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 Paclitaxel, Bevacizumab and Pemetrexed in Patients With Untreated, Advanced Non-Small Cell Lung Cancer Using Web-Based Data Collection, Patient Self-Reporting of Adverse Effects and Automated Response Assessment (NCT00807573), a na phase 2 study. The primary outcome is Objective Response Rate (CR + PR by RECIST) Paclitaxel, Pemetrexed, and Bevacizumab in Patients With Advanced Non-Small Lung Cancer Who Have Received no Prior Treatment for Metastatic Disease..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00807573 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 44 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Memorial Sloan Kettering Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced non-small cell lung cancer who had received no prior treatment for metastatic disease. The registry lists lung cancer as the condition and an enrollment of 44. It does not give further eligibility details or describe the characteristics of participants included in the primary endpoint analysis.
Intervention
A combination of paclitaxel, pemetrexed and bevacizumab. The registry describes administration of the drugs together during 28-day cycles, with treatment on days 1 and 15. It also describes web-based data collection, patient self-reporting of adverse effects and automated response assessment as features of the trial.
Comparator
There was no separate control group. The design is listed as single-group, with allocation not applicable and no masking. The study therefore does not provide a randomized, like-for-like comparison with another treatment or with no treatment.
Primary question
The primary endpoint asks how many participants had an objective response, defined as complete response or partial response under RECIST v1.0. The registry describes assessment by MRI and gives a two-year timeframe. It does not report the number who responded, the response percentage, or an uncertainty interval, so the size and precision of the observed response cannot be established from the registry record.
The clinical question is whether tumors shrink during treatment with this three-drug combination in patients with untreated advanced disease. The trial is Phase II and is designed around observing responses in a treated group, rather than estimating a comparative treatment effect. Without a control group, a response count would describe outcomes among participants receiving the combination; it would not by itself establish how those outcomes compare with another regimen or what would have occurred without the combination. The registry record does not report a primary endpoint result, so it does not permit a quantitative answer to the response question.
3. Trial Design
This is a Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. The registry lists enrollment of 44 and records the study as completed. Allocation is listed as not applicable, and masking as none. Those design features mean participants were not assigned between trial arms in a randomized comparison, and treatment was not masked. The registry record does not describe a comparator group.
Participants received paclitaxel, pemetrexed and bevacizumab together. The regimen description specifies a 28-day cycle, with the drugs given intravenously on days 1 and 15. Paclitaxel is described at 90mg/m² over 60 minutes, and pemetrexed at 500mg/m² over 10 minutes. The bevacizumab description gives 10mg but ends without a complete dose description. Additional dosing details, treatment duration, dose modifications and stopping rules are not reported in the registry record.
The study's stated focus included web-based data collection, patient self-reporting of adverse effects and automated response assessment. Its primary outcome is objective response, defined as complete response plus partial response by RECIST v1.0, with a two-year timeframe. The registry identifies MRI assessment, but does not describe the assessment schedule, confirmatory procedures, or how missing or unevaluable scans were handled.
The record lists a start in 2008-12 and primary completion in 2014-04. It does not report the study protocol, a statistical analysis plan, a sample-size justification, interim analyses, or a detailed account of the analysis population. Those omissions limit interpretation of how the trial was planned and how its endpoint would be analyzed. The single-group structure also means that outcomes, if reported, would be descriptive of this treated cohort and would not constitute a direct estimate of a treatment difference.
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 | Paclitaxel, Bevacizumab & Pemetrexed |
|---|---|
| STARTED | 44 |
| COMPLETED | 42 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Paclitaxel, Bevacizumab & Pemetrexed |
|---|---|
| Withdrawal by Subject | 1 |
| Adverse Event | 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 | Paclitaxel, Bevacizumab & Pemetrexed |
|---|---|---|
| Participants analysed | 44 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 28 |
| Age, Categorical: >=65 years | Participants | 16 |
| Sex: Female, Male: Female | Participants | 22 |
| Sex: Female, Male: Male | Participants | 22 |
| Region of Enrollment: United States | participants | 44 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Response Rate (CR + PR by RECIST) Paclitaxel, Pemetrexed, and Bevacizumab in Patients With Advanced Non-Small Lung Cancer Who Have Received no Prior Treatment for Metastatic Disease., measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is objective response rate, defined as complete response or partial response under RECIST v1.0. The registry describes complete response as disappearance of all target lesions. Partial response is described as a decrease of >=30% in the sum of the longest diameters of target lesions. The record identifies MRI as the assessment method and gives a two-year timeframe.
Objective response is a binary outcome for each participant: the participant either meets the criteria for complete or partial response, or does not. Across a study group, it can be expressed as the count of participants meeting either response category, and often as the proportion of participants assessed. This endpoint is used to characterize tumor shrinkage during a treatment study. It is not the same as a time-to-event outcome, and it does not, by itself, describe how long a response lasts or whether outcomes differ from those under another treatment.
The registry record does not report the response count, the denominator used for the endpoint, a response percentage, or an uncertainty interval. It also does not specify in the result record how participants with missing or unevaluable assessments were classified. Consequently, the endpoint's 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
The registry record identifies objective response as the primary endpoint, but no result for that endpoint is reported. It gives neither the count of complete and partial responses nor the number of participants included in the endpoint analysis. A response rate, confidence interval, or test result therefore cannot be described from the registry record. The study's stated purpose is to determine the percentage of patients whose tumors shrink, but the posted record does not provide that percentage.
This absence is important for interpretation. A binary response outcome can be summarized by the number meeting the response criteria and the total number evaluated. The denominator matters because participants who start treatment may not all have an evaluable response assessment. The registry lists enrollment of 44, but does not establish that all 44 were included in the primary endpoint denominator. The safety risk counts are not a substitute for the endpoint denominator.
Even if a response result were available, the single-group design would support a description of responses in the treated group, not a randomized estimate of comparative benefit. With no comparator, differences attributable to patient characteristics, disease course, or other factors cannot be separated from the treatment group's observed outcomes. The record lists no masking, which is relevant to how assessment procedures might be interpreted; details of automated assessment and safeguards against measurement bias are not reported.
The lack of a reported confidence interval also means the precision of any response estimate cannot be assessed. In general, a confidence interval communicates the range of values compatible with the observed data under the method's assumptions. No interval is reported for this endpoint. Similarly, no p-value or other statistical analysis is reported, and the registry does not state a hypothesis test against a particular reference value. The absence of such statistics should not be interpreted as evidence either for or against an effect.
The serious-adverse-event summary gives a separate description of safety reporting: 20 participants are listed as affected among 44 at risk, while the other adverse-event summary lists 44 affected among 44 at risk. These are not measures of tumor response, and they do not resolve the missing primary endpoint result. The registry also lists selected serious event terms with percentages, but those entries do not provide a full account of all safety outcomes or establish that an event was caused by the regimen.
In general, over-dispersion matters when observed variation is greater than a statistical model expects; ignoring it can make uncertainty estimates too narrow. The registry record does not report a model for the primary endpoint or indicate whether over-dispersion was assessed. No model-based inference should therefore be attributed to this record. Overall, the available information describes the question, design, endpoint definition and selected safety counts, but not the observed objective-response result or its statistical precision.
9. Statistical Methods Explained
The methods can be understood by separating the study question, the endpoint definition, the design and the analysis results. The study asks about tumor response under a three-drug regimen. Its endpoint is binary at the participant level: complete response or partial response counts as an objective response under the stated RECIST v1.0 definition. A binary endpoint can be summarized with a count and a denominator. The registry record does not report either component for the primary outcome, so no response proportion can be interpreted.
What does a single-group design estimate?
A single-group study observes outcomes among participants receiving the study regimen. If response results are reported, the direct estimate is the response frequency in that group, subject to the endpoint definition and analysis population. It is not a treatment contrast. A randomized comparison can help distinguish differences between assigned treatments from other influences by comparing groups formed under the trial's allocation process. This study has no separate comparator, and allocation is listed as not applicable. Its design therefore does not support a randomized comparison with another treatment or control.
The absence of masking is also part of the design context. Masking can limit the influence of knowledge of treatment assignment on conduct or assessment. The registry lists no masking but also describes automated response assessment. It does not explain how the automation operated, whether assessments were reviewed independently, or what safeguards were used. Those details are not reported in the registry record, so the potential role of assessment procedures cannot be evaluated further.
Why does the denominator matter?
For an endpoint expressed as a proportion, the numerator is the number of participants meeting the response definition and the denominator is the number included in the analysis. Enrolled participants and participants with an evaluable assessment need not be the same set. A participant without a recorded assessment might be excluded, counted as a non-responder, or handled in another prespecified way. Each approach can affect the estimate. The registry lists enrollment of 44 but does not report the primary endpoint denominator or how missing assessments were handled. It is therefore not possible to determine which analysis population was used.
In general, a confidence interval describes statistical uncertainty around an estimate under a chosen method and its assumptions. It reflects that a study's observed result is an estimate rather than a perfectly precise value for a broader population. The interval's width depends on the amount of information and the analysis. No confidence interval for objective response is reported, so the precision of the response estimate cannot be assessed. A p-value would address a specified statistical hypothesis under a chosen model; it would not measure the size or clinical importance of an effect. No p-value or hypothesis test for the primary endpoint is reported.
What is and is not reported about planning?
The registry record does not report a sample-size justification, protocol-based power target, interim analysis plan, or statistical analysis plan. It is therefore not possible to explain what assumptions guided the trial's original enrollment target or what analysis was planned for the primary outcome. A planning calculation is not the same as observed power: planning calculations illustrate how many participants might be needed under stated assumptions, whereas observed power is not a substitute for reporting the endpoint estimate and its uncertainty.
The record's planning-illustration assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect defined as a stated fraction of an observed effect. Those are assumptions for planning illustrations, not evidence that this single-group trial used a two-arm design or had those protocol features. The study itself is listed as single-group. In general, planning results depend on the assumed effect and analysis; they do not supply missing observed response data or show that a result was statistically significant.
For a binary outcome, the normal approximation is a general method that can be used in some planning or inference settings. Its suitability depends on the data and assumptions. The registry does not report the response count, the model used, or a method for calculating uncertainty. There is no basis to attribute a particular approximation or inferential procedure to the primary analysis.
How should safety counts be interpreted?
Safety counts describe participants reported as affected by adverse events, not the efficacy endpoint. The registry lists 44 participants at risk for other adverse events and 44 affected, and 44 at risk for serious adverse events with 20 affected. The listed serious event terms include percentages, but the record does not give event-level counts for those terms. In general, one participant may experience more than one event type, so event categories should not be treated as mutually exclusive unless the reporting method establishes that. The listed percentages should be read as the registry's reported figures, not as a complete event distribution or a comparative risk estimate.
Overall, the methods information establishes a single-group Phase II structure and a binary response definition. It does not establish the endpoint analysis population, missing-data approach, inferential method, or precision of the observed response. These gaps prevent reconstruction or independent statistical interpretation of the primary result from the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists one primary outcome measure, objective response, and does not report a primary endpoint analysis. It does not provide a statistical analysis plan or describe whether additional outcomes were tested, how multiple comparisons would be handled, or whether any interim analyses were planned. Because these details are absent, it is not possible to determine whether multiplicity adjustments were used or considered. A single listed primary outcome does not establish that no other analyses took place.
Missing-data handling is also not reported. The record does not state how participants without an evaluable MRI assessment would be treated in the response analysis, whether assessments were missing, or how many participants contributed to the endpoint. These choices matter for a binary endpoint because excluding unevaluable participants and classifying them as non-responders can yield different summaries. The enrollment count of 44 should not be assumed to be the primary endpoint denominator.
The analysis population is not described in the registry record. It does not state whether the primary analysis included all enrolled participants, participants who began treatment, or another defined group. No per-protocol or sensitivity analysis is reported. Without a stated analysis population and missing-data method, the target of any response estimate cannot be established from the record.
In general, multiplicity concerns arise when many outcomes or tests are examined, because the chance of a nominally notable result can change with the number and structure of analyses. Missing-data methods concern how absent outcome information is handled and what assumptions are made about it. These general issues cannot be resolved by the registry entry: the relevant analysis details are not reported in the registry record.
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 |
|---|---|---|---|
| Paclitaxel, Bevacizumab & Pemetrexed | 20 | 44 | 45.5% |
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 |
|---|---|
| Paclitaxel, Bevacizumab & Pemetrexed | 44 of 44 |
Most frequent serious adverse event terms
| Event term | Organ system | Paclitaxel, Bevacizumab & Pemetrexed |
|---|---|---|
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 3 of 44 |
| Infection, other | Infections and infestations | 3 of 44 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 2 of 44 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 2 of 44 |
| Inf norm ANC/gr1/2 neut-Pneumonia(lung) | Infections and infestations | 2 of 44 |
| Pain - Back | Musculoskeletal and connective tissue disorders | 2 of 44 |
| Pain - Chest wall | Musculoskeletal and connective tissue disorders | 2 of 44 |
| Pain - Extremity-limb | Musculoskeletal and connective tissue disorders | 2 of 44 |
| Death not assoc w CTCAE term- Death NOS | General disorders | 1 of 44 |
| Dehydration | Metabolism and nutrition disorders | 1 of 44 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Paclitaxel, Bevacizumab & Pemetrexed |
|---|---|---|
| Glucose, high (hyperglycemia) | Metabolism and nutrition disorders | 35 of 44 |
| Fatigue (asthenia, lethargy, malaise) | General disorders | 30 of 44 |
| Pain - Pain NOS | General disorders | 28 of 44 |
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 22 of 44 |
| ALT, SGPT | Investigations | 21 of 44 |
| Anorexia | Metabolism and nutrition disorders | 21 of 44 |
| Leukocytes (total WBC) | Blood and lymphatic system disorders | 21 of 44 |
| Pain - Muscle | Musculoskeletal and connective tissue disorders | 21 of 44 |
| Lymphopenia | Blood and lymphatic system disorders | 19 of 44 |
| Hemoglobin | Investigations | 18 of 44 |
Serious events with the highest arm-level rate: dyspnea (shortness of breath) (6.8%), infection, other (6.8%), fatigue (asthenia, lethargy, malaise) (4.5%), hypoxia (4.5%), inf norm anc/gr1/2 neut-pneumonia(lung) (4.5%).
The safety summary lists 44 participants at risk for serious adverse events and 20 affected. For other adverse events, it lists 44 at risk and 44 affected. These counts are separate from the primary objective-response endpoint. The record does not report a death count; deaths are not reported in the registry record.
The registry lists selected serious adverse-event terms with percentages: dyspnea (shortness of breath) and infection, other at 6.8% each; fatigue (asthenia, lethargy, malaise), hypoxia, infection with neutropenia and pneumonia, and back pain at 4.5% each. These are reported percentages for listed terms, not a full account of all events. The record does not provide event-level counts alongside these terms or explain whether participants could contribute to more than one category.
These safety summaries describe what the registry reports, but do not establish that the regimen caused a particular event. They also do not allow comparison with an untreated group or another treatment because the trial is single-group. The counts should not be read as a measure of treatment effect or as a complete characterization of safety.
12. What the Result Does, and Does Not, Mean
The record establishes that a Phase II, single-group study evaluated paclitaxel, pemetrexed and bevacizumab in patients described as having untreated advanced non-small cell lung cancer. It defines the primary endpoint as objective response, combining complete and partial response under RECIST v1.0, with a two-year timeframe. It also gives selected safety counts and adverse-event terms. These details clarify what the study set out to assess and the general structure of the evidence.
The record does not report the primary endpoint result. It gives no number of responses, response proportion, confidence interval, or statistical test. Therefore, the posted results do not quantify how often participants met the response definition or how precisely that outcome was estimated. No conclusion about the size of an observed response can be drawn from the endpoint information alone.
The design also places limits on what an outcome could establish. A single-group study has no randomized comparator, so it cannot directly estimate a difference between this combination and another treatment. Any observed outcomes would describe the treated cohort, while causal attribution and comparative interpretation would require evidence not reported in the registry record. The record does not report detailed eligibility criteria, participant characteristics, or the primary analysis population, which further restricts assessment of how broadly an eventual result might apply.
The safety counts are descriptive. They do not establish causation, and the listed serious event percentages are not a randomized comparison. The record does not report a death count or a complete account of safety analyses. Thus the registry supports a description of the study design, endpoint definition and selected reported safety figures, but not a quantitative conclusion about objective response, comparative effects, or the full safety profile.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its clinical question and its design answer different kinds of questions. Objective response is a defined binary endpoint that can describe tumor shrinkage within a treated group. The single-group design is suited to observing outcomes in that cohort, but it does not create a randomized comparison. Keeping the endpoint separate from the design prevents a within-group description from being mistaken for a comparative treatment effect.
The registry record also illustrates why an endpoint definition is not a substitute for an endpoint result. RECIST v1.0 criteria specify what counts as complete or partial response, but interpretation also requires the observed count, the denominator and a measure of uncertainty. Those result details are not reported for the primary endpoint. Without them, neither the magnitude nor the precision of the response estimate can be evaluated.
Safety reporting likewise requires careful separation of affected-participant counts, event terms and causal interpretation. The registry gives counts at risk and affected and selected serious event percentages, but those data do not provide a comparator or establish causation. Treating the safety summary as descriptive avoids making claims beyond the study design and recorded information.
Finally, the record shows the importance of reporting analysis populations, missing-data handling and statistical plans. These choices determine how outcome information is translated into an estimate and how much uncertainty should be attached to it. Their absence limits independent interpretation even when the study's broad objective and endpoint definition are clear. The statistical value of the record is therefore chiefly in its description of a single-group Phase II design and its stated measurement question, alongside the clear boundary that the primary result is not reported.
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 direct limitation for interpreting the primary question is that no objective-response result is reported. The record provides the endpoint definition and timeframe but not the response count, denominator, proportion, or uncertainty interval. It is consequently not possible to assess the endpoint's observed magnitude or precision.
The single-group design has no randomized control group, so it does not support a direct comparison with another treatment. The record lists no masking. Although automated response assessment is part of the study description, details of the assessment process and any safeguards are not reported. The registry also does not provide participant characteristics or detailed eligibility criteria beyond the stated disease setting and absence of prior treatment for metastatic disease.
The analysis population and handling of missing or unevaluable response assessments are not reported. Enrollment of 44 cannot be assumed to be the number assessed for the primary endpoint. The statistical analysis plan, sample-size justification, interim analysis plan and multiplicity approach are also not reported in the registry record. These omissions limit evaluation of how the study was planned and how its results were analyzed.
The treatment description is incomplete for bevacizumab: it gives 10mg but no further dose detail in the registry text. The record also does not report treatment duration, discontinuation details, or dose-modification rules. For safety, the registry gives affected-participant counts and selected serious adverse-event percentages, but not a death count or a full account of all safety results. These limitations constrain interpretation of both efficacy and safety beyond the registry entries explicitly recorded.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Paclitaxel, Bevacizumab and Pemetrexed in Patients With Untreated, Advanced Non-Small Cell Lung Cancer Using Web-Based Data Collection, Patient Self-Reporting of Adverse Effects and Automated Response Assessment — NCT00807573.
- Registry record last updated 2015-12-30; status last verified 2015-11.
- Results first posted 2015-12-30.
- 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
The registry record describes NCT00807573, a completed Phase II, single-group study sponsored by Memorial Sloan Kettering Cancer Center. It lists enrollment of 44 and evaluates paclitaxel, pemetrexed and bevacizumab in untreated advanced non-small cell lung cancer. The study description includes web-based data collection, patient self-reporting of adverse effects and automated response assessment.
The primary endpoint is objective response, defined as complete response plus partial response under RECIST v1.0, with MRI assessment and a two-year timeframe. The registry record does not report an endpoint result or statistical analysis. It lists 20 participants affected by serious adverse events among 44 at risk and 44 affected by other adverse events among 44 at risk, alongside selected serious event terms and percentages. These safety entries are descriptive and do not provide a randomized comparison.
The record does not report the primary endpoint denominator, missing-data handling, analysis population, statistical analysis plan, sample-size justification, or death count.