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 Taselisib and Enzalutamide in Treating Patients With Androgen Receptor Positive Triple-Negative Metastatic Breast Cancer (NCT02457910), a randomized phase 1, phase 2 study. The primary outcome is Clinical Benefit Rate (CBR) - Phase II.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02457910 |
| Phase | Phase 1, Phase 2 |
| Condition | Estrogen Receptor Negative, Estrogen Receptor Positive, HER2/Neu Negative |
| Allocation | Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Vanderbilt-Ingram Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with androgen receptor-positive triple-negative breast cancer that had spread to other parts of the body. The primary endpoint population was evaluable patients with triple-negative breast cancer who had received at least one treatment cycle and had disease re-evaluated at 16 weeks, or whose disease had progressed before that assessment.
Intervention
The registry describes taselisib, a PI3K inhibitor, with enzalutamide, an androgen receptor inhibitor. Dose-finding groups used taselisib at 2 mg, 4 mg, 6 mg, or 8 mg with enzalutamide. A combination arm also started enzalutamide and taselisib at different cycles.
Comparator
Enzalutamide alone was the comparator in the phase II endpoint results. The record also describes patients in the enzalutamide-only arm crossing over to the combination after disease progression, subject to a specified treatment-start window.
Primary question
The primary question was the clinical benefit rate at 16 weeks: the proportion of evaluable patients whose best response was complete response, partial response, or stable disease under RECIST criteria 1.1. The posted results give an estimate for the combination arm and for enzalutamide alone, each with a 95% confidence interval. They do not report a formal between-arm comparison.
The registry describes the study as a phase Ib/II trial and its brief summary as partially randomized. Its stated aims included studying side effects and dose as well as whether the combination could produce clinical benefit. These aims are distinct: a dose-finding study can inform which dose to study further, while an endpoint such as clinical benefit rate describes outcomes for a defined patient group and time point. The posted phase II results are therefore best read as estimates within the two reported arms, rather than as a definitive comparison of treatment strategies.
3. Trial Design
The registry lists NCT02457910 as a phase Ib/II trial with randomized allocation, no masking, and a sequential model. The brief summary describes it as partially randomized. Enrollment was 30. The sponsor was Vanderbilt-Ingram Cancer Center. The record gives a start date of 2015-06, a primary completion date of 2018-12, and a status of terminated; the reason for termination is not reported in the registry record.
The phase Ib component included taselisib dose groups of 2 mg, 4 mg, 6 mg, and 8 mg, each described with enzalutamide. The phase II endpoint results are reported for a combination arm and an enzalutamide-only arm. In the combination dose-group descriptions, taselisib was taken on days 1-28, while enzalutamide began on days 9-28 of the first course and continued on days 1-28 of later courses. Courses repeated every 28 days unless disease progressed or toxicity became unacceptable. The separate combination-arm description starts enzalutamide on day 1 of cycle 1 and taselisib on day 1 of cycle 2.
Patients assigned to enzalutamide alone could cross over to enzalutamide plus taselisib after disease progression. The record specifies that crossover treatment had to begin within 21 days after the clinic visit at which progression was determined. Crossover means that later treatment exposure may differ from initial assignment, a relevant consideration when interpreting outcomes over time. The registry does not report the sequence or rules used to assign participants, how many entered each phase Ib dose group, or the detailed statistical plan for the randomized component.
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: 13 in phase Ib and 17 in phase II
Overall Study
| Stage | PH1b E+T - Level 1 (Cohort 1) | PH1b E+T - Level 2 (Cohort 1 and 2) | PH1b E+T - Level 3 (Cohort 1) | PH1b E+T - Level 4 (Cohort 1) | PHII Enzalutamide | Phase II E+T |
|---|---|---|---|---|---|---|
| STARTED | 3 | 5 | 3 | 2 | 5 | 12 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 | 1 |
| NOT COMPLETED | 3 | 5 | 3 | 2 | 5 | 11 |
Reasons for not completing this period:
| Reason | PH1b E+T - Level 1 (Cohort 1) | PH1b E+T - Level 2 (Cohort 1 and 2) | PH1b E+T - Level 3 (Cohort 1) | PH1b E+T - Level 4 (Cohort 1) | PHII Enzalutamide | Phase II E+T |
|---|---|---|---|---|---|---|
| Withdrawal by Subject | 0 | 0 | 0 | 0 | 0 | 1 |
| Adverse Event | 0 | 2 | 1 | 1 | 1 | 3 |
| Disease progression | 3 | 3 | 1 | 1 | 4 | 6 |
| Other complicating illness | 0 | 0 | 0 | 0 | 0 | 1 |
| Death | 0 | 0 | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Evaluable TNBC patients who had at least one cycle of treatment and had their disease re-evaluated at 16 weeks or progressed prior to the 16-week assessment
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: ER/PR+ or TNBC AR+ women with breast cancer
Only the first 6 of 7 groups are shown.
| Characteristic | Unit | PH1b E+T - Level 1 (Cohort 1) | PH1b E+T - Level 2 (Cohort 1 and 2) | PH1b E+T - Level 3 (Cohort 1) | PH1b E+T - Level 4 (Cohort 1) | PHII Enzalutamide | Phase II E+T |
|---|---|---|---|---|---|---|---|
| Participants analysed | 3 | 5 | 3 | 2 | 5 | 12 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 | 4 | 3 | 2 | 2 | 9 |
| Age, Categorical: >=65 years | Participants | 1 | 1 | 0 | 0 | 3 | 3 |
| Sex: Female, Male: Female | Participants | 3 | 4 | 3 | 2 | 5 | 12 |
| Sex: Female, Male: Male | Participants | 0 | 1 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 2 | 5 | 3 | 2 | 5 | 11 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Clinical Benefit Rate (CBR) - Phase II, measured over At 16 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | proportion of patients |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was clinical benefit rate at 16 weeks, expressed as a proportion of patients. Clinical benefit included a best response of complete response, partial response, or stable disease under RECIST criteria 1.1. The registry defines complete response as disappearance of all target lesions, with pathological lymph nodes reduced to less than 10 mm in short axis. Partial response requires at least a 30% decrease in the sum of target-lesion diameters. Progressive disease includes at least a 20% increase from the smallest sum on study and an absolute increase of at least 5 mm. Stable disease is neither sufficient shrinkage for partial response nor sufficient increase for progressive disease, using the smallest on-study sum as reference.
A rate combining complete response, partial response, and stable disease captures more than tumor shrinkage alone: stable disease can count as benefit at the defined assessment. The time point matters because the measure is tied to the 16-week evaluation, not an unrestricted follow-up period. The endpoint population included evaluable patients who had received at least one cycle and were reassessed at 16 weeks, as well as those who progressed before that assessment. It is not necessarily the same as all enrolled participants. The record describes the proportion as the mean of a binomial distribution and reports a 95% confidence interval for each arm.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
For Arm I, taselisib with enzalutamide, the reported clinical benefit rate was 0.357 among 14 patients, with a 95% confidence interval of 0.163 to 0.612. For Arm II, enzalutamide alone, the reported rate was 0.0 among 5 patients, with a 95% confidence interval of 0.0 to 0.434. These are estimates for the endpoint population and assessment described in the registry, not necessarily for every enrolled participant.
The intervals convey substantial uncertainty around both estimates. In particular, the interval for the enzalutamide-only arm extends from 0.0 to 0.434, so an observed rate of 0.0 does not establish that the underlying clinical benefit rate is exactly zero. The combination-arm interval also spans a broad range of possible values. A confidence interval describes the precision of an estimate under the assumptions of the method used; the interval is not a guarantee that the true rate lies within its endpoints. The record does not identify the method used to calculate these intervals.
The point estimates alone do not establish a treatment difference. The registry does not report a formal between-arm effect estimate, confidence interval for a difference, or p-value. The arms also have different reported endpoint sample sizes. A reader should not infer a comparative effect merely by contrasting the two point estimates, particularly when the estimates are imprecise and the study is described as partially randomized. The exact allocation process is not reported in the registry record.
The trial status is terminated, and the endpoint results should be interpreted as the posted observations rather than as confirmation of a treatment effect. Reasons for termination, the planned target enrollment for the phase II comparison, and whether the study ended according to its intended schedule are not reported in the registry record. The enrollment of 30 does not substitute for the endpoint denominators: the primary analysis was defined in an evaluable population, and the results are presented for 14 and 5 patients in the two arms.
Two secondary outcomes are listed: overall progression-free survival for patients treated with enzalutamide and taselisib, and pharmacokinetic profile. Their detailed numerical findings are not reported in the registry record. Consequently, the clinical benefit rate results do not by themselves describe progression-free survival, drug exposure, or the full pattern of outcomes over follow-up.
9. Statistical Methods Explained
The endpoint is a proportion: each patient in the defined analysis population either met the clinical-benefit definition by the specified assessment or did not. The registry reports the mean of a binomial distribution and a 95% confidence interval for each arm. The clinical benefit rate is therefore read as an estimate of the fraction of evaluable patients meeting the response definition. The registry does not state the interval-calculation method, so it is not appropriate to attribute the limits to a particular formula.
What does a confidence interval express?
General: A confidence interval communicates the statistical uncertainty around an estimated quantity. Its width is affected by the amount of information in the data and by the method used. A broad interval indicates that the estimate is not very precise; it does not mean that all values inside the interval are equally plausible, nor that values outside it are impossible. Here, the intervals are broad relative to the reported rates, and the arm-specific patient counts are limited. That combination calls for cautious interpretation of the point estimates.
The 95% confidence interval is not a measure of individual patient variability, and it does not say what outcome any particular patient will experience. It also does not resolve questions about design, selection, or missing outcomes. Confidence intervals describe sampling uncertainty under statistical assumptions; they do not correct for bias caused by how participants were assigned, how outcomes were measured, or which participants were included.
Why is the arm comparison not a simple subtraction?
The registry reports arm-specific proportions and intervals, but no formal comparative analysis. A between-arm analysis would need to define a contrast and account for the study design and analysis population. The posted results do not provide a comparison estimate or its uncertainty. It would be misleading to treat the difference between the two displayed point estimates as a sponsor-reported treatment effect. The record also does not report an adjusted analysis, covariate strategy, or test for the phase II comparison.
General: A randomized comparison can support causal interpretation when allocation, follow-up, outcome assessment, and analysis are conducted as planned. A description of randomized allocation alone does not supply those details. This study is described as partially randomized, and the available record does not explain the allocation process or identify which participants entered each component. The phase Ib dose groups and phase II arms also address related but distinct questions: dose evaluation is not interchangeable with an arm-level phase II endpoint comparison.
How does crossover affect interpretation?
The record allows participants in the enzalutamide-only arm to cross over to the combination after progression, within the specified treatment-start window. Crossover changes treatment exposure after the initial assigned regimen. It is especially relevant to outcomes measured over longer follow-up, because later outcomes can reflect treatment received after progression as well as initial assignment. The primary endpoint is assessed at 16 weeks, but the record does not state how crossover timing related to each participant's endpoint assessment or how crossover was handled in analyses of the secondary outcomes. Those details are not reported in the registry record.
What does the planning illustration mean?
General: A sample-size calculation can describe how many participants might be needed to detect a specified effect under stated assumptions. It is a planning exercise, not an observed result and not evidence that a trial had a particular probability of success. The planning assumptions described for this analysis use a two-sided significance level of 0.05, equal allocation, and a normal approximation, with the effect to detect defined as a stated fraction of the observed effect. The illustration is not the trial's own design justification, and it should not be called observed power. The particular planning results are not included in the registry entries available for interpretation.
General: When outcomes are proportions, precision generally depends on the number of observed participants and the underlying rate. Small endpoint groups can produce wide uncertainty even when the observed rate is at an extreme such as 0.0. The interval for the enzalutamide-only arm demonstrates why an observed rate of 0.0 should not be treated as proof of a zero underlying rate. The two arms also have different endpoint denominators, which should remain visible when reading the estimates.
The registry lists no analyses in its analysis count, while posting the primary endpoint values and intervals. This means that the record does not provide a fuller set of inferential outputs, such as a comparative test or model-based estimate. It does not establish whether analyses were conducted outside the registry. No information is reported about interim analyses, prespecified stopping boundaries, or the statistical analysis plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary endpoint and two secondary outcomes: overall progression-free survival for patients treated with enzalutamide and taselisib, and pharmacokinetic profile. It does not report a multiplicity strategy, such as a hierarchy of hypotheses or an adjustment for testing multiple outcomes. Without that information, the relationship between any possible secondary analyses and a confirmatory claim cannot be assessed. Numerical secondary outcome results are not reported in the registry record.
The primary endpoint population is defined as evaluable patients who received at least one treatment cycle and either had disease reassessed at 16 weeks or progressed before that assessment. This definition provides some detail about who was counted, but the registry does not report a participant flow, the number excluded from the endpoint population, or reasons for non-evaluability. It also does not describe how missing or unevaluable response assessments were handled beyond the stated population definition.
Missing-data methods can matter when patients without an assessment differ from those with an assessment. General: excluding participants without evaluable outcomes can change the composition of the group being summarized, while other approaches require assumptions about their outcomes. The record does not report whether any sensitivity analyses were used to assess this issue.
The record does not report an intention-to-treat analysis population, per-protocol population, or other analysis-set rules beyond the primary endpoint's evaluable population definition. Nor does it report interim analyses or a detailed prespecified statistical plan. These omissions limit assessment of multiplicity, missing outcomes, and how closely the posted endpoint analysis followed an intended plan.
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 |
|---|---|---|---|
| Taselisib 2 mg | 0 | 3 | 0.0% |
| Taselisib 4 mg | 2 | 5 | 40.0% |
| Taselisib 6 mg | 1 | 3 | 33.3% |
| Taselisib 8 mg | 0 | 2 | 0.0% |
| Enzalutamide | 1 | 2 | 50.0% |
| Enzalutamide + Taselisib | 10 | 12 | 83.3% |
| Cross-Over | 1 | 3 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Taselisib 2 mg | 2 of 3 |
| Taselisib 4 mg | 4 of 5 |
| Taselisib 6 mg | 3 of 3 |
| Taselisib 8 mg | 2 of 2 |
| Enzalutamide | 2 of 2 |
| Enzalutamide + Taselisib | 5 of 12 |
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 |
|---|---|
| Taselisib 2 mg | 3 of 3 |
| Taselisib 4 mg | 5 of 5 |
| Taselisib 6 mg | 3 of 3 |
| Taselisib 8 mg | 2 of 2 |
| Enzalutamide | 2 of 2 |
| Enzalutamide + Taselisib | 12 of 12 |
Most frequent serious adverse event terms
| Event term | Organ system | Taselisib 2 mg | Taselisib 4 mg | Taselisib 6 mg | Taselisib 8 mg | Enzalutamide | Enzalutamide + Taselisib |
|---|---|---|---|---|---|---|---|
| Fever | General disorders | 0 of 3 | 1 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 5 of 12 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 6 of 12 |
| Confusion | Psychiatric disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 2 of 12 |
| Fatigue | General disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 2 of 12 |
| Anorexia | Metabolism and nutrition disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 2 of 12 |
| Back pain | Musculoskeletal and connective tissue disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 1 of 12 |
| Colitis | Gastrointestinal disorders | 0 of 3 | 1 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 0 of 12 |
| Neoplasms benign | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 1 of 12 |
| Hypotension | Vascular disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 1 of 12 |
| Febrile neutropenia | Blood and lymphatic system disorders | 0 of 3 | 0 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 1 of 12 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Taselisib 2 mg | Taselisib 4 mg | Taselisib 6 mg | Taselisib 8 mg | Enzalutamide | Enzalutamide + Taselisib |
|---|---|---|---|---|---|---|---|
| Fatigue | General disorders | 2 of 3 | 5 of 5 | 3 of 3 | 2 of 2 | 2 of 2 | 10 of 12 |
| Nausea | Gastrointestinal disorders | 2 of 3 | 2 of 5 | 2 of 3 | 1 of 2 | 2 of 2 | 7 of 12 |
| Anemia | Blood and lymphatic system disorders | 1 of 3 | 5 of 5 | 3 of 3 | 2 of 2 | 0 of 2 | 4 of 12 |
| Hyperglycemia | Metabolism and nutrition disorders | 0 of 3 | 5 of 5 | 3 of 3 | 2 of 2 | 0 of 2 | 3 of 12 |
| Diarrhea | Gastrointestinal disorders | 1 of 3 | 4 of 5 | 1 of 3 | 2 of 2 | 0 of 2 | 4 of 12 |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | 2 of 3 | 2 of 5 | 1 of 3 | 2 of 2 | 0 of 2 | 4 of 12 |
| Headache | Gastrointestinal disorders | 2 of 3 | 2 of 5 | 2 of 3 | 1 of 2 | 0 of 2 | 4 of 12 |
| Aspartate aminotransferase increased | Investigations | 1 of 3 | 2 of 5 | 2 of 3 | 1 of 2 | 1 of 2 | 2 of 12 |
| Vomitting | Gastrointestinal disorders | 0 of 3 | 4 of 5 | 0 of 3 | 0 of 2 | 0 of 2 | 4 of 12 |
| Gastroesophageal reflux disease | Gastrointestinal disorders | 2 of 3 | 5 of 5 | 0 of 3 | 1 of 2 | 0 of 2 | 0 of 12 |
Serious events with the highest arm-level rate: rash maculo-papular (50.0%), heart failure (50.0%), dyspnea (50.0%), fever (41.7%), pleural effusion (33.3%).
The registry reports serious adverse event counts by listed treatment group, alongside the number at risk. For example, the enzalutamide plus taselisib group lists 12 at risk and 10 affected by serious adverse events. The enzalutamide-only group lists 2 at risk and 1 affected. These are group-level counts as posted; they do not establish that a treatment caused an event. The registry also lists deaths by group, but the figures do not specify cause or establish a relationship to treatment.
The record lists serious adverse event terms with percentages, including rash maculo-papular, heart failure, and dyspnea at 50.0% each; fever at 41.7%; and pleural effusion and left ventricular systolic dysfunction at 33.3% each. These percentages should be read as posted. The record does not give enough information to determine from the term list alone how events were distributed across treatment groups, whether one participant could contribute more than one term, or the denominator used for each term.
Group sizes differ, and the safety groups include dose groups, the combination group, enzalutamide alone, and crossover. The groups should not be combined or compared as though they were equivalent randomized safety populations. The record does not report event timing, severity grading details, exposure-adjusted rates, or causal attribution for the listed events. Counts and percentages describe the registry entries, not an individual risk prediction.
12. What the Result Does, and Does Not, Mean
The posted results show that the registry recorded clinical benefit rate estimates at 16 weeks for two phase II arms. The combination arm estimate was 0.357 among 14 patients, with a 95% confidence interval of 0.163 to 0.612. The enzalutamide-only estimate was 0.0 among 5 patients, with a 95% confidence interval of 0.0 to 0.434. These figures describe the defined evaluable endpoint populations and the response categories specified under RECIST criteria 1.1.
The results do not, on their own, establish that one regimen is better than the other. No formal comparison estimate or p-value is reported, and the confidence intervals indicate considerable uncertainty. The registry's description of a partially randomized design also matters: the record does not explain the allocation details needed to evaluate the comparability of the groups. An arm-level rate is not a causal effect simply because the record also identifies randomized allocation.
The clinical benefit endpoint combines complete response, partial response, and stable disease at a particular assessment time. It does not provide a complete account of tumor response over time, duration of benefit, progression-free survival, or overall survival. Although progression-free survival and pharmacokinetic profile are listed as secondary outcomes, their numerical findings are not reported in the registry record.
The record's terminated status and the small endpoint populations further limit how broadly the estimates can be interpreted. The reason for termination, handling of missing assessments, and detailed analysis plan are not reported. The numbers therefore describe the posted endpoint results, with uncertainty, rather than settling the clinical or comparative question. They should not be interpreted as treatment advice or as proof of benefit or lack of benefit.
13. Why This Trial Matters Statistically
This trial is statistically notable because it combines early dose evaluation with a phase II clinical benefit endpoint and a partially randomized description. Those features make it important to distinguish the questions being addressed. Dose groups can inform tolerability and dose selection, while a phase II endpoint estimates outcomes in a specified patient group. One should not treat all dose groups and the phase II arms as a single homogeneous comparison.
The endpoint is a proportion assessed at a fixed time point, and the reported intervals make precision central to interpretation. The combination-arm estimate and the enzalutamide-only estimate are based on different numbers of evaluable patients. The wide intervals show why point estimates alone are not enough to characterize uncertainty. In particular, a zero observed rate in a small group can coexist with a confidence interval extending to 0.434.
Crossover adds another design consideration for outcomes extending beyond the initial treatment period. Participants in the enzalutamide-only arm could receive the combination after progression, so later outcomes may reflect more than the initial arm. A statistical analysis of time-to-event outcomes would need to explain how this was handled, but such details and numerical secondary outcome results are not reported in the registry record.
Finally, the record illustrates the value of separating posted outcomes from formal comparative evidence. Arm-specific estimates are informative descriptions, but without a reported comparative analysis, allocation details, and fuller information about missing data and multiplicity, their statistical meaning remains limited. The trial's status is terminated, but the reason is not reported; no explanation should be inferred from that status alone.
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 endpoint populations are substantially smaller than the enrollment count, and the registry does not provide participant flow or explain the differences. The clinical benefit results are reported for 14 patients in the combination arm and 5 in the enzalutamide-only arm, within an evaluable population defined by treatment exposure and disease assessment. How many enrolled participants were not included, and why, are not reported in the registry record.
The study is described as partially randomized, but the allocation process, group assignment details, and phase-specific participant counts are not reported. The record does not explain how the phase Ib dose groups relate to the phase II arms or how the combination arm's treatment schedule maps to the dose-group schedules. This makes it difficult to assess comparability and interpret the broader design.
The registry does not state the confidence-interval method, report a formal comparison of the phase II arms, or provide adjusted estimates. It also does not report the statistical analysis plan, multiplicity handling, missing-data sensitivity analyses, interim analyses, or a sample-size justification. The trial status is terminated, but the reason and its statistical implications are not reported.
Safety summaries are given for groups with different numbers at risk, including crossover, and the record does not provide exposure-adjusted rates or event attribution. The secondary outcomes are listed without numerical results. Together, these gaps mean that the posted results support a narrow description of the 16-week endpoint and reported safety entries, but not a complete assessment of comparative efficacy, long-term outcomes, or safety across treatment strategies.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Taselisib and Enzalutamide in Treating Patients With Androgen Receptor Positive Triple-Negative Metastatic Breast Cancer — NCT02457910.
- Registry record last updated 2022-08-22; status last verified 2022-08.
- Results first posted 2020-05-07.
- 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
NCT02457910 is a terminated phase Ib/II trial of taselisib with enzalutamide in androgen receptor-positive triple-negative metastatic breast cancer. The registry describes randomized allocation, no masking, a sequential model, and a partially randomized study. Enrollment was 30.
The primary phase II endpoint was clinical benefit rate at 16 weeks among evaluable patients. The combination arm had a reported rate of 0.357 among 14 patients, with a 95% confidence interval of 0.163 to 0.612; the enzalutamide-only arm had a rate of 0.0 among 5 patients, with a 95% confidence interval of 0.0 to 0.434. A formal arm comparison is not reported in the registry record. The record also lists progression-free survival and pharmacokinetic profile as secondary outcomes but does not report their numerical results.