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 Treating Patients With Prostate Cancer That Did Not Respond to Hormone Therapy (NCT00460031), a na phase 2 study. The primary outcome is Number of Patients With a Partial Response, Progressive Disease, or Stable Disease Based on Prostate-Specific Antigen (PSA) or Measurable Disease.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00460031 |
| Phase | Phase 2 |
| Condition | Prostate Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 34 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Case Comprehensive Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned patients with prostate cancer that had progressed after androgen deprivation, described in the public title as cancer that did not respond to hormone therapy. The registry lists prostate cancer as the condition and identifies the primary outcome population as all patients who completed the study. Enrollment was 34, but the number who completed the study is not reported in the registry record. These details matter because the enrolled group and the outcome population are not necessarily the same; the record does not say how many enrolled participants contributed to the primary endpoint.
Intervention
The study evaluated ketoconazole and lenalidomide with therapeutic hydrocortisone. Its arm is named Ketoconazole Plus Lenalidomide, while the intervention list and study summary also include hydrocortisone. The rationale in the summary describes ketoconazole as intended to stop adrenal androgen production and lenalidomide as intended to inhibit tumor blood flow. Those statements explain the rationale for studying the regimen; they do not establish what happened to participants in the trial.
Comparator
The registry describes a single-group study with no masking and allocation recorded as NA. It does not identify a concurrent control group or a comparison treatment. The trial therefore does not provide a like-for-like randomized treatment comparison. Its outcome information, if reported, would describe participants in the study group rather than estimate a contrast between that regimen and a control.
Primary question
The primary outcome is the number of participants with partial response, progressive disease, or stable disease based on prostate-specific antigen or measurable disease, with a stated timeframe of 28 days. The outcome description also says patients would be evaluated for clinical benefit monthly with PSA values and that response for measurable disease uses RECIST criteria. The registry record does not report the observed count or provide an analysis. The clinical question can therefore be stated, but the primary result cannot be determined from this record.
3. Trial Design
NCT00460031 is a completed phase II study sponsored by Case Comprehensive Cancer Center. The registry lists a start date of 2006-09-01 and a primary completion date of 2010-08-31. Enrollment was 34. The study is classified as single-group, with allocation listed as NA and masking listed as none. These design features mean there is no randomized, masked comparison group described in the registry record.
The study group is named Ketoconazole Plus Lenalidomide. The intervention list also includes therapeutic hydrocortisone, and the brief summary describes giving ketoconazole and hydrocortisone together with lenalidomide. The registry does not give further detail about dose, schedule, treatment duration, adherence, or changes to treatment over time. Those details are not reported in the registry record.
The primary outcome is a participant count based on partial response, progressive disease, or stable disease, determined using PSA or measurable disease. Its stated timeframe is 28 days. The outcome description says clinical benefit would be evaluated monthly with PSA values, that patients benefiting from treatment could receive additional cycles, and that therapy would continue until criteria for progressive disease were met. The description does not report how these statements were operationalized for the posted primary result.
Secondary outcomes include time to progression, grade 3 and 4 toxicity assessed by NCI CTCAE v3.0, change in immune response from baseline, and the ratio of change in immune response from baseline. The registry lists five outcome measures overall, but no outcome analyses are listed. It does not report the number completing the study or give the primary outcome count. The study status is completed; that status alone does not establish that results were posted for each outcome.
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 | Ketoconazole Plus Lenalidomide |
|---|---|
| STARTED | 34 |
| COMPLETED | 23 |
| NOT COMPLETED | 11 |
Reasons for not completing this period:
| Reason | Ketoconazole Plus Lenalidomide |
|---|---|
| Adverse Event | 5 |
| Withdrawal by Subject | 5 |
| Death | 1 |
Analysis population for the primary outcome, as defined in the registry: All patients who completed the study
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 | Ketoconazole Plus Lenalidomide |
|---|---|---|
| Participants analysed | 34 | |
| Age, Customized: 40-49 years | participants | 2 |
| Age, Customized: 50-59 years | participants | 5 |
| Age, Customized: 60-69 years | participants | 10 |
| Age, Customized: 70-79 years | participants | 11 |
| Age, Customized: 80-89 years | participants | 6 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 34 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 33 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| 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 | 3 |
| Race (NIH/OMB): White | Participants | 30 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 1 |
| Region of Enrollment: United States | participants | 34 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With a Partial Response, Progressive Disease, or Stable Disease Based on Prostate-Specific Antigen (PSA) or Measurable Disease, measured over 28 days.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is a count of participants classified as having partial response, progressive disease, or stable disease based on PSA or measurable disease. The record gives a timeframe of 28 days. It also describes monthly PSA evaluation for clinical benefit, so the timeframe and the described follow-up schedule are not fully elaborated in the record. The registry does not report the endpoint count, the number evaluated, or the results by response category.
For measurable disease, the record cites RECIST criteria. It defines partial response as a decrease of at least 30% in the sum of the longest diameters of target lesions from baseline, progressive disease as an increase in that sum from baseline, and stable disease as neither sufficient for partial response nor sufficient for progressive disease. The description does not give the corresponding PSA thresholds or explain how PSA and measurable-disease classifications were combined when both were available.
Response categories can be used to describe tumor status under the stated assessment criteria. However, this endpoint groups together categories that represent different disease states, including progressive disease. The resulting count, without category-specific results or additional definitions, would not by itself reveal how many participants had each type of response or whether the count represents a favorable clinical outcome. The registry record does not provide enough outcome information to interpret the observed result, because no observed result is reported.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record establishes the design and intended outcome but does not report the primary endpoint result. It therefore does not support a numerical description of the observed response categories, a confidence interval around a response estimate, or a statistical test of the primary outcome. The enrollment of 34 is not a substitute for the number evaluated: the primary population is described as all patients who completed the study, and the number completing is not reported in the registry record.
The study is single-group and has no identified concurrent comparator. Even if an outcome count were available, it would describe the observed experience in the study group. Without a control group, the record would not establish how that experience compares with another treatment or with the course of disease in a comparable untreated group. This design limitation is separate from the absence of a posted primary result.
The endpoint description includes partial response, progressive disease, and stable disease. Those classifications are not interchangeable. A combined count across them would not indicate how many participants fell into each category, and the registry record gives no category-specific counts. For measurable disease, the record supplies RECIST definitions; for PSA-based classification, it does not state thresholds. It also does not explain how results based on PSA and measurable disease were reconciled.
General statistical interpretation: a confidence interval describes the uncertainty around an estimated quantity under the assumptions of the analysis. It is useful for assessing precision, not just whether a result crosses a significance threshold. No interval or estimated primary outcome is reported in the registry record, so precision cannot be assessed for this endpoint. A p-value would address compatibility with a specified statistical model or null hypothesis; the record gives no primary analysis or p-value, and no such test should be inferred.
The posted safety information lists serious adverse events for the study group, including 12 affected among 34 at risk in the serious-event category. These figures describe the registry’s safety reporting, not the primary efficacy endpoint. The registry also lists 34 affected among 34 at risk in the other adverse-event category. The meaning of each category and the reporting rules are not further detailed in the record, so these counts should not be combined or treated as a complete account of all treatment-related harms.
The overall interpretation is limited by missing outcome results, missing analysis details, and the single-group design. The record identifies what the trial intended to study, but it does not establish the size, precision, or comparative meaning of an observed primary effect.
9. Statistical Methods Explained
The registry classifies the primary outcome as a participant count. In general, a count outcome records how many people meet a defined event or response criterion. Interpretation depends on a clear definition of who is eligible for assessment, how the event is determined, and what happens when an observation is missing. In this trial, the stated primary population is all patients who completed the study, while enrollment was 34. The number completing and the number with an evaluable primary assessment are not reported in the registry record.
What does the response classification mean?
The outcome description combines partial response, progressive disease, and stable disease, based on PSA or measurable disease. For measurable disease, RECIST definitions are stated: partial response requires a decrease of at least 30% in the sum of longest target-lesion diameters from baseline; progressive disease is defined by an increase in that sum; stable disease meets neither definition. The record does not provide PSA thresholds or specify how the two assessment sources were combined. In general, an endpoint that groups distinct categories can conceal differences in the underlying category distribution. Without separate counts, it is not possible to tell how much of a combined count comes from each classification.
What can a single-group result show?
In general, a single-group study can describe outcomes among participants who received the study regimen. A concurrent comparator helps distinguish the treatment-group experience from background changes, differences in participant characteristics, and other influences. This registry record describes a single-group study, with no masking and allocation listed as NA. It does not identify a like-for-like control. Thus, even a reported outcome would not by itself provide a randomized estimate of the difference between treatments.
How should statistical precision be understood?
In general, an estimate is more informative when its uncertainty is reported alongside it. A confidence interval gives a range of values compatible with the observed data and the chosen statistical method; its width reflects precision under that method. A p-value is a measure tied to a specified hypothesis and model, not a measure of clinical importance. The registry record reports no primary estimate, confidence interval, p-value, or analysis method. None can be reconstructed from the enrollment count alone, and no primary statistical conclusion can be drawn from the record.
What do the planning assumptions represent?
The record describes sample-size planning illustrations using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning assumptions, not a description of the actual single-group trial design. The record does not report a trial-specific sample-size justification or the values needed to interpret a particular illustration. Such calculations are conditional on their assumptions and should not be read as observed power or as evidence that a study had a given probability of detecting an effect. A planning illustration is not a replacement for an observed outcome and its uncertainty.
What does over-dispersion mean in general?
General statistical explanation: for count outcomes, some models assume that variability follows a particular relationship to the expected count. Over-dispersion means the data vary more than that model expects. If it is ignored, standard errors can be too small and confidence intervals or tests can be misleading. Whether over-dispersion is relevant depends on the outcome and the analysis model. The registry record does not identify a count model or report a diagnostic assessment, so no claim can be made about over-dispersion in this trial.
What analysis information is absent?
The registry lists five outcome measures and zero analyses. It does not report the primary statistical method, an adjustment strategy, a confidence interval, or a p-value. It also does not state whether there were interim analyses, how repeated assessments were handled, or whether any covariates were used. Those elements are not reported in the registry record and should not be inferred from the phase or study design. The posted information supports a description of the planned endpoint and design, but not a reconstruction of the sponsor’s statistical analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists five outcome measures, including the primary response-count endpoint and secondary measures concerning time to progression, grade 3 and 4 toxicity, and immune response. It lists zero analyses. The record does not describe a multiplicity strategy, such as whether any adjustment was planned for evaluating several outcomes or comparisons. It also does not report a hierarchy of hypotheses or identify which outcomes were intended to support confirmatory conclusions. In general, testing or interpreting multiple outcomes can increase the chance of a chance finding; the appropriate approach depends on the trial’s aims and analysis plan. That plan is not reported in the registry record.
The primary analysis population is described as all patients who completed the study. Enrollment was 34, but the number completing is not reported. The record does not give a participant flow, the number assessed for the primary endpoint, reasons for non-completion, or the handling of missing outcome data. It also does not specify whether the outcome analysis used all enrolled participants, only evaluable participants, or another population. These omissions prevent assessment of how missing observations may have affected the endpoint.
General statistical explanation: missing-data methods make assumptions about why observations are absent and how those absences relate to outcomes. Complete-case results may be affected if participants with missing assessments differ from those with observed assessments. Sensitivity analyses can examine how conclusions change under alternative assumptions. The registry record does not report missing-data assumptions or sensitivity analyses. No missing-data method should be attributed to the trial based on the information available.
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 |
|---|---|---|---|
| Ketoconazole Plus Lenalidomide | 12 | 34 | 35.3% |
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 |
|---|---|
| Ketoconazole Plus Lenalidomide | 34 of 34 |
Most frequent serious adverse event terms
| Event term | Organ system | Ketoconazole Plus Lenalidomide |
|---|---|---|
| Vomiting | Gastrointestinal disorders | 3 of 34 |
| Hemoglobin | Blood and lymphatic system disorders | 2 of 34 |
| Cardiovascular/General | Cardiac disorders | 2 of 34 |
| Diarrhea patients without colostomy | Gastrointestinal disorders | 2 of 34 |
| Dizziness/lightheadedness | Nervous system disorders | 2 of 34 |
| Blood/Bone Marrow | Blood and lymphatic system disorders | 1 of 34 |
| Cardiovascular/Arrhythmia | Cardiac disorders | 1 of 34 |
| Cardiac-ischemia/infarction | Cardiac disorders | 1 of 34 |
| Edema | General disorders | 1 of 34 |
| Coagulation | Blood and lymphatic system disorders | 1 of 34 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ketoconazole Plus Lenalidomide |
|---|---|---|
| "Fatigue (lethargy, malaise, asthenia)" | General disorders | 27 of 34 |
| Hemoglobin | Investigations | 18 of 34 |
| Lymphopenia | Investigations | 17 of 34 |
| Rash/desquamation | Skin and subcutaneous tissue disorders | 16 of 34 |
| Nausea | Gastrointestinal disorders | 14 of 34 |
| Hypocalcemia | Metabolism and nutrition disorders | 14 of 34 |
| Platelets | Blood and lymphatic system disorders | 13 of 34 |
| Edema | General disorders | 13 of 34 |
| Alkaline phosphatase | Investigations | 13 of 34 |
| SGPT (ALT) (serum glutamic pyruvic transaminase) | Investigations | 13 of 34 |
Serious events with the highest arm-level rate: vomiting (8.8%), hemoglobin (5.9%), cardiovascular/general (5.9%), diarrhea patients without colostomy (5.9%), dizziness/lightheadedness (5.9%).
The registry reports 12 participants affected among 34 at risk in the serious adverse-event category for Ketoconazole Plus Lenalidomide. The listed serious-event terms include Vomiting at 8.8%, Hemoglobin at 5.9%, Cardiovascular/General at 5.9%, Diarrhea patients without colostomy at 5.9%, Dizziness/lightheadedness at 5.9%, and Blood/Bone Marrow at 2.9%. These are registry-listed terms and percentages; the record does not provide term-specific participant counts or additional detail about how the terms were defined.
The other adverse-event category lists 34 affected among 34 at risk. The registry does not explain the category’s precise scope in the information available, so it should not be interpreted as a count of treatment-caused events or as a measure of severity. The reported serious and other-event counts should be read as separate categories, not added together. Doing so could count the same participant more than once, and the registry does not report distinct participant totals across categories.
The death count is not reported in the registry record. The listed serious-event figures do not establish causation, comparative risk, or a complete safety profile. No control group is identified for comparison.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed phase II, single-group study of ketoconazole, lenalidomide and therapeutic hydrocortisone in prostate cancer progressing after androgen deprivation. It defines a primary outcome based on PSA or measurable disease and describes RECIST categories for measurable disease. It also lists safety outcomes and secondary outcomes concerning time to progression and immune response. These details indicate what the study set out to assess.
The record does not report the primary outcome count, a category breakdown, or a statistical analysis. It therefore does not establish how many participants had partial response, progressive disease, or stable disease, and it does not show the precision of an estimated outcome. The stated enrollment of 34 cannot fill that gap: the primary outcome population is all patients who completed the study, and the number completing is not reported.
The study design also limits what could be concluded from a group result. With a single group and no identified concurrent control, outcomes could not be directly contrasted with a randomized comparison group. A within-group observation, if reported, would not by itself distinguish the regimen’s effect from other influences on disease course or assessment.
The safety record lists adverse-event counts and serious-event terms, but it does not report a comparison group, causal attribution, or all details needed to characterize risk. These entries are useful as descriptions of registry reporting, not as proof that an event was or was not caused by treatment. The death count is not reported in the registry record.
In short, the record supports a description of the trial’s design, intervention and intended measurements. It does not support a conclusion about the primary result, its statistical precision, or a comparative treatment effect. Those limits arise from both the single-group design and the absence of posted outcome analyses.
13. Why This Trial Matters Statistically
This trial illustrates why outcome definitions and outcome reporting are both essential to statistical interpretation. The primary endpoint is described as a count across partial response, progressive disease, and stable disease, assessed using PSA or measurable disease. The measurable-disease definitions are given, but the record does not report PSA thresholds or how the assessment sources were combined. Without category-specific results, a combined count would not show the distribution across distinct disease states.
The registry also illustrates the difference between enrollment and an analysis population. Enrollment was 34, while the primary outcome population is described as all patients who completed the study. Because the completion count and number evaluated are not reported, the reader cannot establish the denominator for the primary outcome or assess the role of missing assessments.
Its single-group design is another important statistical feature. A study can describe the outcomes observed among participants, but a concurrent comparator is needed for a direct treatment contrast. The registry gives no such comparison. It is therefore important not to interpret a group-level result, even if one were available, as a randomized estimate of comparative effect.
Finally, the registry’s design and endpoint details do not substitute for an analysis plan or results. It lists five outcome measures and zero analyses, with no reported primary estimate or uncertainty interval. The distinction between a planned measurement, a completed study, and a posted statistical result is central: the first two do not establish the third.
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 immediate limitation is that the primary outcome result is not reported in the registry record. There is no observed count, response-category breakdown, analysis estimate, confidence interval, or p-value. As a result, statistical precision and the direction or magnitude of the observed outcome cannot be assessed.
The outcome definition leaves important operational details unspecified. The record gives RECIST criteria for measurable disease but does not provide PSA thresholds or explain how PSA-based and measurable-disease classifications were combined. The primary timeframe is listed as 28 days, while the description refers to monthly PSA evaluations and additional cycles for patients benefiting from treatment. The relationship between those details is not explained.
The primary population is all patients who completed the study, but the number completing, number assessed, and reasons for missing outcomes are not reported. Enrollment was 34, which does not establish the primary endpoint denominator. The registry also does not report the statistical method, handling of repeated assessments, or missing-data approach.
The study is single-group, with no masking and no identified concurrent comparator. Any group outcome would therefore lack a direct randomized comparison. The record does not report dose, schedule, adherence, or treatment changes, which further limits interpretation of the intervention as delivered. Safety reporting is also incomplete for interpretation: the death count is not reported, and the record does not provide details on event attribution or the precise scope of the other adverse-event category.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treating Patients With Prostate Cancer That Did Not Respond to Hormone Therapy — NCT00460031.
- Registry record last updated 2020-07-24; status last verified 2020-07.
- Results first posted 2015-12-09.
- 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
NCT00460031 is a completed phase II, single-group study sponsored by Case Comprehensive Cancer Center. It enrolled 34 participants and studied ketoconazole and lenalidomide with therapeutic hydrocortisone in prostate cancer progressing after androgen deprivation. The study had no masking and no identified concurrent comparator.
The primary outcome was a count of participants with partial response, progressive disease, or stable disease based on PSA or measurable disease, with a stated timeframe of 28 days. The registry record does not report the primary outcome result or a statistical analysis. It lists five outcome measures and zero analyses. The primary population is described as all patients who completed the study, but the number completing is not reported. Safety information includes 12 affected among 34 at risk for serious adverse events; the death count is not reported.