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 Bendamustine as Second-Line Therapy in Treating Patients With Relapsed or Refractory Small Cell Lung Cancer (NCT00984542), a na phase 2 study. The primary outcome is Time to Progression.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00984542 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 50 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Vanderbilt-Ingram Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with relapsed or refractory small cell lung cancer were the intended population. The brief summary describes treatment in the second- or third-line setting, while the study title describes second-line therapy. The registry record reports an enrollment of 50. It does not give further eligibility details in the information recorded for this analysis.
Intervention
The intervention was bendamustine hydrochloride, listed as a drug. The study had one Bendamustine group. The registry record does not give a treatment schedule or dose in the information recorded for this analysis.
Comparator
There was no concurrent comparator group: the study is recorded as single-group, with allocation listed as not applicable. Its results therefore describe outcomes among patients in the Bendamustine group; they do not provide a randomized comparison with another treatment or with no treatment.
Primary question
The primary question concerned the duration from study entry to disease progression, summarized as median time to progression. Progression was defined using RECIST v1.1 criteria. The registry record specifies Kaplan–Meier estimation and a 95% confidence interval, but does not report the resulting median or confidence interval. The design can describe the observed time-to-progression experience, but by itself cannot establish how that experience compares with an alternative treatment.
3. Trial Design
This was a completed phase II study sponsored by Vanderbilt-Ingram Cancer Center. The registry identifies the design as single-group, with no masking and allocation listed as not applicable. Enrollment was 50. The recorded start was 2009-09, and primary completion was 2012-09. The study title describes second-line bendamustine for relapsed or refractory small cell lung cancer; the brief summary says the therapy was studied in the second- or third-line setting.
The intervention was bendamustine hydrochloride, recorded under one Bendamustine group. There is no control arm in the design information. Consequently, the study can describe the treatment group's outcomes, but cannot make a like-for-like randomized comparison with a concurrent control group. Differences from outcomes in other settings would not be direct trial comparisons and are not reported in this record.
The primary outcome was time to progression, expressed as a median in days with a 95% confidence interval. The planned observation description refers to measurements after cycles 2, 4, and 6 of a 21-day cycle, for 6 cycles, during 126 days. These are details of the recorded timeframe; the registry does not give the actual primary outcome estimate. It describes progression assessment using RECIST v1.1 and estimation with the Kaplan–Meier method.
Secondary outcomes listed were number of patients with each worst-grade toxicity, best response, progression-free survival, and overall survival. The record also lists serious and other adverse-event information. The number of analyses is recorded as 0. A detailed statistical analysis plan, including planned hypothesis tests or handling of interim analyses, is 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: 59 patients consented, 9 were determined to be ineligible to participate
Overall Study
| Stage | Bendamustine |
|---|---|
| STARTED | 50 |
| COMPLETED | 9 |
| NOT COMPLETED | 41 |
Reasons for not completing this period:
| Reason | Bendamustine |
|---|---|
| disease progression | 19 |
| toxicity | 7 |
| Death | 7 |
| complicating disease | 3 |
| Withdrawal by Subject | 3 |
| clinical deterioration | 2 |
Analysis population for the primary outcome, as defined in the registry: All patients are included in the analysis on intention-to treat basis. Analysis is by Kaplan-Meier method, where progression is an event, with censoring for non-progressed patients at greater of off-study date, last known alive date, or date of death not attributable to disease progression.
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.
Population: Patients with Relapsed or Refractory Small Cell Lung Cancer (SCLC)who received study drug
| Characteristic | Unit | Bendamustine |
|---|---|---|
| Participants analysed | 50 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 34 |
| Age, Categorical: >=65 years | Participants | 16 |
| Age, Continuous | years | 62 (SD 8) |
| Sex: Female, Male: Female | Participants | 22 |
| Sex: Female, Male: Male | Participants | 28 |
| Region of Enrollment: United States | participants | 50 |
6. Primary Endpoint
The registry lists the primary outcome as Time to Progression, measured over On-study to date of progression, measured following cycle 2, 4, and 6 of a 21-day cycle for 6 cycles, (during 126 days).
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
Time to progression measures the time from study entry to the date disease progression is identified. In this study the registry specifies a median, in days, estimated using the Kaplan–Meier method, with a 95% confidence interval. The numerical median and interval are not reported in the registry record, so the endpoint's observed value and precision cannot be described from this record.
Progression was defined under RECIST v1.1 as an increase of >=20% in the sum of longest diameters of target lesions, unequivocal progression of non-target lesions, or the appearance of new lesions. The endpoint therefore focuses on documented disease progression rather than survival or toxicity. It is a time-to-event measure: the event is progression, and a patient's follow-up may end without a recorded progression event.
General statistical context: a median time-to-event estimate describes the point by which the estimated event-free proportion has reached one half, if the data support estimation of that point. A confidence interval communicates uncertainty around an estimate; it is not a range containing every individual patient's outcome. With censoring, Kaplan–Meier estimation can use follow-up up to the last relevant observation for people whose event time is not observed. How much precision the estimate has depends on the event and follow-up information, neither of which is summarized numerically for the primary endpoint in the registry record.
Time to progression is used to describe how long participants remain without documented progression under a defined assessment approach. It should not be treated as equivalent to overall survival, and this single-group endpoint does not itself establish a comparative treatment effect.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a phase II, single-group study of bendamustine in relapsed or refractory small cell lung cancer. It names median time to progression as the primary outcome and specifies Kaplan–Meier estimation with a 95% confidence interval. However, it does not report the numerical median, the confidence interval limits, or a primary outcome analysis. As a result, no estimate of the duration to progression, or of its precision, can be interpreted from the record.
This absence matters for statistical interpretation. Naming an endpoint and an analysis method describes what was intended to be measured and how, but does not reveal the observed result. Without a reported estimate, readers cannot assess whether the median was precisely or imprecisely estimated, or how many participants had progression events or were censored. Those event and censoring details are not reported in the registry record.
The design also limits what an outcome description could establish even if an estimate were reported. The study has one treatment group and no concurrent randomized comparator. A time-to-progression estimate would characterize the participants' observed experience under the study conditions, but it would not by itself show what would have happened to comparable participants under another treatment or without the intervention. The record does not report a sponsor-reported hazard ratio or comparative analysis.
The registry lists secondary outcomes including best response, progression-free survival, overall survival, and worst-grade toxicity. Their inclusion identifies additional areas assessed, but does not supply their results. No conclusions about response, survival, or the frequency or severity of specific toxicities should be drawn from an outcome's listing alone. The adverse-event information does give serious and other affected-patient counts and names several serious adverse-event terms; those figures describe recorded events, not a comparative treatment effect.
General statistical context: estimates from a small study can be uncertain, particularly when the number and timing of events provide limited information. This is a general consideration, not a claim about the precision of this study's unavailable endpoint estimate. The enrollment of 50 indicates the scale recorded for the study, but does not state how many participants contributed evaluable follow-up, had progression, or were censored.
In short, the record documents the design, endpoint definition, and some safety summaries, but omits the primary numerical result. It supports a description of what the study set out to assess, not an assessment of the size or precision of the primary endpoint result, nor a claim of comparative benefit or harm.
9. Statistical Methods Explained
What does a single-group design tell us?
The study is recorded as single-group, with one Bendamustine group and no comparator. This design can describe outcomes observed among participants receiving the study intervention. It does not create a concurrent control group that would show how similar participants fared under another treatment. Without that comparison, an observed outcome cannot be separated from other influences on disease course or follow-up. This is a general limitation of uncontrolled designs, not a claim about the cause of any particular outcome in this study.
The record lists allocation as not applicable and masking as none. It does not describe random assignment or blinded assessment. Details beyond those recorded, including additional safeguards against bias, are not reported in the registry record.
How does Kaplan–Meier estimation work?
General statistical explanation: the Kaplan–Meier method estimates the probability of remaining without the event over time. For this endpoint, progression is the event. The method uses the observed event times and incorporates censored follow-up: a censored participant contributes information up to the time their follow-up is censored, without being treated as having progressed at that time. The method can therefore use incomplete follow-up, provided the censoring assumptions and recorded times are appropriate.
The primary outcome description says patients without progression are censored at the greater of the off-study date, last known alive date, or date of death not attributable to disease progression. The registry does not provide a numerical account of event times, censoring times, or the number censored. It therefore does not allow readers to inspect the distribution of follow-up behind the estimate.
What does a median time-to-event estimate mean?
General statistical explanation: the median time-to-event is the time at which the estimated proportion remaining event-free reaches one half, when that point can be estimated from the observed data. It is a summary of the estimated event-time distribution, not an average duration for every participant. It does not mean that all participants experience the event at that time, and it does not describe what happens after the median point.
The registry specifies that the result would be reported in days and accompanied by a 95% confidence interval. A confidence interval expresses statistical uncertainty around an estimate under the method's assumptions. A narrower interval indicates greater precision than a wider interval, while neither interval nor point estimate alone resolves limitations of study design. Here, the numerical median and confidence interval are not reported in the registry record, so their location and width cannot be assessed.
Why does censoring matter?
General statistical explanation: censoring occurs when an event time is not observed during available follow-up, or when observation ends for another reason. Censored participants are not simply discarded; their observed time contributes to the analysis until censoring. Kaplan–Meier estimates rely on the assumption that censoring does not systematically conceal a different event risk than that of participants remaining under observation, conditional on the method and data structure. The registry's stated censoring rule is available, but information needed to evaluate the pattern of censoring is not reported.
Progression assessment also depends on when assessments occur and how measurements are interpreted. The record describes assessment following cycles 2, 4, and 6 of a 21-day cycle for 6 cycles, during 126 days. The progression criteria follow RECIST v1.1. The registry does not report additional details on assessment timing, missed assessments, or adjudication procedures.
What can a confidence interval and a single arm not show?
General statistical explanation: a confidence interval addresses uncertainty in an estimate under a statistical model; it does not correct design limitations, measurement problems, or incomplete reporting. A single-arm result can be descriptive, but a confidence interval around that result would not supply the missing counterfactual outcome under a comparator. The record reports no hazard ratio or comparative estimate, and no primary endpoint confidence interval values. Thus neither comparative magnitude nor primary estimate precision can be evaluated.
Statistical plan details, including any multiplicity approach, planned interim analyses, and sample-size justification, are not reported in the registry record. The enrollment figure is not evidence of a particular power or design rationale. General considerations about power cannot be used to infer the study's planned or achieved ability to detect an effect when those design assumptions and primary results are absent.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several outcomes: time to progression as primary, and worst-grade toxicity, best response, progression-free survival, and overall survival as secondary outcomes. Considering multiple outcomes can raise a multiplicity issue because interpreting several statistical tests can increase the chance of at least one apparently notable result arising by chance. General statistical practice may address this through a prespecified testing hierarchy or adjustment, but the registry record does not report such a strategy. It also does not give the results of statistical tests for these outcomes.
The number of analyses is recorded as 0. This entry does not supply a detailed account of which comparisons, if any, were planned or performed. Interim analyses and stopping rules are not reported in the registry record. A sample-size justification and a complete statistical analysis plan are also not reported, so no rationale for the enrollment figure or planned operating characteristics can be inferred.
The analysis population description says all patients are included on an intention-to-treat basis. It states that progression is the event and that non-progressed patients are censored at the greater of off-study date, last known alive date, or date of death not attributable to disease progression. This describes the stated population and censoring approach, but does not give participant-level disposition or explain any missing endpoint assessments.
Missing-data frequency, reasons for missing assessments, and any imputation or sensitivity analysis are not reported in the registry record. The actual number contributing to the primary endpoint analysis, event count, and censoring count are also not reported. Without those details, readers cannot assess how incomplete follow-up may have affected the endpoint estimate. The registry's stated intention-to-treat approach should not be read as proof that every enrolled participant had complete outcome data.
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 |
|---|---|---|---|
| Bendamustine | 27 | 50 | 54.0% |
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 |
|---|---|
| Bendamustine | 50 of 50 |
Most frequent serious adverse event terms
| Event term | Organ system | Bendamustine |
|---|---|---|
| death not associated with CTCAE term-disease progression | General disorders | 9 of 50 |
| dyspnea | Respiratory, thoracic and mediastinal disorders | 5 of 50 |
| nausea | Gastrointestinal disorders | 4 of 50 |
| pneumonia | Respiratory, thoracic and mediastinal disorders | 4 of 50 |
| diarrhea | Gastrointestinal disorders | 3 of 50 |
| fatigue | General disorders | 3 of 50 |
| hip fracture | Musculoskeletal and connective tissue disorders | 3 of 50 |
| hemoglobin | Blood and lymphatic system disorders | 3 of 50 |
| atrial fibrillation | Cardiac disorders | 2 of 50 |
| fever in the absence of neutropenia, where neutropenia is defined as ANC < 1.0 x 10e9/L | General disorders | 2 of 50 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Bendamustine |
|---|---|---|
| hemoglobin | Blood and lymphatic system disorders | 26 of 50 |
| fatigue | General disorders | 25 of 50 |
| nausea | Gastrointestinal disorders | 21 of 50 |
| platelets | Blood and lymphatic system disorders | 17 of 50 |
| anorexia | Metabolism and nutrition disorders | 16 of 50 |
| constitutional symptoms-other | General disorders | 15 of 50 |
| metabolic, laboratory-other | Metabolism and nutrition disorders | 15 of 50 |
| vomiting | Gastrointestinal disorders | 14 of 50 |
| dyspnea | Respiratory, thoracic and mediastinal disorders | 13 of 50 |
| leukocytes | Blood and lymphatic system disorders | 11 of 50 |
Serious events with the highest arm-level rate: death not associated with ctcae term-disease progression (18.0%), dyspnea (10.0%), nausea (8.0%), pneumonia (8.0%), diarrhea (6.0%).
The registry records 27 patients with serious adverse events among 50 at risk in the Bendamustine group. It records other adverse events affecting 50 patients among 50 at risk. These are group-level counts in the registry record; they do not establish that an event was caused by the intervention. The record also lists serious adverse-event terms and their reported percentages.
The listed terms are death not associated with a CTCAE term-disease progression, 18.0%; dyspnea, 10.0%; nausea, 8.0%; pneumonia, 8.0%; diarrhea, 6.0%; and fatigue, 6.0%. The death category is explicitly described as not associated with a CTCAE term and as disease progression. It should not be interpreted as a treatment-related death based on this record.
General statistical context: a serious adverse event is a safety reporting category and does not by itself establish causality, severity grading for every listed event, or comparative risk. The registry lists number of patients with each worst-grade toxicity as a secondary outcome, but the information recorded does not include a complete toxicity-grade distribution. Because the study has no control group, these safety figures cannot show whether event frequency differs from another treatment or from the underlying disease course. The record does not state attribution details for the listed terms.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of bendamustine was conducted in relapsed or refractory small cell lung cancer, with enrollment of 50 and a primary endpoint of time to progression. It specifies a Kaplan–Meier method, a 95% confidence interval, and a progression definition based on RECIST v1.1. These details describe the study question and planned endpoint framework.
The record does not report the numerical primary endpoint result. It therefore does not show the median time to progression or the confidence interval, and cannot support a statement about the size or precision of that endpoint. Secondary outcomes are named, but their numerical results are not included in the recorded information. Listing an outcome is not the same as reporting its result.
Even if the outcome estimate were available, the single-group design would support a descriptive interpretation rather than a randomized treatment comparison. It cannot establish how outcomes would compare with another treatment or with no treatment. No comparative hazard ratio is reported. Nor does the existence of a completed phase II study, by itself, determine the clinical importance of an outcome.
The safety entries report affected-patient counts and serious adverse-event terms, but do not establish that the intervention caused the events. The record's disease-progression description for the death category is important to preserve when reading that entry. With no concurrent control group, the listed frequencies do not quantify excess risk relative to a comparator.
General statistical context: estimates are best interpreted alongside their uncertainty, event counts, follow-up, missingness, and study design. Here, the record gives the method and endpoint definition but lacks the primary estimate and several supporting details. The appropriate conclusion is therefore bounded: the registry documents what the study assessed and some safety summaries, while leaving the principal efficacy result and its precision unavailable in the registry record. No advice about treatment choice follows from this statistical description.
13. Why This Trial Matters Statistically
This study is statistically useful as an example of the distinction between a clearly defined endpoint and a reported endpoint result. The registry identifies time to progression as primary, specifies RECIST v1.1 progression criteria, names Kaplan–Meier estimation, and states that a 95% confidence interval was intended. Yet the numerical median and interval are not reported in the registry record. A method description cannot replace the estimate needed to assess the result.
The single-group design also makes the role of a comparator especially important. Time-to-event methods can describe observed experience while accounting for censored follow-up, but a well-defined estimator does not create a comparison group. In this study, no concurrent randomized control is recorded, so the design cannot isolate a comparative treatment effect.
The record illustrates why endpoint interpretation depends on more than the endpoint label. Readers would also need the number of events, censoring pattern, follow-up distribution, and information about missing assessments to understand the basis and reliability of an estimate. These details are not reported for the primary endpoint. The stated intention-to-treat approach and censoring rule provide some methodological context, but not enough to reconstruct or evaluate the result.
Safety reporting likewise needs careful interpretation. Patient counts and named serious adverse-event terms offer descriptive information, but they do not determine attribution or comparative risk. The absence of a control group limits what those numbers can say about differences between treatment strategies.
The broader statistical lesson is that study completion, enrollment, endpoint selection, analysis method, and outcome reporting are distinct elements. Each contributes different information. In this record, the design and analytic framework are described, while the central numerical endpoint result is missing. That reporting gap constrains interpretation regardless of what the study may otherwise have measured.
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 important trial-specific limitation for interpreting the primary outcome is that its numerical result is not reported in the registry record. The median time to progression and its 95% confidence interval are specified as the intended summary, but their values are absent. Event counts, censoring counts, and follow-up detail for this endpoint are also not reported, so neither the estimate nor its precision can be evaluated.
The design is single-group and has no concurrent comparator. Accordingly, it can provide a descriptive account of the Bendamustine group's outcomes but cannot support a randomized comparison. Allocation is listed as not applicable, and masking is recorded as none. The record does not report other procedures that might affect assessment or reduce bias.
The population description is limited. The study title refers to second-line treatment, while the brief summary describes second- or third-line therapy. The information recorded for this analysis does not give further eligibility criteria or characterize the enrolled patients in detail. Treatment dose and schedule are also not reported in the record.
The registry lists secondary outcomes but does not report their numerical findings in the information available for this analysis. Statistical-plan details, multiplicity handling, sample-size justification, interim analyses, and missing-data procedures are not reported in the registry record. Although the analysis description states an intention-to-treat population and a censoring rule, participant disposition and endpoint completeness are not described.
Safety information includes counts and selected serious adverse-event terms, but not a full worst-grade toxicity distribution or comparative safety estimate. The registry entries do not establish event attribution. These omissions limit both efficacy and safety interpretation and should not be filled by assumptions about typical phase II study practice.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Bendamustine as Second-Line Therapy in Treating Patients With Relapsed or Refractory Small Cell Lung Cancer — NCT00984542.
- Registry record last updated 2014-03-12; status last verified 2013-08.
- Results first posted 2014-03-12.
- 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
NCT00984542 is a completed phase II, single-group study sponsored by Vanderbilt-Ingram Cancer Center. It investigated bendamustine hydrochloride for relapsed or refractory small cell lung cancer, described as second-line in the title and second- or third-line in the brief summary. Enrollment was 50. The recorded start was 2009-09, and primary completion was 2012-09.
The primary outcome was median time to progression in days, estimated by Kaplan–Meier with a 95% confidence interval and progression defined using RECIST v1.1. The numerical median and interval are not reported in the registry record. Secondary outcomes included worst-grade toxicity, best response, progression-free survival, and overall survival. The record reports safety counts and selected serious adverse-event terms, but does not provide a concurrent comparator or a comparative treatment estimate.