Patients Receiving Induction With Liposomal Daunorubicin and Cytarabine (CPX-351) for Acute Myeloid Leukemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Patients Receiving Induction With Liposomal Daunorubicin and Cytarabine (CPX-351) for Acute Myeloid Leukemia (NCT03988205), a na phase 4 study. The primary outcome is Number of Participants Adherent to Recommendations.

2. Clinical Question

In practical terms, the study was designed to document whether participants could follow the specified outpatient recommendations during induction and follow-up. It also included a care model with education and surveys, rather than testing CPX-351 against another drug: CPX-351 was part of participants’ usual medical care and was a condition for participation. Because there is no randomized control group, the study is descriptive with respect to the outpatient model. It cannot isolate the effect of that model from other features of care or participant circumstances.

3. Trial Design

This is a Phase IV, single-group study sponsored by the Medical College of Wisconsin. The registry record gives a start date of 2019-08-28, a primary completion date of 2021-12-01, and a status of terminated. It reports enrollment of 3 participants. The study is described as single-site for its run-in phase and as a planned multicenter continuation afterward.

The brief summary describes a run-in phase of six subjects followed by a continuation phase of 114 subjects. These are described as the study plan; the registry record reports actual enrollment of 3. It does not report how many participants were in each phase, whether the continuation phase began, or why the study was terminated. Those details should not be inferred from the planned design or the final enrollment.

The intervention combined an outpatient care model with CPX-351. The outpatient model included nurse-teacher education and quality-of-life surveys for participants and caregivers. Induction therapy and medical follow-up were performed without prophylactic inpatient admission, as described in the arm information. CPX-351 was administered as usual medical care according to FDA approval, and receiving it was an eligibility condition for participation.

Allocation is recorded as NA, masking as none, and the model as single group. The record therefore does not describe randomized assignment or blinded assessment. It lists two outcome measures and no statistical analyses. Details such as eligibility criteria beyond the stated treatment condition, recruitment procedures, protocol deviations, and a 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

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 Number of Participants Adherent to Recommendations, measured over Day 60.

The primary endpoint is the number of participants adherent to recommendations at Day 60. It is a count of participants, not a continuous measure or a comparison between treatment groups. The recommendation combines attendance at outpatient follow-up appointments with adherence to readmission recommendations. The description defines adherence as completing 95% of recommended outpatient visits.

This type of endpoint is used to describe whether participants followed a care pathway during a specified period. In this study, it focuses on the feasibility of outpatient management rather than on a direct comparison of clinical efficacy between care settings. It can summarize adherence under the study’s outpatient model, but a count by itself does not explain why participants did or did not meet the criterion. Nor does it establish that the model caused any observed adherence pattern.

The endpoint description identifies the measure and its assessment time, but the registry record does not report the observed number of adherent participants. It also does not report the denominator assessed at Day 60, how adherence was handled for participants with incomplete follow-up, or whether readmission recommendations were evaluated separately from visit completion. Without those details and the observed endpoint result, the primary measure cannot be interpreted as an estimate of adherence in the enrolled group.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The study design and reported outcomes set limits on which statistical interpretations are possible. General statistical concepts can help explain those limits, but they do not replace results that are not reported in the registry record.

What does a single-group design show?

In a single-group study, outcomes are described for participants who receive the study care model. Without a concurrent comparison group, an observed outcome cannot show what would have happened to comparable participants under a different care approach. Differences between people, changes over time, and other features of care can all contribute to outcomes. Randomization is one way to make a comparison more credible because assignment is not chosen by the participants or clinicians, but randomization is not reported in this study.

For this trial, the question is not whether CPX-351 is better than another therapy: participants were receiving CPX-351 as usual medical care. The study intervention was the outpatient care model. Since the registry records a single group and no like-for-like control, the design is suited to reporting observations about the enrolled group, not estimating a causal contrast between outpatient and inpatient care.

How should a count endpoint be read?

A participant count records how many people meet a defined condition. Here, the condition is adherence to outpatient follow-up appointments and readmission recommendations by Day 60, with visit completion defined as 95% of recommended visits. A count is most interpretable when its denominator is also clear: readers need to know how many participants were eligible for assessment and how incomplete follow-up was treated. The registry record does not report the observed count, assessment denominator, or handling of incomplete data.

Counts do not convey the same information as an estimate with a confidence interval. In general, a confidence interval expresses uncertainty around an estimated quantity under a statistical model. It is often used to show a range of values compatible with the observed data and assumptions. No interval for the primary endpoint is reported, and without the endpoint result there is no basis for interpreting such precision.

What do significance tests and analyses contribute?

A statistical test evaluates how compatible data are with a specified null hypothesis under assumptions. A p-value is not the probability that a hypothesis is true, nor does it measure the size or importance of an effect. In this registry record, the number of listed analyses is 0. No p-value, confidence interval, adjusted estimate, or analysis specification is reported. It is therefore not possible to assess a hypothesis test or determine which statistical model, if any, was used for the planned outcomes.

In general, a statistical analysis plan can identify the primary analysis, define analysis populations, state how missing observations will be handled, and specify any adjustments or interim reviews. Such details matter because analytic choices can affect estimates and their uncertainty. These details are not reported in the registry record. The record also does not report a sample-size justification. The planned run-in and continuation enrollment figures do not, by themselves, establish the statistical rationale for those targets.

What do planning illustrations mean?

Sample-size planning is different from analyzing observed results. A planning calculation asks how many participants might be needed under stated assumptions about an effect, significance level, allocation, and statistical approximation. The registry record’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of an observed effect. Those assumptions are described as a planning illustration, not as the trial’s own design.

Such illustrations should not be described as observed power. Power is a design quantity based on assumptions about a target effect and variability; it does not establish whether a finding is true. A calculation based on an observed effect can also be unstable, especially when the underlying group is small. The registry record does not report the underlying effect values or a two-arm trial design for this study. Its actual design is single-group, so equal allocation between two arms is not a description of this trial.

For count or binary outcomes, statistical models may need to account for the way outcomes arise and for differences in exposure or follow-up. General statistical methods can be appropriate only when their assumptions match the data and design. The registry record does not identify a model for the primary count endpoint or explain how it would handle variation in observation time. No model-based estimate is reported for this outcome.

These concepts clarify what a complete analysis would ordinarily need to address; they do not establish that any particular method was used. The registry record does not report an analysis plan, model, confidence intervals, or inferential results. Interpretation should therefore remain at the level of the study description and the posted participant counts.

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("Intervention")
n      <- c(3)
events <- c(3)
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  Intervention  3  3  100.0  29.2  100.0  43.9  100.0  39.8  99.4

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists two outcome measures, but it does not report a multiplicity strategy. Multiplicity arises when several outcomes or statistical tests are considered: the more opportunities there are to identify an apparently notable result, the more important it becomes to specify which questions are primary and how additional tests will be interpreted. The primary endpoint is identified as adherence to recommendations, but no statistical analysis is reported and no approach to controlling or describing multiplicity is given.

Missing-data handling is also not reported in the registry record. This matters for the Day 60 endpoint because interpreting a count of adherent participants requires knowing who was assessed and what happened when visit or follow-up information was incomplete. The record does not state whether participants with incomplete follow-up were excluded, counted as not adherent, or handled in another way. None of these approaches should be assumed.

Analysis populations are not described. The record does not specify whether the primary analysis would include all enrolled participants, only those who began the outpatient model, or another defined group. It also does not report whether the primary endpoint was analyzed or whether participants were removed from any analysis.

The listed number of analyses is 0. The absence of reported analyses means there are no inferential results to evaluate for multiplicity or sensitivity to missing data. It should not be taken as evidence that no analyses were performed outside the registry record. Interim analyses and stopping rules are not reported in the registry record either, so the reason for termination and any role of interim review cannot be determined from the listed information.

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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: febrile neutropenia (66.7%), fever (33.3%).

The safety results report that 3 participants were at risk for serious adverse events and 3 were affected. For other adverse events, the record likewise reports 3 at risk and 3 affected. Deaths are reported as 0. These figures describe participant-level counts in the enrolled group; they do not give the number of individual event episodes or explain whether a participant experienced more than one event.

The most frequently listed serious adverse event terms are febrile neutropenia, reported as 66.7%, and fever, reported as 33.3%. These percentages refer to the terms in the registry listing. They should not be added or treated as mutually exclusive categories unless the registry record establishes that they are. The information described does not report timing, duration, severity grades, relatedness to the care model, or event-level detail.

Safety counts in a single-group study do not establish a comparative risk. The absence of a control group means there is no study-based comparison with inpatient management, and the small enrollment limits how broadly the counts can be generalized. The zero deaths entry is a recorded count for this study, not evidence about mortality risk beyond the enrolled participants or the reported observation period. The registry record does not report the safety observation period in the information described.

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

The study addresses a practical care-delivery question: whether a specified outpatient model could be used for people receiving CPX-351 induction, with adherence to outpatient recommendations assessed by Day 60. The registry record makes clear what the primary endpoint was intended to capture and describes the educational and survey components of the care model.

However, the primary endpoint’s observed result is not reported. Without the number of participants meeting the adherence definition and the number assessed, the record cannot quantify adherence or its precision. It also cannot show whether the model met a feasibility target, because no such target or primary endpoint result is reported.

The safety entries describe serious and other adverse events among the enrolled participants, with 0 deaths reported. They do not estimate comparative safety, establish a causal relationship with the outpatient model, or characterize risk in a broader population. These are descriptive registry counts, not evidence that one care setting is safer than another.

The design also limits interpretation. This was a single-group study without a randomized comparator or masking. Any outcomes cannot be separated from factors other than the outpatient model, and the record does not report participant characteristics needed to judge how representative the enrolled group might be. The status is terminated and enrollment is reported as 3, while the brief summary describes a larger planned run-in and continuation. The registry record does not explain the termination or how much of the planned design was completed.

Accordingly, the posted information documents the study’s design, its limited enrollment, and safety counts. It does not provide the primary feasibility result or an analysis that would support a comparative conclusion. The findings should be read as a limited registry record of a single-group study, rather than as a definitive estimate of what would occur in other settings.

14. Why This Trial Matters Statistically

This trial is statistically instructive because its clinical question and its design need to be kept distinct. It focuses on an outpatient care model, but the record describes no control group. Even if the primary adherence result were available, a single-group result would describe the participants observed rather than establish how outcomes compare with inpatient care.

The registry record also illustrates why endpoint definitions and denominators matter. The primary measure has a specified time point, Day 60, and a stated adherence threshold of 95% of recommended outpatient visits. Yet the observed count and assessment denominator are not reported. Without both, readers cannot understand how many participants met the criterion or how incomplete follow-up affects the interpretation.

Enrollment and study status are important context for precision. Enrollment is reported as 3, and the study is listed as terminated. The brief summary describes a run-in and a larger continuation plan, but the registry record does not explain the relationship between that plan and the final enrollment. The listed safety counts must therefore be understood as a small descriptive record, not as precise estimates for a wider population.

Finally, the record shows the difference between a planned statistical illustration and an analysis of trial data. Planning assumptions can illustrate what sample sizes might be required under selected conditions, but they do not substitute for observed endpoint results, uncertainty estimates, or a stated analysis plan. No analyses are listed for this study. That absence makes transparent reporting of the endpoint result, missing-data approach, analysis population, and any comparison especially important for interpretation.

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.

Several limitations are specific to this study record. The reported enrollment is 3, and the status is terminated. The study is single-group, with allocation recorded as NA and masking recorded as none. There is therefore no concurrent control group for comparison, and outcomes cannot be attributed specifically to the outpatient model on the basis of this design.

The planned study structure in the brief summary differs in scale from the reported enrollment: it describes a six-subject single-site run-in followed by a 114-subject multicenter continuation. The registry record does not report how many participants were enrolled in either phase, whether the multicenter phase occurred, or why the study was terminated. Those uncertainties prevent assessment of how closely the planned design was implemented.

The primary endpoint is described, but its observed result is not reported. The record also does not report the assessment denominator, participant-level adherence information, or handling of incomplete follow-up. No statistical analyses are listed, and an analysis plan, sample-size justification, analysis population, multiplicity approach, and interim analyses are not reported in the registry record.

Safety information is limited to participant counts, listed adverse-event terms, and deaths. Event-level timing, severity grades, duration, attribution, and the safety observation period are not reported in the information described. Participant characteristics and details needed to assess representativeness are also not reported. Together, these limitations restrict both precision and generalizability; they do not establish what the unreported results would have been.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Patients Receiving Induction With Liposomal Daunorubicin and Cytarabine (CPX-351) for Acute Myeloid Leukemia — NCT03988205.
• Registry record last updated 2022-07-19; status last verified 2022-07.
• Results first posted 2022-07-19.
• 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

NCT03988205 is a Phase IV, single-group study sponsored by the Medical College of Wisconsin, with the title referring to outpatient management during CPX-351 induction for acute myeloid leukemia. The registry record reports a start date of 2019-08-28, primary completion date of 2021-12-01, terminated status, and enrollment of 3.

The intervention was an outpatient care model that included nurse-teacher education and quality-of-life surveys for participants and caregivers. The primary endpoint was the number of participants adhering to outpatient follow-up and readmission recommendations by Day 60, with adherence defined as completing 95% of recommended outpatient visits. Its observed result is not reported in the registry record.

The safety entries report 3 of 3 participants affected by serious adverse events, 3 of 3 affected by other adverse events, and 0 deaths. The record lists no statistical analyses and does not report why the study was terminated. The planned run-in and continuation structure is described, but the extent of its completion is not reported.