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 Brivanib Alaninate in Treating Patients With Persistent or Recurrent Cervical Cancer (NCT01267253), a na phase 2 study. The primary outcome is Objective Tumor Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01267253 |
| Phase | Phase 2 |
| Condition | Cervical Adenocarcinoma, Cervical Adenosquamous Carcinoma, Cervical Squamous Cell Carcinoma, Not Otherwise Specified |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 31 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Gynecologic Oncology Group |
| Results posted | Yes |
2. Clinical Question
Population
Eligible and treated participants with persistent or recurrent cervical cancer were the primary endpoint population. The listed conditions include cervical adenocarcinoma, cervical adenosquamous carcinoma, cervical squamous cell carcinoma not otherwise specified, persistent disease, and recurrent cervical carcinoma. The registry reports enrollment of 31 participants; it does not report the number included in the primary endpoint analysis.
Intervention
Participants received brivanib alaninate by mouth once daily on days 1-28. Courses repeated every 28 days while disease progression or unacceptable toxicity was absent. Laboratory biomarker analysis is also listed as an intervention, but its role in the primary clinical question is not described in the registry record.
Comparator
This was a single-group study, with no randomized comparator group listed. The registry record does not describe a placebo, usual-care, or other concurrent control arm. Consequently, the trial's primary response outcome is an observed proportion in the treated study population, not a randomized estimate of a difference between treatments.
Primary question
The primary question was what proportion of eligible and treated participants had an objective tumor response, defined as complete or partial response assessed using RECIST 1.1. The registry specifies a 90% confidence interval for this proportion. It does not report the numerical response estimate or its confidence interval in the record summarized for this analysis.
This question is descriptive of tumor response during treatment in the enrolled study group. Without a concurrent comparison group, the response proportion cannot separate the course of disease under treatment from other influences, including participant characteristics, prior treatment, or the natural course of disease. Those factors are not detailed in the registry record.
3. Trial Design
This was a phase II, single-group study sponsored by the Gynecologic Oncology Group. The registry lists the study as completed and reports enrollment of 31. The study title concerns brivanib alaninate in patients with persistent or recurrent cervical cancer. The registry record lists no randomized allocation and no masking; the design is recorded as single group.
The treatment description specifies brivanib alaninate by mouth once daily on days 1-28, with courses repeating every 28 days in the absence of disease progression or unacceptable toxicity. Laboratory biomarker analysis is also listed. The registry record does not give further detail about the biomarker procedures or how those results were related to the clinical endpoint.
The primary outcome was objective tumor response in eligible and treated participants. Response was defined as complete or partial tumor response assessed by RECIST 1.1. The assessment schedule was every other cycle for the first 6 months, then every 3 months thereafter until disease progression was confirmed, and at other times if symptoms or physical signs suggested progressive disease. The listed secondary outcomes were progression-free survival and overall survival.
Because the design has no concurrent control arm, it can describe outcomes among participants treated in this study but cannot directly estimate a randomized treatment contrast. The registry record does not report a protocol-based comparison with a historical control, a sample-size justification, or the assumptions used to plan enrollment. It also does not report a detailed statistical analysis plan. Those omissions prevent a full assessment of how the study's design choices were linked to a prespecified decision rule.
The recorded start date was 2011-04-04 and the primary completion date was 2014-02-28. These dates describe the registry timeline; they do not establish the duration of treatment or follow-up for any individual participant.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Brivanib |
|---|---|
| STARTED | 31 |
| COMPLETED | 28 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Brivanib |
|---|---|
| Ineligible - required test not done | 1 |
| Inevaluable-never received study treatme | 2 |
Analysis population for the primary outcome, as defined in the registry: Eligible and Treated participants
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: Eligible and treated participants
| Characteristic | Unit | Brivanib |
|---|---|---|
| Participants analysed | 28 | |
| Age, Customized: 30-39 years | Participants | 8 |
| Age, Customized: 40-49 years | Participants | 9 |
| Age, Customized: 50-59 years | Participants | 7 |
| Age, Customized: 60-69 years | Participants | 2 |
| Age, Customized: 70-79 years | Participants | 2 |
| Sex/Gender, Customized: Female | Participants | 28 |
| Sex/Gender, Customized: Male | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Objective Tumor Response, measured over Every other cycle for first 6 months; then every 3 months thereafter until disease progression confirmed; and at any other time if clinically indicated based on symptoms or physical signs suggestive of progressive disease..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 90% Confidence Interval |
| Unit | proportion |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the proportion of eligible and treated participants with an objective tumor response. A participant meets the response definition by having a complete or partial tumor response assessed according to RECIST 1.1. The registry specifies a 90% confidence interval around the proportion, but it does not report the numerical estimate or interval in the record summarized for this analysis.
In general, an objective response endpoint provides a defined way to count tumor shrinkage using specified response categories and assessment procedures. Reporting the proportion makes the outcome interpretable as the share of the analyzed population meeting the response definition. The assessment schedule matters because tumor measurements are made at particular points during follow-up; the registry describes assessments every other cycle for the first 6 months, then every 3 months thereafter until progression is confirmed, with additional assessment when symptoms or physical signs suggest progression.
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the analysis. A 90% confidence interval is the registry's stated precision measure for this endpoint. Its width and limits cannot be evaluated without the response estimate and interval endpoints, which are not reported in the registry record. Nor can the observed response proportion be interpreted as a treatment effect relative to another approach, because this study has a single group and no concurrent comparator.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies objective tumor response as the primary endpoint and defines it as complete or partial response assessed by RECIST 1.1. It also specifies that the endpoint is a proportion among eligible and treated participants and that uncertainty is to be expressed with a 90% confidence interval. However, the numerical response result is not reported in the registry record available for this analysis. The proportion, the confidence interval limits, and the number of participants contributing to that analysis therefore cannot be described.
This absence is central to interpretation. The study design and endpoint definition explain what the investigators intended to measure, but they do not establish the observed response frequency. No conclusion about the size or precision of the response estimate can be drawn from the endpoint definition alone. The registry lists enrollment of 31, but enrollment is not necessarily the same as the number eligible, treated, or included in a particular outcome analysis. The primary population is identified as eligible and treated participants, while its analyzed count is not reported in the registry record.
In general, a response proportion is a descriptive summary of how many analyzed participants met a specified response criterion. Its interpretation depends on the population denominator, the response assessment rules, the follow-up and assessment schedule, and the handling of participants whose response status is not available. The registry describes the response criteria and assessment timing, but does not report the numerical endpoint result or how missing assessments were handled.
The single-group design also limits what the endpoint can establish. An observed response proportion in a treated group is not a randomized comparison and cannot by itself show how that proportion differs from what would have occurred under another treatment or without the study treatment. No concurrent control group is listed. The registry record does not report a historical-control comparison or a prespecified benchmark against which to judge the response proportion.
The registry lists progression-free survival and overall survival as secondary outcomes, but numerical results for these outcomes are not reported in the record summarized for this analysis. Six outcome measures are listed, while no analysis records are listed. This does not establish that no analyses were conducted; it means the registry record does not contain analysis entries from which to assess estimates, methods, or uncertainty.
The available information supports a description of the trial's design, treatment schedule, endpoint definition, and assessment schedule. It does not support an assessment of the observed efficacy result, its precision, or the consistency of the primary and secondary outcomes. Any sample-size illustrations based on an observed effect should be interpreted as planning exercises rather than as evidence of the primary result or as observed power.
9. Statistical Methods Explained
The registry record describes a single-group phase II study with objective tumor response as its primary endpoint. It identifies the endpoint as a proportion and specifies a 90% confidence interval. It does not report the numerical response estimate, the confidence interval limits, or an analysis entry. The statistical interpretation is therefore limited to explaining the stated endpoint and the general methods concepts relevant to it.
What does a response proportion represent?
In general, a proportion is the number of participants who meet a defined condition divided by the number of participants in the analysis population. For this trial, the condition is a complete or partial tumor response assessed using RECIST 1.1, and the intended population is described as eligible and treated participants. The registry does not report the numerator, the primary endpoint denominator, or the resulting proportion. Enrollment of 31 should not be substituted for the analysis denominator without evidence that all enrolled participants were eligible, treated, and assessed for this endpoint.
In general, the definition of the event and the population denominator are both necessary for a reproducible estimate. A response assessment depends on tumor measurements, the criteria used to classify them, and the timing of assessment. The record gives the response categories and schedule, but does not supply participant-level assessments or enough detail to determine how incomplete assessments were handled.
What does the confidence interval add?
In general, a confidence interval describes the statistical uncertainty associated with an estimate under a specified method and its assumptions. The registry identifies a 90% confidence interval for the response proportion. That confidence level does not tell the reader the interval's actual width or whether the estimate is precise; those require the estimate and interval limits, which are not reported in the registry record.
In general, interval width is affected by the amount of information available and by the variability of the quantity being estimated. For a binary response outcome, the number of participants with an evaluable response and the observed proportion are relevant. Because neither the response count nor the analyzed denominator is reported, the precision of this endpoint cannot be reconstructed from the enrollment count alone. No recalculated endpoint estimate is available in the registry information.
Why does the design affect interpretation?
In general, a single-group study estimates outcomes in the group that received the study intervention. A concurrent randomized comparator can support an estimate of the difference between groups by balancing treatment assignment across measured and unmeasured characteristics, on average. This study has no such comparator. Its response proportion, if reported, would describe observed responses among participants in the study, rather than a comparative effect attributable solely to treatment.
In general, external comparisons can be difficult to interpret because populations, eligibility rules, assessment schedules, follow-up, and supportive care may differ. The registry record does not report a historical-control analysis or a benchmark comparison for the primary endpoint. No comparative conclusion should therefore be inferred from the single-group design.
How should planning illustrations be read?
The registry information describes planning illustrations based on a stated fraction of an observed effect, using a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation. These are planning assumptions, not the actual single-group design. The record states that non-time-to-event illustrations are expressed as participants per arm, while time-to-event illustrations refer to a total number of events using the Schoenfeld approach. Such illustrations are not observed sample sizes, results, or observed power, and they do not show that the trial was planned using those assumptions.
In general, a sample-size calculation asks how much information a future study might need to detect a defined effect under specified assumptions. The resulting planning quantity depends on the effect being targeted, the error rate, allocation, outcome type, and calculation method. A planning illustration does not establish that the observed trial had adequate precision, nor does it substitute for an analysis of its endpoint. The registry record does not report a protocol sample-size justification or a basis for treating any planning illustration as the original design calculation.
When over-dispersion is relevant to a statistical model, it means that the observed variability is greater than the model's assumed variability. In general, ignoring over-dispersion can make uncertainty appear smaller than it is. The registry record does not identify an over-dispersion analysis for this binary response endpoint, and no model details are reported. This general concept should not be mistaken for a reported feature of the trial analysis.
What can be assessed from the record?
The endpoint definition, confidence-interval level, treatment schedule, and assessment schedule are recorded. The numerical result, analysis population count, missing-response method, and detailed statistical model are not reported in the registry record. The record lists six outcome measures and no analysis records. Those details support a distinction between a prespecified or registered outcome description and an available numerical analysis. They do not show whether analyses were conducted outside the registry.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists objective tumor response as the primary outcome and progression-free survival and overall survival as secondary outcomes. It also lists six outcome measures in total. The registry record does not report how multiplicity across outcomes or analyses was handled. It does not identify a hierarchy of tests, an adjustment procedure, or a rule for interpreting multiple outcomes. Without that information, the relationship between any secondary findings and the primary endpoint cannot be evaluated statistically.
The registry record lists no analysis records. This means numerical analysis details, including any reported estimates or inferential tests for the outcomes, are not available in the record. It does not establish whether analyses were conducted elsewhere or whether a statistical analysis plan existed.
The primary population is described as eligible and treated participants. The registry record does not report a broader intention-to-treat population or explain how eligibility and treatment status were applied in the analysis. It also does not report the number of participants with evaluable response data or the handling of participants who discontinued treatment, missed assessments, or had unclassifiable response status.
In general, missing outcome data can affect an estimate if participants without recorded outcomes differ systematically from those with observed outcomes. The direction and size of any effect depend on why data are missing and how the analysis treats them. The registry record does not report missing-data assumptions, imputation, sensitivity analyses, or a complete-case rule for the primary endpoint. No conclusions about the impact of missingness can therefore be drawn.
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 |
|---|---|---|---|
| Brivanib | 14 | 28 | 50.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Brivanib | 28 of 28 |
Most frequent serious adverse event terms
| Event term | Organ system | Brivanib |
|---|---|---|
| Neoplasms Benign, Malignant And Unspecified (Incl | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 2 of 28 |
| Anemia | Blood and lymphatic system disorders | 1 of 28 |
| Pulmonary Valve Disease | Cardiac disorders | 1 of 28 |
| Colonic Hemorrhage | Gastrointestinal disorders | 1 of 28 |
| Diarrhea | Gastrointestinal disorders | 1 of 28 |
| Small Intestinal Obstruction | Gastrointestinal disorders | 1 of 28 |
| Rectal Hemorrhage | Gastrointestinal disorders | 1 of 28 |
| Mucositis Oral | Gastrointestinal disorders | 1 of 28 |
| Bile Duct Stenosis | Hepatobiliary disorders | 1 of 28 |
| Sepsis | Infections and infestations | 1 of 28 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Brivanib |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 23 of 28 |
| Fatigue | General disorders | 22 of 28 |
| Nausea | Gastrointestinal disorders | 20 of 28 |
| Vomiting | Gastrointestinal disorders | 15 of 28 |
| Anorexia | Metabolism and nutrition disorders | 15 of 28 |
| White Blood Cell Decreased | Investigations | 12 of 28 |
| Hypertension | Vascular disorders | 12 of 28 |
| Diarrhea | Gastrointestinal disorders | 11 of 28 |
| Headache | Nervous system disorders | 11 of 28 |
| Abdominal Pain | Gastrointestinal disorders | 10 of 28 |
Serious events with the highest arm-level rate: neoplasms benign, malignant and unspecified (incl (7.1%), anemia (3.6%), pulmonary valve disease (3.6%), colonic hemorrhage (3.6%), diarrhea (3.6%).
The registry reports 28 participants at risk for serious adverse events and 14 affected. For other adverse events, it reports 28 at risk and 28 affected. These are separate summaries of participants at risk and affected; the registry record does not provide event-level counts or explain whether a participant could contribute more than one event. The enrollment count of 31 should not be treated as the denominator for these safety summaries.
The listed serious adverse event terms and percentages are Neoplasms Benign, Malignant And Unspecified (Incl, 7.1%; Anemia, 3.6%; Pulmonary Valve Disease, 3.6%; Colonic Hemorrhage, 3.6%; Diarrhea, 3.6%; and Small Intestinal Obstruction, 3.6%. The first term appears in that abbreviated form in the registry record. The record does not provide further detail on event timing, severity grading, relatedness assessment, or the number of events per participant.
In general, adverse-event summaries describe events recorded during a study and should not automatically be interpreted as caused by the intervention. The registry record reports no death count; it does not report that there were no deaths. These safety figures do not provide a comparison with another group, because the study is single group.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a phase II, single-group study of brivanib alaninate in patients with persistent or recurrent cervical cancer. It identifies objective tumor response as the primary endpoint, defines response as complete or partial response by RECIST 1.1, and states that the response proportion was to be accompanied by a 90% confidence interval. It also provides the assessment schedule and treatment regimen.
The record does not report the numerical primary response result, its confidence interval, or the number of participants analyzed for that endpoint. Accordingly, the observed frequency of objective response and its precision cannot be described from the registry record. The listed enrollment of 31 is not a substitute for those missing endpoint details.
Even if the response proportion were available, the single-group design would make it a description of outcomes in the treated study population, not a randomized estimate of treatment benefit relative to a control. The study does not list a concurrent comparator. Differences from outcomes in another population or study could reflect differences in participants, care, assessment, or follow-up rather than the intervention alone.
The listed secondary outcomes are progression-free survival and overall survival, but numerical results for them are not reported in the record. The registry also does not report how missing outcome data were handled or how multiple outcomes were considered. These gaps limit assessment of the completeness and statistical interpretation of the study results.
The safety record lists serious and other adverse event summaries, including the number at risk and affected, as well as serious-event terms with percentages. These are descriptive safety data from the study group. They do not establish causality or comparative safety, and the record does not report a death count.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined endpoint and a numerical result are both necessary for statistical interpretation. The registry specifies objective response, the response categories, the assessment criteria, the target population, and a 90% confidence interval. Those elements describe what the primary analysis was intended to summarize. Without the numerical estimate, interval limits, and analysis denominator, however, neither the frequency of response nor its precision can be evaluated.
The single-group design also makes the estimand important. A response proportion in treated participants is a within-group description. It is not the same quantity as a difference in response between treatment groups, and it cannot by itself establish what would have happened to comparable participants under another approach. This distinction remains important even when an endpoint is objective and uses standardized criteria.
The registry record also separates study enrollment from the endpoint population. Enrollment is 31, whereas the primary population is described as eligible and treated participants. The safety summaries separately identify 28 participants at risk. These counts serve different purposes and should not be interchanged. A transparent report would make the denominator and outcome status clear for each analysis.
Finally, registry outcome descriptions do not replace analysis reporting. The record lists multiple outcomes but no analysis records, and it does not report the treatment of missing data or multiplicity. This limits the ability to assess how estimates were obtained and how conclusions should be calibrated. The statistical value of the record is therefore chiefly in documenting the design and intended endpoint, while its numerical outcome evidence remains unavailable.
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 interpretation is that the registry record does not report the objective response estimate or its 90% confidence interval. It also does not report the number of eligible and treated participants included in that endpoint analysis. Although enrollment is listed as 31, the endpoint population is not numerically characterized, so enrollment cannot be assumed to be the analysis denominator.
The single-group design has no concurrent comparator. It can describe outcomes in the study group but cannot estimate a randomized between-group contrast. The registry record does not report a historical-control analysis or a prespecified benchmark, so no comparative reference for the primary response proportion is available.
The record does not report how missing or unevaluable response assessments were handled, or whether sensitivity analyses were performed. It gives an assessment schedule but not participant-level completion or follow-up information. It also does not report a detailed statistical analysis plan, multiplicity approach, sample-size justification, or analysis records. The absence of those details in the registry record prevents evaluation of those methods; it does not establish what may have been done outside the record.
For safety, the registry gives participant counts at risk and affected and lists serious-event terms with percentages, but does not report event-level counts, timing, severity grading, relatedness, or a death count. The available summary therefore does not permit a detailed assessment of event patterns or comparative safety. The study's listed secondary outcomes also lack numerical results in the record summarized for this analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Brivanib Alaninate in Treating Patients With Persistent or Recurrent Cervical Cancer — NCT01267253.
- Registry record last updated 2019-03-20; status last verified 2019-03.
- Results first posted 2017-01-04.
- 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
NCT01267253 is a completed phase II, single-group study sponsored by the Gynecologic Oncology Group. The registry reports enrollment of 31 and a treatment regimen of brivanib alaninate by mouth once daily on days 1-28, with 28-day courses continuing in the absence of progression or unacceptable toxicity.
The primary outcome is the proportion of eligible and treated participants with complete or partial tumor response assessed by RECIST 1.1, with a 90% confidence interval. Assessments were scheduled every other cycle for the first 6 months, then every 3 months until confirmed progression, with additional assessments when clinically indicated. Progression-free survival and overall survival are listed as secondary outcomes.
The registry record does not report the numerical primary outcome result, its interval, or analysis records. Safety summaries list 28 participants at risk and 14 affected for serious adverse events, and 28 at risk and 28 affected for other adverse events. No death count is reported.