This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Bortezomib in Treating Patients With Stage IIIB or Stage IV Lung Cancer (NCT00118144), a na phase 2 study. The primary outcome is Objective Response Rate With Bortezomib Evaluated by Both RECIST Criteria and Computer-assisted Image Analysis..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00118144 |
| Phase | Phase 2 |
| Condition | Adenocarcinoma of the Lung, Bronchoalveolar Cell Lung Cancer, Non-small Cell Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with stage IIIB or stage IV lung cancer were the focus of the study. The official title specifies advanced bronchiolo-alveolar carcinoma (BAC) or adenocarcinoma with BAC features. The registry also lists several lung cancer conditions, including recurrent and non-small cell lung cancer. Further eligibility details and the population included in the primary endpoint analysis are not reported in the registry record.
Intervention
Patients in Arm I received bortezomib intravenously over 3-5 seconds on days 1 and 8. The registry record does not specify additional dosing-cycle details in the arm description.
Comparator
There was no separate comparator arm. The study is listed as single-group, with allocation marked NA and masking listed as none. Its results therefore do not provide a randomized, like-for-like comparison with another treatment or a control group.
Primary question
The primary question was whether bortezomib was associated with confirmed complete or partial responses, assessed using RECIST criteria and computer-assisted image analysis. The registry states that a response rate of 20% or more would be of interest for further evaluation, while a rate less than 5% would be of no interest. The observed response rate is not reported in the registry record.
3. Trial Design
This was a phase II, single-group study sponsored by the National Cancer Institute (NCI). The registry lists 42 enrolled participants and one treatment arm, Arm I, in which participants received bortezomib intravenously over 3-5 seconds on days 1 and 8. Allocation is recorded as NA, and masking as none. No randomized control group is described, so the design does not support a direct treatment comparison within the study.
The trial title concerns patients with stage IIIB or stage IV lung cancer, and its official title identifies advanced bronchiolo-alveolar carcinoma (BAC) or adenocarcinoma with BAC features. The registry conditions also include adenocarcinoma of the lung, bronchoalveolar cell lung cancer, non-small cell lung cancer, recurrent non-small cell lung cancer, and stage IIIB and stage IV non-small cell lung cancer. Detailed eligibility criteria and the exact analysis population are not reported in the registry record.
The recorded start was 2005-06, primary completion was 2010-12, and the status is completed. The primary endpoint was objective response rate, with progression-free survival and overall survival listed as secondary outcomes. The primary response assessment was specified as using RECIST criteria and computer-assisted image analysis. The registry record does not report the detailed assessment schedule, follow-up completeness, or the statistical plan. It also does not report whether participants received other care that could affect interpretation of outcomes.
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
| Stage | Arm I |
|---|---|
| STARTED | 42 |
| COMPLETED | 41 |
| NOT COMPLETED | 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.
| Characteristic | Unit | Arm 1 |
|---|---|---|
| Participants analysed | 42 | |
| Age, Continuous | years | 68 (54 to 75) |
| Sex: Female, Male: Female | Participants | 23 |
| Sex: Female, Male: Male | Participants | 19 |
| Region of Enrollment: United States | participants | 42 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate With Bortezomib Evaluated by Both RECIST Criteria and Computer-assisted Image Analysis., measured over Up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of responders |
| Endpoint classification | Binary Endpoint |
The primary endpoint was objective response rate, expressed as the percentage of responders. A responder was defined as a participant with a confirmed complete response (CR) or partial response (PR). Response was to be evaluated using both RECIST criteria and computer-assisted image analysis, with a timeframe of up to 5 years.
In general, an objective response endpoint describes the proportion of participants meeting prespecified criteria for a tumor response. It is useful in a phase II study because it focuses on whether responses are observed under a defined assessment method. Confirmation is intended to distinguish a sustained qualifying response from a finding that is not confirmed, although the confirmation procedures and assessment schedule are not reported in the registry record.
The record states that a response rate of 20% or more would be of interest for further evaluation, whereas a rate less than 5% would be of no interest. These are stated decision thresholds, not the observed result. The registry record does not report the response count, response percentage, confidence interval, or a comparison between the two assessment approaches. It therefore does not establish how the observed endpoint related to those thresholds.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a phase II study with 42 enrolled participants and a single bortezomib arm. This design can describe outcomes among participants treated in the study, but it does not provide a concurrent control group for estimating how outcomes would have differed without bortezomib or with another treatment. Differences between the study participants and any outside group could reflect patient characteristics, disease course, assessment practices, or other factors, rather than treatment.
The primary endpoint was a confirmed complete or partial response assessed by RECIST criteria and computer-assisted image analysis. The registry record gives a response-rate threshold of 20% or more as being of interest for further evaluation and a rate less than 5% as being of no interest. It does not report the observed response count or rate. Consequently, the posted record cannot show whether the response rate reached either threshold, nor how precisely a response rate was estimated.
Progression-free survival and overall survival are listed as secondary outcomes, but their results and analysis details are not reported in the registry record. No time-to-event estimates, confidence intervals, or event counts for these outcomes are available in the recorded details. These endpoints therefore cannot add an outcome-based comparison or clarify the primary response finding from the information recorded.
The safety information lists 12 participants affected by serious adverse events among 42 at risk, and 42 affected by other adverse events among 42 at risk. The serious-event terms include disease progression, dyspnea, vomiting, fatigue, back pain, and hemoglobin decreased, with percentages recorded for those terms. These are descriptive counts and term frequencies; they do not establish that bortezomib caused an event. The record does not give a comparator group, detailed event-level information, or a death count.
Overall, the registry record identifies what the trial intended to measure and provides selected safety counts, but it does not report the primary efficacy result. The study design and available posted results support description of the trial’s structure and recorded safety information, not a conclusion about comparative effects or the magnitude and precision of an observed response rate.
9. Statistical Methods Explained
The primary endpoint is binary at the participant level: each participant either meets the definition of confirmed CR or PR, or does not. In general, a response rate is the proportion of participants classified as responders in the analysis population. The registry describes the endpoint as a percentage of responders, but the number included in the primary endpoint analysis and the response count are not reported in the registry record. Enrollment alone does not establish how many participants had an evaluable response assessment.
RECIST criteria and computer-assisted image analysis are both named as assessment approaches. In general, prespecified response criteria help make outcome classification more consistent across participants and assessments. Using two approaches can allow the endpoint to be examined in more than one way, but the record does not explain how results from the approaches were combined, whether one was primary for adjudication, how disagreements were handled, or whether readings were independent. Those details are not reported in the registry record.
The endpoint definition requires confirmation of CR or PR. In general, confirmation is a rule about how an initial qualifying response is checked before it counts as an endpoint event. Its practical interpretation depends on the assessment schedule, confirmation window, handling of missed assessments, and rules for participants who discontinue follow-up. Those procedural details are not reported in the registry record. The stated timeframe is up to 5 years, but the record does not describe how response follow-up within that timeframe was organized.
A confidence interval, in general, expresses statistical uncertainty around an estimate under the assumptions of the analysis. For a response rate, it would help readers judge how compatible the data are with a range of underlying response rates. The registry record does not report an observed response estimate or its confidence interval, so the precision of the primary endpoint cannot be assessed from the record. No primary-endpoint p-value or statistical test is reported either.
The registry gives decision thresholds: a response rate of 20% or more would be of interest for further evaluation, and a response rate less than 5% would be of no interest. These statements provide context for the trial’s objective, but they are not a substitute for the observed estimate and its uncertainty. The record does not provide a statistical plan explaining how the thresholds were to be assessed or how uncertainty around the endpoint would inform interpretation.
In general, a single-group study estimates outcomes in the enrolled group without a randomized concurrent comparator. The observed response rate, if reported, would describe that group under the study’s conditions. It would not by itself distinguish an effect of treatment from other explanations, such as the natural course of disease, participant selection, or assessment influences. The registry lists the study as single-group, with allocation marked NA and masking as none. Further design details that could affect measurement and analysis are not reported in the registry record.
The registry also includes sample-size planning illustrations based on assumptions that specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. These are planning assumptions for hypothetical illustrations, not a description of this single-group trial’s design or an observed-power calculation. The illustrations are not the trial’s own design justification, and a sample-size justification for the trial is not reported in the registry record. No sample-size illustration should be interpreted as evidence that the study had a particular probability of detecting an effect.
Over-dispersion is a general concern when binary outcomes vary more across participants or groups than a model assumes. If present, a model that understates this variation can yield uncertainty estimates that are too narrow. The registry does not report a model-based analysis for the response endpoint, and no evidence about over-dispersion is reported in the registry record. The endpoint is presented as a response percentage, so readers should not assume a more elaborate model was used.
The registry lists progression-free survival and overall survival as secondary outcomes. In general, time-to-event methods account for the time until an event and may also account for participants whose event status is not observed by the end of follow-up. The record does not report the event definitions, censoring rules, estimation methods, or results for these outcomes. It is therefore not possible to assess their statistical treatment from 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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Arm 1 | 42 | 2 | 4.8% | 0.6% to 16.2% | 1.3% to 15.8% | 1.5% to 15.8% |
R code and output
arm <- c("Arm 1")
n <- c(42)
events <- c(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 Arm 1 42 2 4.8 0.6 16.2 1.3 15.8 1.5 15.811. Multiplicity, Missing Data and Analysis Populations
The registry identifies objective response rate as the primary endpoint and lists progression-free survival and overall survival as secondary outcomes. It reports three outcome measures in total, but the third measure is not identified in the recorded details. The registry record does not report a multiplicity strategy, such as an adjustment to account for evaluating several outcomes or assessment approaches. It also does not report whether any outcomes were designated as exploratory or how they were ordered for interpretation.
In general, multiplicity matters because evaluating multiple endpoints or repeated analyses can increase the chance of a seemingly notable result arising by chance. A prespecified approach can clarify which findings are confirmatory and how uncertainty should be interpreted across outcomes. Without a reported plan, the registry record does not establish how the primary endpoint, secondary outcomes, or two named response-assessment approaches were handled together statistically.
Missing-data procedures and analysis populations are not reported in the registry record. The record does not state whether the primary analysis included all enrolled participants, only those with evaluable assessments, or another defined population. It also does not describe how missing response evaluations, withdrawals, or incomplete follow-up were handled. This matters because the endpoint depends on classifying participants as responders or nonresponders, and absent assessments can affect the denominator and interpretation.
The registry reports zero analysis records. No interim analyses or stopping rules are reported in the registry record. Their absence from the record should not be taken as evidence that a particular procedure was or was not used outside what is recorded; the statistical plan is not reported in the registry record.
12. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Arm I | 12 | 42 | 28.6% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Arm I | 42 of 42 |
Most frequent serious adverse event terms
| Event term | Organ system | Arm I |
|---|---|---|
| Disease progression | General disorders | 4 of 42 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 3 of 42 |
| Vomiting | Gastrointestinal disorders | 2 of 42 |
| Fatigue | General disorders | 2 of 42 |
| Back pain | Musculoskeletal and connective tissue disorders | 2 of 42 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 1 of 42 |
| Abdominal pain | Gastrointestinal disorders | 1 of 42 |
| Constipation | Gastrointestinal disorders | 1 of 42 |
| Diarrhea | Gastrointestinal disorders | 1 of 42 |
| Ear, nose and throat examination abnormal | Gastrointestinal disorders | 1 of 42 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Arm I |
|---|---|---|
| Fatigue | General disorders | 29 of 42 |
| Nausea | Gastrointestinal disorders | 22 of 42 |
| Diarrhea | Gastrointestinal disorders | 20 of 42 |
| Peripheral sensory neuropathy | Nervous system disorders | 18 of 42 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 18 of 42 |
| Hemoglobin decreased | Blood and lymphatic system disorders | 15 of 42 |
| Disease progression | General disorders | 15 of 42 |
| Constipation | Gastrointestinal disorders | 14 of 42 |
| Anorexia | Metabolism and nutrition disorders | 14 of 42 |
| Cough | Respiratory, thoracic and mediastinal disorders | 13 of 42 |
Serious events with the highest arm-level rate: disease progression (9.5%), dyspnea (7.1%), vomiting (4.8%), fatigue (4.8%), back pain (4.8%).
The safety record lists 42 participants at risk for serious adverse events and 12 affected. For other adverse events, it lists 42 at risk and 42 affected. These counts describe the registry’s recorded safety groups; the record does not provide a detailed account of how events were collected or whether participants could contribute more than one event.
The listed serious adverse event terms are disease progression (9.5%), dyspnea (7.1%), vomiting (4.8%), fatigue (4.8%), back pain (4.8%), and hemoglobin decreased (2.4%). These are term-specific percentages in the registry record. They should be read as descriptive safety information, not as estimates of comparative risk, because no control arm is described. Disease progression is included among the listed serious terms, but the record does not explain the event classification or relationship to treatment.
The record gives no death count. It also does not report event duration, severity grades, timing, recurrence, or causal attribution in the information recorded. The safety figures therefore provide limited descriptive context and do not establish that bortezomib caused any listed event.
13. What the Result Does, and Does Not, Mean
The record establishes that a phase II study evaluated bortezomib in a single group of participants, with objective response rate as the primary endpoint. It also specifies how response was defined and gives response-rate thresholds that would inform interest in further evaluation. However, it does not report the observed response result. No conclusion about whether the endpoint met either threshold can be drawn from the recorded information.
Even if a response estimate were available, a single-group design would describe outcomes in the treated group rather than provide a randomized comparison. Without a comparator, the study cannot separate treatment-associated outcomes from other influences on response. The lack of reported analysis details also means the precision, population basis, and handling of missing assessments cannot be evaluated.
The registry lists progression-free survival and overall survival as secondary outcomes, but their results are not reported in the registry record. The available safety information gives counts and selected serious-event terms, but no comparative safety estimates or causal assessments. These registry entries should be interpreted as a limited description of what the record contains, not as a complete account of clinical benefit or harm.
The study’s stated aim was to investigate bortezomib in advanced lung cancer. The registry record does not provide enough outcome information to determine the magnitude or precision of an observed response, or to compare outcomes with another treatment. Its design and posted results therefore have a descriptive interpretation, with important efficacy and analysis information not reported.
14. Why This Trial Matters Statistically
This trial illustrates why endpoint definitions and outcome reporting need to be read alongside study design. The registry specifies a confirmed-response definition, names RECIST criteria and computer-assisted image analysis, and states thresholds for further interest. Those details clarify what the investigators intended to measure, but without the observed response count and rate they cannot show what the study found or how precisely it estimated the endpoint.
The single-group design also shows the distinction between describing outcomes and estimating comparative effects. An outcome observed among treated participants does not, by itself, show what would have happened under a different treatment or without treatment. Randomization and a concurrent comparator address different questions from a single-arm response assessment; neither can be inferred from this record, which lists one group and allocation as NA.
The record’s missing statistical details are also consequential. The analysis population, missing-data approach, multiplicity strategy, and endpoint results are not reported in the registry record. Without these elements, readers cannot fully assess the denominator, uncertainty, or how multiple outcomes were interpreted. The safety counts remain descriptive and lack a comparison group.
Statistically, the study is a useful example of why a stated decision threshold is not itself a result, why planning illustrations are not observed power, and why endpoint estimates require both a defined analysis population and an account of uncertainty. The record identifies a trial question and selected design and safety details, while leaving key outcome and analysis information unavailable.
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 most important limitation for interpreting efficacy is that the registry record does not report the observed primary response result. It supplies the endpoint definition and decision thresholds but not the responder count, response rate, or confidence interval. As a result, the primary question cannot be answered from the recorded results.
The single-group design has no concurrent control arm. Any outcomes observed in the group would not provide a randomized comparison, and the design cannot by itself distinguish treatment-associated outcomes from other influences. The registry lists masking as none; further details about outcome assessment and any safeguards against measurement bias are not reported in the registry record.
The registry does not report the analysis population, handling of missing response assessments, detailed response-assessment schedule, or method for reconciling RECIST and computer-assisted image analysis. These omissions limit assessment of how response classification and the endpoint denominator were determined. A statistical analysis plan, multiplicity procedure, interim-analysis details, and sample-size justification are also not reported in the registry record.
Although progression-free survival and overall survival are listed as secondary outcomes, their definitions, results, and analysis methods are not reported. The safety record provides selected counts and serious-event terms, but no death count or detailed event-level context. These limitations leave the available record insufficient for a complete evaluation of efficacy, safety, or statistical precision.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Bortezomib in Treating Patients With Stage IIIB or Stage IV Lung Cancer — NCT00118144.
- Registry record last updated 2014-08-21; status last verified 2013-04.
- Results first posted 2014-08-21.
- 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.
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19. Record Summary
This completed phase II study enrolled 42 participants and evaluated bortezomib in a single group. The registry lists the start as 2005-06 and primary completion as 2010-12. Participants in Arm I received bortezomib intravenously over 3-5 seconds on days 1 and 8.
The primary endpoint was objective response rate, defined by confirmed CR or PR and assessed using RECIST criteria and computer-assisted image analysis, with a timeframe of up to 5 years. Progression-free survival and overall survival were listed as secondary outcomes. The registry record does not report the observed response result or secondary-outcome results.
Safety information lists 12 participants affected by serious adverse events among 42 at risk and 42 affected by other adverse events among 42 at risk. The record reports no analysis records and does not report the statistical plan, analysis population, or missing-data approach.