NT-I7 for Kaposi Sarcoma in Patients With or Without HIV: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for NT-I7 for Kaposi Sarcoma in Patients With or Without HIV (NCT04893018), a na phase 1 study. The primary outcome is Percentage of Participants With Adverse Events.

2. Clinical Question

The study asks whether NT-I7, also called efineptakin alfa, can be given to patients with Kaposi sarcoma and what effects are observed, including adverse events. The registry describes a phase 1 study in patients with or without HIV infection. Its primary outcome is the percentage of participants with adverse events, assessed using Common Terminology Criteria for Adverse Events version 5.0.

The registry also names secondary outcomes: objective response rate, duration of response, progression-free survival, overall survival, and changes in absolute lymphocyte counts and CD4+ and CD8+ T cells. These outcomes describe different aspects of tumor response, disease course and immune-cell kinetics. Their names indicate what was intended for assessment; they do not establish what was observed. Results for those secondary outcomes are not reported in the registry record.

3. Trial Design

This was a phase 1 study of efineptakin alfa for Kaposi sarcoma. The registry lists Fred Hutchinson Cancer Center as sponsor and records enrollment of 8. The study began on 2022-05-23, had a primary completion date of 2023-12-15, and is now listed as terminated. The reason for termination is not reported in the registry record.

The design is single-group, with allocation listed as not applicable and masking listed as none. In practical terms, the record does not describe a randomized assignment or a blinded comparison. Participants received efineptakin alfa at a dose level. One dose level is titled Dose Level 1 and the other Dose Level 2. Safety summaries are listed for both dose levels, but the registry explicitly cautions that the arms are not a like-for-like control comparison. Differences between the dose-level summaries therefore cannot be treated as randomized evidence about dose effects.

The treatment description specifies intramuscular efineptakin alfa on day 1. Cycles repeat every 9 weeks for up to 4 cycles in the absence of disease progression or unacceptable toxicity. The record does not report further details of dose selection, escalation rules, or how decisions about progression or toxicity affected treatment.

The primary outcome is the percentage of participants with adverse events, assessed using Common Terminology Criteria for Adverse Events version 5.0. Its analysis population is described as all enrolled participants who received at least one dose. The observation period is up to 30 days after the last dose, approximately 31 weeks total. This is a safety-focused endpoint, not a randomized estimate of comparative efficacy.

The registry lists nine outcome measures and no analysis entries. The results available in this record do not include a numerical result for the primary endpoint, a confidence interval, or a statistical test. Those omissions restrict interpretation to the design and the safety figures that are recorded.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Analysis is conducted on all enrolled participants who received at least one dose of NT-I7.

5. Baseline Characteristics

The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.

Population: Population included all enrolled participants.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants With Adverse Events, measured over Up to 30 days after last dose of NT-I7, approximately 31 weeks total.

The primary endpoint is the percentage of participants with adverse events. It is assessed among enrolled participants who received at least one dose of NT-I7, with events measured using Common Terminology Criteria for Adverse Events version 5.0. The registry sets the observation period as up to 30 days after the last dose, approximately 31 weeks total.

A percentage endpoint expresses how common an event was in the defined analysis population during the specified observation period. For a safety-focused phase 1 study, documenting adverse events is relevant because it describes events reported during exposure and follow-up. The endpoint does not, by itself, establish that treatment caused an event. In a single-group study, there is no control group to estimate how often similar events would occur without the study treatment.

The endpoint also differs from the named secondary outcomes. Objective response rate concerns tumor response; duration of response and progression-free survival concern time-related disease outcomes; overall survival concerns survival; and the blood-cell outcomes concern immune-cell kinetics. These are not substitutes for the primary safety measure, and their results are not reported in the registry record.

A numerical overall result for the primary endpoint is not reported in the registry record. The available safety details instead list serious-event counts by dose level and selected serious-event terms. Those details should be read as limited descriptive information, not as a complete estimate of the primary endpoint across the stated analysis population.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registered primary measure is a binary outcome summarized as a percentage of participants: whether a participant experienced an adverse event during the specified assessment period. A binary endpoint records whether an event occurred, rather than measuring a continuous quantity such as a laboratory value. The registry states that adverse events are measured using Common Terminology Criteria for Adverse Events version 5.0, but the detailed rules used to combine events into the primary percentage are not reported in the registry record.

What does a percentage estimate?

In general, a percentage for a binary outcome describes the proportion of people in a defined analysis population who meet the outcome definition during a defined period. The interpretation depends on the numerator, denominator, event definition and follow-up. Here, the stated population is all enrolled participants who received at least one dose, and the stated period extends to 30 days after the last dose, approximately 31 weeks total. The registry does not report an overall primary-endpoint percentage, so that estimate cannot be interpreted from the record.

When a percentage is estimated from a limited group, uncertainty matters as well as the point estimate. In general, a confidence interval gives a range of values compatible with the observed data under the method’s assumptions. It can communicate how imprecise an estimate may be. A confidence interval for the primary endpoint is not reported in the registry record. Without the endpoint result and its uncertainty, the record cannot show how stable or precise an overall safety estimate would be.

Why does the single-group design matter?

In general, a control group helps distinguish events that occur during treatment from events that might also occur without that treatment. This study is listed as single-group, with allocation not applicable and masking none. Consequently, the adverse-event summaries describe events recorded among participants receiving the study intervention, but they do not estimate a treatment-versus-control contrast. The record does not include a control arm against which to compare event frequency.

The safety data are separated by dose level, but the registry says these are not a like-for-like control comparison. In general, comparisons between nonrandomized groups can reflect differences in participant characteristics, exposure, follow-up or other factors as well as any dose-related effect. The record does not provide enough information to evaluate such differences. The dose-level counts should therefore remain descriptive rather than being interpreted as a formal test of dose effects.

What do the serious-event summaries convey?

The record lists the number at risk and number affected for serious events within each dose level, and it lists selected serious-event terms with percentages. These figures can describe what was recorded in the registry entry. They do not replace the primary endpoint, which is defined as the percentage of participants with adverse events, nor do they establish causation. The registry does not report a complete event-by-event dataset or the calculation details for the listed term percentages.

In general, over-dispersion is an issue when observed variation in count data exceeds what a simple statistical model expects. It can make standard errors too small if a model assumes less variation than the data contain. That concept is relevant to some count-based analyses, but the primary outcome is a participant-level binary percentage. The registry record does not report a count model, an over-dispersion adjustment, or a statistical method for the primary endpoint. It would be inappropriate to infer that such a method was used.

What can the planning illustration establish?

The record describes a sample-size planning illustration based on the observed effect, not the trial’s own design. It specifies a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. For time-to-event outcomes, the illustration is described as using a total number of events; for other outcomes, it is described as participants per arm.

Such a planning calculation is hypothetical: it asks what sample size might be needed under stated assumptions. It is not the observed power of this study, and it does not show that the trial was planned with those assumptions. The actual planning rationale and statistical plan are not reported in the registry record. The record does not include numerical outputs for the illustration in the registry record for this analysis, so no planning sample-size figure can be interpreted.

Are formal analyses reported?

The registry lists no analysis entries. That means no formal analysis result is recorded in the information available for this account. It does not establish which analyses investigators may have conducted outside the registry record. The statistical plan, handling of repeated events, confidence-interval method, and any interim analysis are not reported in the registry record.

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("NT-I7 (Efineptakin Alfa) Dose Level 1", "NT-I7 (Efineptakin Alfa) Dose Level 2")
n      <- c(6, 2)
events <- c(6, 2)
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  NT-I7 (Efineptakin Alfa) Dose Level 1  6  6  100.0  54.1  100.0  61.0  100.0  59.0  99.6
ARM  NT-I7 (Efineptakin Alfa) Dose Level 2  2  2  100.0  15.8  100.0  34.2  100.0  29.2  99.2

11. Multiplicity, Missing Data and Analysis Populations

The registry names a primary adverse-event outcome and several secondary outcomes. These include objective response rate, duration of response, progression-free survival, overall survival, and changes in absolute lymphocyte counts, CD4+ T cells and CD8+ T cells. It also lists nine outcome measures overall. The results for the named secondary outcomes are not reported in the registry record.

When a study examines multiple outcomes, there can be more than one opportunity to observe a result that appears notable by chance. In general, multiplicity methods set out how investigators will interpret or adjust for that possibility. The statistical plan and any multiplicity adjustment are not reported in the registry record. No analysis entries are listed, so the record does not establish which endpoints were formally analyzed or how results across endpoints were handled.

The registry identifies the primary endpoint population as enrolled participants who received at least one dose. However, it does not provide a participant flow account, endpoint-specific denominators, or an overall primary endpoint result. The handling of missing observations, withdrawals, incomplete follow-up, and participants who did not receive a dose is not reported in the registry record. No imputation or other missing-data method is specified.

These omissions matter because percentages depend on the population and follow-up used in the calculation. A stated analysis population is useful, but without the endpoint result and denominator details it is not possible to assess how missing or incomplete observations affected the estimate. It is also not possible to determine from the registry entry whether all planned follow-up was completed before termination.

The reason for termination, interim analyses and a detailed statistical analysis plan are not reported in the registry record. Their absence limits assessment of whether the recorded outcomes and analyses correspond to the study’s original plans. It does not, by itself, establish that any particular procedure was or was not conducted outside the registry.

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: bacteremia (50.0%), chylothorax (50.0%), pleural effusion (50.0%), injection site reaction (16.7%).

The serious-event information is presented by dose level. Dose Level 1 lists 1 affected participant among 6 at risk, and Dose Level 2 lists 1 affected participant among 2 at risk. These are the counts recorded for the serious-event summary; they are not the overall primary adverse-event percentages.

The registry also lists bacteremia, chylothorax and pleural effusion at 50.0% each, and injection site reaction at 16.7%. The denominator and calculation details for these term percentages are not reported in the registry record. The percentages should not be treated as if they necessarily describe the same population or measure as the primary endpoint.

Neither dose-level group is a control group, and the registry cautions against treating the arms as a like-for-like comparison. The figures are descriptive. They do not establish that the intervention caused a listed event, nor do they allow a reliable comparison between dose levels. No deaths are listed in either dose-level serious-event summary. The record does not report an overall primary endpoint result or a complete account of all adverse events, so these selected entries do not fully characterize safety.

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

The registry record establishes that a phase 1, single-group study of efineptakin alfa for Kaposi sarcoma was conducted, with enrollment of 8, and is now marked terminated. It describes treatment by intramuscular injection on day 1 of cycles that repeat every 9 weeks for up to 4 cycles, subject to disease progression or unacceptable toxicity. It also defines a primary safety outcome and lists selected serious-event summaries by dose level.

The record does not report an overall numerical result for the primary adverse-event endpoint, its confidence interval, or a statistical test. It also does not report results for the listed secondary outcomes. As a result, the available information does not quantify the overall frequency of adverse events under the stated endpoint definition, establish how precise that estimate might be, or answer the listed tumor-response, survival and immune-cell questions.

The serious-event counts and terms are descriptive observations. A single-group study cannot show how event frequencies would compare with those in an untreated or differently treated group. The dose-level summaries do not provide a randomized comparison, and the registry explicitly says the arms are not a like-for-like control comparison. The figures therefore should not be used to claim that one dose level is safer or that treatment caused a particular event.

Likewise, the study’s stated purpose and the registry’s secondary outcome names do not demonstrate clinical benefit. The reason the study was terminated is not reported in the registry record, so termination status alone should not be taken as evidence of a specific outcome or safety concern. The cautious conclusion is that this record documents the study design and selected safety data but does not report enough outcome information to determine the primary endpoint result or treatment effects.

14. Why This Trial Matters Statistically

Phase 1 studies are important statistically because they focus on early evaluation, including how treatment is administered and what safety events are observed. In this study, the registry defines a primary adverse-event endpoint and specifies an analysis population and follow-up period. Those definitions are essential for interpreting a safety percentage, but the numerical result for that endpoint is not reported in the registry record.

The study also illustrates why design shapes the questions that can be answered. A single-group study can document events among participants receiving the intervention, but it does not provide a concurrent control group for estimating comparative safety or efficacy. The listed dose-level groups do not substitute for a randomized comparison; the registry cautions that they are not like-for-like control groups. Their results should be understood descriptively.

Another statistical issue is the distinction between an endpoint’s name and its result. The registry lists outcomes spanning tumor response, disease progression, survival and immune-cell measures, but those outcomes are not reported in the registry record. Listing planned measures does not show that a treatment effect was observed, nor how uncertain any estimate might be.

The record’s limited reporting also underscores the importance of complete endpoint results, analysis populations, missing-data handling and statistical methods. Without these details, readers cannot assess precision, evaluate whether all participants contributed to an endpoint, or understand how analyses were conducted. The study remains informative about its registered design and the form of safety summaries recorded, while the missing outcome results constrain stronger statistical 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.

The principal limitation is that the numerical result for the primary endpoint, the percentage of participants with adverse events, is not reported in the registry record. No confidence interval or statistical test is reported for that endpoint. The available serious-event summaries cannot fill that gap because they are not presented as the overall primary outcome.

The study has no control group. Its single-group design, allocation listed as not applicable and lack of masking limit comparative interpretation. The registry further cautions that the dose-level groups are not a like-for-like control comparison. The serious-event counts by dose level should not be interpreted as evidence of a dose effect.

Enrollment was 8, and the safety summaries list 6 at risk in Dose Level 1 and 2 at risk in Dose Level 2. With limited group counts, descriptive event summaries provide a restricted basis for judging the range or precision of safety estimates. The registry does not report an overall adverse-event estimate or an uncertainty interval.

Details about missing observations, participant flow, endpoint-specific denominators and completion of follow-up are not reported in the registry record. The reason for termination is also not reported. These gaps make it difficult to assess how termination and incomplete information may relate to the reported outcomes.

The registry lists secondary outcomes but does not report their results. It also does not report the detailed statistical plan, interim analyses, multiplicity handling or missing-data methods. These limitations are specific to what can be interpreted from this record; they do not establish what investigators may have documented elsewhere.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: NT-I7 for Kaposi Sarcoma in Patients With or Without HIV — NCT04893018.
• Registry record last updated 2024-06-27; status last verified 2024-04.
• Results first posted 2024-06-27.
• 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

NCT04893018 is a phase 1, single-group study of NT-I7, identified as efineptakin alfa, for Kaposi sarcoma in patients with or without HIV. Fred Hutchinson Cancer Center is listed as sponsor. Enrollment was 8. The study began on 2022-05-23, had a primary completion date of 2023-12-15 and is listed as terminated.

The primary outcome is the percentage of participants with adverse events, assessed among enrolled participants who received at least one dose and measured using Common Terminology Criteria for Adverse Events version 5.0. The observation period is up to 30 days after the last dose, approximately 31 weeks total. Selected serious-event data are listed by dose level, but the registry does not report an overall primary endpoint result, its uncertainty, or results for the listed secondary outcomes.

The single-group design has no control arm, and the dose-level summaries are not a like-for-like comparison. The reason for termination, detailed statistical plan and handling of missing data are not reported in the registry record. The available information supports descriptive reading of the registered design and selected safety figures, not conclusions about comparative effects.