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 Oral Calcitriol With Ketoconazole in CRPC (NCT03261336), a na phase 2 study. The primary outcome is PSA Response Rate.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03261336 |
| Phase | Phase 2 |
| Condition | Castration-resistant Prostate Cancer |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Donald Trump, MD |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned men with castration-resistant prostate cancer whose disease had progressed despite primary androgen-deprivation therapy and abiraterone. The registry describes only one patient as enrolled; that patient was discontinued early due to an adverse event. The record does not report further details about the enrolled patient's characteristics or whether the planned endpoint could be assessed.
Intervention
The experimental regimen combined oral calcitriol, ketoconazole, and hydrocortisone. The arm description specifies calcitriol 10mcg QD X3 weekly, ketoconazole 400mg QD, and hydrocortisone 20mg AM, 10 mg PM. The study's stated aim describes estimating the PSA response rate with ketoconazole and hydrocortisone among men whose disease progressed despite abiraterone.
Comparator
There was no concurrent comparison group. The trial is identified as single-group, with allocation listed as NA and masking as NONE. Consequently, the design does not estimate a contrast between the regimen and a control treatment or usual care.
Primary question
The primary endpoint was PSA response rate, with a timeframe of 2 years and PSA assessment every 4 weeks. The intended question was whether PSA responses could be observed during treatment in this clinical setting. The registry does not report a response estimate, a definition of response, or an analysis of the endpoint. With one enrollee who was discontinued early due to an adverse event, the posted record does not establish an interpretable estimate of response in the intended population.
3. Trial Design
This was a phase II, single-group study of a drug combination for castration-resistant prostate cancer. Its official title describes oral calcitriol in combination with ketoconazole in disease progressing despite primary androgen-deprivation therapy and abiraterone. The intervention arm included calcitriol, ketoconazole, and hydrocortisone. The registry describes the regimen as calcitriol 10mcg QD X3 weekly, ketoconazole 400mg QD, and hydrocortisone 20mg AM, 10 mg PM.
The record lists allocation as NA and masking as NONE. A single-group design has no concurrent control arm, so it can describe outcomes among enrolled participants but cannot directly compare outcomes under the regimen with outcomes under another treatment. The study's stated aim was to estimate PSA response rate among men whose disease had progressed despite abiraterone. The primary endpoint was PSA Response Rate, assessed every 4 weeks over a timeframe of 2 years. Tumor Response and Toxicity and Tolerability of Experimental Arm were listed as secondary outcomes.
The registry gives an enrollment of 1 and a status of TERMINATED. It records a start date of 2017-01-06 and a primary completion date of 2018-02-28. The primary outcome description states that only one patient enrolled and was discontinued early due to AE. The record does not report why the study was terminated, a target enrollment, or a recruitment history beyond the enrollment figure.
Details about randomization are not applicable to the listed single-group design. The protocol's eligibility criteria, planned treatment duration, adherence procedures, and monitoring schedule beyond PSA assessment are not reported in the registry record. The sponsor is listed as Donald Trump, MD. No registered analysis records are listed, and no endpoint estimate or inferential result is reported. Accordingly, the design and posted data do not support a comparative treatment-effect conclusion.
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 | Calcitriol, Ketoconazole, Hydrocortisone |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Calcitriol, Ketoconazole, Hydrocortisone |
|---|---|
| Adverse Event | 1 |
Analysis population for the primary outcome, as defined in the registry: Only one patient enrolled to study and was discontinued early due to AE.
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 | Calcitriol, Ketoconazole, Hydrocortisone |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 1 |
| Age, Continuous | years | 78 (78 to 78) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | 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 | 0 |
| Race (NIH/OMB): White | Participants | 1 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as PSA Response Rate, measured over 2 years.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was PSA response rate. A response-rate endpoint is generally a proportion: it counts participants who satisfy a prespecified response definition and relates that count to a defined analysis population. PSA measurements are used in prostate cancer studies to track a disease marker over time; changes that meet a response criterion can serve as an indicator of biological activity. The registry says PSA was assessed every 4 weeks and gives a timeframe of 2 years.
The endpoint's interpretation depends on details that are not reported in the registry record, including the PSA response threshold, confirmation requirements, baseline reference, and rules for handling treatment discontinuation or unavailable measurements. The record does not provide a response count, a response proportion, or a confidence interval. Its primary outcome description notes that only one patient enrolled and was discontinued early due to AE, but does not state whether that patient's PSA response could be determined.
In general, a response rate is meaningful only when the response definition, follow-up period, and denominator are clear. Repeated PSA assessment can characterize whether measurements meet a criterion during follow-up, but the schedule alone does not establish that a response occurred or how it should be classified. With no endpoint analysis reported, the primary outcome cannot be quantified from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted information is principally a description of the intended study and its limited enrollment, rather than a report of an analyzed efficacy outcome. The trial is listed as phase II and single-group, and the registry gives enrollment as 1. It also states that the sole enrolled patient was discontinued early due to an adverse event. No primary endpoint estimate, confidence interval, test result, or analysis record is reported.
The primary question concerned PSA response rate in men with castration-resistant prostate cancer progressing despite abiraterone. The record specifies PSA assessment every 4 weeks and a 2 years timeframe, but it does not give the response definition or report whether a response was observed. Therefore, the endpoint's value and its precision cannot be assessed from the posted record. The absence of a reported estimate is not evidence that a response did or did not occur.
The single-group design also limits interpretation. Without a concurrent comparator, an outcome observed during treatment could not by itself distinguish the regimen's effect from the natural course of disease, other care, or differences in participant characteristics. In this instance, the very limited enrollment and early discontinuation add further uncertainty about what can be learned. These registry entries do not permit an estimate of treatment benefit or a reliable characterization of outcomes for the intended population.
The safety record reports 1 participant at risk for serious adverse events and 1 affected, with hypercalcemia listed as a serious event term at 100.0%. The registry also reports 1 participant at risk for other adverse events and 0 affected, and deaths of 0. These counts describe the posted safety record, but the number of participants is too limited to characterize the range or frequency of harms in a broader population. The serious-event percentage is tied to the single participant represented in the record and should not be treated as a stable population rate.
The study status is TERMINATED, but the reason for termination is not reported in the registry record. No sample-size justification, statistical analysis plan, interim-analysis plan, or analysis population definition is reported. The sample-size illustrations use stated planning assumptions, not results from a completed comparative trial, and do not alter the fact that the actual study was single-group. Overall, the record documents an attempted study with minimal enrollment and an early discontinuation, while leaving the primary efficacy result unreported. It cannot support a firm conclusion about efficacy, comparative effectiveness, or the frequency of adverse events.
9. Statistical Methods Explained
Statistical methods should connect the clinical question, the endpoint definition, the enrolled population, and the analysis. In this study, the endpoint was PSA response rate, a proportion-type outcome. General statistical interpretation of a response rate requires a clear rule for classifying each participant as a responder or non-responder, a defined denominator, and a specified time period. The registry names the endpoint, gives a 2 years timeframe, and says PSA was assessed every 4 weeks. It does not report the response threshold, confirmation rules, or a statistical method for estimating the rate.
What would an estimate describe?
In general, a response-rate estimate describes the share of participants in a defined population who meet a response criterion. The denominator matters: it could be all enrolled participants or a specified subset, depending on the protocol and analysis plan. That choice is especially consequential when participants stop treatment early or lack follow-up measurements. The registry notes that the only patient enrolled was discontinued early due to AE, but does not state how the endpoint was classified or whether that patient had usable PSA measurements. The analysis population and handling of unavailable endpoint data are not reported in the registry record.
A confidence interval is a statistical range that reflects uncertainty around an estimate under a specified method and assumptions. In general, an interval based on very limited observations will be highly uncertain. No response estimate or confidence interval is reported in the registry record, so there is no interval to interpret and no basis for quantifying the endpoint's precision. The record also lists no analysis records. Statistical significance cannot be evaluated without a reported test and result, and a test would not resolve the design's lack of a comparator.
Why does the design matter?
General principles of comparative inference require a credible contrast between groups to estimate how outcomes differ under alternative interventions. This study is listed as single-group, with allocation NA and masking NONE. Its results, if fully reported, could describe outcomes among treated participants, but could not provide a randomized comparison. A single-arm response rate also cannot by itself establish that the regimen caused a response. Factors such as disease course and participant selection would remain possible explanations for outcomes.
The registry's sample-size assumptions describe a planning illustration rather than the trial's own design. Those assumptions specify a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is described as a stated fraction of the observed effect. Time-to-event illustrations are described as totals of events, while other illustrations are described as participants per arm. These are general planning assumptions, not evidence that the trial enrolled two groups or used that analysis. Because the study is single-group and no endpoint effect estimate is reported, the planning assumptions do not establish the trial's actual power or provide an observed treatment effect. Sample-size calculations are planning illustrations, never observed power.
What is not documented?
The registry record does not report the statistical analysis plan, sample-size justification, interim analyses, a planned hypothesis test, an analysis population, or the method for estimating uncertainty. It also does not report a multiplicity strategy for the primary and secondary outcomes. These absences matter because analytical choices can affect which observations contribute to an estimate, how uncertainty is quantified, and how multiple outcomes are interpreted. They should not be filled in by assuming standard methods.
Over-dispersion is a general concern when observed variation is greater than a statistical model expects. It can make model-based uncertainty too narrow if ignored. The registry does not report a model for these data or evidence that over-dispersion was assessed; with no outcome analysis reported, there is no model fit to evaluate. More generally, any method should be judged against the endpoint type, amount of information, study design, and missing-data process. Here, the posted record does not provide the analysis needed to make those assessments.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists PSA Response Rate as the primary outcome and Tumor Response and Toxicity and Tolerability of Experimental Arm as secondary outcomes. Testing or estimating several outcomes can create multiplicity: when several inferential questions are pursued, the chance of at least one apparently notable result can increase. General approaches include prespecifying a primary hypothesis, adjusting inference, or treating secondary findings as exploratory. Which approach was intended is not reported in the registry record. No multiplicity adjustment or hierarchy of secondary outcomes is described.
The record does not report a statistical analysis plan, analysis populations, a missing-data method, or rules for handling early treatment discontinuation. These details are important for an endpoint based on serial PSA assessment, because a participant may have some measurements without completing the planned follow-up. The primary outcome description says the sole enrolled patient was discontinued early due to AE; it does not state whether PSA measurements were available, how the endpoint was classified, or whether any observations were excluded.
In general, missingness can affect an estimate when the chance of having a measured outcome is related to the outcome itself or to participant characteristics. Methods such as imputation, complete-case analysis, or a prespecified nonresponse rule make different assumptions. None is identified in the registry record, so no such method should be attributed to the study. The posted information also does not establish whether the analysis used all enrolled participants or another population.
Given the absence of reported outcome analyses, multiplicity adjustments and missing-data handling cannot be evaluated empirically. The limited enrollment and early discontinuation make these reporting gaps particularly relevant, but they do not justify assuming an unreported analytic choice.
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 |
|---|---|---|---|
| Calcitriol, Ketoconazole, Hydrocortisone | 1 | 1 | 100.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Calcitriol, Ketoconazole, Hydrocortisone | 0 of 1 |
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 |
|---|---|
| Calcitriol, Ketoconazole, Hydrocortisone | 0 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Calcitriol, Ketoconazole, Hydrocortisone |
|---|---|---|
| hypercalcemia | Blood and lymphatic system disorders | 1 of 1 |
Serious events with the highest arm-level rate: hypercalcemia (100.0%).
The serious adverse event record lists 1 participant at risk and 1 participant affected in the Calcitriol, Ketoconazole, Hydrocortisone group. Hypercalcemia is the listed serious adverse event term, reported as 100.0%. This percentage corresponds to the sole participant represented in the safety record; it should not be read as a precise estimate of risk in a broader patient population. With only one participant, the record cannot describe how often the event might occur among other patients receiving the regimen.
For other adverse events, the record lists 1 participant at risk and 0 affected. It records deaths as 0. These figures describe the posted counts, not a comprehensive safety profile. The registry does not report event onset, duration, severity grading beyond the serious classification, relationship assessment, or further clinical detail. The primary outcome description states that the only enrolled patient was discontinued early due to AE, but does not identify that event in that description. Hypercalcemia is listed in the serious-event data; the record does not provide further information about the discontinuation decision.
Safety counts need to be interpreted alongside the number exposed, duration of observation, and completeness of reporting. Here, the limited exposure represented in the record means that the counts cannot establish a population-level event rate or rule out other risks.
12. What the Result Does, and Does Not, Mean
The record establishes that a phase II, single-group study of calcitriol with ketoconazole and hydrocortisone was registered for castration-resistant prostate cancer progressing despite abiraterone. It records an enrollment of 1, a TERMINATED status, and an early discontinuation due to AE for the sole enrolled patient. It also identifies PSA Response Rate as the primary outcome, with assessment every 4 weeks and a timeframe of 2 years.
Those details do not establish whether the primary endpoint was met. The registry does not report a PSA response estimate, the number of responses, or an uncertainty interval. The response definition and endpoint analysis are not reported in the registry record. Consequently, the posted record cannot tell readers how often a response occurred or how precisely a response rate was estimated.
The study also cannot answer whether the regimen performed better than another treatment. It had no concurrent comparator, and it was not designed as a like-for-like control comparison. Even if a participant's outcome had been reported, outcomes from a single group would not isolate the effect of the regimen from other possible explanations. The absence of a reported endpoint result leaves even a descriptive efficacy estimate unavailable.
The safety information documents 1 serious adverse event among 1 participant at risk, with hypercalcemia reported at 100.0%, as well as 0 other adverse events among 1 participant at risk and 0 deaths. These observations should be read as counts from a very limited record, not as reliable estimates of the frequency of harm in a larger population. The study's early termination and limited enrollment constrain what can be inferred about efficacy and safety.
The record does not explain why the trial was terminated, give a sample-size justification, or report an analysis plan. It therefore cannot show whether termination resulted from an adverse event, recruitment difficulty, a decision about study feasibility, or another reason. Those explanations should not be inferred. The most defensible interpretation is that the registry documents study intent and limited enrollment, but not a usable primary outcome analysis or a comparative treatment-effect estimate.
13. Why This Trial Matters Statistically
This trial is statistically instructive because its clinical aim and its available evidence are not the same thing. The stated aim was to estimate PSA response rate in a defined treatment setting, but the registry reports only one enrollee and an early discontinuation due to AE. It does not report a response estimate or analysis. A planned endpoint, even one with a stated assessment schedule, does not become an observed result unless outcome data and their analysis are reported.
The design also clarifies the distinction between describing outcomes and estimating a treatment effect. A single-group study can in principle describe what happened among participants who received a regimen. It does not provide a concurrent comparison, so it cannot directly estimate how outcomes differ under another treatment. Statistical significance or a confidence interval would not remove this design limitation. In this case, there is no reported primary endpoint analysis to interpret in the first place.
Enrollment and follow-up determine how much information a study can contribute. Here, the only enrolled patient was discontinued early, which makes endpoint availability and analysis rules particularly important. Those details are not reported in the registry record. The safety figures likewise show why a percentage based on a single participant can be numerically striking yet provide little precision about a broader population.
Finally, the registry distinguishes study-specific methods from planning illustrations. The stated sample-size assumptions involve two-arm planning and are not the design of this single-group study. Recognizing that distinction helps prevent a planning calculation from being mistaken for observed power or for evidence that a comparative analysis took place. The record is therefore useful for examining reporting limits, endpoint definition, and the gap between a trial's intended question and the conclusions its posted data can support.
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 the enrollment of 1, together with the registry statement that the only patient enrolled was discontinued early due to AE. The record does not report whether that participant had sufficient follow-up to assess the primary endpoint. It also does not provide a PSA response definition, a response count, or an estimate of PSA response rate. The primary outcome therefore cannot be interpreted quantitatively.
The single-group design has no concurrent comparator. It cannot support a direct estimate of the difference between the regimen and another treatment, and it offers no randomized basis for separating treatment effects from other influences. The registry gives allocation as NA and masking as NONE. No controlled comparison should be inferred from the planning assumptions described for sample-size illustrations.
The study is marked TERMINATED, but the reason for termination is not reported in the registry record. The record does not give a target enrollment, recruitment details, treatment exposure duration, or reasons for the patient's discontinuation beyond AE. It also does not state whether the adverse event causing discontinuation was hypercalcemia. While hypercalcemia is listed among serious adverse events, the record provides no further linkage or clinical detail.
Important analysis details are also absent. The statistical analysis plan, analysis population, missing-data rules, response criteria, confidence intervals, and handling of multiple outcomes are not reported. Without those details, readers cannot assess how the endpoint would have been defined and analyzed or whether incomplete follow-up affected the result. The safety counts are also based on the limited enrollment and do not characterize a wider population. Taken together, these limitations prevent reliable conclusions about the regimen's efficacy, comparative effect, or broader safety profile.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Oral Calcitriol With Ketoconazole in CRPC — NCT03261336.
- Registry record last updated 2021-02-08; status last verified 2021-01.
- Results first posted 2021-02-08.
- 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 describes a phase II, single-group study of oral calcitriol with ketoconazole and hydrocortisone in castration-resistant prostate cancer progressing despite primary androgen-deprivation therapy and abiraterone. The listed regimen was calcitriol 10mcg QD X3 weekly, ketoconazole 400mg QD, and hydrocortisone 20mg AM, 10 mg PM. The stated primary outcome was PSA Response Rate, assessed every 4 weeks over 2 years.
The study is marked TERMINATED, with enrollment of 1. The primary outcome description says the sole enrolled patient was discontinued early due to AE. The registry does not report a PSA response result, statistical analysis, or reason for termination. Serious adverse event data list 1 participant affected among 1 at risk and hypercalcemia at 100.0%; the other adverse event count is 0 among 1 at risk, and deaths are 0. These figures are limited to the participant represented in the record and do not establish broader outcome rates.