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 Ixabepilone in Treating Patients With Metastatic Prostate Cancer (NCT00087139), a na phase 2 study. The primary outcome is Proportion of Patients With PSA Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00087139 |
| Phase | Phase 2 |
| Condition | Adenocarcinoma of the Prostate, Recurrent Prostate Cancer, Stage IV Prostate Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 124 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Cancer Institute (NCI) |
| Results posted | Yes |
2. Clinical Question
Population
Patients with metastatic prostate cancer that had not responded to previous hormone therapy were the study population. The registry also lists adenocarcinoma of the prostate, recurrent prostate cancer, and stage IV prostate cancer as conditions. Patients were stratified by prior chemotherapy: none, 1 prior taxane-containing regimen, or 2 prior cytotoxic regimens.
Intervention
Patients received ixabepilone intravenously over 1 hour on days 1, 8, and 15. Courses were scheduled to repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Comparator
This was a single-group study, with no concurrent comparison group described. The registry lists allocation as not applicable and masking as none, so the study does not provide a randomized, masked comparison with another treatment or control.
Primary question
The primary endpoint was the proportion of patients with a PSA response. A response required a decline from baseline by >=50%, or normalization of PSA to <0.2 ng/lm, confirmed by a second measurement >=4 weeks later. The registry description says the response proportion was calculated separately for 3 strata and only among the first cohort.
The clinical question is therefore whether PSA responses were observed in the defined study population receiving the specified ixabepilone schedule, considered separately by prior-chemotherapy stratum. The registry record does not report the response proportions or their confidence intervals, so the size and precision of any response cannot be assessed from the posted results. The single-group design also means that the study does not estimate a treatment contrast against a concurrent comparator. Its primary outcome is a biomarker response, rather than a direct comparison of survival or another clinical outcome.
3. Trial Design
This completed Phase II study evaluated a weekly schedule of ixabepilone in patients with metastatic prostate cancer that had not responded to previous hormone therapy. The registry records enrollment of 124. It identifies the National Cancer Institute (NCI) as sponsor and gives a study start of 2004-09 and primary completion of 2009-04.
The design is listed as single-group, with allocation not applicable and masking none. Accordingly, the study describes outcomes among patients receiving ixabepilone; it does not contain a concurrent randomized control group. The arm description says patients were stratified according to prior chemotherapy: none, 1 prior taxane-containing regimen, or 2 prior cytotoxic regimens. These strata describe patient history, not separate randomized treatment assignments.
Ixabepilone was administered intravenously over 1 hour on days 1, 8, and 15, with courses repeating every 28 days unless disease progression or unacceptable toxicity occurred. The registry lists 4 outcome measures, including the primary PSA-response outcome and secondary outcomes for measurable disease response, duration of PSA response, and duration of measurable disease response.
The registry description says additional patients could be accrued if the regimen met a protocol-specified PSA response rate, with a target of 25 eligible patients with measurable disease in each stratum. It also says the PSA response rate was calculated only among the first cohort, excluding additional patients accrued to the study. The precise analysis population and the numbers analyzed within each stratum are not reported in the registry record. The protocol’s full statistical plan, including the rationale for its enrollment target, is not reported in the registry record.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Ixabepilone - no Prior Chemo | Ixabepilone - Prior Taxane | Ixabepilone - Two Prior Chemo |
|---|---|---|---|
| STARTED | 39 | 49 | 36 |
| Treated | 39 | 48 | 36 |
| Eligible and Treated | 35 | 42 | 32 |
| Patients for the Analysis of PSA Respons | 30 | 34 | 22 |
| Patients With Measurable Disease | 22 | 25 | 24 |
| Patients With PSA Response | 10 | 8 | 4 |
| Patients w/ Measurable Disease Response | 5 | 2 | 0 |
| COMPLETED | 0 | 0 | 0 |
| NOT COMPLETED | 39 | 49 | 36 |
Reasons for not completing this period:
| Reason | Ixabepilone - no Prior Chemo | Ixabepilone - Prior Taxane | Ixabepilone - Two Prior Chemo |
|---|---|---|---|
| Adverse Event | 18 | 17 | 13 |
| Death | 2 | 1 | 0 |
| Withdrawal by Subject | 3 | 2 | 2 |
| Physician Decision | 1 | 1 | 1 |
| Disease progression | 9 | 19 | 12 |
| Ineligible | 4 | 7 | 4 |
| Symptomatic deterioration | 1 | 1 | 3 |
| Use of coumadin | 1 | 0 | 0 |
| Response achieved | 0 | 1 | 0 |
| Extradural disease | 0 | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: If the regimen demonstrated a PSA response rate specified in the protocol, additional patients would be entered so the total number of eligible patients with measurable disease in each stratum is 25. But the PSA response rate was only calculated among the first cohort of patients, not including the additional patients with measurable disease.
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 | Ixabepilone - no Prior Chemo | Ixabepilone - Prior Taxane | Ixabepilone - Two Prior Chemo | Total |
|---|---|---|---|---|---|
| Participants analysed | 35 | 42 | 32 | 109 | |
| Age, Continuous | years | 72 (45 to 82) | 69 (50 to 81) | 67.5 (49 to 90) | 70 (45 to 90) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 35 | 42 | 32 | 109 |
6. Primary Endpoint
The registry lists the primary outcome as Proportion of Patients With PSA Response, measured over Every 4 weeks during treatment; then every 3 months if <2 years from study entry; then every 6 months if 2-5 years from study entry.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 90% Confidence Interval |
| Unit | Proportion of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the proportion of patients with a PSA response. PSA is a biomarker, and this endpoint classifies whether a participant met a prespecified response definition during treatment. The definition required either a decline from baseline by >=50% or normalization to <0.2 ng/lm, with the qualifying change confirmed by a second measurement >=4 weeks later. Requiring confirmation helps distinguish a sustained qualifying measurement from an isolated result.
The registry specifies PSA assessments every 4 weeks during treatment, then every 3 months if <2 years from study entry, and then every 6 months if 2-5 years from study entry. It reports that the response proportion was evaluated separately for the 3 prior-chemotherapy strata. The description also states that only the first cohort contributed to this calculation, even though additional patients could be accrued under the protocol’s stated condition.
In general, a response proportion offers a straightforward way to describe how often a defined binary outcome occurred in a study group. It does not, by itself, measure how long a response lasts or establish a comparative treatment effect. The registry lists duration of PSA response and measurable disease outcomes separately as secondary outcomes. The primary response estimates and their 90% confidence intervals are not reported in the registry record, so neither the response frequency nor its precision can be evaluated from the available results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies a clinically defined PSA-response endpoint and states that the response proportion was calculated separately for 3 prior-chemotherapy strata. However, it does not report the corresponding proportions or the 90% confidence intervals. As a result, the posted results do not establish how often a response occurred in any stratum, how uncertain an estimate might be, or whether the response proportions differed across strata.
The enrollment figure of 124 describes the study, but it should not be treated as the denominator for the primary endpoint. The registry says that the PSA analysis used only the first cohort and excluded additional patients accrued to the study. The numbers contributing to the primary analysis, both overall and within each stratum, are not reported in the registry record. Enrollment, eligibility, receipt of treatment, and inclusion in an endpoint analysis can describe different groups; the record does not provide enough detail to reconcile them for this outcome.
The study’s single-group design is important when interpreting any outcome. A response observed among treated patients would describe what happened in this study group, but without a concurrent comparator it would not quantify how the outcome would have differed under another treatment or no treatment. Stratification by prior chemotherapy provides separate descriptions by treatment history, but it does not create randomized comparison groups. The registry record does not report adjusted or unadjusted comparisons among those strata.
The endpoint definition includes a confirmation requirement, which makes the classification depend on repeat measurement. The schedule for PSA assessment is specified, but the record does not say how missing or delayed measurements were handled, how participants without confirmation were classified, or whether all participants had sufficient follow-up to be assessed. These omissions limit interpretation of the endpoint even if an estimate were available.
The registry lists secondary outcomes for best overall measurable disease response and duration of both PSA and measurable disease response. It does not provide their results in the registry record recorded for this study. No analysis entries are reported, and the primary response estimates themselves are absent. Therefore, conclusions about response frequency, durability, uncertainty, or differences by stratum are not supported by the registry information available. The safety summary is a separate descriptive record and does not fill that efficacy-reporting gap.
9. Statistical Methods Explained
What does a response proportion represent?
In general, a binary endpoint places each participant into a defined outcome category, such as response or no response. A response proportion describes the share of the analyzed group classified as having the event. For this study, the registry defines the event using a PSA decline from baseline by >=50% or normalization to <0.2 ng/lm, with confirmation by a second measurement >=4 weeks later. The definition matters because a different threshold, confirmation rule, or analysis population could produce a different proportion.
The registry calls the endpoint a proportion and specifies a 90% confidence interval as its dispersion measure. In general, a confidence interval conveys uncertainty around an estimated quantity: narrower intervals indicate greater precision, while wider intervals indicate less precision. It is not a guarantee that a particular interval contains the underlying value. No interval limits or response estimates are reported in the registry record, so precision cannot be judged for this endpoint.
Why are the prior-chemotherapy strata relevant?
The study description separates patients according to prior chemotherapy: none, 1 prior taxane-containing regimen, or 2 prior cytotoxic regimens. The PSA response proportion was reported separately for 3 strata according to the primary outcome description. In general, stratified reporting can show whether outcome patterns vary across clinically relevant groups. It does not, on its own, demonstrate that the groups differ: participants in the strata may have different treatment histories, and the study is not described as randomizing patients between those histories or between treatments.
The record does not report the response estimate or denominator for each stratum. It therefore does not permit assessment of the precision of stratum-specific estimates or a formal comparison across strata. The total enrollment figure should not be substituted for stratum-specific analysis denominators.
What does confirmation contribute?
The endpoint requires the qualifying PSA change or normalization to be confirmed by a second measurement at least 4 weeks later. In general, confirmation rules can reduce classification based on a transient or unverified measurement. They also mean that response status depends on having a later measurement available. The registry does not report how participants lacking a confirmatory measurement were handled, or how missing PSA observations were treated.
The record specifies a PSA assessment schedule: every 4 weeks during treatment; then every 3 months if <2 years from study entry; then every 6 months if 2-5 years from study entry. It does not report adherence to that schedule, the timing of individual measurements, or whether outcome assessment was complete for all participants included in the endpoint analysis.
What can be learned from the design?
This is a single-group study with masking listed as none. In general, a single-group endpoint estimate describes outcomes within the treated study group. It does not provide a randomized estimate of the difference between ixabepilone and a concurrent control. Differences between historical groups or between the prior-chemotherapy strata would not be equivalent to a randomized treatment comparison.
The registry reports a 90% confidence interval for the primary endpoint, but the method used to calculate it is not reported in the registry record. The statistical analysis plan, any adjustment procedures, and any interim analyses are also not reported in the registry record. The record describes a protocol condition for accruing additional patients and says the primary PSA response calculation used only the first cohort. It does not give the decision rule’s numerical response threshold or a full account of the accrual and analysis process.
General planning calculations, when used, are not observed power. The planning assumptions recorded for this study describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects defined as fractions of an observed effect. Those are planning assumptions, not features of this single-group study’s treatment allocation. The record does not report a trial-specific sample-size justification or an observed power analysis.
10. Exact Estimates for Each Arm
No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Ixabepilone - no Prior Chemo | 30 | 0 | 0.0% | 0.0% to 11.6% | 0.0% to 11.4% | 0.1% to 11.2% |
| Ixabepilone - Prior Taxane | 34 | 0 | 0.0% | 0.0% to 10.3% | 0.0% to 10.2% | 0.1% to 10.0% |
| Ixabepilone - Two Prior Chemo | 22 | 0 | 0.0% | 0.0% to 15.4% | -0.0% to 14.9% | 0.1% to 14.8% |
R code and output
arm <- c("Ixabepilone - no Prior Chemo", "Ixabepilone - Prior Taxane", "Ixabepilone - Two Prior Chemo")
n <- c(30, 34, 22)
events <- c(0, 0, 0)
for (i in seq_along(n)) {
ci <- binom.test(events[i], n[i])$conf.int # Clopper-Pearson exact interval
cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i]) # Bayesian, uniform Beta(1,1) prior
wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}
ARM Ixabepilone - no Prior Chemo 30 0 0.0 0.0 11.6 0.0 11.4 0.1 11.2
ARM Ixabepilone - Prior Taxane 34 0 0.0 0.0 10.3 0.0 10.2 0.1 10.0
ARM Ixabepilone - Two Prior Chemo 22 0 0.0 0.0 15.4 -0.0 14.9 0.1 14.811. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary PSA-response outcome and secondary outcomes for measurable disease response, duration of PSA response, and duration of measurable disease response. Having several outcomes raises the general statistical issue of multiplicity: when many hypotheses are examined, the chance of at least one apparently notable result can increase. Whether and how to address that issue depends on the prespecified analysis plan. A multiplicity strategy is not reported in the registry record, so it is not possible to determine whether any adjustment was planned or used.
The record does not report the results for the primary endpoint or the listed secondary outcomes. It also does not report analysis entries, and no statistical analysis plan is available in the registry information. The exact analysis population, stratum-specific denominators, and handling of participants with incomplete endpoint measurements are not reported in the registry record.
Missing-data handling is particularly relevant to a response definition requiring confirmation by a second measurement. In general, participants who lack a confirmatory measurement may be excluded, classified under a prespecified rule, or handled through another approach. Those choices can affect the estimated response proportion. The registry record does not state which approach was used, whether missingness was assessed, or whether sensitivity analyses were conducted.
The description says additional patients could be accrued if a protocol-specified PSA response rate was demonstrated, but the response calculation was restricted to the first cohort and excluded additional accrued patients. The numerical accrual criterion, the relationship between that criterion and the reported analysis, and the analysis-population details are not reported in the registry record. No conclusion about interim monitoring or stopping rules can be drawn from the information recorded.
12. 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 |
|---|---|---|---|
| Ixabepilone | 90 | 123 | 73.2% |
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 |
|---|---|
| Ixabepilone | 122 of 123 |
Most frequent serious adverse event terms
| Event term | Organ system | Ixabepilone |
|---|---|---|
| Neuropathy-sensory | Nervous system disorders | 32 of 123 |
| Fatigue | General disorders | 28 of 123 |
| Leukopenia | Investigations | 27 of 123 |
| Neutropenia | Investigations | 22 of 123 |
| Neuropathy-motor | Nervous system disorders | 13 of 123 |
| Anorexia | Metabolism and nutrition disorders | 12 of 123 |
| Anemia | Blood and lymphatic system disorders | 8 of 123 |
| Diarrhea w/o prior colostomy | Gastrointestinal disorders | 7 of 123 |
| Nausea | Gastrointestinal disorders | 7 of 123 |
| Dehydration | Metabolism and nutrition disorders | 6 of 123 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ixabepilone |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 112 of 123 |
| Fatigue | General disorders | 89 of 123 |
| Leukopenia | Investigations | 84 of 123 |
| Neuropathy-sensory | Nervous system disorders | 66 of 123 |
| Anorexia | Metabolism and nutrition disorders | 56 of 123 |
| Nausea | Gastrointestinal disorders | 56 of 123 |
| Neutropenia | Investigations | 51 of 123 |
| Alopecia | Skin and subcutaneous tissue disorders | 45 of 123 |
| Diarrhea w/o prior colostomy | Gastrointestinal disorders | 41 of 123 |
| Taste disturbance | Nervous system disorders | 31 of 123 |
Serious events with the highest arm-level rate: neuropathy-sensory (26.0%), fatigue (22.8%), leukopenia (22.0%), neutropenia (17.9%), neuropathy-motor (10.6%).
The registry’s safety summary lists 123 participants at risk for serious adverse events, with 90 affected, and 123 at risk for other adverse events, with 122 affected. These are descriptive counts in the ixabepilone group. They are not comparisons with a control group, and they do not establish whether an event was caused by treatment. The registry’s serious-event summary also lists neuropathy-sensory (26.0%), fatigue (22.8%), leukopenia (22.0%), neutropenia (17.9%), neuropathy-motor (10.6%), and anorexia (9.8%).
In general, a serious adverse event is a safety classification and should not be treated as interchangeable with every adverse event or with an event’s severity rating. The registry record does not specify the denominator used for the listed term percentages or provide additional detail about event definitions and timing. The percentages should therefore be read as reported, without inferring a different denominator or calculating new rates. The safety figures describe reported events in this study group; they do not establish comparative safety.
13. What the Result Does, and Does Not, Mean
The registry record establishes that this was a completed Phase II, single-group study of ixabepilone and describes a prespecified PSA-response definition. It also records the schedule of treatment, prior-chemotherapy strata, and a safety summary. Those details clarify what the study set out to measure and the context in which outcomes should be interpreted.
The record does not report the primary PSA-response proportions or their 90% confidence intervals. It therefore does not show the observed response frequency, how precisely it was estimated, or whether responses varied by prior-chemotherapy stratum. Although the registry lists secondary outcomes, their results are not reported in the information available. The absence of these results means the record cannot support a conclusion about the magnitude or duration of response.
Even if response estimates were available, the single-group design would limit what they could establish. An observed response proportion would describe participants in this study; it would not measure a randomized difference against another treatment. The study also stratified by prior chemotherapy rather than assigning those histories at random, so differences between strata could not be interpreted as treatment effects.
The endpoint is a biomarker response defined by a threshold and confirmation rule. In general, a biomarker response is not itself a complete account of patient experience, disease course, or comparative benefit. The registry lists measurable disease and response-duration outcomes separately, but their results are not reported. The available safety counts and listed event percentages are descriptive and likewise do not provide a comparative assessment.
Accordingly, the posted record is informative about design, endpoint definition, and recorded safety events, but it does not provide the efficacy estimates needed to characterize the primary result. It also does not report the analysis details needed to assess missing measurements, denominator selection, or statistical adjustment.
14. Why This Trial Matters Statistically
This study illustrates why a clear endpoint definition is only one part of interpreting a clinical trial. The registry specifies a PSA response threshold, a normalization criterion, and a confirmation measurement. Those details make the intended outcome understandable, but the response proportions and confidence intervals are not reported. Without the estimates and their analysis denominators, neither the frequency nor precision of the endpoint can be evaluated.
The study also highlights the distinction between stratification and randomization. Prior chemotherapy was used to define 3 strata, and the primary outcome description says response proportions were calculated separately for them. This can be useful for describing outcomes across different treatment histories, but it does not remove the limitations of a single-group design or create a controlled treatment comparison.
Its accrual description makes analysis populations especially important. The registry says additional patients could be enrolled under a protocol condition, while the PSA response calculation used only the first cohort. Enrollment and endpoint-analysis populations therefore cannot be assumed to be identical. The reported enrollment figure alone does not reveal how many participants contributed to the primary endpoint or to each stratum.
Finally, the record distinguishes efficacy outcomes from safety summaries. The safety counts and listed event percentages offer descriptive information, while the missing response estimates prevent assessment of the primary efficacy outcome. Together, these features show why registry interpretation requires attention to design, endpoint definitions, denominators, uncertainty, and completeness of outcome reporting—not just the trial’s title or enrollment figure.
15. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most important limitation in the registry information is that the primary PSA-response proportions and their 90% confidence intervals are not reported. Although the endpoint is defined and the analysis is described as stratified, the results needed to assess response frequency and precision are absent. The listed secondary outcomes also have no reported results in the record.
The single-group design provides no concurrent treatment comparison. The registry lists allocation as not applicable and masking as none, so the study cannot estimate a randomized contrast. The 3 prior-chemotherapy strata are descriptive categories and do not substitute for randomized groups.
The analysis population is also unclear. The registry reports enrollment of 124, but says the primary PSA calculation used only the first cohort and excluded additional patients accrued to the study. The number analyzed overall, the stratum-specific denominators, and the handling of participants without a confirmed response measurement are not reported in the registry record. The description’s account of the population is incomplete, and the full analysis criteria are not reported.
The registry record does not report the statistical method used for the confidence intervals, a multiplicity adjustment, the missing-data approach, or a complete statistical analysis plan. It does not report the numerical protocol response criterion governing additional accrual. These gaps make it impossible to reconstruct the primary analysis or assess whether all planned outcomes and analysis decisions are fully represented.
The safety summary is descriptive and lacks a concurrent comparator. The denominator for the listed serious-event term percentages and further event-level context are not reported in the registry record. These limitations constrain interpretation of both efficacy and safety information.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Ixabepilone in Treating Patients With Metastatic Prostate Cancer — NCT00087139.
- Registry record last updated 2014-05-09; status last verified 2013-04.
- Results first posted 2012-12-27.
- 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.
19. Record Summary
NCT00087139 is a completed Phase II, single-group study sponsored by the National Cancer Institute (NCI), with enrollment of 124. The registry records a study start of 2004-09 and primary completion of 2009-04. Patients received ixabepilone intravenously on days 1, 8, and 15, with courses scheduled every 28 days in the absence of progression or unacceptable toxicity. Prior chemotherapy defined 3 strata.
The primary endpoint was the proportion of patients with PSA response, defined by a decline from baseline by >=50% or normalization to <0.2 ng/lm, confirmed by a second measurement >=4 weeks later. The registry says the primary response calculation used the first cohort and excluded additional accrued patients. The response estimates, confidence intervals, exact analysis population, and results for the listed secondary outcomes are not reported in the registry record.
The safety summary reports affected counts for serious and other adverse events and lists serious-event terms with percentages. Because the study has no concurrent comparator, these safety figures are descriptive.