Ph II OSI-774 (Erlotinib,Tarceva) In Advanced Bronchioloalveolar Cell Lung Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Ph II OSI-774 (Erlotinib,Tarceva) In Advanced Bronchioloalveolar Cell Lung Cancer (NCT00590902), a na phase 2 study. The primary outcome is Overall Objective Response of OSI-774.

2. Clinical Question

The study asks a response question in a defined advanced lung cancer population: how many participants had an objective response while receiving the study drug? The primary objective, as described in the registry, was to determine the major objective response rate. The outcome measure is recorded as a number of participants, rather than as a reported response rate or a comparison between treatment groups.

The response criteria described in the record are based on RECIST. A complete response means disappearance of all target lesions; a partial response requires at least a 30% decrease in the sum of target lesions. Stable disease is defined as meeting neither the criteria for progressive disease nor those for partial response. The registry does not report how many participants met any of these categories, so the central clinical question cannot be answered from the posted results.

This is not a randomized comparison. It can describe outcomes among participants treated in the study, but the design does not provide a concurrent control group for estimating how outcomes would have differed under another treatment or no study treatment.

3. Trial Design

The registry describes a multicenter phase II study of OSI-774, also identified as erlotinib and Tarceva, in patients with advanced bronchioloalveolar cell lung cancer. The condition is recorded as the bronchioloalveolar cell variant of non-small cell lung cancer. The study has a single-group model, allocation listed as not applicable, and no masking. Enrollment is recorded as 81, and the study status is completed.

The recorded start was 2002-03, and primary completion was 2013-02. The primary endpoint timeframe is 53 weeks. These registry dates and the timeframe describe different features of the record: the dates refer to the study timeline, while the timeframe belongs to the listed outcome measure. The registry does not describe the visit schedule or the timing of individual response assessments beyond that timeframe.

Because the trial has no concurrent comparison group, it is not a randomized treatment comparison. The study can report the number of participants meeting response criteria during the study, but a single-group design cannot by itself separate an observed outcome from the course participants might have experienced without the intervention. The registry does not report a prespecified response threshold for declaring the study successful, a sample-size justification, or further details about the statistical design.

The sponsor entry names Memorial Sloan Kettering Cancer Center. The brief summary also says that the study is sponsored by Genentech. The registry does not explain the relationship between these descriptions. It identifies OSI-774 as the intervention but does not report its dose, route, schedule, treatment duration, or rules for changing or stopping treatment. These omissions limit how fully the intervention and operational design can be reconstructed from the 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 Overall Objective Response of OSI-774, measured over 53 weeks.

The primary outcome is titled Overall Objective Response of OSI-774. It is recorded as a number of participants and includes complete and partial responses. The registry describes response assessment using criteria proposed by the Response Evaluation Criteria in Solid Tumors Committee. Under that description, complete response is disappearance of all target lesions, while partial response requires at least a 30% decrease in the sum of target lesions. Stable disease means that neither the progressive disease nor partial response criteria are met.

Objective response is used in cancer trials to record measurable tumor change according to stated criteria. It turns imaging assessments into categories that can be counted, rather than relying only on a subjective impression of improvement. A response count can describe how many participants met the specified criteria, but it does not by itself describe how long responses lasted, whether participants lived longer, or how symptoms changed.

The endpoint timeframe is 53 weeks. The registry does not report the response count, the denominator used for the analysis, the response rate, or the handling of participants without an evaluable assessment. As a result, the endpoint definition is available, but its observed result and precision are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical methods help translate observed outcomes into estimates and characterize the uncertainty around those estimates. In this study, however, the registry record contains no reported primary analysis. The general explanations below describe how methods of this kind are commonly interpreted; they do not establish which methods the investigators used.

What does an objective response count represent?

The endpoint is recorded as the number of participants with complete or partial response. A count is a descriptive summary. To interpret it as a frequency among participants, the count must be considered with an appropriate denominator: for example, the participants eligible for the endpoint analysis or those with an evaluable assessment. The registry lists enrollment of 81, but does not report the denominator used for the response outcome. Enrollment alone cannot establish how many participants were assessed or included in an analysis.

A response proportion is often accompanied by a confidence interval. In general, a confidence interval represents uncertainty in an estimate under the statistical model and sampling assumptions used to calculate it. It communicates a range of values compatible with the observed data and method; it is not a guarantee that the true value lies in that range. No response proportion or confidence interval is reported in the registry record, so the precision of the primary outcome cannot be assessed.

How should a single-group result be understood?

In a single-group study, an outcome is observed among participants receiving the intervention, without a concurrent randomized comparator. A response count or proportion can describe that group. It cannot directly estimate a between-group treatment effect because there is no concurrent group representing what would have happened under a different assignment. General differences between groups are not the issue; rather, the counterfactual outcome for the enrolled participants is unobserved. This is why a single-group response result should not be expressed as a randomized comparative effect.

Randomization, when used, aims to balance both measured and unmeasured characteristics between assigned groups on average. This study is recorded as having allocation not applicable and a single-group model, so that protection against confounding by randomized assignment is not present. The registry does not describe an external control or a historical comparison used in the primary analysis.

What does the response definition add?

The record specifies categories based on tumor changes under RECIST criteria. A complete response requires disappearance of all target lesions. A partial response requires at least a 30% decrease in the sum of target lesions. Stable disease is described as fitting neither the criteria for progressive disease nor those for partial response. Clear categories can make assessments more reproducible than an undefined judgment, but the record does not report assessment procedures, independent review, or the handling of ambiguous or missing measurements.

Response is a categorical endpoint, while the primary measure is recorded as a count of participants. Such data are often analyzed using methods for binary outcomes, where each participant is classified as having or not having the event of interest. The registry record does not identify a particular model or test, and it does not report an estimate, uncertainty interval, or p-value. No specific method should therefore be attributed to the study based on the available record.

Why can analysis populations matter?

Different analysis populations can answer different questions. An analysis might include all enrolled participants, only participants who received treatment, or only those with evaluable response assessments. Those choices can change the denominator and interpretation of a response summary. The registry does not name an analysis population or provide participant flow information. The outcome denominator, exclusions, and reasons for missing assessments are not reported in the registry record.

Missing outcome assessments can matter because a participant without an assessment cannot automatically be classified as a responder or nonresponder without a prespecified rule. Complete-case analysis, imputation, or a conservative classification can lead to different summaries. These are general possibilities, not claims about this trial. The registry does not report the missing-data rule or the number of participants with unavailable response assessments.

What can planning calculations say?

Sample-size calculations are planning tools that depend on assumptions about the expected effect, variability, significance level, power, allocation, and analysis method. A planning illustration is not an observed result and is not observed power. The registry record does not report a sample-size justification or the assumptions used to plan enrollment. Nor does it report a primary response analysis from which an observed effect could be assessed. Thus, no trial-specific planning conclusion or power interpretation can be drawn from the recorded information.

Over-dispersion is a general issue for count outcomes when the observed variation exceeds what a chosen probability model expects. It can make standard errors too small if the model does not account for the extra variation. It is not possible to determine whether over-dispersion is relevant to this endpoint from the registry description: the primary outcome is a participant count in a single group, and no model or participant-level data are reported. In general, model choice should follow the design and data structure rather than be inferred from an endpoint label alone.

Finally, a statistical significance test, if conducted, would address compatibility of the data with a stated null hypothesis under its assumptions. It would not replace the need to report the effect estimate, its uncertainty, the analysis population, and the endpoint definition. No test result is reported in the registry record, and the record does not state a null hypothesis or analysis plan. The most defensible interpretation is therefore descriptive: the design and endpoint are documented, while the primary statistical result is unavailable.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary objective response outcome and one outcome measure. It reports no analyses. The record does not describe a statistical analysis plan, any planned secondary efficacy hypotheses, or a procedure for controlling multiplicity. It is therefore not possible to determine whether multiple comparisons were planned, whether any adjustment was used, or how the primary endpoint was intended to guide interpretation. The number of outcome measures alone does not establish the full set of analyses conducted.

Missing data require particular attention for a response endpoint because a participant without an evaluable assessment cannot be assigned a response category from the information recorded. The registry does not report how many participants had missing or unevaluable response assessments, whether assessments were performed for all enrolled participants, or what rule was used for participants without an assessment. It also does not report any imputation or sensitivity analysis. These details are not reported in the registry record.

The record lists enrollment of 81 but does not identify the primary analysis population or the number included in an efficacy analysis. It does not state whether the analysis was based on all enrolled participants, participants who received treatment, or participants with evaluable assessments. Those populations have different interpretations, and a reader should not assume that the enrollment count is the endpoint denominator.

The registry does not report interim efficacy analyses, stopping rules, or prespecified handling of protocol deviations. Their presence or absence cannot be inferred from the completion status. Without these analysis details and a reported endpoint result, neither multiplicity nor missing-data assumptions can be evaluated for their effect on the primary conclusion.

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.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: dyspnea (7.4%), pulmonary, other (4.9%), cough (2.5%), diarrhea (2.5%), abdominal pain/cramping (1.2%).

The safety group lists 81 participants at risk for both serious and other adverse events. It records 22 participants affected by serious adverse events and 66 affected by other adverse events. These are participant counts as recorded for the group. The registry does not report whether an individual could appear in both categories, how event severity was determined, or whether every participant had the same duration of safety observation.

The listed serious adverse event terms and percentages are dyspnea, 7.4%; pulmonary, other, 4.9%; cough, 2.5%; diarrhea, 2.5%; abdominal pain/cramping, 1.2%; and Adult Respiratory Distress Syndrome (ARDS), 1.2%. These are the terms reported in the registry. The denominator used for each percentage, the corresponding term counts, and whether participants could contribute to multiple terms are not reported in the registry record. The listed percentages should not be summed.

The record does not provide a death count. A null entry does not establish that no deaths occurred; the death result is not reported in the registry record. These safety summaries describe recorded events in the study group and do not support a comparison with another treatment group.

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

The trial record establishes that a completed phase II, single-group study enrolled 81 participants with advanced bronchioloalveolar cell variant of non-small cell lung cancer and evaluated OSI-774, identified as erlotinib and Tarceva. It also defines the primary response categories and records a timeframe of 53 weeks. These details show what the study set out to assess, but they do not supply the observed primary outcome.

The record does not report the number of participants with complete or partial response, the denominator for that outcome, or an uncertainty estimate. It therefore cannot show the magnitude or precision of the response result. This absence must not be read as a finding of no responses. It means that the response analysis is not reported in the registry record.

Even if an objective response count were available, its meaning would depend on the analysis population, assessment completeness, and response-assessment procedures. A single-group result would describe the participants observed in the study. It would not, by itself, establish what would have happened to the same participants under a comparator or demonstrate a causal difference from another treatment.

The safety entries provide counts for serious and other adverse events and percentages for selected serious event terms. These are useful descriptive details, but the registry does not provide all denominators and event-level information needed to fully interpret them. The record also does not report a death count. Safety data should therefore be read as a limited summary of recorded events, not as a complete account of risk or a comparative assessment.

In general, evidence from a clinical trial is interpreted using the study design, endpoint definition, results, precision, and analysis methods together. Here, the design and outcome definition are available, while the primary estimate and key analysis details are not. No conclusion about the size or certainty of an objective response effect can be drawn from the posted results alone.

13. Why This Trial Matters Statistically

This trial illustrates an important distinction between a documented endpoint and a reported result. The registry specifies an objective response outcome, explains the RECIST categories, and gives a timeframe of 53 weeks. But it does not report the response count or the analysis. A precisely described outcome is not enough to assess the size or uncertainty of an effect when the result itself is absent.

It also demonstrates why design matters to interpretation. A single-group phase II study can describe response experience among treated participants, but it does not include a concurrent randomized comparator. Even a clear response summary would not have the same interpretation as a randomized estimate of a difference between groups. Statistical precision and causal interpretation are separate questions: an estimate can be precise without being a randomized comparison.

The record offers another practical lesson about denominators. Enrollment is listed as 81, yet the number analyzed for the primary endpoint is not reported. Statistical summaries depend on knowing who was included and how participants without evaluable assessments were handled. Without those details, a result cannot be reconstructed or independently checked.

Finally, the safety summary shows the importance of distinguishing counts from percentages and of understanding their denominators. The record reports serious and other adverse event counts alongside selected serious-event percentages, but does not document all term-specific denominators or overlap. These limitations matter because descriptive safety figures should be read in the context of the population and follow-up represented by the data.

For biostatistical interpretation, the central issue is not the complexity of a method but the completeness of the reporting. The registry allows a reader to identify the trial question and several design features, while leaving the primary result and substantial analysis details unavailable.

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 main limitation of the registry record is that the primary response result is not reported. Although the outcome definition and timeframe are given, the record does not provide the response count, response proportion, confidence interval, or test result. It is therefore not possible to evaluate the magnitude or precision of the primary outcome from the posted results.

The analysis denominator and population are also not reported. Enrollment is 81, but the record does not say how many participants were treated, assessed, or included in an efficacy analysis. It does not explain whether participants without an evaluable assessment were excluded or assigned a category. These details are important for interpreting a participant-count endpoint.

The single-group design has no concurrent comparator, so it cannot provide a randomized between-group treatment effect. The registry does not describe a historical or external comparator. It also does not report the statistical analysis plan, sample-size justification, response decision threshold, interim analyses, or missing-data methods. These omissions prevent assessment of how the investigators planned to analyze and interpret the primary endpoint.

Additional operational details are not reported in the registry record, including dose, administration schedule, assessment schedule, response review procedures, and the handling of protocol deviations. The safety summary lists participant counts and selected event percentages, but term-specific denominators and overlap are not reported. A death count is not reported either.

The sponsor information is not fully consistent within the record: the sponsor entry names Memorial Sloan Kettering Cancer Center, while the brief summary says the study is sponsored by Genentech. The record does not explain this distinction. These limitations do not establish that the study lacked procedures or analyses; they mean that those details and results are unavailable in the registry record.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Ph II OSI-774 (Erlotinib,Tarceva) In Advanced Bronchioloalveolar Cell Lung Cancer — NCT00590902.
• Registry record last updated 2023-10-16; status last verified 2023-10.
• Results first posted 2016-01-20.
• 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 describes a completed multicenter phase II, single-group study of OSI-774, also identified as erlotinib and Tarceva, in advanced bronchioloalveolar cell variant of non-small cell lung cancer. Enrollment was 81. The recorded start was 2002-03, primary completion was 2013-02, and the primary outcome timeframe was 53 weeks.

The primary outcome was overall objective response, recorded as a number of participants with complete or partial response under the RECIST description. The registry does not report the response result, its denominator, or an analysis. The listed safety group records 22 participants with serious adverse events and 66 with other adverse events among 81 at risk; selected serious event terms are reported with percentages. The death result is not reported in the registry record.

The record documents the study question and several design features, but it does not provide the primary efficacy estimate or key statistical analysis details. Its single-group design also means that it is not a randomized comparison.