Laboratory-Treated (Central Memory/Naive) CD8+ T Cells in Treating Patients With Newly Diagnosed or Relapsed Acute Myeloid Leukemia: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Laboratory-Treated (Central Memory/Naive) CD8+ T Cells in Treating Patients With Newly Diagnosed or Relapsed Acute Myeloid Leukemia (NCT02770820), a na phase 1, phase 2 study. The primary outcome is Number of Participants Who Experienced at Least One Serious Adverse Event (SAE).

2. Clinical Question

3. Trial Design

This was a phase I/II study of autologous WT1-TCRc4 gene-transduced CD8+ central memory/naive T cells for acute myeloid leukemia. The registry describes the target population as patients with newly diagnosed or relapsed disease. The listed conditions additionally include EBV-positive neoplastic cells, HLA-A*0201-positive cells, blasts under 5 percent of bone marrow nucleated cells, and elevated WT1. The record does not give a complete description of how those conditions relate to participant eligibility.

The design is recorded as single group, with allocation marked NA and masking listed as none. These features mean that the registry does not describe assignment between distinct treatment groups or a masked comparison. It lists one treatment group, titled Treatment (Autologous CD8 T Cells). The registry lists autologous modified CD8 T cells, aldesleukin, and laboratory biomarker analysis as interventions. The available arm description is incomplete, so further treatment details are not reported in the registry record.

Enrollment is recorded as 9. The study start date is 2017-11-06, primary completion is recorded as 2020-06-01, and the status is terminated. These record details describe the study’s administrative course; they do not establish why the trial was terminated or what conclusions its investigators reached. The reason for termination is not reported in the registry record.

The single-group structure is important for interpreting the results. Counts from the treatment group can describe events recorded among participants at risk, but there is no concurrent control group from which to estimate a comparative treatment effect. The registry lists seven outcome measures and zero analyses. A protocol, statistical analysis plan, analysis population definitions, and detailed study-flow information 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:

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 Who Experienced at Least One Serious Adverse Event (SAE), measured over 12 months after the last infusion.

The primary endpoint is the number of participants who experienced at least one serious adverse event, with the timeframe specified as 12 months after the last infusion. It is a participant count: each person is counted according to whether they had at least one event meeting the endpoint definition, rather than by the total number of events experienced. The registry says toxicities are measured according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0.

Safety outcomes are commonly included in early-phase studies because they describe harms observed during and after exposure to an intervention. A count of participants with at least one serious adverse event gives a direct summary of how many participants met that event criterion in the recorded group and timeframe. It does not, by itself, describe the number, types, timing, duration, or causes of events. Nor does a count alone establish whether an event was related to a treatment.

The posted primary outcome lists 7 participants at risk and 0 participants affected by at least one serious adverse event. The record does not provide a confidence interval or a formal analysis of this endpoint. The registry’s description uses the wording “adverse event (SAE)” while naming the measure as a serious adverse event; interpretation should follow the named endpoint and the registry’s stated timeframe. Further endpoint definitions or adjudication procedures are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation begins with the question the endpoint actually answers. This trial’s primary outcome is a count of participants with at least one serious adverse event within the specified follow-up period. It is not a count of all serious events, because one participant could experience more than one event and still contribute only once to a measure defined as “at least one.” The distinction matters: participant counts describe how many people experienced the endpoint, whereas event counts describe the total number of occurrences.

What a single-group count can describe

For a binary outcome, each participant is classified as having or not having the event during the defined period. The posted group has 7 participants at risk and 0 with the primary event. Such a result is a descriptive summary of the observed group. In general, a proportion can be calculated by dividing the number with the event by the number at risk, but no additional rate is needed to state the posted count, and no rate should be read as a comparison when the design has no control group.

General statistical explanation: an estimate from a finite group has sampling uncertainty. A confidence interval describes a range of values compatible with the data under a specified method and assumptions. When no events are observed, a confidence interval can still have an upper bound above zero; the absence of observed events does not eliminate uncertainty. The registry does not report a confidence interval for this endpoint, so the precision of the result is not quantified in the posted analysis.

Why the design constrains inference

The design is recorded as single group, with allocation marked NA and masking listed as none. There is no concurrent control group in the posted design. General statistical explanation: a randomized comparison can help separate differences associated with treatment assignment from differences that may arise from participant characteristics, the course of disease, other care, or chance. In a single-group study, those alternative explanations cannot be assessed through a treatment-versus-control contrast within the trial. The safety counts can be described, but they do not establish what would have happened to the same participants under another treatment.

The trial is listed as phase I/II, and its registry summary says it studies side effects and how well the intervention works. However, the primary outcome described in the record is a safety count. The registry lists seven outcome measures and zero analyses. The statistical methods used for any efficacy outcome, as well as the results of such analyses, are not reported in the registry record. It is therefore not possible to infer an efficacy estimate or a test of treatment effect from the primary safety count.

Follow-up and event definitions

The primary outcome timeframe is 12 months after the last infusion. General statistical explanation: a defined follow-up period makes an outcome interpretable by specifying when events count toward the measure. Follow-up can also affect whether an event is observed; if observation is incomplete, a simple count may not capture the same period for every participant. The registry does not describe the completeness of follow-up, censoring, or handling of participants with incomplete observation for this outcome. Those details are not reported in the registry record.

The record states that toxicities are measured according to National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0. A standardized grading framework can support consistent classification of adverse events. The record does not provide the event-level data, grading distribution, attribution assessments, or adjudication procedures for the outcomes summarized. The primary count therefore should not be extended to claims about event severity beyond the named serious-event endpoint.

Planning assumptions are not trial results

Planning illustrations use assumptions of 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. These are described as planning assumptions, not as the trial’s own design. They do not match the recorded single-group design, which has no two-arm allocation. General statistical explanation: sample-size planning asks how many participants might be needed under chosen assumptions to detect a specified effect with a chosen operating characteristic. Such an illustration is not evidence that a study had that power, and it is not an observed treatment effect.

The registry does not report the trial’s own sample-size justification or enough analysis detail to reconstruct a formal analysis plan. In particular, a planning calculation cannot substitute for an analysis of the posted observations. The record contains no posted confidence interval, hypothesis test, adjusted analysis, or effect estimate for the primary outcome. A conclusion should therefore remain descriptive and tied to the listed group and follow-up period.

What is unavailable for independent review

To assess an analysis fully, readers generally need to know the prespecified outcomes and analysis populations, how missing observations were handled, whether follow-up differed among participants, and which statistical methods were applied. General statistical explanation: prespecification helps distinguish analyses planned before results were known from analyses chosen after looking at the data. Definitions of analysis populations clarify who contributes to each estimate. Missing-data methods describe how incomplete observations affect the analysis. These are general considerations; the relevant trial-specific procedures are not reported in the registry record.

Because the registry lists zero analyses, the posted safety count should be read as a reported descriptive outcome, not as a complete statistical evaluation. The record does not provide enough information to establish whether the displayed count is based on all enrolled participants, a defined treated population, or another analysis set. The endpoint itself lists 7 participants at risk, and that is the denominator to retain when describing this result.

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("Treatment (Autologous CD8 T Cells)")
n      <- c(7)
events <- c(0)
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  Treatment (Autologous CD8 T Cells)  7  0  0.0  0.0  41.0  0.0  35.4  0.3  36.9

11. Multiplicity, Missing Data and Analysis Populations

The registry lists seven outcome measures and zero analyses. It does not provide a multiplicity plan describing how statistical testing across outcomes would be handled. General statistical explanation: when multiple outcomes or multiple comparisons are tested, the chance of at least one apparently notable result can increase unless the analysis plan addresses multiplicity. This consideration does not establish that formal tests were conducted in this study; the registry does not post any analyses.

Missing-data procedures are not reported in the registry record. The available outcome entry gives 7 participants at risk, but the record does not explain how that analysis group relates to the enrollment of 9, whether all participants completed the stated follow-up, or how incomplete follow-up was handled. No assumptions about missing observations, imputation methods, censoring, or sensitivity analyses can be inferred from the posted count.

Analysis populations are also not described in detail. The registry does not report whether analyses were defined by enrollment, treatment received, or another criterion. The outcome-specific group count of 7 should be used when describing the endpoint; the reason it differs from the enrollment entry is not reported in the registry record.

The record does not report a statistical analysis plan, prespecified hierarchy among outcomes, or inferential methods for the safety endpoint. Consequently, the available information supports a description of the recorded counts but not an evaluation of multiplicity control, missing-data robustness, or analysis-population sensitivity.

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 non-serious adverse event terms

The treatment group lists 7 participants at risk for the serious adverse event outcome and 0 participants affected. The endpoint is defined as experiencing at least one serious adverse event during 12 months after the last infusion. The record also lists 6 participants with other adverse events and 1 death, each among 7 participants at risk. These are distinct reported counts; the registry does not state whether the participants overlap across outcomes.

The counts do not identify specific non-serious adverse events, the circumstances of the death, or whether any event was considered related to the intervention. The registry’s serious-event terms list is empty. A death recorded in the group should not, without information about cause and attribution, be interpreted as caused by treatment. Similarly, the absence of a participant counted under the primary serious-event endpoint does not mean that no adverse events occurred: the separate other-adverse-event count is 6.

General statistical explanation: adverse-event counts describe observed outcomes in a defined group and follow-up period. Their interpretation depends on how events were defined, how completely participants were followed, and how event attribution was assessed. Further details on those matters are not reported in the registry record. The counts are descriptive and do not provide a comparison with another group.

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

The result means that the registry records 0 participants with at least one serious adverse event among 7 participants at risk for that outcome, over the stated period of 12 months after the last infusion. It also records 6 participants with other adverse events and 1 death among 7 participants at risk. These figures describe the posted outcomes for the listed treatment group; they are not estimates from a randomized treatment comparison.

The result does not mean that the underlying chance of a serious adverse event is zero. General statistical explanation: an observed count is not the same as a guarantee about future observations. A finite group can have no recorded events even when the true event probability is not zero. A confidence interval would help express uncertainty, but none is reported for this endpoint. The record also does not provide enough follow-up detail to assess whether observation was complete for every participant.

The result does not show that the intervention caused, prevented, or changed the likelihood of an event. Without a concurrent control group, an outcome count cannot distinguish effects of treatment from other influences. The death count likewise does not establish cause or treatment attribution. The reasons for termination, details of the adverse events, and the relationship between the 9 enrolled participants and 7 at risk are not reported in the registry record.

Nor does this primary safety outcome establish whether the intervention worked against leukemia. Although the registry summary describes a study of side effects and how well the intervention works, the posted primary outcome is a serious adverse event count and the record lists zero analyses. Efficacy results and their statistical analysis are not reported in the registry record. Conclusions should therefore stay within the narrow scope of the posted safety counts and should not be generalized beyond the recorded group and outcome timeframe.

14. Why This Trial Matters Statistically

This trial is statistically notable because its design and endpoint call for a careful distinction between description and comparison. A single-group study can report what was observed among participants receiving an intervention, but it does not provide a concurrent control against which to estimate a treatment contrast. That distinction remains important even when a registry lists several outcomes or describes both safety and potential activity.

The primary endpoint is a participant-level safety count. It asks whether each participant experienced at least one serious adverse event during a defined period, rather than how many total events occurred. The registry reports 0 affected among 7 at risk, alongside 6 participants with other adverse events and 1 death among 7 at risk. Keeping these outcomes distinct prevents a zero count for the named serious-event endpoint from being misread as an absence of all adverse events.

The record also illustrates why denominators and study flow matter. Enrollment is 9, while the primary outcome group lists 7 at risk. Without an explanation for that difference, the outcome count should be described with its endpoint-specific denominator rather than silently substituted with the enrollment total. Reasons for termination and participant disposition are not reported in the registry record.

Finally, zero posted analyses limits what can be inferred beyond the counts. No confidence interval, hypothesis test, or efficacy analysis is reported for this endpoint. Statistical interpretation is strongest when it separates observed data, uncertainty, and causal claims: the registry gives a descriptive safety result, but does not provide the analysis needed for broader conclusions.

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 specific to the registry record constrain interpretation. The trial is listed as terminated, but the reason for termination is not reported in the registry record. Enrollment is recorded as 9, whereas the primary outcome lists 7 participants at risk; the record does not explain this difference or provide participant disposition. As a result, the relationship between enrollment and the outcome population cannot be determined from the posted information.

The design is single group, with allocation marked NA and masking listed as none. There is no recorded concurrent comparator, so the outcome counts do not estimate a treatment effect relative to another group. The registry lists zero analyses and does not post a confidence interval, inferential test, adjusted estimate, or detailed efficacy analysis. The study’s sample-size justification and statistical analysis plan are not reported in the registry record.

The primary endpoint is described as a count of participants with at least one serious adverse event during 12 months after the last infusion. The record does not provide event-level descriptions, event attribution, completeness of follow-up, or methods for handling incomplete observations. It lists 6 participants with other adverse events and 1 death but does not give event details or establish whether the outcomes overlap. The serious-event terms list is empty.

The available arm description is incomplete. Additional details about treatment delivery, adherence, participant characteristics, and the reasons for termination are not reported in the registry record. These limitations mean the posted figures can be read as descriptive counts for the listed group, but not as a complete account of safety, effectiveness, or the trial’s conduct.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Laboratory-Treated (Central Memory/Naive) CD8+ T Cells in Treating Patients With Newly Diagnosed or Relapsed Acute Myeloid Leukemia — NCT02770820.
• Registry record last updated 2021-10-29; status last verified 2021-08.
• Results first posted 2021-06-23.
• 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

NCT02770820 is a phase I/II, single-group study of autologous CD8+ T cells transduced to express a WT1-specific T-cell receptor in acute myeloid leukemia. The registry describes patients with newly diagnosed or relapsed disease and lists additional conditions related to EBV, HLA-A*0201, bone marrow blasts, and WT1. The record lists aldesleukin and laboratory biomarker analysis among the interventions.

The study began on 2017-11-06, has a primary completion date of 2020-06-01, and is listed as terminated. Enrollment is 9. The primary outcome counts participants with at least one serious adverse event during 12 months after the last infusion. The treatment group lists 7 participants at risk and 0 affected; it also lists 6 participants with other adverse events and 1 death among 7 at risk.

The registry lists seven outcome measures and zero analyses. The reason for termination, the relationship between enrollment and the outcome population, and detailed statistical methods are not reported in the registry record. The posted counts are descriptive and do not provide a randomized comparison or an efficacy analysis.