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 Carboplatin, Gemcitabine, and Thalidomide in Patients Undergoing Surgery for Stage II or III Non-Small Cell Lung Cancer (NCT00281827), a non-randomized phase 2 study. The primary outcome is Number of Patients Reporting Clinical Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00281827 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Masonic Cancer Center, University of Minnesota |
| Results posted | Yes |
2. Clinical Question
Population
Patients with stage II or III non-small cell lung cancer who were undergoing surgery. The registry record lists lung cancer as the condition and describes the study population as having stage II and IIIA disease. It records enrollment of 22 participants. For the primary endpoint, the record says that 2 participants did not receive all 3 drugs for all 3 cycles, so the evaluable population analysis included 20 participants.
Intervention
Neoadjuvant treatment with carboplatin, gemcitabine, and thalidomide before surgery. The treatment arm description specifies the three drugs every 21 days for 3 courses. Conventional surgery is also listed as an intervention. The registry rationale describes the treatment as an attempt to affect tumor size before surgery; that rationale is not itself evidence of an observed effect.
Comparator
There was no concurrent control arm listed. The study was non-randomized and used a single-group model, with no masking. Consequently, the registry record does not describe a randomized comparison of the drug combination against another treatment or against surgery alone.
Primary question
The primary endpoint asks how many participants reported a clinical response at the end of the 3 -21 day cycles of treatment. Tumor assessments classified response using complete response, pathological complete response, partial response, progressive disease, or stable disease criteria. The registry record does not report the number of participants in each category or the overall clinical response count, so the primary result cannot be described from the record.
3. Trial Design
This was a phase II, non-randomized, single-group study with no masking. The registry lists enrollment of 22 and identifies Masonic Cancer Center, University of Minnesota as the sponsor. The study began in 2002-05, had primary completion in 2008-03, and is recorded as terminated. The reason for termination is not reported in the registry record.
The treatment was carboplatin, gemcitabine, and thalidomide, administered every 21 days for 3 courses. Conventional surgery was also listed. The study title and rationale describe treatment before surgery for stage II or III non-small cell lung cancer; the official title specifies stage II and IIIA disease. The registry record does not describe a separate control group or random assignment. This means the study design can describe outcomes in the treated group, but it does not create a like-for-like treatment comparison.
The primary endpoint was a count of participants reporting clinical response at the end of the treatment courses. The record identifies an evaluable population of 20 for that endpoint: 2 of the 22 participants did not receive all 3 drugs for all 3 cycles. The reason those participants did not complete the treatment as specified is not reported in the registry record. The count of participants who responded is also not reported.
Secondary outcomes covered disease-free status at 1 year and 2 years, survival at 1 year and 2 years, and survival at 56 months, described as the end of the study. Although these outcomes are named, their results are not reported in the registry record. No analysis details are listed. In particular, the record does not report the statistical plan, the reason for selecting the enrollment target, or whether interim analyses were planned or performed. The terminated status alone does not explain why the study ended.
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 | Intent-to-Treat |
|---|---|
| STARTED | 22 |
| COMPLETED | 22 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: 2 of 22 patients did not receive all 3 drugs for all 3 cycles - only 20 patients achieved this and are thereby included here in the evaluable population analysis.
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 | Intent-to-Treat |
|---|---|---|
| Participants analysed | 22 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 19 |
| Age, Categorical: >=65 years | Participants | 3 |
| Age, Continuous | years | 53 (32 to 78) |
| Sex: Female, Male: Female | Participants | 12 |
| Sex: Female, Male: Male | Participants | 10 |
| Region of Enrollment: United States | participants | 22 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients Reporting Clinical Response, measured over At end of 3 -21 day cycles of treatment.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants reporting clinical response, assessed at the end of the 3 -21 day cycles of treatment. It is a count endpoint: it describes how many participants met the study’s response definition, rather than measuring the size of an average change across all participants. The endpoint description refers to tumor assessments and defines response categories. Complete response requires disappearance of target and non-target lesions and normalization of a tumor marker level, if applicable. Pathological complete response means no viable tumor cells in the specimen by light microscopy. Partial response requires at least 30% decrease in the sum of the longest diameters of target lesions from baseline. Progressive disease requires at least 20% increase in that sum or new lesions. Stable disease is neither partial response nor progressive disease.
In general, a tumor-response endpoint can provide a structured way to describe tumor status over a specified assessment period. It can distinguish categories of change or lack of change using predefined assessment rules. Its interpretation depends on how assessments were conducted and how participants with incomplete treatment or unavailable assessments were handled. Those operational details are not reported in the registry record beyond the evaluable-population note.
The endpoint’s result count and the distribution across response categories are not reported in the registry record. Therefore, the description establishes what was intended to be counted, but it does not establish how many participants met any response category.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a clinical-response count as the primary endpoint and says that 20 participants were included in the evaluable population analysis. It does not report the number of those participants who had a clinical response. Without that count, there is no primary response result to interpret, and no estimate of response frequency or its precision can be stated. The response category counts are also not reported in the registry record.
The design is important when considering what any reported outcome could establish. This was a single-group, non-randomized study without masking. Outcomes in the group could describe what occurred among participants receiving the treatment and proceeding through the study assessments. Without a concurrent control group, the study does not provide a randomized comparison that separates the treatment experience from other explanations for observed outcomes. The registry record does not report a comparator or another basis for a causal treatment contrast.
The enrollment figure is 22, while the primary endpoint’s evaluable population is 20 because 2 participants did not receive all 3 drugs for all 3 cycles. These figures refer to different analysis populations. The record does not explain whether the 2 participants had any response assessments, why their treatment differed from the specified courses, or how they were handled for outcomes other than the primary endpoint. It would therefore be inappropriate to assume that the evaluable population applies to every outcome.
The listed secondary outcomes concern disease-free status and survival at specified follow-up times, including 56 months, which the record describes as the end of the study. Their result counts are not reported in the registry record. Follow-up completeness, definitions for these outcomes, and handling of participants whose status was unknown are also not reported. The terminated status does not by itself indicate whether follow-up was complete or what results were obtained.
The record lists 6 outcome measures and 0 analyses. It does not report estimates, confidence intervals, p-values, or a statistical analysis plan. In general, a confidence interval describes the uncertainty around an estimate under the assumptions of the analysis; no such interval is available for the primary response endpoint in this record. The absence of a reported analysis means there is no basis in the record for judging statistical precision or testing a hypothesis. These limitations concern what can be concluded from the registry record, not whether the treatment had an effect.
9. Statistical Methods Explained
What does a single-group design estimate?
In general, a single-group study records outcomes among participants who receive the study treatment. A count endpoint can report how many participants meet a specified outcome definition within that group. Such a count is descriptive unless the analysis also defines a suitable comparison and a method for estimating a contrast. In a non-randomized, single-group design, there is no randomized control arm to provide that contrast. Differences between the observed experience and what might have happened under another treatment cannot be isolated from the registry record alone.
For this study, the primary measure is the number of participants reporting clinical response. The record gives an endpoint assessment time and response definitions, but does not report the response count or a statistical analysis. As a result, a reader can understand what was intended to be measured, but cannot assess the observed response frequency, uncertainty, or any comparison with another group.
Why define response categories?
In general, prespecified categories make clinical assessments more consistent by linking labels to explicit criteria. The definitions in this record distinguish disappearance of lesions, a pathological assessment of the specimen, a decrease in measured lesion diameters, an increase or new lesions, and a state meeting neither the partial-response nor progressive-disease definition. These are different clinical classifications and should not be collapsed into a claim about an overall response result unless the analysis specifies how they are combined. The registry record does not report category-specific counts or further details about the classification process.
What does an analysis population mean?
An analysis population is the set of participants included in a particular outcome analysis. The record states that 20 participants were included in the evaluable population analysis for the primary endpoint because 2 of the 22 participants did not receive all 3 drugs for all 3 cycles. That is a specific statement about the primary endpoint. It does not establish how other endpoints were analyzed or whether all enrolled participants had complete data. The registry record does not report a general analysis-population plan, an intention-to-treat analysis for the primary endpoint, or the handling of missing outcome assessments.
In general, excluding participants from an evaluable analysis can change the group represented by the result, especially when the reasons for exclusion are related to treatment or outcome. The record does not describe why the 2 participants did not complete all specified treatment or whether their exclusion could be related to their clinical course. No direction or size of any resulting bias can therefore be inferred.
How should statistical uncertainty be read?
In general, an estimate is more informative when accompanied by a measure of uncertainty, such as a confidence interval. A confidence interval represents a range of values compatible with the observed data under the method’s assumptions. It does not replace the underlying design, and it cannot remove limitations such as the lack of a control group. No confidence interval or other uncertainty estimate for the primary response count is reported in the registry record.
For count outcomes, the number of participants with an event and the number assessed are both important to interpretation. The registry record provides the evaluable denominator for the primary endpoint but not the number with a response. The reported enrollment alone cannot be used to fill in that missing result. No unadjusted recalculation is available in the record for the primary endpoint.
What do the planning assumptions mean?
In general, sample-size planning translates a stated effect, significance level, allocation plan, and statistical model into an illustrative number of participants or events. The planning assumptions recorded for this study describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. They also describe effects as fractions of the observed effect. The record explicitly characterizes these calculations as a planning illustration computed from the observed effect, not as the trial’s own design.
Those assumptions do not describe the actual trial design, which was single-group and non-randomized. The two-arm equal-allocation assumption is not a like-for-like description of the study. Moreover, because the primary observed effect is not reported in the registry record, the planning illustration cannot be independently interpreted from this record. Planning numbers are hypothetical design aids, not observed power and not evidence that the study had a particular probability of detecting an effect.
In general, over-dispersion occurs when outcome variability is greater than a statistical model expects. If present and ignored, uncertainty estimates can be too small and statistical conclusions overly confident. The registry record does not report an over-dispersion assessment or a model-based analysis for the primary endpoint. No conclusion about whether over-dispersion mattered in this study can be drawn.
What analyses are documented?
The record lists 0 analyses. It does not report a statistical analysis plan, inferential tests, adjustment for multiple outcomes, interim analyses, or estimates with uncertainty. Those details are not reported in the registry record; they should not be inferred from the phase, title, or endpoint definitions. The available statistical interpretation is therefore limited to describing the design, the endpoint definition, and the stated analysis population.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 6 outcome measures: the primary clinical-response count and secondary outcomes for disease-free status at 1 year and 2 years, survival at 1 year and 2 years, and survival at 56 months, described as the end of the study. Examining multiple outcomes can raise a multiplicity concern in general, because repeated statistical tests can increase the chance of at least one apparently noteworthy result when no adjustment is made. The registry record does not report a multiplicity strategy, a hierarchy among the secondary outcomes, or statistical tests for these measures. It lists 0 analyses.
Missing-data handling is also not reported in the registry record. The record identifies 2 of 22 participants who did not receive all 3 drugs for all 3 cycles and says they were not included in the evaluable population analysis of the primary endpoint. It does not state why they did not receive the complete treatment, whether they had an endpoint assessment, or how they were treated in analyses of the secondary outcomes. The record does not describe imputation, censoring rules, or a sensitivity analysis.
The endpoint-specific evaluable population should not be assumed to be the same as an intention-to-treat population. The safety group is separately labelled Intent-to-Treat, but the primary endpoint population is described as evaluable. Whether the primary endpoint was also analyzed for all enrolled participants is not reported in the registry record. These missing analysis details limit assessment of how exclusions and unavailable outcomes might affect the reported findings.
11. 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 |
|---|---|---|---|
| Intent-to-Treat | 1 | 19 | 5.3% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Intent-to-Treat | 22 of 22 |
Most frequent serious adverse event terms
| Event term | Organ system | Intent-to-Treat |
|---|---|---|
| Carotid stenosis | Vascular disorders | 1 of 19 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Intent-to-Treat |
|---|---|---|
| Constipation | Gastrointestinal disorders | 17 of 22 |
| Nausea | Gastrointestinal disorders | 14 of 22 |
| Fatigue | General disorders | 13 of 22 |
| Neutropenia | Blood and lymphatic system disorders | 12 of 22 |
| Rash, body | Skin and subcutaneous tissue disorders | 10 of 22 |
| Anemia | Blood and lymphatic system disorders | 7 of 22 |
| Dizziness | Nervous system disorders | 7 of 22 |
| Diarrhea | Gastrointestinal disorders | 6 of 22 |
| Rash, peripheral | Skin and subcutaneous tissue disorders | 6 of 22 |
| Cough | Respiratory, thoracic and mediastinal disorders | 5 of 22 |
Serious events with the highest arm-level rate: carotid stenosis (5.3%).
The registry record lists a safety group labelled Intent-to-Treat. It reports 19 participants at risk for serious adverse events and 1 affected, alongside 22 participants at risk for other events and 22 affected. These are distinct entries with distinct denominators; they should not be combined into a single adverse-event count. The record also lists carotid stenosis at 5.3% among the top serious adverse event terms.
The term-level percentage describes the registry’s listing, but the record does not provide additional detail about event timing, severity grading, duration, attribution to treatment, or whether participants experienced more than one event. The serious-event group’s at-risk count is not the same as the enrollment count, and the reason for that denominator is not reported in the registry record. The other-event figures should not be read as a count of serious events.
In general, safety summaries describe recorded events within the stated population and follow-up context. They do not establish that an event was caused by a treatment, and a listed event does not by itself describe its clinical course. The record does not provide a broader safety analysis or enough information to compare event frequencies with another group.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase II study of carboplatin, gemcitabine, and thalidomide before surgery in patients with stage II or III non-small cell lung cancer. It specifies a clinical-response endpoint, describes the response categories, and states that the primary evaluable population included 20 participants. It also lists secondary disease-free and survival outcomes and a safety summary.
Those design and endpoint details do not reveal the treatment outcomes. The primary response count, category-specific counts, and results for the listed secondary outcomes are not reported in the registry record. No statistical analyses or uncertainty estimates are reported. Therefore, the record does not support a statement about the size or precision of a clinical response result.
The single-group, non-randomized design also limits what an outcome could establish. In general, a descriptive outcome among treated participants is not the same as a randomized estimate of the difference between treatment options. Without a concurrent comparator in this study, observed outcomes cannot be attributed to the treatment combination on the basis of this record alone. This does not determine what the outcomes were; it defines the limits of the design for comparative interpretation.
The difference between enrollment and the primary endpoint’s evaluable population matters for interpreting any eventual response count. The record says that 2 participants did not receive all 3 drugs for all 3 cycles, but does not report why or how their outcome data were handled. The stated 20-person evaluable population should not be extended automatically to other analyses.
The sample-size assumptions describe a planning illustration rather than the trial’s own design. Such planning values are not observed power, and they do not replace actual outcome estimates. Overall, the registry record makes the intended question and some design features clear, but leaves the principal outcome results and several analysis details unreported.
13. Why This Trial Matters Statistically
This trial illustrates why a registry entry needs to be read at several levels: the clinical question, the design, the endpoint definition, the analysis population, and the reported results. A clearly defined endpoint does not guarantee that its observed result is available. Here, the record describes a clinical-response count and detailed response criteria, while the count itself is not reported in the registry record.
The study’s single-group, non-randomized design also shows the distinction between describing outcomes and estimating a comparative treatment effect. A single-group result can characterize outcomes in the participants studied, but without a concurrent randomized comparison it cannot provide a like-for-like control contrast. This design feature remains important regardless of how an endpoint is defined or how large an observed result might be.
The record also distinguishes enrollment from the primary endpoint’s evaluable population. It records 22 enrolled participants and says that 20 were included in the primary evaluable population because 2 did not receive all 3 drugs for all 3 cycles. Readers need that context to understand who a reported primary outcome would describe. The record does not provide enough information to assess the reasons for those exclusions or their possible effect.
Finally, the entry names multiple secondary outcomes and gives planning assumptions, but does not report outcome analyses. This makes clear why planning illustrations, endpoint labels, and safety summaries should not be mistaken for a complete results analysis. For biostatistical interpretation, the missing outcome estimates and analysis details are central: without them, the record supports a careful account of the study’s structure, not a quantitative conclusion about its primary endpoint.
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.
Several trial-specific limitations follow directly from the registry record. The study was non-randomized, had a single group, and used no masking. No comparator arm is listed, so there is no randomized control comparison described. The record does not report the primary clinical-response count, response-category counts, or results for the named disease-free and survival outcomes. It also does not report confidence intervals, p-values, or other inferential results.
The primary endpoint analysis included 20 participants, while enrollment was 22. The record attributes the difference to 2 participants not receiving all 3 drugs for all 3 cycles, but does not report the reasons, their outcome assessments, or how they were handled outside the evaluable analysis. The record does not report a broader missing-data plan or establish that the primary analysis included all enrolled participants.
The study is recorded as terminated, but the reason for termination is not reported in the registry record. Follow-up completeness for the secondary outcomes is not described, including the outcomes at 1 year, 2 years, and 56 months. The registry record also does not report the statistical analysis plan, multiplicity handling, interim analyses, or the rationale for the enrollment target. The planning assumptions describe a two-arm illustration, although the actual study was single-group; those assumptions should not be treated as the trial’s design or as observed power.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Carboplatin, Gemcitabine, and Thalidomide in Patients Undergoing Surgery for Stage II or III Non-Small Cell Lung Cancer — NCT00281827.
- Registry record last updated 2017-12-28; status last verified 2017-12.
- Results first posted 2009-12-03.
- 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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18. Record Summary
NCT00281827 is a phase II, non-randomized, single-group study of carboplatin, gemcitabine, and thalidomide before surgery for stage II or III non-small cell lung cancer. The registry lists enrollment of 22, no masking, and a treatment schedule of every 21 days for 3 courses. It records the study as terminated, with no termination reason reported.
The primary endpoint is the number of participants reporting clinical response at the end of treatment. The record says 20 participants were included in the evaluable population analysis because 2 did not receive all 3 drugs for all 3 cycles. The primary response count and the results for the listed secondary outcomes are not reported in the registry record. No statistical analyses are listed. The design and endpoint definitions are documented, but the outcome results and several analysis details are unavailable in the record.