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 Docetaxel, Prednisone, and Pasireotide in Treating Patients With Metastatic Hormone-Resistant Prostate Cancer (NCT01468532), a na phase 1, phase 2 study. The primary outcome is Maximum Tolerated Dose (MTD) of Pasireotide in Combination With Docetaxel and Prednisone by the Occurrence of Adverse Events and the Associated Grade Per NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01468532 |
| Phase | Phase 1, Phase 2 |
| Condition | Adenocarcinoma of the Prostate, Hormone-resistant Prostate Cancer, Recurrent Prostate Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 18 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Barbara Ann Karmanos Cancer Institute |
| Results posted | Yes |
2. Clinical Question
Population
The trial enrolled patients with metastatic castrate-resistant prostate cancer, also described in the registry record as metastatic hormone-resistant prostate cancer. The listed conditions include adenocarcinoma of the prostate, recurrent prostate cancer, and stage IV prostate cancer. Further details about participant eligibility and baseline characteristics are not reported in the registry record.
Intervention
The treatment combined pasireotide with docetaxel and prednisone. The described regimen gives pasireotide by intramuscular injection at 40 mg on day 1, docetaxel intravenously at 75mg/m2 over 1 hour, and prednisone orally at 5mg twice daily continuously. Docetaxel courses repeat every 21 days and pasireotide courses every 28 days, in the absence of disease progression or unacceptable toxicity.
Comparator
There was no comparator group. The trial is recorded as single-group, with no masking and no randomized allocation. Its findings therefore describe the participants who received the combination; they do not estimate a contrast against another treatment or a control group.
Primary question
The primary question was what maximum tolerated dose of pasireotide could be given with docetaxel and prednisone, based on adverse events and their grades under NCI CTCAE Version 4.0. The stated assessment timeframe was up to day 57. The registry record also lists secondary questions concerning toxicity, tumor measurements, PSA change, time to progression, overall survival, pharmacokinetics, biomarkers, and circulating tumor cell counts. A numerical MTD result is not reported in the registry record.
3. Trial Design
This was a phase I/II, single-group trial sponsored by Barbara Ann Karmanos Cancer Institute. The registry record lists an enrollment of 18 and a completed status. The recorded start was 2011-10, and primary completion was 2017-07-20. The design has no randomized allocation, no masking, and no like-for-like control arm. It is therefore a study of a treatment combination rather than a comparative trial.
Participants in the treatment group received pasireotide, docetaxel, and prednisone. The regimen describes pasireotide 40 mg intramuscularly on day 1, docetaxel 75mg/m2 intravenously over 1 hour, and prednisone 5mg orally twice daily continuously. Docetaxel courses were to repeat every 21 days, while pasireotide courses were to repeat every 28 days, provided there was no disease progression or unacceptable toxicity.
The primary outcome was the maximum tolerated dose of pasireotide with the two other drugs, assessed through adverse events and their grades under NCI CTCAE Version 4.0 up to day 57. Listed secondary outcomes included toxicity, tumor measurements using RECIST, PSA change, time to progression, overall survival, pharmacokinetics, changes in IGF-1, serum chromogranin A and neuron-specific enolase, and changes in circulating tumor cell counts.
The registry record does not report the dose-escalation rules, cohort structure, sample-size justification, or a detailed statistical analysis plan. It lists no analyses. These omissions limit what can be reconstructed about how the trial was conducted and how its outcomes were intended to be analyzed.
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 | Treatment (Chemotherapy, Receptor Agonist) |
|---|---|
| STARTED | 18 |
| COMPLETED | 18 |
| NOT COMPLETED | 0 |
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 | Treatment (Chemotherapy, Receptor Agonist) |
|---|---|---|
| Participants analysed | 18 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 14 |
| Age, Continuous | years | 65 (49 to 75) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 18 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 6 |
| Race (NIH/OMB): White | Participants | 11 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 18 |
6. Primary Endpoint
The registry lists the primary outcome as Maximum Tolerated Dose (MTD) of Pasireotide in Combination With Docetaxel and Prednisone by the Occurrence of Adverse Events and the Associated Grade Per NCI Common Terminology Criteria for Adverse Events (CTCAE) Version 4.0, measured over Up to day 57.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | mg |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the maximum tolerated dose of pasireotide when combined with docetaxel and prednisone. It is defined in terms of adverse events and the associated grade under NCI Common Terminology Criteria for Adverse Events Version 4.0, with assessment up to day 57. The endpoint is expressed in mg, but the registry record does not report the selected dose or a numerical endpoint result.
In general, a maximum tolerated dose is a safety-focused dose-selection measure: it seeks to identify a dose level that can be given without an unacceptable pattern of toxicity under the study’s rules. The registry record does not state those dose-escalation or stopping rules, so the precise operational definition used in this trial cannot be assessed. The endpoint is relevant to a phase I component because it concerns tolerability and dose selection, rather than a direct estimate of comparative treatment benefit.
The registry description says binary endpoints, such as specific toxicity types at a dose level and severity grade, would be described using point estimates and 90% confidence intervals for proportions, with Wilson’s method. In general, a confidence interval expresses uncertainty around an estimate; it is not a guarantee that the true value lies within that particular interval. Without the posted endpoint estimate and interval, the precision of the MTD assessment cannot be judged from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The trial’s design supports descriptive interpretation of outcomes among participants who received the combination. Because it was a single-group study without randomized allocation or a control arm, its results cannot establish how outcomes compare with another treatment. Any changes in tumor measures, PSA, time to progression, or survival would need to be interpreted as observations in this group, not as estimates of a treatment effect relative to a comparator.
The central endpoint was a safety-based dose question: the maximum tolerated dose of pasireotide with docetaxel and prednisone, assessed through adverse events and their grades up to day 57. Although the registry describes how binary endpoints were intended to be summarized, it does not report a numerical MTD result. The dose selected, the dose-specific toxicity estimates, and their uncertainty are therefore not available in the registry record.
The record lists secondary outcomes spanning toxicity, tumor measurements, PSA change, time to progression, overall survival, pharmacokinetics, biomarkers, and circulating tumor cells. It does not provide numerical results for these outcomes in the registry record presented for this analysis. Their inclusion as planned or listed outcomes should not be mistaken for evidence that a particular result was observed, or that a treatment effect was established. The registry record lists no analyses, which further limits statistical interpretation.
Safety summaries report 14 of 18 participants with serious adverse events, 18 of 18 with other adverse events, and 13 deaths. These counts describe reported events in the registry record; they do not, on their own, establish whether an event was caused by treatment. The registry record does not provide event timing, exposure-adjusted rates, or individual participant histories in the information available for interpretation. The listed serious-event terms and percentages also do not replace a complete account of event severity, duration, and attribution.
Any planning illustrations based on an observed effect are not observed power and are not the trial’s original design justification. In particular, a hypothetical equal-allocation, two-arm calculation does not match this trial’s single-group design. The illustrations can show how assumed effect size and design choices affect planning, but they do not change what the trial observed or provide a valid treatment comparison.
9. Statistical Methods Explained
The registry description gives a limited account of the intended statistical methods. It states that occurrence rates for binary endpoints would be described with point estimates and 90% confidence intervals for proportions using Wilson’s method. The primary outcome is a dose in mg, based on adverse events and their grades up to day 57. The record does not report the numerical endpoint result, the dose-specific event counts, or a full analysis plan.
What does a point estimate convey?
A point estimate is a single summary calculated from observed data. For a binary outcome, the relevant data distinguish participants or observations that meet a defined event criterion from those that do not. A point estimate can make the observed frequency easier to interpret, but it does not show how much uncertainty remains. That uncertainty depends on the amount and pattern of available data as well as on the statistical method. The registry record does not provide the dose-specific data needed to assess such estimates for the primary endpoint.
Why report a confidence interval?
In general, a confidence interval gives a range of values compatible with the data under a specified statistical procedure. A wider interval indicates less precision than a narrower one, all else being equal. The registry description specifies 90% confidence intervals for proportions and identifies Wilson’s method. It does not give the resulting intervals. Accordingly, readers cannot use the record to judge the precision of toxicity estimates at particular dose levels or to determine how much the estimates vary across doses.
A confidence interval is not the probability that a fixed parameter lies inside a particular calculated interval. Rather, the stated confidence level describes the long-run behavior of the interval procedure under its assumptions. In general, the Wilson method is one approach for intervals around a proportion. The method does not remove uncertainty caused by limited information, incomplete reporting, or ambiguity about how outcomes were defined.
How does this relate to the maximum tolerated dose?
The maximum tolerated dose is not simply a proportion or a single toxicity count. It depends on how toxicity findings are linked to dose levels and on the rules used to decide whether a dose is acceptable. The registry identifies adverse-event occurrence and grade under NCI CTCAE Version 4.0 as the basis for the primary endpoint. However, the dose levels, escalation scheme, dose-limiting toxicity definition, and decision rules are not reported in the registry record. Without those details and the endpoint result, the MTD decision cannot be independently reconstructed from the record.
In general, dose-finding decisions balance information about toxicity at candidate doses with the need to avoid exposing participants to doses judged unacceptable under the protocol. A dose-finding design can be rule-based or model-based, but the registry record does not identify the method used in this trial. It would therefore be inappropriate to infer a particular dose-escalation design or to describe the MTD as having been established according to any specific algorithm.
What can a single-group design estimate?
A single-group trial can describe outcomes observed among enrolled participants. It cannot by itself distinguish treatment effects from changes that might have occurred without the treatment combination, or from other differences over time. In general, randomized comparison groups help address such questions by providing a concurrent reference, though this trial did not have one. The absence of a comparator does not make descriptive counts meaningless; it limits the claims that can be supported by them.
The listed planning illustration uses a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect stated as a fraction of the observed effect. For time-to-event calculations, it describes total events; for other calculations, it describes participants per arm. These are planning assumptions, not the recorded design of this single-group trial. Any calculated sample sizes under those assumptions are planning illustrations, not observed power, and are not evidence about this trial’s efficacy or precision.
What statistical details remain unavailable?
The registry record does not report a numerical primary endpoint result, dose-level toxicity estimates, confidence intervals, or analyses. It also does not report the statistical model, handling of repeated measurements, analysis populations, or strategies for missing data. The absence of these details means that readers cannot assess model fit, sensitivity to analytic choices, or the impact of incomplete observations. General statistical methods can explain how estimates and uncertainty are interpreted, but they cannot supply results or methods that the registry does not report.
Over-dispersion is a general concern when count data vary more than a chosen statistical model expects; ignoring it can make uncertainty appear too small. That concept is not enough to identify the appropriate model for any outcome in this trial. The registry record does not report a count model or the information needed to assess over-dispersion. Likewise, the listed biomarker and circulating tumor cell outcomes involve measurements at multiple time points, but the analysis method for those repeated measurements is not reported. No model or adjustment should be presumed.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists several secondary outcomes: toxicity, RECIST tumor measurements, PSA change, time to progression, overall survival, pharmacokinetics, changes in IGF-1, serum chromogranin A and neuron-specific enolase, and changes in circulating tumor cell counts. Considering many outcomes can create multiplicity: when numerous statistical tests are performed, the chance of at least one apparently notable result can increase. In general, an analysis plan can address this by identifying primary and secondary questions, setting priorities, and specifying any adjustments or interpretive strategy. The registry record does not report a multiplicity strategy or analyses for these outcomes.
The primary endpoint is identified as the maximum tolerated dose, but the record does not report a numerical result or the methods used to select the dose. It also does not report whether interim analyses were planned or conducted. No interim analysis should be inferred from the phase I/II designation or from the fact that dose finding was a study objective.
Missing-data methods and analysis populations are not reported in the registry record. The record does not state how participants with incomplete follow-up, unavailable measurements, or treatment discontinuation were handled in analyses. This matters in general because the amount and pattern of missing information can affect the interpretation of estimates, especially for outcomes measured over time. The registry’s enrollment count alone does not reveal how many participants contributed data to each outcome.
These reporting gaps prevent assessment of whether the outcome definitions, analysis populations, multiplicity approach, and missing-data handling were prespecified and consistently applied. They also mean that the listed secondary outcomes should be treated as outcome areas, not as evidence of a statistically supported finding.
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 |
|---|---|---|---|
| Treatment (Chemotherapy, Receptor Agonist) | 14 | 18 | 77.8% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Treatment (Chemotherapy, Receptor Agonist) | 13 of 18 |
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 |
|---|---|
| Treatment (Chemotherapy, Receptor Agonist) | 18 of 18 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Chemotherapy, Receptor Agonist) |
|---|---|---|
| Anemia | Blood and lymphatic system disorders | 1 of 18 |
| Anorexia | Gastrointestinal disorders | 1 of 18 |
| Dyspnea | Respiratory, thoracic and mediastinal disorders | 1 of 18 |
| Edema limbs | Blood and lymphatic system disorders | 1 of 18 |
| Generalized muscle weakness | Musculoskeletal and connective tissue disorders | 1 of 18 |
| Hematoma | Blood and lymphatic system disorders | 1 of 18 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 18 |
| Hyponatremia | Metabolism and nutrition disorders | 1 of 18 |
| Hypotension | Vascular disorders | 1 of 18 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 1 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment (Chemotherapy, Receptor Agonist) |
|---|---|---|
| Fatigue | General disorders | 17 of 18 |
| Hyperglycemia | Metabolism and nutrition disorders | 14 of 18 |
| Diarrhea | Gastrointestinal disorders | 13 of 18 |
Serious events with the highest arm-level rate: anemia (5.6%), anorexia (5.6%), dyspnea (5.6%), edema limbs (5.6%), generalized muscle weakness (5.6%).
The registry safety summary reports serious adverse events in 14 of 18 participants and other adverse events in 18 of 18 participants. It also reports 13 deaths. These are counts of participants, not measures adjusted for length of exposure, and the registry information does not give event timing or individual participant histories. The record does not report the cause of each death or establish whether events were attributed to study treatment.
The serious adverse-event terms listed are anemia, anorexia, dyspnea, edema limbs, generalized muscle weakness, and hematoma; each is reported at 5.6%. These percentages describe the registry’s reported summary and should not be converted into additional counts or interpreted as comparative risks. Several terms can occur in the same participant, so the individual entries should not be treated as mutually exclusive categories or added together.
In general, an adverse event occurring during a trial is not by itself proof that treatment caused it. Seriousness, severity grade, timing, and attribution are distinct aspects of safety reporting. The primary endpoint refers to adverse-event grades under NCI CTCAE Version 4.0, but the safety summary does not provide the full event-level detail needed to understand those dimensions. The counts therefore describe reported safety experience without establishing causation.
12. What the Result Does, and Does Not, Mean
The registry record describes a phase I/II investigation of pasireotide with docetaxel and prednisone in metastatic castrate-resistant prostate cancer. Its central question was safety-oriented: identifying the maximum tolerated dose of pasireotide with the combination, using adverse events and their grades through day 57. The listed secondary outcomes span additional safety, tumor, PSA, survival, pharmacokinetic, biomarker, and circulating tumor cell measures.
The record does not report the numerical MTD result or numerical results for those secondary outcomes in the information available for this analysis. It therefore does not support a conclusion about the size or precision of an observed efficacy outcome. The stated treatment purpose and listed endpoints are not themselves results. The record’s completion status indicates that the trial is marked completed; it does not fill in missing outcome estimates.
The reported safety counts describe participants with serious and other adverse events and deaths. They do not establish that treatment caused those events, nor do they compare the observed counts with what might have occurred under another treatment or without treatment. Attribution, event timing, and exposure-adjusted rates are not reported in the registry record.
Because the trial had one treatment group, there is no randomized control group for estimating comparative effects. Observations in the treated group may be useful as descriptive evidence, but they cannot isolate the contribution of the combination from other influences. The record also does not report enough statistical detail to evaluate the dose-selection process, missing data, or analyses of the secondary outcomes.
Accordingly, the most defensible reading is narrow: the registry records a completed, single-group phase I/II trial with a stated dose-finding endpoint and several listed secondary outcomes, but the numerical efficacy and primary endpoint results are not reported in the record. Neither a treatment benefit nor a lack of benefit can be inferred from these omissions.
13. Why This Trial Matters Statistically
This trial illustrates why the question being asked and the design used must be read together. A phase I/II, single-group study can address a dose and safety question in the participants who receive the treatment combination. Without a comparator, it cannot provide a like-for-like estimate of how outcomes differ from another treatment or from a control condition. Descriptive evidence and comparative evidence answer different questions.
The primary endpoint also highlights the importance of reporting the full dose-finding method. Adverse-event type and grade are central to interpreting a maximum tolerated dose, but the dose levels, escalation rules, toxicity thresholds, and resulting dose decision are not reported in the registry record. Without those details, the endpoint’s operational meaning and the basis for the selected dose cannot be independently evaluated.
Several additional outcomes cover distinct domains, from tumor measurements and PSA to survival, pharmacokinetics, biomarkers, and circulating tumor cells. These outcomes may be informative for different purposes, but their listing does not make them interchangeable or establish that results were analyzed. The record lists no analyses, and it does not report multiplicity or missing-data methods. That limits assessment of how robustly any secondary finding could be interpreted.
The trial’s record is also a reminder that a completed status is not a substitute for posted numerical results. Without outcome estimates and their uncertainty, readers cannot judge precision or weigh the findings. Clear reporting of endpoint definitions, analysis methods, and results is essential for distinguishing what a trial set out to investigate from what its data ultimately showed.
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 key limitation is the absence of a reported numerical result for the primary endpoint. The registry describes the MTD question and its assessment timeframe, but does not report the selected dose, dose-specific event patterns, or confidence intervals. As a result, the primary dose-finding conclusion cannot be evaluated from the registry record.
The single-group design has no randomized comparator, so the study cannot estimate a comparative treatment effect. This limits interpretation of outcomes such as tumor measurements, PSA change, time to progression, and overall survival. The registry record lists these outcomes but does not report their numerical results in the information.
The record also omits details needed to assess how the primary endpoint was operationalized: dose levels, dose escalation and stopping rules, and the definition of dose-limiting toxicity are not reported. Although the description names NCI CTCAE Version 4.0 and states a timeframe up to day 57, it does not provide the dose-finding procedure or the endpoint estimate.
For safety, participant counts are reported, but the available summary does not give event timing, treatment attribution, duration, exposure-adjusted rates, or event-level overlap. The deaths are not accompanied by causes. The record does not report analysis populations, missing-data handling, multiplicity methods, or detailed statistical analyses. These limitations prevent a fuller assessment of precision, robustness, and the relationship between observed events and treatment.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Docetaxel, Prednisone, and Pasireotide in Treating Patients With Metastatic Hormone-Resistant Prostate Cancer — NCT01468532.
- Registry record last updated 2021-03-25; status last verified 2021-03.
- Results first posted 2021-03-25.
- 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 a completed phase I/II, single-group trial of pasireotide with docetaxel and prednisone for metastatic castrate-resistant prostate cancer. Enrollment was 18. The primary endpoint was the maximum tolerated dose of pasireotide based on adverse events and grades under NCI CTCAE Version 4.0, assessed up to day 57.
The record lists secondary outcomes covering toxicity, tumor measurements, PSA change, time to progression, overall survival, pharmacokinetics, biomarkers, and circulating tumor cell counts. It reports serious adverse events in 14 of 18 participants, other adverse events in 18 of 18, and 13 deaths. Numerical results for the primary endpoint and the listed secondary outcomes are not reported in the registry record. The design has no comparator group, and the record lists no analyses.