted estimates, hypothesis tests, or a rationale for the enrollment target. In general, a dose-escalation study may use prespecified rules to guide movement between dose levels, but the rules for this study are not reported in the registry record. The available endpoint definition is not a substitute for a complete analysis plan: it does not specify the analysis population, handling of missing outcomes, or uncertainty calculations.
A planning illustration, if present alongside posted results, is not an observed trial result. General statistical explanation: sample-size illustrations describe how many participants might be needed under stated assumptions to detect a specified effect with a chosen error rate and allocation. They are not evidence that a study had that power after enrollment, and they do not replace an analysis of observed data. The registry’s design assumptions state a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation for the illustration; time-to-event illustrations use a total number of events. These assumptions describe planning calculations, not the design of a randomized comparison in this trial. No sample-size justification for AML-02 is reported in the registry record.
Overall, the distinction between design, endpoint definition, and results is essential. The design and thresholds are described, but statistical analyses and primary outcome findings are not. The safety counts can be read descriptively, with the limitations of a non-randomized sequential study and incomplete analysis detail.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and secondary outcomes covering toxicity assessed by CTCAE grading, time to hematologic recovery, overall participant survival, event-free survival, and progression-free survival. It also reports six outcome measures in total. The number of analyses reported is zero. Results for the listed secondary outcomes are not reported in the registry record.
Multiplicity is the issue of interpreting results when several outcomes, time points, or statistical tests are examined. In general, testing many outcomes can increase the chance of a seemingly notable result arising by chance. A statistical plan may address this through an outcome hierarchy or other prespecified procedures, but the registry record does not report whether such methods were planned or used. With no outcome analyses reported, it is not possible to assess how multiplicity was handled or whether any particular finding was prespecified.
Missing-data handling is also not reported. The record does not specify whether participants who lacked a response assessment, did not complete follow-up, or were otherwise unevaluable were included in the primary endpoint analysis. It does not report imputation methods, censoring rules, or sensitivity analyses. These details matter because outcome estimates can depend on who is included and how unavailable measurements are treated.
The analysis populations are not reported in the registry record. The primary endpoint says the population is the number of participants in each cohort, but this does not establish the actual population analyzed or whether all enrolled participants contributed to each outcome. The record also does not report analysis sets for survival or adverse events. As a result, the denominator for a primary response estimate and the handling of follow-up losses cannot be established from the posted information.
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 |
|---|---|---|---|
| Patients With Newly Diagnosed AML, Cohort 1 | 2 | 5 | 40.0% |
| Patients With Newly Diagnosed AML, Cohort 2 | 3 | 4 | 75.0% |
| Patients With Newly Diagnosed AML, Cohort 3 | 4 | 10 | 40.0% |
| Patients With Newly Diagnosed AML, Cohort 4 | 2 | 3 | 66.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Patients With Newly Diagnosed AML, Cohort 1 | 2 of 5 |
| Patients With Newly Diagnosed AML, Cohort 2 | 3 of 4 |
| Patients With Newly Diagnosed AML, Cohort 3 | 4 of 10 |
| Patients With Newly Diagnosed AML, Cohort 4 | 2 of 3 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Patients With Newly Diagnosed AML, Cohort 1 | 5 of 5 |
| Patients With Newly Diagnosed AML, Cohort 2 | 4 of 4 |
| Patients With Newly Diagnosed AML, Cohort 3 | 10 of 10 |
| Patients With Newly Diagnosed AML, Cohort 4 | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | Patients With Newly Diagnosed AML, Cohort 1 | Patients With Newly Diagnosed AML, Cohort 2 | Patients With Newly Diagnosed AML, Cohort 3 | Patients With Newly Diagnosed AML, Cohort 4 |
|---|---|---|---|---|---|
| Death | Blood and lymphatic system disorders | 2 of 5 | 3 of 4 | 4 of 10 | 2 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Patients With Newly Diagnosed AML, Cohort 1 | Patients With Newly Diagnosed AML, Cohort 2 | Patients With Newly Diagnosed AML, Cohort 3 | Patients With Newly Diagnosed AML, Cohort 4 |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 3 of 5 | 4 of 4 | 9 of 10 | 2 of 3 |
| Nausea | Gastrointestinal disorders | 3 of 5 | 4 of 4 | 6 of 10 | 1 of 3 |
| Vomiting | Gastrointestinal disorders | 2 of 5 | 4 of 4 | 6 of 10 | 1 of 3 |
| Diarrhea | Gastrointestinal disorders | 2 of 5 | 2 of 4 | 6 of 10 | 0 of 3 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 5 | 2 of 4 | 5 of 10 | 1 of 3 |
| Hypernatremia | Metabolism and nutrition disorders | 1 of 5 | 2 of 4 | 5 of 10 | 1 of 3 |
| Abdominal pain | Gastrointestinal disorders | 0 of 5 | 3 of 4 | 4 of 10 | 1 of 3 |
| Headache | Nervous system disorders | 2 of 5 | 1 of 4 | 5 of 10 | 0 of 3 |
| Chills | Infections and infestations | 3 of 5 | 2 of 4 | 2 of 10 | 0 of 3 |
| Oral mucositis | Gastrointestinal disorders | 0 of 5 | 1 of 4 | 5 of 10 | 1 of 3 |
Serious events with the highest arm-level rate: death (75.0%).
The registry lists serious adverse-event counts by cohort as affected participants and participants at risk. Cohort 1 has 2 serious-event-affected participants among 5 at risk; cohort 2 has 3 among 4; cohort 3 has 4 among 10; and cohort 4 has 2 among 3. For other adverse events, the corresponding counts are 5 among 5, 4 among 4, 10 among 10, and 3 among 3. Deaths are listed separately by cohort as 2, 3, 4, and 2.
These are descriptive safety figures, not estimates of treatment-caused harm. The record does not report whether the deaths were considered related to treatment, when events occurred, how long participants were observed, or whether an affected participant could contribute multiple event reports. The at-risk counts are stated for the event categories, but details of the specific denominator used for the top serious-event term are not reported.
The registry identifies Death as the top serious-event term at 75.0%. That figure should be read as a reported event-term statistic, not automatically as the proportion of participants who died. Its denominator and method of calculation are not reported in the registry record. The cohort death counts are separate descriptive entries and do not establish attribution or comparative risk.
12. What the Result Does, and Does Not, Mean
The record documents a study designed to evaluate four omacetaxine dose levels alongside cytarabine and idarubicin induction in newly diagnosed AML. It also states the criteria intended to define an optimally active and safe dose. Those details explain the study question, but they do not show whether the criteria were met. The primary endpoint result and selected dose are not reported in the registry record.
The safety information gives cohort-level counts for serious adverse events, other adverse events, and deaths. Those counts can describe what the registry reports, but they cannot establish that omacetaxine caused an event or death. The record does not provide attribution, event timing, exposure-adjusted measures, or a randomized control group. The sequential cohorts are not a like-for-like comparison, so their counts should not be used to claim that one dose level is safer or more hazardous than another.
In general, a treatment effect is assessed by comparing outcomes under a design and analysis that support that comparison, while also describing uncertainty. Here, the non-randomized design and lack of a reported control group limit comparative interpretation, and the absence of reported analyses prevents assessment of statistical precision. No effect estimate, confidence interval, or inferential test for the primary endpoint is reported.
The registry also does not report results for its listed secondary outcomes, including survival and hematologic recovery. The planned follow-up description does not establish that all participants completed follow-up or that all outcomes were collected for every participant. Missing-data and analysis-population details are not reported.
Accordingly, the record supports a description of the trial’s design, dose cohorts, endpoint criteria, and posted safety counts. It does not establish efficacy, treatment causation, a comparative benefit or harm, or which dose best met the stated criteria. Those conclusions would require outcome results and analysis details not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable because its main question is dose selection rather than a randomized comparison of treatment strategies. The primary endpoint combines a hematologic toxicity threshold with a CR threshold. That structure makes the decision rule explicit, but a rule alone is not an outcome: the registry must also report cohort results and how they were analyzed for readers to know whether a dose met the criteria. The primary endpoint result is not reported in the registry record.
The sequential structure also illustrates why cohort counts need careful interpretation. Cohorts correspond to different omacetaxine dose levels, but they were not formed through random allocation. Comparisons among their event counts can be affected by differences in cohort size, participant composition, observation, and other factors. A numerical pattern across cohorts would not, by itself, demonstrate a dose-related causal effect.
Small cohorts make uncertainty especially important. General statistical point: estimates from limited data can be imprecise, and a point estimate alone may conceal a wide range of plausible values. The registry does not report confidence intervals or other uncertainty estimates for the primary outcome or the safety results. Readers therefore cannot judge precision from the reported counts alone.
Finally, the record lists several secondary outcomes but does not report their results or an analysis plan addressing multiple outcomes, missing data, and analysis populations. Those omissions matter to the interpretation of any dose-finding study, because selection depends not only on observed counts but also on clear definitions, complete outcome assessment, and transparent uncertainty. In this record, the design and decision criteria are available, while the statistical evidence needed to evaluate the intended dose-selection conclusion is not reported.
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.
Several limitations specific to this study constrain interpretation. The trial was non-randomized and sequential, with no like-for-like control group listed. As a result, the cohort safety counts cannot establish comparative effects of the dose levels or isolate the contribution of omacetaxine from the standard induction regimen. Cohort sizes differ, and the registry does not provide enough information to assess differences in participant characteristics across cohorts.
The registry lists enrollment of 22, but it does not report the number included in each outcome analysis or whether participants completed their planned treatment and follow-up. Although the arm descriptions state planned outpatient follow-up at least every 2 months for a total of 6 months and a final visit 6 months (+/-1 week) after the last dose, actual follow-up completeness is not reported. Missing outcome data and the handling of participants without an assessment are also not reported.
The primary endpoint definition is available, but the primary endpoint result, the selected OD, and the component CR and hematologic toxicity findings are not reported in the registry record. Results for the listed secondary outcomes are likewise not reported. The record does not provide confidence intervals, hypothesis tests, adjusted estimates, or a statistical analysis plan. It also does not report the rules used to guide dose escalation or the reason for termination.
The adverse-event entries give counts, but not event timing, duration, detailed attribution, or the denominator and calculation basis for the 75.0% Death term. The counts therefore cannot support causal conclusions or reliable dose comparisons. These limitations mean the registry record describes the study framework and selected safety entries, but does not provide the evidence needed to assess the primary dose-finding conclusion or its precision.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: AML-02: Omacetaxine With Standard-of-Care Induction With Cytarabine & Idarubicin in Newly-Diagnosed AML Patients — NCT02440568.
- Registry record last updated 2021-07-06; status last verified 2021-07.
- Results first posted 2021-07-06.
- 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
AML-02 was a phase 1 and phase 2, non-randomized, unmasked, sequential study of omacetaxine added to cytarabine and idarubicin induction for newly diagnosed AML. The registry lists enrollment of 22, four omacetaxine dose cohorts, and terminated status. The record gives a start date of 2015-06-05 and a primary completion date of 2018-11-30.
The primary endpoint sought an optimally active and safe dose, defined using hematologic toxicity and CR thresholds within 50 days. The record reports cohort-level serious and other adverse-event counts and deaths, but does not report the primary endpoint result, secondary outcome results, or statistical analyses. It does not report the reason for termination, analysis populations, missing-data methods, or uncertainty estimates. The available record therefore supports a description of the design and posted safety counts, not a conclusion about comparative treatment effects or the dose-selection outcome.