Trial of Nab-sirolimus in Patients With Well-differentiated Neuroendocrine Tumors (NETs) of the Gastrointestinal Tract, Lung, or Pancreas Who Have Not Received Prior Treatment With mTOR Inhibitors: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Trial of Nab-sirolimus in Patients With Well-differentiated Neuroendocrine Tumors (NETs) of the Gastrointestinal Tract, Lung, or Pancreas Who Have Not Received Prior Treatment With mTOR Inhibitors (NCT05997056), a na phase 2 study. The primary outcome is Percentage of Participants With Objective Response Rate.

2. Clinical Question

3. Trial Design

This was a Phase 2, multi-center, open-label, single-arm study sponsored by Aadi Bioscience, Inc. It enrolled 12 participants with well-differentiated neuroendocrine tumors of the gastrointestinal tract, lung, or pancreas who had not received prior treatment with mTOR inhibitors. Nab-sirolimus was the listed intervention. The registry status is completed; the recorded study start date is 2023-10-20 and the primary completion date is 2025-10-03.

Because there was only one study group, the design can describe outcomes among participants receiving nab-sirolimus, but it does not estimate a treatment difference against a concurrent control group. The study was open-label, so masking was listed as none. The registry record does not describe an allocation procedure beyond noting that allocation was not applicable.

The primary outcome was the percentage of participants with objective response, defined as a confirmed complete or partial response based on the Investigator’s assessment under RECIST v1.1. The assessment timeframe began at study treatment initiation and extended to radiographically confirmed disease progression, with assessments at screening, every 6 weeks (±7 days) for the first 12 weeks, and every 12 weeks (±7 days) thereafter, up to 22 months.

The registry also lists outcomes covering treatment-emergent and treatment-related adverse events, duration of response, disease control rate, time to response, progression-free survival, and overall survival. The posted record contains no analyses. It does not report an enrollment rationale, a protocol-level statistical analysis plan, or further details on how these outcomes were analyzed.

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 Percentage of Participants With Objective Response Rate, measured over From the start of study treatment to radiographically confirmed disease progression (PD) per RECIST v1.1, assessed at screening, every 6 weeks (±7 days) for the first 12 weeks, and every 12 weeks (±7 days) thereafter, up to 22 months..

The primary endpoint is the percentage of participants with an objective response. A response counted when the best overall response was a confirmed partial response or complete response, as assessed by the Investigator using RECIST v1.1. The registry defines progression under those criteria as an increase in target-lesion measurements meeting the stated thresholds, unequivocal progression of non-target lesions, or the appearance of new lesions.

In general, an objective response endpoint compresses tumor-response assessments into a proportion: participants meeting the response definition are counted relative to the group assessed. It can provide a direct description of how often confirmed shrinkage meeting the specified criteria was observed during the assessment period. It does not, by itself, describe how long a response lasts, whether disease remained stable, or how outcomes compare with another treatment.

The registry specifies a 95% confidence interval as the dispersion measure for the primary endpoint. In general, a confidence interval conveys the uncertainty around an estimated response proportion. However, the registry record does not post the response count, percentage, or interval. The endpoint’s result and its precision therefore cannot be evaluated from the available record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The statistical information in the registry is limited. It defines the primary outcome and states that dispersion is expressed as a 95% confidence interval, but no outcome analyses are posted. The following explains the relevant methods in general and distinguishes those concepts from the analysis details that are absent from the registry record.

What does the response percentage describe?

In general, a binary outcome assigns each participant to one of two categories for a specified endpoint: the participant meets the response definition or does not. The response percentage is an estimate of the proportion meeting that definition in the analyzed group. Here, the registry’s definition counts participants with a confirmed complete response or confirmed partial response as the best overall response, assessed by the Investigator using RECIST v1.1.

The endpoint depends on the prespecified response rules and the assessments used to apply them. The registry describes progression criteria and the timing of assessments, but it does not report the number of participants classified as responders, the number included in the endpoint analysis, or whether any participants had unevaluable response data. Without those items, neither the observed proportion nor its calculation can be checked.

What does a confidence interval add?

In general, a confidence interval communicates the precision of an estimated quantity. A point estimate alone does not show how much uncertainty is associated with the estimate. The registry names a 95% confidence interval for the primary percentage, but does not post its bounds. As a result, the interval’s width and the precision of the response estimate are not available for interpretation.

A confidence interval is not a guarantee that the true value lies within a particular range, nor does it make a single-arm result comparative. It describes uncertainty under the statistical method and assumptions used to construct it. The exact interval method and any adjustments are not reported in the registry record, so the stated confidence level is the only interval detail that can be cited.

How does the design affect inference?

In general, a single-arm study estimates outcomes in the treated group without a concurrent randomized control. It can describe the observed response proportion, but does not estimate a between-group difference. The registry lists allocation as not applicable and masking as none. Therefore, its design does not support a randomized comparison, and no like-for-like control comparison is available in the record.

Open-label assessment can matter for outcomes that require judgment. The registry says response was determined by the Investigator under RECIST v1.1. It does not report whether independent review, adjudication, or other procedures were used. Such procedures must not be assumed. The record also does not describe the analysis population used for the response endpoint, including how participants who discontinued or lacked assessments were treated.

What do the sample-size assumptions mean?

The record describes a planning illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says that the effect to detect is a stated fraction of the observed effect. For time-to-event outcomes, the illustration is described in terms of the total number of events; for other outcomes, it is described in participants per arm. These are planning assumptions, not a description of the trial’s single-arm enrollment or an observed-power calculation.

No resulting planning values are reported in the registry record. The assumptions therefore cannot be used to infer the trial’s original sample-size justification, nor do they establish that the trial was designed or analyzed as a two-arm comparison. The record does not report a protocol-level sample-size rationale or explain how the planning illustration relates to the enrolled cohort.

More broadly, the registry does not report an analysis plan, interim analyses, adjustment methods, or the handling of missing data. General statistical concepts can explain why those details may matter, but they cannot fill in the study’s actual procedures. Any claim about the analyses performed beyond the endpoint definition and posted safety figures would go beyond what the registry record reports.

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("Neuroendocrine Tumors")
n      <- c(12)
events <- c(1)
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  Neuroendocrine Tumors  12  1  8.3  0.2  38.5  1.5  35.4  1.9  36.0

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary objective response outcome and secondary outcomes covering treatment-emergent and treatment-related adverse events, duration of response, disease control rate, time to response, progression-free survival, and overall survival. It reports seven outcome measures, but no analyses. The posted record does not state how many hypotheses were formally tested or whether any multiplicity procedure was used.

In general, when several outcomes are examined, the chance of obtaining at least one apparently notable result can increase if each is assessed independently. Statistical plans may address this through prespecified testing sequences or adjustment methods. No such approach is reported in the registry record, so no claim can be made about how multiplicity was handled.

The record also does not report missing-data rules, analysis populations, censoring conventions for time-to-event outcomes, or the number of participants contributing data to each outcome. These details can affect estimates and their interpretation. The assessment schedule for the primary endpoint is specified, but that alone does not say whether assessments were missed or how missing assessments were treated.

Consequently, the primary result cannot be reconstructed, and the secondary outcome findings cannot be evaluated from the posted analyses. The registry record does not report an interim-analysis plan or a fuller statistical analysis plan.

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: coronavirus infection (8.3%), pneumonia (8.3%), urinary tract infection (8.3%), vomiting (8.3%), acute kidney injury (8.3%).

The registry lists one group of 12 participants. It records serious adverse events for 4 participants, other adverse events for 12 participants, and 2 deaths. These are descriptive safety figures in the registry record; they do not establish that an event was caused by nab-sirolimus. The record does not provide further details about event timing, severity grading, exposure duration, or the attribution process.

The listed serious-event terms are Coronavirus infection, Pneumonia, Urinary tract infection, Vomiting, Acute kidney injury, and Pneumonitis. Each term is listed as 8.3%. The record does not give counts alongside these term percentages, so the percentages should be read as posted rather than converted into counts. It also does not explain whether participants could contribute more than one event term.

In general, adverse-event summaries describe events observed during a study and require context about definitions, follow-up, and attribution. The registry lists treatment-emergent and treatment-related adverse events among its outcomes, but does not post a full analysis of those outcomes. The safety entries therefore provide only a limited account of events recorded in this group and do not support comparisons with another treatment.

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

The registry record establishes that a completed, open-label, single-arm Phase 2 study enrolled 12 participants with well-differentiated neuroendocrine tumors of the gastrointestinal tract, lung, or pancreas who had not received prior mTOR inhibitor treatment. It identifies nab-sirolimus as the intervention and defines objective response as a confirmed complete or partial response under RECIST v1.1.

It does not report the primary response percentage, the number of responders, or the 95% confidence interval. The absence of those results cannot be interpreted as no response, and it cannot support a conclusion about the size or precision of the response estimate. The record also lists secondary outcomes but does not post their analyses.

In general, a single-arm result can describe what happened among participants in the study, but it does not establish how outcomes would have differed under a comparator. This study has no randomized control group, and masking is listed as none. Thus, even if outcome estimates were available, the posted design would not support a direct treatment comparison or a causal estimate of treatment effect.

The safety figures are likewise descriptive. The registry reports serious adverse events in 4 of 12 participants, other adverse events in 12 of 12, and 2 deaths, along with serious-event terms and their posted percentages. These entries do not by themselves establish causation or provide a complete account of safety over time.

The clearest conclusion is a boundary on interpretation: the record describes the trial’s population, design, endpoint, assessment schedule, and selected safety entries, but it does not report the primary efficacy estimate or statistical analyses. It therefore cannot answer how frequently objective responses occurred or how precisely that rate was estimated.

14. Why This Trial Matters Statistically

This trial is statistically informative as an example of the distinction between an endpoint definition and an endpoint result. The registry carefully defines objective response, identifies RECIST v1.1, specifies a 95% confidence interval, and describes the assessment schedule. Yet the response estimate and interval are not posted. Knowing how an outcome is defined does not reveal its observed value.

The single-arm design is also central to interpretation. A response proportion, if reported, would describe the enrolled group, not a randomized difference from another treatment. This distinction matters when readers are tempted to interpret a percentage as evidence of comparative benefit. No comparator group is listed, and the registry record does not report analyses that could supply one.

The enrollment of 12 makes it especially important to have the actual estimate and its uncertainty available. In general, the precision of a proportion depends on the data contributing to it, and a confidence interval helps readers assess that precision. The registry identifies the intended interval measure but omits its bounds, leaving readers unable to judge the estimate’s uncertainty.

Finally, the record illustrates why a protocol-level statistical plan and transparent results matter. Information about analysis populations, missing assessments, multiplicity, and the handling of time-to-event outcomes is not reported. Those omissions do not justify guessing how analyses were conducted; they mark what cannot be evaluated from the registry record.

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 for interpreting efficacy is that the registry record does not report the primary objective response result, its participant count, or its 95% confidence interval. It also lists no analyses. The record therefore cannot support a numerical description of the primary outcome or an assessment of its precision.

The study enrolled 12 participants and used a single-arm, open-label design. There is no randomized control group for comparison, and allocation is listed as not applicable. Outcomes can therefore not be interpreted as a randomized estimate of a treatment difference. The open-label design is relevant because response was determined by the Investigator; the registry record does not report whether independent review or other assessment safeguards were used.

The enrolled population included tumors of the gastrointestinal tract, lung, or pancreas, but the record does not provide outcome results by tumor site or other participant characteristics. It also does not report the analysis populations, the handling of missing or unevaluable response assessments, or the number of participants assessed for each endpoint. Although an assessment schedule and timeframe are given, completed assessment data are not reported.

For safety, the record lists selected serious-event terms and summary figures, but not a detailed account of event timing, severity, exposure, or attribution. The registry also does not report a statistical analysis plan, multiplicity procedures, or interim-analysis details. These limitations constrain interpretation; they do not establish that a particular analysis or procedure was or was not performed outside what is posted in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Trial of Nab-sirolimus in Patients With Well-differentiated Neuroendocrine Tumors (NETs) of the Gastrointestinal Tract, Lung, or Pancreas Who Have Not Received Prior Treatment With mTOR Inhibitors — NCT05997056.
• Registry record last updated 2026-08-25; status last verified 2026-07.
• Results first posted 2026-08-25.
• 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

The registry record describes NCT05997056, a completed, multi-center, open-label, single-arm Phase 2 study sponsored by Aadi Bioscience, Inc. It enrolled 12 patients with well-differentiated neuroendocrine tumors of the gastrointestinal tract, lung, or pancreas who had not received prior mTOR inhibitor treatment. Nab-sirolimus was the listed intervention.

The primary endpoint was the percentage of participants with a confirmed complete or partial response under RECIST v1.1, with a 95% confidence interval specified. The record gives the assessment schedule but does not report the primary result or any analyses. It lists secondary outcomes including adverse events, duration of response, disease control rate, time to response, progression-free survival, and overall survival.

Safety entries include serious adverse events in 4 participants, other adverse events in 12 participants, and 2 deaths. The design has no comparator group, so the record does not provide a randomized treatment comparison.