Vyxeos for Induction of Low- or Intermediate-risk.: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Vyxeos for Induction of Low- or Intermediate-risk. (NCT05599360), a na phase 2 study. The primary outcome is Response Rate for Low/Intermediate Risk Acute Myeloid Leukemia (AML) After Induction With Vyxeos With or Without Mylotarg..

2. Clinical Question

The study asks whether induction with Vyxeos, used with or without Mylotarg, can produce a response in people with newly diagnosed low- or intermediate-risk acute myeloid leukemia. The primary outcome includes complete remission and complete remission with incomplete hematologic recovery. The registry record specifies that the response is assessed up to 1 month.

The study description focuses on de novo AML patients who are FLT3-negative and frames the target population as age 18-70. It also identifies the feasibility and efficacy of combining Mylotarg with Vyxeos as a study goal. Whether Mylotarg is used is determined by physician decision, rather than random assignment. This means the observed groups may differ for reasons related to treatment choice as well as treatment itself.

The registry record does not report the observed response count or response rate, so the clinical question cannot be answered from the posted outcome data. The design can describe experience in the enrolled participants, but the physician-directed use of Mylotarg limits direct interpretation of differences between treatment groups.

3. Trial Design

NCT05599360 is registered as a phase 2 pilot study sponsored by Shaare Zedek Medical Center. The record identifies the condition as acute myeloid leukemia and lists Vyxeos and Mylotarg as drug interventions. The enrollment is 20. The study is classified as a single-group model, with allocation recorded as NA and masking recorded as none.

The treatment description distinguishes patients receiving Vyxeos with Mylotarg from those receiving Vyxeos without Mylotarg. The decision to give Mylotarg is made by the physician. Thus, the record includes treatment groups for descriptive purposes, but does not describe random assignment or a like-for-like control comparison. The single-group classification and the treatment-group descriptions should be read together: the record does not establish a randomized comparative trial.

The study began on 2022-08-07, and the primary completion date is 2023-10-23. Its registry status is active, not recruiting. The record lists 20 participants enrolled, but does not provide an account of how many participants completed treatment, contributed data to each endpoint, or were included in an analysis.

The primary endpoint is response after induction with Vyxeos with or without Mylotarg, including complete remission and complete remission with incomplete hematologic recovery, assessed up to 1 month. Listed secondary outcomes include participants with negative measurable residual disease, overall survival, and disease-free survival. The record notes five outcome measures, but does not report analyses for them.

The study summary discusses Vyxeos as a liposomal combination of cytarabine and daunorubicin, and describes a rationale for evaluating it in low- or intermediate-risk disease. It also describes the intended examination of the combination with Mylotarg. These are study rationales and aims, not evidence of a treatment effect. The registry record does not report detailed eligibility criteria, a full treatment schedule, a statistical analysis plan, or a sample-size justification.

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

Analysis population for the primary outcome, as defined in the registry: 1. Patients treated with Vyxeos without Mylotarg. 2. Patients treated with Vyxeos, with Mylotarg.

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.

6. Primary Endpoint

The registry lists the primary outcome as Response Rate for Low/Intermediate Risk Acute Myeloid Leukemia (AML) After Induction With Vyxeos With or Without Mylotarg., measured over up to 1 month.

The primary endpoint concerns response after induction with Vyxeos, with or without Mylotarg. The registry defines the response assessment to include complete remission and complete remission with incomplete hematologic recovery, with a timeframe of up to 1 month. These categories describe hematologic response while distinguishing a complete remission from one in which hematologic recovery is incomplete. The endpoint is intended to assess whether disease response is observed after the induction treatment.

Response endpoints are used in clinical studies because they provide a defined, relatively near-term measure of treatment activity. They can help describe the proportion or number of participants meeting specified response criteria within a stated assessment period. Interpreting such an endpoint depends on the criteria used, the timing and completeness of assessments, and how participants without an evaluable assessment are handled. Those operational details are not reported in the registry record.

The registered outcome is described as a response rate, while its measurement unit is participants and its parameter is a count of participants. The record does not report the response count, the denominator used to calculate a rate, or the outcome results. It also does not provide a confidence interval or an inferential comparison. Therefore, the endpoint definition indicates what the investigators intended to measure, but the posted registry information does not establish the observed response or its precision.

The record separately lists negative measurable residual disease, overall survival, and disease-free survival as secondary outcomes. Those outcomes address different aspects of disease status and follow-up. Their definitions, assessment schedules, and results are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry record identifies a phase 2, single-group pilot study with no masking. Allocation is recorded as NA. These design labels matter because they describe how treatment assignment and outcome assessment are structured. In this study, the use of Mylotarg is determined by physician decision. The groups receiving Vyxeos with or without Mylotarg therefore should not be interpreted as randomized arms.

What does nonrandom treatment assignment mean?

General explanation: random assignment is used to make treatment groups comparable, on average, with respect to both measured and unmeasured characteristics. When treatment is chosen by a physician, group membership may instead be associated with prognosis, eligibility, clinical judgment, or other factors. A simple comparison of group outcomes can then mix treatment differences with pre-existing differences between patients. Statistical adjustment can sometimes address measured differences, but cannot guarantee removal of bias from unmeasured factors. The registry record does not report an adjusted comparison or the information needed to evaluate one.

The record specifically cautions that the groups are not a like-for-like control comparison. This caution applies even if outcome counts are displayed separately by treatment group. A numerical contrast between groups would be descriptive unless a suitable analysis and defensible assumptions supported a stronger interpretation. No such comparative analysis is reported.

What can the primary endpoint establish?

The primary endpoint is response, including complete remission and complete remission with incomplete hematologic recovery, assessed up to 1 month. General explanation: a response endpoint summarizes whether participants meet prespecified response criteria during a defined assessment period. Its interpretation depends on a clear definition of response, consistent measurement, and a transparent denominator. The record gives the response categories and timeframe, but does not report the detailed assessment rules, the response count, the denominator, or an estimate of uncertainty.

The endpoint is described as a response rate but is recorded with a participant count as its measurement. A count alone does not show how frequent the outcome was unless its denominator is also known. The enrolled total is 20, but the record does not establish how many participants were evaluable for the primary endpoint or how any missing assessments were handled. No response rate should be inferred from enrollment or adverse-event denominators.

How should uncertainty be understood?

General explanation: a point estimate is a summary of the observed data, while a confidence interval conveys sampling uncertainty around an estimate under the assumptions of the analysis. Wider intervals indicate less precision. In small studies, an estimate may be sensitive to the outcome of a small number of participants. Confidence intervals do not account automatically for selection bias, outcome misclassification, missing data, or differences between nonrandomized groups. The registry record does not report a confidence interval for the primary outcome.

The record lists zero analyses. It does not identify an inferential test, a regression model, covariate adjustment, a confidence-interval method, or a plan for handling time-to-event outcomes. It would be inappropriate to infer which methods were used or intended. The sample-size assumptions described for planning illustrations are not the trial’s own design justification. Such illustrations should be understood as planning scenarios based on an observed effect, not as observed power or as evidence that the trial was adequately powered.

Why do event counts need careful interpretation?

General explanation: event counts describe what was recorded in a defined group, but the size of a group affects how stable the apparent event frequency is. With limited observations, a small change in the count can substantially change the apparent pattern. Comparisons between nonrandomized groups have the additional problem that participants may differ before treatment. A count should therefore be read with its denominator, event definition, follow-up period, and ascertainment procedures in mind. The registry record reports adverse-event denominators and counts, but does not describe detailed ascertainment or follow-up rules.

The record lists a serious adverse-event term of sepsis at 7.1%. It does not state the time window, attribution rules, or the denominator underlying that term in the information available. It should not be treated as a group-specific rate or compared directly with the serious-event counts without further detail.

What is not specified?

The registry record does not report the statistical analysis plan, sample-size justification, interim-analysis plan, analysis populations, missing-data procedures, or multiplicity strategy. It also does not report outcome analyses for the primary or secondary endpoints. These omissions prevent assessment of whether analyses followed a prespecified plan, how incomplete follow-up was handled, or how multiple outcomes were interpreted. General methodological principles can explain why these details matter, but they cannot replace the missing study-specific methods.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Vyxeos Without Mylotarg", "Vyxeos With Mylotarg")
n      <- c(14, 6)
events <- c(13, 6)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Vyxeos Without Mylotarg  14  13  92.9  66.1  99.8  68.5  98.7  68.1  98.3
ARM  Vyxeos With Mylotarg  6  6  100.0  54.1  100.0  61.0  100.0  59.0  99.6

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists a primary response outcome and several secondary outcomes, including negative measurable residual disease, overall survival, and disease-free survival. It does not report a multiplicity strategy. When a study examines multiple outcomes, repeated statistical testing can increase the chance of finding an apparently notable result by chance. A prespecified hierarchy or adjustment can help control that concern, but no such approach is reported. Because the outcome analyses are not reported, the extent to which multiplicity would affect interpretation cannot be assessed.

Missing-data procedures are also not reported. The registry record does not state how participants with no response assessment, incomplete follow-up, or unavailable outcome data would be handled. It does not identify an analysis population, such as all enrolled participants or only participants with evaluable assessments. The enrollment of 20 does not establish the denominator for any endpoint.

General explanation: excluding participants with missing outcomes can bias results if missingness is related to prognosis or treatment experience. Treating missing outcomes as nonresponse is another possible convention, but it also requires a stated rationale and can alter the estimate. The appropriate approach depends on the endpoint and the reasons data are missing. No approach should be assumed for this study because the registry record does not describe one.

The record lists zero analyses and provides no primary or secondary outcome results. Consequently, it is not possible to determine how outcome multiplicity, missing observations, or analysis-set choices affected the posted findings. These are reporting gaps, not evidence that a particular method was or was not used outside the registry record.

12. Safety Results

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

Deaths

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 (7.1%).

The recorded serious adverse-event counts are 0 of 6 participants in the Vyxeos with Mylotarg group and 1 of 14 participants in the Vyxeos without Mylotarg group. Recorded deaths are 0 in the group receiving Mylotarg and 1 in the group not receiving Mylotarg. The other adverse-event category records 6 of 6 and 14 of 14 participants, respectively. The registry record does not provide further definition of that category or a detailed account of event severity and timing.

These figures are descriptive counts, not evidence that one treatment group is safer than the other. The groups are small and treatment assignment is based on physician decision, so they may differ in ways that influence adverse events. The registry record itself cautions that the arms are not a like-for-like control comparison. The top serious adverse-event term is sepsis, listed as 7.1%; no group-specific context or denominator for that percentage is reported in the available record.

General explanation: adverse-event counts are most informative when interpreted alongside the observation period, event definitions, ascertainment methods, attribution rules, and participant-level context. Those details are not reported. The counts should not be read as complete evidence about safety, nor should the absence of a recorded serious event in a small group be interpreted as proof that such events cannot occur.

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

This pilot study is designed to describe response after induction with Vyxeos, with or without Mylotarg, among low- or intermediate-risk AML patients. It also identifies feasibility and the combination with Mylotarg as study interests. The registry record provides the intended primary endpoint and some adverse-event counts, but it does not report primary response results. Therefore, no response rate, response comparison, or degree of precision can be stated from the posted record.

The design is important to what can be concluded. Mylotarg use is decided by the physician, and the registry cautions that the treatment groups are not like-for-like controls. Any observed difference between groups would not by itself isolate the effect of adding Mylotarg. Differences in participant characteristics, treatment selection, or follow-up could contribute. The record does not report an adjusted analysis that might address measured differences.

The safety counts document events recorded in the two groups, including serious adverse events and deaths. They do not provide a robust comparative safety estimate, especially without detailed event definitions, follow-up information, or a randomized comparison. The sepsis term listed at 7.1% is not accompanied by enough context to interpret it as a group-specific result.

General explanation: a pilot study can provide descriptive information and help characterize feasibility, but its findings require careful consideration of sample size, design, outcome completeness, and analysis methods. A lack of reported efficacy analyses is not evidence of no effect; it means the registry record does not supply the results needed to evaluate the endpoint. Similarly, a posted count should not be treated as a causal conclusion.

The record identifies secondary outcomes, but does not report their analyses. It does not state a statistical plan, analysis populations, missing-data methods, or multiplicity approach. These limits mean the record supports understanding what the study intended to assess and the safety counts it reports, but not a conclusion about treatment effectiveness or comparative benefit.

14. Why This Trial Matters Statistically

This study raises a useful statistical distinction between describing outcomes in a treatment cohort and estimating comparative treatment effects. Its primary question concerns response after induction with Vyxeos, with or without Mylotarg, but the physician chooses whether Mylotarg is used. The resulting groups can describe observed experience, while lacking the exchangeability that random assignment is intended to support. Recognizing that distinction prevents a descriptive contrast from being mistaken for a causal comparison.

The registry record also illustrates why endpoint labels and endpoint results are different. It defines a response outcome that includes complete remission and complete remission with incomplete hematologic recovery, and specifies a timeframe up to 1 month. Yet the record does not report the response count or rate. An endpoint definition alone cannot establish what was observed, how complete the assessment was, or how precisely the response was estimated.

Small pilot studies can be valuable for describing feasibility and identifying questions for further investigation, but their estimates may be unstable and sensitive to a limited number of outcomes. That general consideration is particularly relevant when the recorded groups contain 6 and 14 participants for the adverse-event reporting. These denominators are not evidence of response denominators, and they do not establish the analysis population.

The record lists multiple secondary outcomes and safety information, while leaving analysis methods and missing-data handling unreported. This makes transparent reporting of denominators, outcome definitions, follow-up, uncertainty, and analysis populations especially important. The statistical significance of a result, if later reported, would not by itself resolve confounding in a physician-directed treatment comparison.

For clinical researchers, the main statistical lesson is to separate what a registry record specifies, what it reports as an observed count, and what remains unavailable. That separation preserves the study’s descriptive value without overstating what the design or posted results can establish.

15. 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 primary limitation for interpreting efficacy is that response results are not reported in the registry record. There is no response count, rate, confidence interval, or reported analysis. Although enrollment is 20, the record does not state the number assessed for the primary endpoint or the denominator used for a response rate.

The treatment groups are not randomized, and Mylotarg use is determined by physician decision. The record explicitly cautions that they are not like-for-like controls. Differences between groups may therefore reflect treatment selection or participant characteristics as well as treatment. No adjusted comparison is reported.

The registry record does not provide detailed eligibility criteria, treatment schedules, response assessment rules, follow-up procedures, or operational definitions for all outcomes. It also does not describe analysis populations, missing-data handling, an analysis plan, interim analyses, multiplicity control, or the sample-size justification. These omissions limit assessment of bias, precision, and whether analyses followed prespecified methods.

Safety interpretation is also limited by the small recorded group denominators and the lack of event timing, ascertainment detail, or context for the sepsis listing. The adverse-event counts are descriptive and should not be interpreted as a comparative safety estimate. Secondary outcomes are named, but their results and methods are not reported.

The study is classified as a single-group pilot while describing patients treated with and without Mylotarg. This underscores the need to interpret the group-specific figures in light of physician-directed treatment selection rather than as evidence from a controlled comparison. The registry status and primary completion date do not fill the gaps in outcome reporting.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Vyxeos for Induction of Low- or Intermediate-risk. — NCT05599360.
• Registry record last updated 2026-10-08; status last verified 2026-09.
• Results first posted 2026-09-03.
• 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.

19. Record Summary

NCT05599360 is a phase 2 pilot study sponsored by Shaare Zedek Medical Center, with enrollment of 20 and a registry status of active, not recruiting. The study concerns newly diagnosed low- or intermediate-risk AML and evaluates induction with Vyxeos, with or without Mylotarg at physician discretion. The record gives a primary response endpoint that includes complete remission and complete remission with incomplete hematologic recovery, assessed up to 1 month.

The record classifies the study as single group, with allocation recorded as NA and no masking. It lists negative measurable residual disease, overall survival, and disease-free survival as secondary outcomes. Primary and secondary outcome analyses are not reported in the registry record. Adverse-event counts are posted for the two treatment groups, but the nonrandomized design limits comparative interpretation. Statistical analysis methods, missing-data procedures, and the multiplicity approach are not reported.