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, Pemetrexed Disodium, and Bevacizumab for Patients With Stage III or IV Non-Small Cell Lung Cancer Who Are Light/Never Smokers (NCT01344824), a na phase 2 study. The primary outcome is Progression-free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01344824 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 38 |
| Arms in the primary outcome | n/a |
| Lead sponsor | UNC Lineberger Comprehensive Cancer Center |
| Results posted | Yes |
2. Clinical Question
The registry record describes a phase II study of carboplatin, pemetrexed and bevacizumab for patients with stage III or stage IV non-small cell lung cancer who had a light or never smoking history. Its central question was how the regimen performed in this selected population, with progression-free survival named as the primary outcome. The stated treatment approach included maintenance therapy with pemetrexed and bevacizumab; the arm description also mentions an option for second-line erlotinib hydrochloride.
Population
Patients with stage III or stage IV non-small cell lung cancer and a light or never smoking history. The registry record does not give an operational definition of light smoking or further eligibility details.
Intervention
Bevacizumab, carboplatin and pemetrexed disodium, followed by pemetrexed and bevacizumab maintenance therapy as described in the official title. The arm description includes an option for second-line erlotinib hydrochloride.
Comparator
No concurrent comparator is listed. The study is recorded as single-group, with allocation marked NA and masking listed as none.
Primary question
What progression-free survival was observed with this treatment approach in the eligible population? The registry names progression-free survival as the primary outcome but does not report its median estimate or 95% confidence interval.
This is a within-group clinical question, not a randomized comparison of the regimen against another treatment. Without a concurrent control group, the registry record cannot establish how outcomes would have differed under an alternative treatment. The clinical question is further limited by the outcome information available: the record specifies the endpoint label and some assessment details, but does not report a numerical primary result or a statistical comparison.
3. Trial Design
This was a multicenter phase II trial sponsored by UNC Lineberger Comprehensive Cancer Center. The registry lists the study as completed, with a start date of 2010-03 and primary completion on 2016-05-24. Registry enrollment is 38. Its title and brief description identify patients with stage III or IV non-small cell lung cancer who were light or never smokers.
The design is recorded as single-group. Allocation is NA and masking is none, so the record does not describe random assignment or blinded treatment. The arm description names bevacizumab, carboplatin and pemetrexed disodium, with an option for second-line erlotinib hydrochloride. The official title describes carboplatin, pemetrexed and bevacizumab followed by pemetrexed and bevacizumab maintenance therapy. The record does not provide doses, administration schedules or details about how the treatment option was selected.
With no concurrent comparator, the study can describe outcomes among people treated in this group, but it does not directly estimate a treatment contrast against another regimen or usual care. Comparisons with results from other studies would be indirect and could be affected by differences in patient selection, follow-up and outcome measurement. The registry record does not report a statistical plan explaining how such comparisons might be handled.
The listed secondary outcomes are overall survival and subjects experiencing toxicity. The record reports 3 outcome measures, but it does not provide analysis results for them. It records 0 analyses. Consequently, the design details establish what kind of study was registered, but do not establish the numerical findings or the methods used to analyze them. Details such as eligibility criteria beyond the population description, follow-up completeness and analysis populations are not reported in the registry 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.
Before assignment: From March 2010 to November 2013, 52 patients consented to treatment. 14 patients were consented and screened for eligibility but found ineligible.
Overall Study
| Stage | Bevacizumab, Carboplatin, and Pemetrexed Disodium, With Option |
|---|---|
| STARTED | 38 |
| COMPLETED | 1 |
| NOT COMPLETED | 37 |
Reasons for not completing this period:
| Reason | Bevacizumab, Carboplatin, and Pemetrexed Disodium, With Option |
|---|---|
| Adverse Event | 15 |
| Withdrawal by Subject | 6 |
| Other complicating disease | 1 |
| Progression | 15 |
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 | Single Arm Trial |
|---|---|---|
| Participants analysed | 38 | |
| Age, Continuous | years | 63.5 (27 to 80) |
| Sex: Female, Male: Female | Participants | 25 |
| Sex: Female, Male: Male | Participants | 13 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 6 |
| Race (NIH/OMB): White | Participants | 30 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 38 |
| Eastern Cooperative Oncology Group (ECOG) Performance Status: grade 0 | Participants | 18 |
| Eastern Cooperative Oncology Group (ECOG) Performance Status: grade 1 | Participants | 14 |
| Eastern Cooperative Oncology Group (ECOG) Performance Status: not performed | Participants | 6 |
| Smoking Status: Never smoker | Participants | 24 |
| Smoking Status: Former light smoker | Participants | 14 |
| Histology: Adenocarcinoma | Participants | 37 |
| Histology: Neuroendocrine carcinoma | Participants | 1 |
| Previous erlotinib treatment | Participants | 10 |
| Mutation status: EGFR | Participants | 6 |
| Mutation status: KRAS | Participants | 5 |
| Mutation status: EML4/ALK | Participants | 0 |
| Mutation status: MET del14 | Participants | 1 |
| Mutation status: None (tested and no driver found) | Participants | 22 |
| Mutation status: Unknown | Participants | 4 |
6. Primary Endpoint
The registry lists the primary outcome as Progression-free Survival, measured over 1400 days.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | months |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival is the named primary endpoint, with the parameter specified as a median, the unit as months and the uncertainty measure as a 95% confidence interval. In general, a time-to-event endpoint records the time until a defined event occurs; progression-free survival is used to describe the time participants remain alive without documented disease progression under the study's endpoint definition. The registry record does not report the median estimate or its confidence interval, so the observed duration and its precision cannot be assessed from the posted results.
The endpoint description says that radiographic response was assessed using RECIST v1.0. It describes complete response as disappearance of target lesions and partial response as a >=30% decrease in the sum of their longest diameters; overall response is described as complete response plus partial response. The description also says assessments occurred each year until subject death and gives a timeframe of 1400 days.
These details leave some ambiguity: the endpoint is labeled progression-free survival, while the description focuses on radiographic response categories. The registry record does not explain how response assessment was used to define progression-free survival, how the event date was assigned, or how follow-up and censoring were handled. In general, time-to-event endpoints can capture both the timing of events and the duration before an event, while a median summarizes the estimated event-time distribution. That general purpose does not resolve the missing operational details in this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies progression-free survival as the primary outcome and specifies a median in months with a 95% confidence interval. It does not report the median, the interval limits or a hazard ratio. The record also lists 0 analyses. Thus, there is no posted numerical primary result to interpret for magnitude, precision or statistical evidence. The absence of a reported estimate is not evidence that progression-free survival was either favorable or unfavorable; it means the registry record does not provide that result.
Even if a within-group median were available, it would describe outcomes in the treated group, not the causal effect of the regimen relative to an alternative. The study is registered as single-group, with allocation marked NA. No concurrent control is listed. A change over time in a single group cannot be separated from the effects of patient characteristics, disease course, other care or other influences using this design alone. The record therefore does not support a randomized treatment comparison.
Precision also cannot be judged from the endpoint specification alone. A confidence interval expresses uncertainty around an estimate; its width and location would be needed to understand how compatible the result is with a range of underlying values. Although the record names a 95% confidence interval, its limits are not reported. The listed enrollment of 38 does not substitute for an outcome estimate or indicate how many participants contributed information to a particular analysis.
The record names overall survival and subjects experiencing toxicity as secondary outcomes, but does not report their analysis results. It reports 3 outcome measures and 0 analyses. The serious adverse-event summary includes affected counts and a list of terms with percentages, but that information cannot fill in the missing efficacy results. Nor does it establish a treatment-related cause for an event.
The stated sample-size assumptions describe planning illustrations based on a fraction of an observed effect, a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation. They are not observed power. The assumed equal allocation between two arms differs from the registered single-group design, so such illustrations should be understood as hypothetical planning scenarios rather than a description of how this trial was conducted. Since no effect estimate or numerical planning results are reported in the registry record, those assumptions do not supply evidence about the treatment outcome.
Overall, the record is informative about the population, planned treatment approach, study structure and endpoint label. It is not sufficient to determine the observed progression-free survival, its precision, or whether outcomes differed from those under another approach. Those questions remain unanswered by the posted results.
9. Statistical Methods Explained
The registry describes a time-to-event primary outcome and specifies the median and a 95% confidence interval. These are summary and uncertainty measures, not treatment comparisons by themselves. A median for a time-to-event outcome summarizes the point at which the estimated event-time distribution reaches its midpoint. A confidence interval communicates uncertainty around an estimate under the statistical method used. A narrower interval generally indicates greater precision than a wider one, but neither the estimate nor the interval is reported in this record.
What does a time-to-event endpoint capture?
In general, time-to-event methods use information about when an event occurs, rather than only classifying whether it occurred. Participants who have not had the event by the end of their observed follow-up may contribute information up to that point; this is commonly handled through censoring methods. The registry record does not state how censoring was defined or analyzed in this study, so no particular handling method can be attributed to the investigators.
The endpoint description refers to radiographic response under RECIST v1.0 and defines complete and partial response using target-lesion measurements. However, progression-free survival is the named primary endpoint, while the description emphasizes response categories. The record does not explain the operational link between those categories and the progression event used for the time-to-event analysis. It also does not specify assessment timing beyond the description that criteria were assessed each year until death, alongside the timeframe of 1400 days. Without further information, the endpoint construction and event ascertainment cannot be reconstructed.
What does the single-group design allow?
A single-group study can report outcomes observed among participants receiving the listed treatment approach. It does not directly compare those outcomes with a contemporaneous control group. Allocation is listed as NA, and the study model is single-group. Accordingly, there is no randomized contrast in the registry record to attribute a difference in outcome to treatment. General comparisons against external studies would not have the same protection from confounding as a randomized comparison; the record does not state that such a comparison was planned.
Masking is listed as none. The record does not explain whether endpoint assessment was independently reviewed or what steps, if any, were taken to reduce assessment bias. This matters in general because knowledge of treatment and clinical circumstances may influence decisions or measurements, although the record does not provide enough detail to assess whether that occurred.
How should confidence intervals and hazard ratios be read?
In general, a confidence interval gives a range of values compatible with an estimate under a specified method and its assumptions. It is a way to describe statistical uncertainty, not a guarantee that the true value lies within the reported limits. Interpretation requires the estimate, both limits, and relevant analysis details. The registry names a 95% confidence interval for the median but does not report the estimate or limits.
Hazard ratios are comparative summaries used in some time-to-event analyses. They relate event rates over follow-up under a model and do not have the same interpretation as a difference between medians. The registry record does not report a hazard ratio for this trial. It also does not report an analysis model, model assumptions or a comparative group, so no hazard-ratio interpretation is available. A model-specific result should not be inferred from the endpoint name alone.
What do the planning assumptions describe?
The record's sample-size assumptions describe planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. For time-to-event tables, the stated convention is a total number of events; other planning tables use participants per arm. These are planning assumptions, not observed trial results and not observed power.
There is a design distinction to keep in mind: the planning setup assumes equal allocation between two arms, whereas the registered study is single-group. The illustrations therefore do not describe the registered allocation. The registry record does not report the numerical planning outputs, the effect estimate on which they depend, or the trial's own sample-size justification. It also does not describe whether any sample-size calculation formed part of the original design. These omissions prevent a reader from using the planning assumptions to infer the study's intended statistical power or the precision of its actual outcome estimate.
Which analysis details are absent?
The registry reports 0 analyses. It does not report a statistical analysis plan, endpoint-specific analysis populations, missing-data methods, interim analyses or multiplicity procedures. Those methods can affect the interpretation of clinical trial estimates, but their use should not be presumed from the phase or endpoint. General statistical principles can explain why such details matter; they cannot establish which methods investigators used in this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry record names progression-free survival as the primary outcome and lists overall survival and subjects experiencing toxicity as secondary outcomes. It reports 3 outcome measures and 0 analyses. It does not describe a statistical plan for handling multiple outcomes or tests, including whether any multiplicity adjustment was planned or used. Without reported analysis results or a plan, the potential influence of multiple testing on statistical interpretation cannot be assessed.
The record also does not report missing-data methods, loss-to-follow-up details, or the number of participants included in each outcome analysis. For a time-to-event endpoint, follow-up and censoring rules are relevant to how the estimate is constructed; the registry does not specify them. The endpoint description refers to assessment until death, but does not explain how incomplete assessments, missed visits or other missing observations were treated.
Analysis populations are not identified. The registry does not say whether analyses used all enrolled participants, only those who began treatment, or another defined group. These are not interchangeable populations, and no particular approach should be inferred. The record likewise does not state whether participants were excluded from any analysis or how deviations from treatment were handled.
Consequently, the posted information supports a description of the registered outcomes and the stated design, but not a reconstruction of the analysis process. It is not possible to determine from the registry record how missing observations, multiple outcomes or analysis-set choices may have affected 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 |
|---|---|---|---|
| Single Arm Trial | 14 | 38 | 36.8% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Single Arm Trial | 28 of 38 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0.5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Single Arm Trial | 38 of 38 |
Most frequent serious adverse event terms
| Event term | Organ system | Single Arm Trial |
|---|---|---|
| Dyspnea (shortness of breath) | Respiratory, thoracic and mediastinal disorders | 3 of 38 |
| Diarrhea | Gastrointestinal disorders | 2 of 38 |
| Hemoglobin | Blood and lymphatic system disorders | 2 of 38 |
| Supraventricular and nodal arrhythmia - Atrial fibrillation | Cardiac disorders | 2 of 38 |
| Ataxia (incoordination) | Nervous system disorders | 1 of 38 |
| Dehydration | Metabolism and nutrition disorders | 1 of 38 |
| Extremity-lower (gait/walking) | General disorders | 1 of 38 |
| Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L) | General disorders | 1 of 38 |
| Infection with Grade 3 or 4 neutrophils (ANC <1.0 x 10e9/L) - Lung (pneumonia) | Infections and infestations | 1 of 38 |
| Infection with normal ANC or Grade 1 or 2 neutrophils - Kidney | Infections and infestations | 1 of 38 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Single Arm Trial |
|---|---|---|
| Fatigue (asthenia, lethargy, malaise) | General disorders | 29 of 38 |
| Nausea | Gastrointestinal disorders | 21 of 38 |
| Hemoglobin | Blood and lymphatic system disorders | 19 of 38 |
| Constipation | Gastrointestinal disorders | 14 of 38 |
| Hypertension | Vascular disorders | 12 of 38 |
| Glucose, serum-high (hyperglycemia) | Metabolism and nutrition disorders | 11 of 38 |
| Neutrophils/granulocytes (ANC/AGC) | Blood and lymphatic system disorders | 10 of 38 |
| AST, SGOT(serum glutamic oxaloacetic transaminase) | Metabolism and nutrition disorders | 9 of 38 |
| Pain - Head/headache | Nervous system disorders | 9 of 38 |
| Pain - Joint | Musculoskeletal and connective tissue disorders | 9 of 38 |
Serious events with the highest arm-level rate: dyspnea (shortness of breath) (7.9%), diarrhea (5.3%), hemoglobin (5.3%), supraventricular and nodal arrhythmia - atrial fibrillation (5.3%), ataxia (incoordination) (2.6%).
The serious adverse-event summary is for the single arm. It lists 38 at risk and 14 affected for serious events, alongside 38 at risk and 38 affected for other events. It also reports 28 deaths. These are registry summary counts; the record does not explain the relationship between the death count and the adverse-event summaries or give additional context for interpreting them.
The listed serious-event terms and percentages are dyspnea (shortness of breath), 7.9%; diarrhea, 5.3%; hemoglobin, 5.3%; supraventricular and nodal arrhythmia - atrial fibrillation, 5.3%; ataxia (incoordination), 2.6%; and dehydration, 2.6%. The term list does not give the number of cases for each term or state a term-specific denominator. The percentages should therefore be read as the registry reports them, without deriving counts or assuming a denominator beyond what is explicitly recorded.
In general, a serious adverse event is a regulatory seriousness category and is not simply another way to say that an event was severe in intensity. The registry lists event terms but does not establish that treatment caused them. With no comparator group, these summaries describe events reported in the study arm and do not estimate excess risk relative to another treatment. They also do not provide a complete account of event timing, duration or management.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a multicenter phase II, single-group study evaluated a treatment approach involving carboplatin, pemetrexed and bevacizumab in patients with stage III or IV non-small cell lung cancer and a light or never smoking history. The official title describes maintenance therapy with pemetrexed and bevacizumab, and the arm description includes an option for second-line erlotinib hydrochloride. Progression-free survival is named as the primary outcome.
What the record does not establish is the numerical progression-free survival result. No median estimate or 95% confidence interval is reported, and the record lists 0 analyses. The result's magnitude and precision therefore cannot be assessed from the posted results. The record also does not report an overall survival result or analysis results for the toxicity outcome.
The design limits what can be concluded even if an outcome estimate were available. A single-group study has no concurrent control in the record, so outcomes cannot be used to determine how the same population would have fared under another treatment approach. A within-group result would not by itself show that treatment caused an outcome or that it was better or worse than an alternative.
The serious adverse-event summary provides counts for the arm and percentages for selected terms. These data describe reported events in the registry record, but do not establish causality, comparative safety or the frequency of every possible event. The listed death count is not, by itself, a treatment-effect estimate.
Accordingly, the record is useful for understanding the study's population, design, treatment description, endpoint wording and reported safety summaries. It does not support a conclusion about treatment benefit, comparative effectiveness or comparative harm. Those conclusions require outcome estimates and analysis details that are not reported in the registry record, as well as an appropriate basis for comparison.
13. Why This Trial Matters Statistically
This trial illustrates why a study's clinical question, design and outcome reporting need to be read together. The registry identifies progression-free survival as the primary outcome and specifies a median with a 95% confidence interval, but does not report either the median or the interval. An endpoint label alone does not provide a result, and a named uncertainty measure does not reveal precision without its limits.
The single-group design is also central to interpretation. It can describe outcomes among participants treated in the study, but it does not provide a concurrent control for estimating a treatment contrast. This distinction separates a descriptive outcome from a causal comparative claim. The trial's phase II label does not remove that design limitation or supply missing efficacy estimates.
There is also a statistical reporting issue in the endpoint description. The primary outcome is labeled progression-free survival, while the accompanying description defines radiographic response categories and mentions assessments through follow-up. The registry does not explain how those response criteria map to the time-to-event outcome. Clear definitions are important because an endpoint's event rules determine what the resulting estimate means.
The sample-size assumptions offer another useful distinction: planning calculations are not trial findings. The described setup assumes equal allocation between two arms, while the registered design is single-group. Planning illustrations cannot be read as observed power, and without numerical outcomes or stated design justification they do not establish what precision the study achieved.
Finally, the record lists toxicity-related summaries but does not provide a comparative safety analysis. Counts and term percentages are useful descriptive information, yet they do not on their own establish causality or relative risk. These features make the record a useful case for separating what a registry documents from what an outcome analysis would need to show.
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 primary limitation for interpreting efficacy is the absence of numerical outcome results. Although progression-free survival is identified as the primary endpoint, its median and 95% confidence interval are not reported in the registry record. The record lists 0 analyses, so no primary analysis can be evaluated for magnitude, precision or statistical method.
The endpoint description is not fully aligned with its label. It gives RECIST v1.0 response categories and assessment language, but does not explain how progression-free survival events were defined or how radiographic response informed the event time. It gives a timeframe of 1400 days and says criteria were assessed each year until death; the record does not clarify the relationship between these statements or explain censoring.
The study is single-group and has no concurrent comparator listed. This means the record cannot support a direct estimate of relative efficacy or safety. The population is described as having a light or never smoking history, but the registry record does not operationally define light smoking. Further eligibility, treatment exposure, adherence and follow-up details are not reported in the information summarized by the registry.
Analysis methods are also not described. The record does not state analysis populations, missing-data procedures, multiplicity handling, interim analyses or the statistical plan. It does not report numerical results for the named secondary outcomes. These omissions constrain reproducibility and make it difficult to assess possible sources of bias or uncertainty.
Safety information is limited to the arm-level summary, a list of serious-event terms with percentages, and the reported counts. Term-specific case counts and denominators are not listed, and the record does not establish treatment causality. The planning assumptions describe a two-arm allocation despite the single-group registered design; they should not be mistaken for the study's actual design or for observed power.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Carboplatin, Pemetrexed Disodium, and Bevacizumab for Patients With Stage III or IV Non-Small Cell Lung Cancer Who Are Light/Never Smokers — NCT01344824.
- Registry record last updated 2017-10-30; status last verified 2017-06.
- Results first posted 2017-07-11.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01344824 is a completed multicenter phase II study sponsored by UNC Lineberger Comprehensive Cancer Center. The registry lists a start date of 2010-03, primary completion on 2016-05-24 and enrollment of 38. The population is described as patients with stage III or IV non-small cell lung cancer who were light or never smokers.
The study is recorded as single-group, with allocation marked NA and masking listed as none. The treatment description includes bevacizumab, carboplatin and pemetrexed disodium, with an option for second-line erlotinib hydrochloride; the official title describes pemetrexed and bevacizumab maintenance therapy. Progression-free survival is the primary outcome, specified as a median in months with a 95% confidence interval. Overall survival and subjects experiencing toxicity are named as secondary outcomes.
The registry record reports 0 analyses and does not include the primary outcome estimate or its confidence interval. It includes an arm-level adverse-event summary, but no concurrent comparator. The posted information therefore describes the study and some safety reporting, while leaving the efficacy results and key analysis methods unreported.