Study to Evaluate Using Nelfinavir With Chemoradiation for Non-small Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study to Evaluate Using Nelfinavir With Chemoradiation for Non-small Cell Lung Cancer (NCT00791336), a na phase 2 study. The primary outcome is Pathologic Complete Response.

2. Clinical Question

In general, a clinical question about a treatment regimen asks whether an outcome occurs after the regimen is given and, when there is a suitable comparator, whether its frequency differs between groups. This study was intended to examine nelfinavir alongside chemoradiation in the specified population, but its single-group design could not separate any observed outcome from the effects of the other treatments or from the course of disease. In this case, the absence of collected and analyzed outcome data also prevents a descriptive estimate for the primary outcome. The registry record lists characterization of overall and disease-free survival and safety and tolerability as secondary outcomes, but it does not report analyses for them. The study record consequently documents a research question and a planned treatment context, not an estimate of treatment effect.

3. Trial Design

The registry record describes a phase II trial sponsored by the University of Iowa. Its title identifies nelfinavir with concurrent radiation and chemotherapy for patients with stage III non-small cell lung cancer. The brief summary describes candidates for pre-operative treatment. The recorded model is single-group, allocation is not applicable, and masking is none. There is no control arm, so the study is not a randomized comparison and does not support a direct comparison of the regimen with another treatment.

The record gives a study start of 2008-08 and a primary completion date of 2011-03. The status is terminated. Enrollment was 1, and the primary outcome description says that one subject was enrolled and treated. It also says the study was terminated because of poor enrollment and that data were not collected or analyzed because of termination and low enrollment. Thus, the record contains no analyzable outcome result for the primary endpoint.

Nelfinavir is listed as the intervention, while the study summary and title place it alongside radiation therapy and chemotherapy. The arm description is empty. Dose, schedule, eligibility details beyond the stated population, treatment delivery, and additional protocol procedures are not reported in the registry record. The registry lists overall and disease-free survival characterization and safety and tolerability as secondary outcomes. It reports no analyses. The available information establishes the design and reason for termination, but not the full protocol or the details of how each outcome was to be assessed.

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: Only 1 subject was enrolled and treated. Study was terminated due to poor enrollment. Data were not collected or analyzed due to study termination and low enrollment count (n=1).

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 Pathologic Complete Response, measured over 30 days.

The primary endpoint is pathologic complete response, with a timeframe of 30 days. The registry record does not give a unit, operational definition, assessment criteria, or result. It therefore does not specify what findings were required to classify a participant as having a pathologic complete response, or how and when the assessment was to be performed within the stated timeframe.

In general, a pathologic response endpoint uses examination of tissue to describe whether evidence of cancer remains after treatment. It can serve as a measure of response to a treatment regimen, distinct from outcomes such as survival. The exact meaning of the endpoint depends on the tissue sampled, the criteria used, and the timing and method of evaluation; those details are not reported in the registry record. This distinction matters because the endpoint name alone is not enough to reproduce or interpret an assessment.

For this trial, the registry states that outcome data were not collected or analyzed because the study ended with one treated participant. No response count, proportion, or estimate is reported. The endpoint therefore identifies what the study intended to assess, but it supplies no evidence about the observed response in the enrolled participant or in the intended patient population.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The methods that can be described from the registry record are limited. It identifies a phase II, single-group trial with no masking, one enrolled and treated participant, no reported analyses, and termination because of poor enrollment. It does not report a statistical analysis plan, a sample-size justification for the trial, an interim-analysis plan, or detailed outcome definitions. The concepts below explain how such methods generally work; they should not be read as methods that this trial used unless the registry record explicitly says so.

What does a single-group design estimate?

In general, a single-group study can describe outcomes among participants who receive the study regimen. It does not have a concurrent control group from which to estimate a treatment contrast. If an outcome is observed, its interpretation may also depend on participant selection, other care received, and the natural course of the condition. This study is recorded as single-group, and its allocation is not applicable. The registry therefore does not describe randomized assignment to treatment groups or a like-for-like control comparison.

What does the primary endpoint require?

The named primary endpoint is pathologic complete response, with a 30-day timeframe. In general, an endpoint needs a clear operational definition, an assessment method, and a rule for classifying each participant before its results can be summarized consistently. The registry record does not report those criteria or methods. More importantly, it states that data were not collected or analyzed. There is consequently no endpoint value from which to estimate the frequency of response or quantify uncertainty.

What would an estimate and confidence interval mean?

In general, an estimate is a numerical summary of a quantity of interest, such as an outcome frequency. A confidence interval expresses statistical uncertainty around an estimate under the assumptions of the selected method. Neither an estimate nor a confidence interval for pathologic complete response is reported. Without an observed outcome, no interval can characterize the precision of the primary result. The record also reports no p-value or inferential test. A p-value, in general, measures how incompatible data are with a specified statistical model or null hypothesis; it does not measure the probability that a treatment works. Here, no such trial result is available to interpret.

How should the planning illustration be read?

The sample-size assumptions describe 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. The described effect to detect is a stated fraction of the observed effect. These assumptions are not reported as the trial's own design. The trial is single-group, and no observed primary outcome effect is reported, so the illustration cannot be treated as a calculation based on trial results. In general, planned sample size is a design quantity; it is not the number of participants actually analyzed, and it is not observed power. The registry does not report the trial's own sample-size justification or power calculation.

What can be inferred from the secondary outcomes?

The registry lists overall and disease-free survival characterization and safety and tolerability as secondary outcomes. In general, survival analysis requires information about follow-up time and whether and when an event occurred; the record reports no survival analyses or results. A time-to-event method such as a proportional-hazards model should not be assumed. Nor is any count model, regression method, or adjustment procedure reported. Over-dispersion is a general concern when count data vary more than a selected probability model expects; no such model or count outcome analysis is reported for this study, so it cannot be evaluated as a trial method.

What is the scope of the reported analysis?

The record reports zero analyses. It does not identify an analysis population, describe how participants would be handled in an efficacy analysis, or state whether the planned summaries would include all enrolled participants or only treated participants. The primary outcome description does say one subject was enrolled and treated, but that does not establish a formal analysis population. With outcome data not collected or analyzed, there is no basis for reconstructing an analysis set or checking model assumptions. The appropriate statistical reading is therefore descriptive: the design and termination are recorded, but no outcome analysis supports an estimate of benefit, harm, or precision.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary outcome, pathologic complete response, and secondary outcomes concerning overall and disease-free survival and safety and tolerability. It also reports three outcome measures. The record does not describe a hierarchy among outcomes, how many statistical tests were planned, or whether any adjustment for multiple comparisons was intended. These details are not reported in the registry record.

In general, testing multiple outcomes can increase the chance of a chance-positive finding if each is interpreted independently. A protocol may address this through a prespecified hierarchy, adjusted significance thresholds, or a clear distinction between confirmatory and exploratory outcomes. No such strategy is reported. Since the record reports zero analyses and no outcome data, there are no reported tests to adjust or interpret; multiplicity cannot be evaluated from observed results.

The primary outcome description states that data were not collected or analyzed because the study was terminated after low enrollment. This is not described as a set of collected outcome values with some values missing, nor does the record report an imputation method or another missing-data procedure. In general, missing-data methods depend on why measurements are unavailable and what information was collected. The record does not provide enough detail to classify missingness or assess a method.

An analysis population, such as all enrolled participants or a defined treated group, is not reported in the registry record. Although one subject was enrolled and treated, that fact alone does not specify an analysis rule. The record thus offers neither a multiplicity plan nor an analysis-population definition, and it contains no outcome analysis from which to assess their consequences.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

The adverse-event figures list the Nelfinavir group with one participant at risk for serious adverse events and zero affected. For other adverse events, the figures also list one at risk and zero affected. These are the recorded counts for the group; they should be read as limited descriptive entries, not as evidence that adverse events are impossible or absent from a broader population.

With only one participant enrolled and treated, the safety information is extremely limited. A zero affected count in this record cannot characterize uncommon events or provide a stable estimate of event frequency. The record does not list any top serious adverse-event terms. The death count is not reported in the registry record; it should not be interpreted as zero. The registry lists safety and tolerability as a secondary outcome, but it reports no analyses. These entries therefore do not support a broad safety conclusion about the combined regimen.

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

The record establishes that a phase II, single-group study of nelfinavir with radiation and chemotherapy was initiated for patients with stage III non-small cell lung cancer. It also records that the study was terminated because of poor enrollment after one participant was enrolled and treated. The primary outcome was pathologic complete response, but the registry says data were not collected or analyzed. These registry entries describe the study's status and the absence of an outcome result.

They do not show whether the participant had a pathologic complete response. They do not provide an estimate of response among a wider population, nor do they show whether the regimen performed better or worse than another approach. Because there was no comparator group, even a reported outcome in a single group would not establish a causal difference between treatment strategies. The listed survival and safety outcomes also have no reported analyses.

In general, a treatment effect requires an outcome contrast or another defensible design and analysis suited to the question. This record contains neither a comparative estimate nor an analyzed primary outcome. It also provides no confidence interval with which to judge precision. The safety figures record zero affected among one participant at risk for each event category, but those counts are too limited to establish a general safety profile. The death count is not reported.

The most defensible conclusion is therefore narrow: this trial record documents an intended clinical question and a study that ended before outcome data were collected or analyzed. It does not establish benefit, lack of benefit, or the safety of nelfinavir with chemoradiation.

13. Why This Trial Matters Statistically

This trial matters statistically because it illustrates the difference between a registered research question and an analyzable result. A study may identify a population, intervention, endpoint, and timeframe, yet still produce no estimate if enrollment is too low and outcome data are not collected or analyzed. Here, one participant was enrolled and treated, and the registry attributes termination to poor enrollment. The primary endpoint therefore remains a stated objective rather than an observed result.

It also highlights the limits of a single-group design. Without a comparator, the study could not estimate a between-group contrast. That limitation is separate from the absence of outcome data: even a completed single-group study would need careful interpretation, while this record does not provide an analyzed outcome to describe at all.

Finally, the record helps distinguish outcome reporting from safety evidence and from planning calculations. The adverse-event counts are descriptive and based on one participant. The planning assumptions are not the trial's own design and cannot substitute for observed data. Statistically, the important result is the absence of an estimable clinical outcome, not a favorable or unfavorable finding.

14. 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 most consequential limitation is the termination after one participant was enrolled and treated. The registry states that data were not collected or analyzed because of termination and low enrollment. As a result, there is no primary outcome estimate, no reported secondary outcome analysis, and no basis for assessing statistical precision.

The single-group design offers no concurrent comparator, and masking is recorded as none. The study therefore cannot support a like-for-like comparison. The record does not report detailed eligibility criteria, a full treatment schedule, nelfinavir dose, endpoint assessment criteria, or the operational definitions used for the listed survival outcomes. Those details are not reported in the registry record.

Statistical planning details for the trial itself are also absent: a sample-size justification, analysis plan, interim-analysis plan, analysis population, and approach to missing data are not reported. The separate planning illustration is not the trial's own design and cannot resolve those omissions. Safety information is limited to the reported counts for one participant; the death count is not reported. These limitations mean the record should be read as documentation of a terminated study, not as an efficacy or safety evaluation.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Study to Evaluate Using Nelfinavir With Chemoradiation for Non-small Cell Lung Cancer — NCT00791336.
• Registry record last updated 2017-07-28; status last verified 2017-06.
• Results first posted 2017-07-28.
• 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.

18. Record Summary

The registry record describes a phase II, single-group study sponsored by the University of Iowa, examining nelfinavir with radiation therapy and chemotherapy for stage III non-small cell lung cancer. The record lists no masking and no comparator. It gives a start date of 2008-08, a primary completion date of 2011-03, and a terminated status.

Enrollment was 1, and one subject was enrolled and treated. The study was terminated because of poor enrollment; the record says data were not collected or analyzed due to termination and low enrollment. The primary outcome was pathologic complete response with a 30-day timeframe. Overall and disease-free survival characterization and safety and tolerability were listed as secondary outcomes. No analyses are reported, so the record provides no analyzed outcome result.