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 BATTLE-FL: Front-Line Biomarker-Integrated Treatment Study in Non Small Cell Lung Cancer (NCT01263782), a randomized phase 2 study. The primary outcome is Progression Free Survival.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01263782 |
| Phase | Phase 2 |
| Condition | Lung Cancer |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 64 |
| Arms in the primary outcome | 4 |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
Patients with advanced non-small cell lung cancer treated in the front-line setting. The primary endpoint population is described as patients who had treatment. The registry record does not report further eligibility details in the information summarized for this analysis.
Intervention
The study explored using biomarker status to guide selection of a biologic drug alongside carboplatin and pemetrexed. The brief summary names bevacizumab or cixutumumab. The posted endpoint arm labels also include a cetuximab group, creating an inconsistency in the treatment descriptions that should be kept in mind when reading the results.
Comparator
The posted groups include carboplatin and pemetrexed without a biologic drug, as well as groups combining chemotherapy with bevacizumab, cetuximab, or cixutumumab. The registry identifies the carboplatin and pemetrexed group as the control. The allocation ratio and the exact biomarker-based assignment rules are not reported in the registry record.
Primary question
The main endpoint is progression-free survival, measured from treatment start until progression or death, whichever occurs first, with last contact used when neither event has occurred. The study’s stated aim was to investigate whether biomarker status could help researchers choose treatment combinations. Posted arm-level medians describe time to progression or death, but the registry record does not provide a reported hazard-ratio comparison establishing the relative effects of the groups.
3. Trial Design
BATTLE-FL is a randomized, parallel-group phase 2 study in the front-line setting for advanced non-small cell lung cancer. The registry lists an enrollment of 64 and a completed status. The study sponsor is M.D. Anderson Cancer Center. The registry records the study as unmasked, so the design is not described as blinded.
The study’s stated purpose was to examine whether biomarker status could inform the choice of a treatment combination, and to study the safety of those combinations. Carboplatin and pemetrexed form the chemotherapy backbone in the arm descriptions. The registry’s brief summary describes adding bevacizumab or cixutumumab according to biomarker status. Posted progression-free survival arm labels, however, include carboplatin and pemetrexed alone, combinations with bevacizumab, cetuximab, and cixutumumab. Cetuximab is not included in the intervention list or the brief summary. This difference between the record’s treatment descriptions is important context; the available information does not explain it.
The chemotherapy-only arm description specifies four cycles of carboplatin and pemetrexed followed by maintenance pemetrexed. The bevacizumab and cixutumumab arm descriptions specify the drugs and schedules, while the posted arm labels describe treatment followed by maintenance. The chemotherapy-only arm description is truncated in the registry information, so its remaining text cannot be interpreted. The registry does not report the allocation ratio, the randomization procedure, or the operational rules for assigning treatment based on biomarkers.
The primary outcome is progression-free survival among patients who had treatment. Overall response rate is listed as a secondary outcome, but a result for that outcome is not included in the information summarized for this analysis. The primary completion date is 2017-08-15. The available registry information does not state whether the study used interim analyses or how the phase 2 design informed later research.
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: The patients had pre-treatment evaluations, which were used to determine the patients' study eligibility, within 4 weeks prior to initiating therapy. Out of 64 participants, 40 was screen failed.
Overall Study
| Stage | Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance |
|---|---|---|---|---|
| STARTED | 6 | 6 | 6 | 6 |
| COMPLETED | 6 | 6 | 3 | 5 |
| NOT COMPLETED | 0 | 0 | 3 | 1 |
Reasons for not completing this period:
| Reason | Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance |
|---|---|---|---|---|
| Death | 0 | 0 | 1 | 0 |
| Progressive disease | 0 | 0 | 0 | 1 |
| Too early | 0 | 0 | 1 | 0 |
| Inevaluable | 0 | 0 | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Patients who had treatment
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 | Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 6 | 6 | 6 | 6 | 24 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 3 | 3 | 2 | 11 |
| Age, Categorical: >=65 years | Participants | 3 | 3 | 3 | 4 | 13 |
| Age, Continuous | years | 66 (54 to 71) | 65 (56 to 75) | 68 (58 to 84) | 68 (55 to 73) | 66 (54 to 84) |
| Sex: Female, Male: Female | Participants | 3 | 1 | 3 | 0 | 7 |
| Sex: Female, Male: Male | Participants | 3 | 5 | 3 | 6 | 17 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 | 2 | 0 | 0 | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 5 | 4 | 6 | 6 | 21 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 0 | 1 | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 1 | 1 | 3 |
| Race (NIH/OMB): White | Participants | 5 | 5 | 5 | 4 | 19 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 6 | 6 | 6 | 6 | 24 |
6. Primary Endpoint
The registry lists the primary outcome as Progression Free Survival, measured over From treatment start to the time of progression or death, whichever occurred first, or to the time of last contact, assessed up to 5 years.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | month |
| Endpoint classification | Time-to-Event Analysis |
Progression-free survival measures the time from treatment start until disease progression or death, whichever occurs first. If neither event is recorded before the last contact, the endpoint definition uses the time of last contact. Progression is defined using RECIST v1.0: a 20% increase in the sum of the longest diameter of target lesions, a measurable increase in a non-target lesion, or the appearance of new lesions.
This time-to-event endpoint is used because it captures how long participants remain alive without documented progression, rather than recording only whether progression occurred by one fixed assessment time. It can account for participants whose follow-up ends before progression or death is observed by treating their last known contact as the end of observed follow-up. That does not mean their later outcome is known.
The registry reports the endpoint as a median with a 95% confidence interval, in months. A median is a summary of the event-time distribution: it describes the point by which half of the estimated outcome distribution has reached the event. The confidence interval conveys uncertainty around that estimate. The registry’s primary endpoint population is patients who had treatment, so the reported population is not simply described as every enrolled participant. The registry information does not report the detailed analysis procedure used to estimate the medians or intervals.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted progression-free survival medians are 5.2 months for carboplatin and pemetrexed, 14.5 months for carboplatin and pemetrexed with bevacizumab, 20.6 months for the arm labeled carboplatin and pemetrexed with cetuximab, and 8.0 months for carboplatin and pemetrexed with cixutumumab. Their respective 95% confidence intervals are 1.3 to 11.2 months, 1.5 to 53.7 months, 1.4 to 26.0 months, and 3.2 to 22.09 months. These estimates are arm-level summaries, not a reported set of treatment-effect comparisons.
The intervals are broad, particularly for the bevacizumab group. Broad intervals indicate that the median estimates are imprecise: the observed data are compatible with a range of underlying median values. The intervals overlap, but overlap or lack of overlap between separate confidence intervals is not a substitute for a formal comparison. The registry record does not report sponsor hazard ratios or inferential comparisons between the arms in the results.
Each posted endpoint arm has a reported group size of 6. These small arm-level numbers make the estimates especially sensitive to individual event times and follow-up patterns. They also limit how much detail can be learned from the arm summaries alone. The record gives no basis for concluding that a difference between the displayed medians is statistically significant, or that one regimen is superior to another.
The registry lists 64 as enrollment, while the endpoint results describe patients who had treatment and show arm-level group sizes of 6. These figures refer to different registry summaries, and the information does not explain how the enrollment count relates to the endpoint analysis groups. It would be inappropriate to infer a participant flow or explain the difference without additional reporting.
The treatment-label inconsistency also constrains interpretation. The summary and intervention list describe bevacizumab and cixutumumab, whereas the endpoint arm labels include cetuximab. The registry information does not clarify this discrepancy. The arm medians can be reported as labeled, but the discrepancy should not be silently resolved by assuming a particular drug was intended.
Overall, the posted data describe observed time-to-event summaries and uncertainty, not a decisive comparative result. They do not report the hazard ratios, statistical tests, biomarker-specific results, or response-rate results needed to answer broader questions about treatment selection.
9. Statistical Methods Explained
Progression-free survival is a time-to-event outcome. General explanation: for this type of outcome, each participant contributes information about the length of follow-up and whether progression or death was observed. When follow-up ends without either event, the observation is censored at the last known contact. Censoring preserves the observed follow-up information without treating an unobserved later event time as if it were known. The registry definition explicitly identifies last contact as the endpoint time when progression or death has not occurred.
What does a median time-to-event estimate mean?
General explanation: a median time-to-event estimate marks the midpoint of the estimated distribution of event times. It is not the average time, and it does not mean that every participant experienced the event at that time. The registry reports medians in months for each posted treatment group. Because the endpoint population is patients who had treatment, the results should be understood in that population as described, not automatically as an analysis of all enrolled participants.
The registry reports a 95% confidence interval with each median. General explanation: a confidence interval expresses uncertainty in an estimate under the statistical procedure used to construct it. A wider interval indicates less precision than a narrower interval. It is not a range containing a fixed proportion of individual participant outcomes. The posted intervals are broad, so the medians should not be read as highly precise descriptions of the underlying event-time distributions.
Why are the arm medians not treatment comparisons?
A median for one arm and a median for another arm summarize each group separately. A comparative analysis requires an estimate of the difference or relative event experience between groups, together with an appropriate measure of uncertainty. For time-to-event outcomes, a hazard ratio is one possible comparative measure, but the registry information summarized for this analysis does not report sponsor hazard ratios. It also lists no analyses. The record indicates that values were missing or not estimable for a two-arm comparison of this median-time endpoint. Therefore, no comparative estimate or significance test can be interpreted from the posted results.
General explanation: the hazard ratio, when reported, compares event rates over follow-up according to a statistical model; it is not the ratio of the two medians. A hazard ratio would need its own uncertainty interval and analysis context. No such sponsor result is available in the registry record, so it would be inappropriate to reconstruct or imply one from the medians and confidence intervals.
How should the sample-size illustrations be understood?
The planning illustrations use the observed effect as a starting point, equal allocation between two arms, a two-sided significance level of 0.05, and a normal approximation. For time-to-event outcomes, the method is described as using the total number of events. These are planning calculations, not the trial’s own design justification and not observed power. They do not show that the trial had a particular probability of detecting an effect. Their interpretation depends on assumptions, including the chosen fraction of the observed effect and the planning framework. The registry record does not report the trial’s sample-size justification, so the illustrations should not be attributed to the study protocol.
The record specifies that only sponsor-reported hazard ratios would be used for time-to-event comparisons. Since no such results are available in the summarized registry information, the displayed medians cannot be turned into a sponsor comparison. Recalculated quantities, where applicable for other kinds of outcomes, would be simple unadjusted calculations from posted summary statistics; no recalculated time-to-event comparison is appropriate. The record does not identify an analysis plan that would support a different interpretation.
Why does small group size matter?
General explanation: in small groups, a few event times can have a substantial influence on a median and its uncertainty. Time-to-event estimates also depend on the amount and timing of follow-up and on how many participants remain under observation over time. The posted endpoint group sizes are 6 in each arm, but the registry information does not provide risk sets over follow-up, event counts by arm, or the shape of the event-time distributions. Those details would help readers understand how much information supports each estimate.
General explanation: a confidence interval and an analysis method are useful only in context. Details such as the estimator, handling of missing or incomplete follow-up, censoring assumptions, and any adjustment for prognostic factors can affect interpretation. Those specific analytic details are not reported in the registry record. The reported figures should therefore be treated as descriptive registry results with the stated endpoint definition, rather than as a complete account of the statistical analysis.
10. Multiplicity, Missing Data and Analysis Populations
The study includes a primary endpoint of progression-free survival and lists overall response rate as a secondary outcome. The registry information does not report a multiplicity strategy, such as how the chance of false-positive findings across outcomes or treatment comparisons was addressed. It also does not state whether any outcomes or comparisons were designated as exploratory. Without that information, readers cannot determine how to interpret a collection of possible tests as a single inferential program.
The registry lists no analyses for the results, and sponsor hazard ratios are not reported. For the median time-to-event endpoint, the record notes that values were missing or not estimable for a two-arm comparison. Consequently, there is no reported comparative estimate or p-value to interpret. The registry does not report results for the listed secondary outcome in the information summarized for this analysis.
The primary endpoint population is described as patients who had treatment. The record does not describe an intention-to-treat analysis population, explain participant disposition, or report how participants who did not receive treatment were handled. It also does not provide details on missing outcome data, reasons for loss to follow-up, or sensitivity analyses. These points are not reported in the registry record and should not be filled in by assumption.
The enrollment figure and the arm-level endpoint group sizes are not reconciled in the available information. No account of screening, randomization, treatment receipt, exclusions, or analysis-set construction is reported. As a result, the registry figures do not support a complete reconstruction of the analysis population or the handling of missing 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 |
|---|---|---|---|
| Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | 2 | 6 | 33.3% |
| Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | 0 | 6 | 0.0% |
| Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | 2 | 3 | 66.7% |
| Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance | 0 | 5 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | 1 of 6 |
| Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | 0 of 6 |
| Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | 0 of 3 |
| Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance | 0 of 5 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's .26% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | 6 of 6 |
| Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | 6 of 6 |
| Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | 3 of 3 |
| Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance | 4 of 5 |
Most frequent serious adverse event terms
| Event term | Organ system | Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance |
|---|---|---|---|---|---|
| Cardiac arrest | Cardiac disorders | 1 of 6 | 0 of 6 | 0 of 3 | 0 of 5 |
| Creatinine increased | Investigations | 0 of 6 | 0 of 6 | 1 of 3 | 0 of 5 |
| Hypercalcemia | Metabolism and nutrition disorders | 0 of 6 | 0 of 6 | 1 of 3 | 0 of 5 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 6 | 0 of 6 | 0 of 3 | 0 of 5 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 1 of 6 | 0 of 6 | 0 of 3 | 0 of 5 |
| Sepsis | Infections and infestations | 1 of 6 | 0 of 6 | 0 of 3 | 0 of 5 |
| Other, Endarterectomy | Surgical and medical procedures | 0 of 6 | 0 of 6 | 1 of 3 | 0 of 5 |
| Urinary tract infection | Infections and infestations | 1 of 6 | 0 of 6 | 0 of 3 | 0 of 5 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Carboplatin + Pemetrexed x 4 Cycles Followed by Maintenance Pe | Carboplatin + Pemetrexed + Bevacizumab Followed by Maintenance | Carboplatin + Pemetrexed + Cetuximab Followed by Maintenance P | Carboplatin + Pemetrexed + Cixutumumab Followed by Maintenance |
|---|---|---|---|---|---|
| Fatigue | General disorders | 4 of 6 | 4 of 6 | 3 of 3 | 4 of 5 |
| Anemia | Blood and lymphatic system disorders | 3 of 6 | 3 of 6 | 2 of 3 | 4 of 5 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 4 of 6 | 4 of 6 | 2 of 3 | 2 of 5 |
| Cough | Respiratory, thoracic and mediastinal disorders | 5 of 6 | 2 of 6 | 2 of 3 | 1 of 5 |
| Hypomagnesemia | Metabolism and nutrition disorders | 3 of 6 | 2 of 6 | 3 of 3 | 2 of 5 |
| Constipation | Gastrointestinal disorders | 3 of 6 | 2 of 6 | 1 of 3 | 2 of 5 |
| Nausea | Gastrointestinal disorders | 2 of 6 | 4 of 6 | 1 of 3 | 1 of 5 |
| Pain | General disorders | 1 of 6 | 4 of 6 | 1 of 3 | 2 of 5 |
| Anxiety | Psychiatric disorders | 1 of 6 | 2 of 6 | 2 of 3 | 2 of 5 |
| Hyperglycemia | Metabolism and nutrition disorders | 2 of 6 | 1 of 6 | 1 of 3 | 3 of 5 |
Serious events with the highest arm-level rate: creatinine increased (33.3%), hypercalcemia (33.3%), other, endarterectomy (33.3%), cardiac arrest (16.7%), hyperglycemia (16.7%).
The registry reports serious adverse events among participants at risk in each listed group. For carboplatin and pemetrexed, 2 of 6 participants at risk had a serious adverse event, and 6 of 6 had another adverse event. For the bevacizumab group, 0 of 6 had a serious adverse event, and 6 of 6 had another adverse event. For the cetuximab-labeled group, 2 of 3 had a serious adverse event, and 3 of 3 had another adverse event. For the cixutumumab group, 0 of 5 had a serious adverse event, and 4 of 5 had another adverse event. Deaths are listed as 1, 0, 0, and 0, respectively.
These counts describe the registry’s reported categories and at-risk denominators; they do not establish that an event was caused by treatment. The record does not provide enough information in this summary to interpret event timing, duration, severity beyond the serious-event classification, or attribution. The serious-event terms listed include Creatinine increased, Hypercalcemia, Other, Endarterectomy, Cardiac arrest, Hyperglycemia, and Pneumonitis, with the registry reporting percentages of 33.3% or 16.7% for the listed terms. Those term percentages are not mapped to treatment groups in the information.
The group sizes are small, so the counts should be read descriptively rather than as stable comparative safety rates. Differences in the counts do not establish comparative risk, and absence of a recorded serious event in a group does not mean that a regimen is free of serious adverse events.
12. What the Result Does, and Does Not, Mean
The results establish what the registry reports: arm-level medians and confidence intervals for progression-free survival, alongside selected adverse-event counts. They show that the posted median estimates differ among the labeled groups, while the broad intervals indicate substantial uncertainty around those estimates. The figures can help readers understand the scale and precision of the recorded summaries, but they do not by themselves answer whether one treatment strategy changed progression-free survival relative to another.
The registry record does not report sponsor hazard ratios or a statistical comparison for the median-time endpoint. It also does not report biomarker-stratified progression-free survival results or provide the biomarker assignment rules. The study’s stated biomarker-guided purpose therefore cannot be evaluated from the arm-level medians alone. The listed secondary outcome, overall response rate, has no result in the information.
Randomized allocation is a feature of the recorded design, but it does not replace the need for transparent reporting of analysis populations, outcome data, and comparative estimates. The discrepancy between treatment descriptions—particularly the appearance of cetuximab in endpoint arm labels while the summary and intervention list name bevacizumab and cixutumumab—adds uncertainty about how to connect the posted results to the study’s stated treatment strategy.
These data do not establish that any treatment works or does not work, and they do not justify a treatment recommendation. They are registry results from a phase 2 study with small arm-level endpoint groups and no reported two-arm comparison in the information. A reader should distinguish descriptive estimates from evidence of a comparative treatment effect, and should regard unreported methods or results as unknown rather than assume how they were handled.
13. Why This Trial Matters Statistically
This trial raises an important statistical question: how should a study be interpreted when its scientific aim involves biomarker-guided treatment selection, but the posted results are arm-level outcome summaries without a reported comparative analysis? The distinction matters because a biomarker-guided strategy is evaluated not only by outcomes in separate treatment groups, but also by the logic and results of the assignment approach and by evidence that supports comparisons relevant to that strategy.
The registry’s randomized, parallel design and its stated focus on advanced non-small cell lung cancer provide context for the reported data. At the same time, the endpoint summaries come from small groups, have broad confidence intervals, and are not accompanied by sponsor hazard ratios. These features illustrate why numerical differences between medians should not be treated as proof of a treatment effect. Estimation, uncertainty, and comparative inference are related but distinct parts of a statistical result.
The record also highlights the importance of consistency in trial reporting. The stated treatment aim and intervention list name bevacizumab and cixutumumab, but an endpoint arm is labeled with cetuximab. Clear alignment between protocol descriptions, intervention records, arm labels, and posted analyses helps readers understand which groups were compared and what the results mean. When that alignment is not clear, interpretation should remain cautious rather than resolve the discrepancy by assumption.
Finally, the registry record distinguishes enrollment from the endpoint population and reports arm-level endpoint group sizes without a participant-flow explanation. That makes transparent accounting of analysis populations and missing data particularly relevant. These are general lessons in statistical reporting: readers need both estimates and a clear account of who contributed to them, how uncertainty was handled, and which comparisons were planned and 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.
Several limitations specific to this trial constrain interpretation. The primary endpoint is reported for patients who had treatment, but the registry information does not explain how that group was formed or how it relates to the enrollment figure of 64. The posted arm-level endpoint groups each have a reported size of 6, and no participant-flow account is available in the information. Reasons for exclusions, incomplete follow-up, or missing outcome data are not reported in the registry record.
The primary result is a median with a 95% confidence interval for each arm. The registry does not report sponsor hazard ratios, a p-value, or a two-arm comparison for this time-to-event endpoint. It also does not report details of the statistical estimator, analysis plan, censoring assessment, or adjustment for other factors. The confidence intervals are broad, so the arm medians are imprecise, and they should not be interpreted as a formal ranking of treatments.
The biomarker-based assignment rules and biomarker-specific outcome results are not reported in the information, even though the study’s stated aim concerns use of biomarker status. The registry also lists overall response rate as a secondary outcome without a result in this summary. The treatment descriptions are inconsistent: cetuximab appears in a posted endpoint arm label, while the brief summary and intervention list name bevacizumab and cixutumumab. The record does not explain the discrepancy.
Safety counts are descriptive and have small denominators. The available summary does not establish event attribution or provide detailed timing and follow-up for adverse events. Multiplicity handling, sample-size justification, and interim analyses are not reported in the registry record. These limitations mean the posted results offer a restricted view of the trial and do not support stronger conclusions than the reported estimates allow.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: BATTLE-FL: Front-Line Biomarker-Integrated Treatment Study in Non Small Cell Lung Cancer — NCT01263782.
- Registry record last updated 2019-05-15; status last verified 2019-05.
- Results first posted 2019-05-08.
- 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
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18. Record Summary
BATTLE-FL is a completed, randomized, open-label, parallel-group phase 2 study sponsored by M.D. Anderson Cancer Center. It enrolled 64 participants and studied front-line treatment for advanced non-small cell lung cancer. The stated aim was to examine biomarker-guided selection of treatment combinations using carboplatin and pemetrexed with biologic therapy.
The primary endpoint was progression-free survival among patients who had treatment, reported as a median with a 95% confidence interval. The posted arm medians range from 5.2 months to 20.6 months, but the registry does not report a sponsor hazard ratio or a two-arm comparison for this endpoint. The arm-level endpoint groups each have a reported size of 6, and their confidence intervals are broad.
The record’s treatment descriptions are inconsistent because cetuximab appears in an endpoint arm label but not in the brief summary or intervention list. Overall response rate is listed as a secondary outcome without a result in the information. The registry does not report the biomarker assignment rules, detailed missing-data handling, or multiplicity approach.