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 A Study of Pertuzumab in Participants With Prostate Cancer (NCT02480010), a non-randomized phase 2 study. The primary outcome is Percentage of Participants With Confirmed, Objective Response, Non-Response or Progressive Disease by PSA Levels Within the First 24 Weeks of Treatment With Pertuzumab.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02480010 |
| Phase | Phase 2 |
| Condition | Prostate Cancer |
| Allocation | Non-Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 68 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hoffmann-La Roche |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned participants with hormone-refractory prostate cancer who had not previously been treated with chemotherapy. The primary outcome specified the ITT population. The registry record lists prostate cancer as the condition and reports total enrollment of 68; it does not give additional eligibility details or a breakdown of participants included in the primary outcome analysis.
Intervention
Participants received intravenous pertuzumab in one of two dose cohorts. Cohort A used a loading dose of 840 mg followed by 420 mg on Day 1 of each 3-week cycle. Cohort B used 1050 mg on Day 1 of each 3-week cycle. The study summary says the higher-dose cohort was based on observations in Cohort A. The registry description of further enrollment decisions is incomplete, so the full decision process is not reported in the registry record.
Comparator
The two cohorts received different pertuzumab doses, but the study was nonrandomized and had no like-for-like control comparison. Assignment procedures and any adjustments intended to make the cohorts comparable are not reported in the registry record. Differences between cohorts therefore cannot be interpreted as an unbiased causal estimate of dose effects.
Primary question
The primary endpoint asked what percentage of participants had a confirmed objective PSA response, non-response, or progressive disease within the first 24 weeks of pertuzumab treatment. A response required a PSA decline of at least 50% from baseline confirmed by a later value under the specified criteria. The registry record states how these categories were defined, but does not report the number or percentage of participants in each category. The primary clinical question is therefore identifiable, while its numerical answer is not reported in the registry record.
3. Trial Design
This was a Phase II, open-label, multicenter study with a parallel structure and nonrandomized allocation. The registry record lists Hoffmann-La Roche as sponsor, a start date of 2003-09, primary completion of 2005-09, and a terminated status. It reports enrollment of 68 participants. The study title and summary describe participants with hormone-refractory prostate cancer who had not received chemotherapy.
Participants entered one of two pertuzumab cohorts. Cohort A received an 840-mg intravenous loading dose, followed by 420 mg on Day 1 of each 3-week cycle. Cohort B received 1050 mg intravenously on Day 1 of each 3-week cycle. The summary describes the second cohort as using a higher dose based on observations in Cohort A. Response was to be evaluated after 3 treatment cycles, but the available arm descriptions are truncated and do not fully state the subsequent enrollment rules.
Because allocation was not randomized and both cohorts received pertuzumab, this design is not a randomized comparison against an untreated or active control group. Cohort comparisons may reflect differences in who entered each cohort, timing, or other factors, as well as dose. The registry record does not report a protocol-level account of allocation procedures, any adjustment strategy, or a statistical analysis plan. It also does not report why the study was terminated. These omissions limit interpretation of both the design decisions and any between-cohort contrasts.
The registry lists 20 outcome measures, including the primary PSA classification and secondary outcomes such as time to disease progression, objective response by RECIST criteria, overall survival, and pain progression. The presence of an outcome in the registry does not by itself establish that results were reported or analyzed. No analysis results are listed 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 | Pertuzumab 420 Milligrams (mg) - Cohort A | Pertuzumab 1050 mg - Cohort B |
|---|---|---|
| STARTED | 35 | 33 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 35 | 33 |
Reasons for not completing this period:
| Reason | Pertuzumab 420 Milligrams (mg) - Cohort A | Pertuzumab 1050 mg - Cohort B |
|---|---|---|
| Adverse Event | 3 | 1 |
| Death | 1 | 0 |
| Insufficient therapeutic response | 31 | 29 |
| Refused Treatment | 0 | 3 |
Analysis population for the primary outcome, as defined in the registry: ITT population
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: Intent-to-Treat (ITT) population consisted of all participants enrolled and who received at least one dose of study medication.
| Characteristic | Unit | Pertuzumab 420 mg - Cohort A | Pertuzumab 1050 mg - Cohort B | Total |
|---|---|---|---|---|
| Participants analysed | 35 | 33 | 68 | |
| Age, Continuous | years | 70.8 (SD 7.45) | 71.2 (SD 6.01) | 71.0 (SD 6.74) |
| Sex: Female, Male: Female | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Male | Participants | 35 | 33 | 68 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Confirmed, Objective Response, Non-Response or Progressive Disease by PSA Levels Within the First 24 Weeks of Treatment With Pertuzumab, measured over Screening, Every 3 weeks up to Week 24.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint classified the percentage of participants with confirmed objective PSA response, non-response, or progressive disease within the first 24 weeks of treatment. PSA is the biomarker used for this endpoint; the registry describes response using Prostate Specific Antigen Working Group guidelines. A response required a decline of at least 50% from baseline, confirmed by a second value at least 4 weeks later. The confirmatory value had to remain at least 50% below baseline, could be higher than the first decline, and could not be 50% higher than that first decline. The response date was the date of the first qualifying decline.
Progressive disease was defined using at least three consecutive serum PSA measurements, each at least 7 days apart within the 3 months before trial start, documenting progressively increasing values. Non-response meant meeting neither the response nor progressive-disease definition. The endpoint therefore distinguishes categories rather than reporting a single response-only result.
PSA-based classification can provide a structured way to track biomarker change over a specified period. It is distinct from outcomes based on imaging, symptoms, or survival, which are also listed among the secondary outcomes. The registry record gives the endpoint definitions and timeframe but not the observed counts or percentages, uncertainty intervals, or analysis details. Consequently, the magnitude and precision of the primary outcome are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies the main efficacy question and its measurement rules, but does not post the result. It gives no number or percentage of participants classified as having a confirmed PSA response, non-response, or progressive disease. It also does not report an estimate of uncertainty, such as a confidence interval, or a test of the primary endpoint. The primary result therefore cannot be quantified from the registry record.
This absence matters for interpretation. A point estimate would describe the observed proportion in the analyzed population; an uncertainty interval would convey how much precision that estimate has. Neither is reported for the primary endpoint. Without them, readers cannot assess the observed frequency of each category or how uncertain those frequencies might be. The registry's description of a response threshold is not evidence that any particular number of participants met it.
The design also limits comparative interpretation. Participants were not randomly assigned, and the two cohorts received different pertuzumab doses rather than a control treatment. The summary says the higher dose was based on observations in the lower-dose cohort. That makes the cohorts sequentially informed rather than independent randomized groups. Any difference in outcomes between them could be related to participant composition, study timing, or other factors; the registry record does not report methods to separate these possibilities from a dose effect.
Although 68 participants were enrolled overall, this total alone does not show how many contributed to each primary endpoint category or whether all enrolled participants were included in the ITT analysis. The primary outcome names the ITT population, but the analysis count, treatment of missing measurements, and reasons for any exclusions are not reported in the registry record.
The study was terminated, but the reason is not reported in the registry record. Termination alone does not establish whether it was related to efficacy, safety, recruitment, operational considerations, or another cause. The record also lists secondary outcomes, but does not report their results or analyses. Thus, the posted information supports a description of the question, design, and selected safety counts—not a conclusion about the size or reliability of an efficacy effect.
9. Statistical Methods Explained
General: interpreting a percentage endpoint. A percentage endpoint expresses how many participants in a defined analysis population fall into a specified category. In this study, the categories were confirmed objective PSA response, non-response, and progressive disease. Interpreting such an endpoint requires knowing both the number in each category and the denominator used. The registry names the ITT population but does not post the category counts, percentages, or analysis denominator. The endpoint definition alone cannot substitute for these results.
General: uncertainty around an estimate. An estimate from a study population is not necessarily an exact reflection of the population from which participants were drawn. A confidence interval is a way of expressing the precision of an estimate: narrower intervals indicate greater precision, while wider intervals indicate less precision. The registry record does not report a confidence interval for the primary endpoint. It therefore does not show how precisely the PSA outcome categories were estimated.
General: categorical outcome analysis. For a binary outcome, analysis commonly focuses on the proportion with the event and its uncertainty. This primary endpoint has three named classifications, so the results would need to make clear how the categories were counted and how the denominator was handled. The record describes definitions but does not report the category results, statistical model, or any covariate adjustment. No particular analysis method should be inferred from the endpoint wording alone.
General: interpreting a nonrandomized cohort comparison. Random assignment is a design method for reducing systematic differences between comparison groups. Here, allocation was nonrandomized, and both cohorts received pertuzumab at different doses. Cohort outcomes can be described separately, but an unadjusted contrast between them would not by itself establish that dose caused any difference. Participants may differ for reasons related to how or when they entered each cohort. The registry record does not report adjustment methods or other procedures intended to address this concern.
General: the role of a control group. A concurrent control group can help distinguish changes associated with a study intervention from changes that might also occur without it or under another intervention. No randomized control group is described in this record. The design therefore does not support a randomized treatment-versus-control estimate. The two dose cohorts should not be treated as equivalent to a randomized control comparison.
General: staged enrollment. The study summary describes enrollment in two stages, with the higher-dose cohort based on observations in the lower-dose cohort. It also says response would be evaluated at the end of 3 treatment cycles to determine whether additional participants would be enrolled. The available cohort descriptions are truncated, so the full decision rules and any formal stopping boundaries are not reported in the registry record. It would be inappropriate to assume a particular adaptive design or decision threshold.
General: sample-size planning. A planning calculation asks how many participants might be needed under specified assumptions to detect a target effect with a chosen statistical procedure. It is not a measure of whether an already completed study had an effect, and it is not observed power. The registry record does not report the trial's own sample-size justification. Any planning illustration based on an observed effect is a separate hypothetical exercise, not evidence about the trial's design rationale or a replacement for the trial's reported results.
General: over-dispersion. Over-dispersion occurs when outcome variability is greater than a statistical model expects. If present, standard errors and uncertainty intervals from a model that assumes less variability may be too small. Whether this issue is relevant depends on the model and data. The registry record does not report a model or diagnostics for the primary endpoint, so no claim about over-dispersion can be made for this study.
General: multiplicity. When several outcomes or comparisons are assessed, the chance of finding an apparently notable result by chance can increase if many tests are interpreted together. The registry lists 20 outcome measures, but does not report which secondary analyses were conducted, how results were prioritized, or whether any multiplicity procedure was used. The registry does not report an analysis list or results for these outcomes. No multiplicity adjustment should be presumed.
The primary endpoint is defined according to PSA rules, while the listed secondary outcomes include measures based on RECIST criteria, disease progression, pain progression, and survival. These outcomes measure different aspects of disease course and should not be treated as interchangeable. The registry record does not provide results with which to assess agreement or divergence across them.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 20 outcome measures, including the primary PSA classification and multiple secondary outcomes. It reports no analyses. The statistical plan, the hierarchy used to prioritize outcomes, the handling of multiple comparisons, and any adjustment to control the chance of false-positive findings are not reported in the registry record. The existence of several registered outcomes does not establish that all were analyzed.
The primary endpoint identifies the ITT population, but the number of participants included in that analysis and the handling of missing PSA measurements are not reported in the registry record. The record does not describe imputation, censoring, exclusion rules, or sensitivity analyses for missing data. It is therefore not possible to determine whether participants without complete follow-up were included in a particular category or excluded from a denominator.
Missingness can affect an estimate if the participants with recorded outcomes differ from those without them. This is a general concern, not a finding about this trial. Without the number and pattern of missing observations and the analysis rules, the extent and direction of any impact cannot be assessed.
The registry does not report the reason for termination, whether any interim analyses occurred, or whether the study's analysis plan changed. These details are not reported in the registry record and should not be inferred from the terminated status. The posted record is therefore insufficient to evaluate outcome multiplicity, missing-data assumptions, or adherence to a prespecified analysis 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 |
|---|---|---|---|
| Pertuzumab 420 mg - Cohort A | 9 | 35 | 25.7% |
| Pertuzumab 1050 mg - Cohort B | 6 | 33 | 18.2% |
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 |
|---|---|
| Pertuzumab 420 mg - Cohort A | 28 of 35 |
| Pertuzumab 1050 mg - Cohort B | 24 of 33 |
Most frequent serious adverse event terms
| Event term | Organ system | Pertuzumab 420 mg - Cohort A | Pertuzumab 1050 mg - Cohort B |
|---|---|---|---|
| Urinary retention | Renal and urinary disorders | 3 of 35 | 0 of 33 |
| Renal Failure | Renal and urinary disorders | 1 of 35 | 0 of 33 |
| Anemia | Blood and lymphatic system disorders | 1 of 35 | 0 of 33 |
| Hematolytic Uremic Syndrome | Blood and lymphatic system disorders | 1 of 35 | 0 of 33 |
| Atrial Fibrillation | Cardiac disorders | 1 of 35 | 0 of 33 |
| Oedema Peripheral | General disorders | 1 of 35 | 0 of 33 |
| Electrocardiogram wave Inversions | Investigations | 1 of 35 | 0 of 33 |
| Hypoglycemia | Metabolism and nutrition disorders | 1 of 35 | 0 of 33 |
| Cauda Equina Syndrome | Nervous system disorders | 1 of 35 | 0 of 33 |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | 1 of 35 | 0 of 33 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Pertuzumab 420 mg - Cohort A | Pertuzumab 1050 mg - Cohort B |
|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 18 of 35 | 17 of 33 |
| Fatigue | General disorders | 15 of 35 | 4 of 33 |
| Nausea | Gastrointestinal disorders | 11 of 35 | 2 of 33 |
| Anorexia | Metabolism and nutrition disorders | 8 of 35 | 2 of 33 |
| Rhinorrhoea | Respiratory, thoracic and mediastinal disorders | 3 of 35 | 4 of 33 |
| Backpain | Musculoskeletal and connective tissue disorders | 5 of 35 | 2 of 33 |
| Pain in Extremity | Musculoskeletal and connective tissue disorders | 3 of 35 | 4 of 33 |
| Rash | Skin and subcutaneous tissue disorders | 3 of 35 | 4 of 33 |
| Asthenia | General disorders | 3 of 35 | 2 of 33 |
| Dysguesia | Nervous system disorders | 4 of 35 | 1 of 33 |
Serious events with the highest arm-level rate: urinary retention (8.6%), abdominal pain (3.0%), hemastemisis (3.0%), ileus paralytic (3.0%), nausea (3.0%).
The safety record lists serious adverse events by dose cohort. For the 420-mg Cohort A, 35 participants were at risk for serious adverse events and 9 were reported as affected. For the 1050-mg Cohort B, 33 were at risk and 6 were reported as affected. The record also lists other adverse events: 28 of the 35 participants at risk in Cohort A and 24 of the 33 at risk in Cohort B were affected. These are descriptive counts; they do not establish a causal comparison between cohorts.
The listed serious adverse-event terms include urinary retention at 8.6%, and abdominal pain, hemastemisis, ileus paralytic, nausea, and central line infection, each at 3.0%. The registry excerpt does not explain the calculation basis for each listed percentage or give further event-level detail. These terms and percentages should be read as registry-listed serious-event information, not as a complete account of all safety outcomes.
Serious adverse events are not the same as all adverse events, and participant counts do not necessarily convey the number, duration, or severity of individual events. Death counts are not reported in the registry record. Since the cohorts were nonrandomized, their safety counts should not be interpreted as a reliable comparative estimate of the effects of the two doses.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this Phase II study evaluated intravenous pertuzumab in participants with hormone-refractory prostate cancer who had not received chemotherapy. It describes two nonrandomized dose cohorts and defines a primary endpoint based on PSA response, non-response, and progressive disease within the first 24 weeks. The record also lists a range of secondary outcomes and reports selected adverse-event counts.
It does not report the primary endpoint results. In particular, it gives no percentage of participants in any primary PSA category, no confidence interval, and no analysis result. The registry record therefore does not permit a numerical assessment of the primary outcome or its precision. The endpoint criteria state how responses were to be classified; they do not reveal how many participants met those criteria.
The study also does not provide a randomized control comparison. Its two cohorts received different pertuzumab doses, and the higher-dose cohort was described as based on observations in the lower-dose cohort. Any raw difference between their outcomes would need cautious interpretation and could not, by itself, demonstrate a dose effect. The record does not report adjusted comparative analyses.
The reported serious adverse-event counts are descriptive and do not establish that an event was caused by pertuzumab. The registry does not report death counts, a full analysis of adverse events, or the reason the study was terminated. Those gaps limit interpretation of safety and study conduct.
Overall, the available record supports a description of the trial question, endpoint definitions, design, and selected safety information. It does not support a conclusion about the magnitude, precision, or comparative nature of an efficacy result. The absence of posted results is not itself evidence for or against an effect.
13. Why This Trial Matters Statistically
This trial illustrates the difference between a clearly defined outcome and a reported outcome result. The primary endpoint gives detailed rules for classifying PSA response, non-response, and progressive disease. Those rules improve clarity about what was intended to be measured, but without category counts or estimates they cannot answer how participants fared.
It also shows why design features matter when interpreting dose cohorts. The study was nonrandomized, and the higher-dose cohort followed observations in the lower-dose cohort. These features can be important to the conduct of a Phase II study, but they make a direct cohort contrast vulnerable to differences other than dose. Descriptive comparisons should not be mistaken for randomized evidence.
The registry lists several distinct secondary outcomes, spanning PSA, RECIST response, progression, pain, and survival. This range highlights that clinical outcomes can capture different dimensions of disease. Their interpretation depends on their definitions, analysis populations, follow-up, and reporting. The registry record does not provide results for these outcomes or explain how their multiplicity was handled.
Finally, the record includes selected safety counts but lacks the reason for termination and death counts. Such gaps reinforce a core statistical principle: interpretation depends not only on the design and definitions but also on complete reporting of estimates, uncertainty, denominators, and analysis decisions. In this case, the posted information is informative about what the study set out to assess, but not about the numerical primary efficacy result.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most direct limitation is that the registry record does not report the primary endpoint results. Counts, percentages, uncertainty intervals, and analysis results for the PSA categories are absent. The record also lists no analyses, so results for the secondary outcomes cannot be assessed from this information.
The nonrandomized design and absence of a control group restrict causal interpretation. The two cohorts differed in dose and were not described as randomized; the higher-dose cohort was based on observations in the lower-dose cohort. The record does not report cohort-specific primary outcome estimates or adjustment methods that might address differences between participants.
Although the primary endpoint names the ITT population, the registry does not report the number analyzed or how missing PSA values were handled. It does not give details on follow-up completeness, exclusions, protocol deviations, or sensitivity analyses. These omissions make it difficult to assess whether the analysis population and observed outcomes correspond to the intended study population.
The cohort descriptions are incomplete, and the registry record does not report the full rules for further enrollment after response evaluation. The reason for the terminated status, any interim analyses, and the statistical plan are also not reported in the registry record. No explanation of the listed serious adverse-event percentage basis or death count is included. These limitations apply to interpretation of the registry record; they do not establish what may have been analyzed or reported elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of Pertuzumab in Participants With Prostate Cancer — NCT02480010.
- Registry record last updated 2015-09-18; status last verified 2015-08.
- Results first posted 2015-09-18.
- 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
The registry record describes NCT02480010, a Phase II, open-label, multicenter, nonrandomized study of intravenous pertuzumab in participants with hormone-refractory prostate cancer who had not received chemotherapy. It lists enrollment of 68 and a terminated status. Cohort A received an 840-mg loading dose followed by 420 mg on Day 1 of each 3-week cycle; Cohort B received 1050 mg on Day 1 of each 3-week cycle.
The primary endpoint classified participants by confirmed PSA response, non-response, or progressive disease within the first 24 weeks. Its definitions are reported, but its results are not. The registry lists 20 outcome measures and no analyses. It reports selected serious and other adverse-event counts by cohort; death counts and the reason for termination are not reported in the registry record. Because the cohorts were nonrandomized and there was no control group, they do not provide a like-for-like treatment comparison.