Improving Quality of Life of Prostate Cancer Survivors With Androgen Deficiency: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Improving Quality of Life of Prostate Cancer Survivors With Androgen Deficiency (NCT03716739), a randomized phase 2 study. The primary outcome is Number of Participants With Biochemical Recurrence of Prostate Cancer.

2. Clinical Question

3. Trial Design

This completed phase II study compared testosterone cypionate with placebo in men with prostate cancer who had undergone radical prostatectomy. The registry lists randomized allocation, double masking, and a parallel design. Its enrollment is 136. Dana-Farber Cancer Institute is identified as the sponsor. The recorded study start is 2019-03-19, and the primary completion date is 2025-05-16.

The treatment arm received weekly intramuscular administration of 100 mg testosterone cypionate for 12 weeks. The control arm received weekly intramuscular placebo for 12 weeks. The study summary describes the purpose as assessing efficacy and safety in improving symptoms of androgen deficiency and health-related quality of life. The registry also lists safety and other clinical outcomes, including change in PSA, lower urinary tract symptoms measured with the International Prostate Symptom Score, hemoglobin, hematocrit, erythrocytosis, clinical prostate cancer recurrence, sexual desire measured with the DeRogatis Inventory of Sexual Function Sexual Desire questionnaire, and erectile function measured with the International Index of Erectile Function questionnaire.

The primary endpoint is specified as the number of participants with biochemical recurrence during the 12-week treatment and the 3-month follow-up. Its analysis population is defined by receipt of at least 1 study treatment administration. That population definition is distinct from the overall enrollment figure. The registry record does not report the number included in the primary endpoint analysis, nor does it describe a separate efficacy analysis population. It lists 20 outcome measures and 0 analyses. The study status is completed, but a completed status alone does not establish that every planned outcome has a posted comparative result.

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.

Before assignment: We initially planned to enroll 71 men per arm for a total of 142 men. As the study progressed, the loss-to-follow rates were substantially lower. Accordingly, with the concurrence of the trial's Data and Safety Monitoring Board (DSMB) and the NIA program staff, the target sample size was revised to 134 subjects as planned enrollment anticipating that it will still provide the same statistical power as originally assumed. In the end,136 subjects were enrolled and randomized.

Overall Study

Analysis population for the primary outcome, as defined in the registry: The study safety sample includes participants who received at least 1 study treatment administration. The participants who discontinued prior to study treatment administration due to COVID-19 disruption or other reasons are not included in the analysis for the primary safety endpoint.

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.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Biochemical Recurrence of Prostate Cancer, measured over the 12-week treatment and the 3-month follow-up.

The primary endpoint counts participants with biochemical recurrence of prostate cancer during the 12-week treatment and the 3-month follow-up. The registry defines biochemical recurrence as two or more consecutive serum PSA levels of 0.2 ng/mL or higher. This is a binary event for each participant: the endpoint concerns whether the specified recurrence criterion was met, not the size of a PSA change.

The registry labels this outcome as the primary safety endpoint. Counting participants who meet a prespecified event definition can provide a direct description of an event of safety interest during the stated observation period. It is important to retain the definition and timeframe when interpreting the count: a PSA value alone does not meet the registry definition, which requires consecutive measurements at or above the stated threshold.

The endpoint population includes participants who received at least 1 study treatment administration. Participants who discontinued before study treatment because of COVID-19 disruption or other reasons were excluded from the primary safety endpoint analysis. This population rule affects what the endpoint count represents. It is not a count for every person included in the enrollment figure.

The registry record does not provide a primary endpoint comparison or analysis. Therefore, it does not establish from the posted primary endpoint data how the arms compare under this definition. Nor does it provide enough information to assess the precision of any such comparison.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes a randomized, double-masked, parallel-group phase II trial. Randomization is intended to allocate participants to study arms using a chance-based process, while masking is intended to limit knowledge of assignment during the study. These are general design concepts; the registry record does not provide further details about how randomization or masking was implemented. It lists weekly intramuscular treatment for 12 weeks in both arms, with testosterone cypionate in the treatment arm and placebo in the control arm.

What does a participant count represent?

The primary endpoint is a count of participants meeting a specified biochemical recurrence definition. The definition requires two or more consecutive serum PSA levels of 0.2 ng/mL or higher during the 12-week treatment and the 3-month follow-up. A count endpoint records whether each participant meets that definition within the specified period. It is not the same as measuring average PSA change, and the recurrence definition should not be replaced with a single elevated measurement.

For a binary outcome, a descriptive summary can report the event count and the number of participants evaluated. A comparative analysis might express the arm contrast using an appropriate measure of difference or relative occurrence. Those are general options, not methods documented for this trial. The registry record does not identify a statistical model, a test, an effect measure, or an adjustment procedure for the primary endpoint. It reports no analysis entry for the primary outcome.

How should uncertainty be understood?

In general, a confidence interval describes a range of values compatible with the observed data under a specified statistical method and its assumptions. Its width communicates precision: a wider interval indicates less precision than a narrower interval. A point estimate without an interval can conceal substantial uncertainty, particularly when event counts are limited. No primary endpoint confidence interval or comparative estimate is reported in the registry record, so the precision of the primary outcome cannot be assessed from this record.

Likewise, a p-value, when reported, addresses compatibility between data and a specified null hypothesis under a statistical model. It does not describe the size or clinical importance of an effect by itself. No primary endpoint p-value or hypothesis test is reported. It would be inappropriate to infer statistical significance from the arm assignment, study completion status, enrollment, or adverse-event counts.

What do the planning assumptions mean?

The record describes sample-size illustrations as planning calculations based on the observed effect, with a two-sided significance level of 0.05, equal allocation between the arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. These are planning illustrations, not observed power and not proof that the trial had a particular probability of detecting an effect. The registry record does not provide the trial’s own sample-size justification or a statistical plan establishing that these assumptions were used to design the trial. No planning values are stated in the registry record.

In general, planning calculations depend on the target effect, variability or event frequency, the chosen error rate, allocation, and analysis method. Changing an assumed effect changes the implied requirements. An illustration based on an observed effect is especially dependent on the estimate used as its starting point; it should not be read as a prospective guarantee or as evidence that the observed effect is reliable. For time-to-event calculations, the record describes event totals as the planning quantity; for other calculations, it describes participants per arm. Those conventions do not add information about this trial’s observed primary endpoint.

Why do population definitions matter?

The primary safety population includes participants who received at least 1 study treatment administration. Participants who discontinued before treatment because of COVID-19 disruption or other reasons are excluded from that endpoint analysis. This is the population definition stated in the registry, not an assumption that all randomized participants were analyzed. The number included in that population is not reported. The registry record also does not report the analysis population used for the study’s efficacy or quality-of-life questions.

In general, different analysis populations can answer different questions. An analysis of participants according to treatment received can differ from one that retains all randomized participants in their assigned groups. Missing outcomes and withdrawals can also affect interpretation, especially if their occurrence relates to treatment or health status. The registry record does not report missing-data methods, handling of withdrawals after treatment began, or sensitivity analyses.

There are multiple outcome measures listed, but the registry record does not report a multiplicity strategy. In general, evaluating multiple outcomes or repeated comparisons can increase the chance of at least one apparently unusual result if each is considered separately. Prespecified prioritization or statistical adjustment can address that issue, but neither is documented for this trial in the registry record. No claim about the trial’s actual testing strategy can therefore be made.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists 20 outcome measures, including the primary biochemical recurrence safety endpoint and secondary outcomes covering PSA, urinary symptoms, hemoglobin, hematocrit, erythrocytosis, clinical recurrence, sexual desire, and erectile function. It does not report a multiplicity strategy, such as a hierarchy for interpreting outcomes or an adjustment for multiple comparisons. The registry record therefore does not establish how results across these measures were intended to be interpreted together. This is a reporting gap, not evidence that the trial did or did not use a particular approach.

Missing-data methods are not reported in the registry record. The primary safety population is defined as participants who received at least 1 study treatment administration, with those who discontinued before treatment due to COVID-19 disruption or other reasons excluded. The number meeting that population definition is not reported. The record does not describe missing endpoint assessments after treatment began, how withdrawals were handled, whether outcomes were imputed, or whether sensitivity analyses were conducted.

The analysis population for efficacy and quality-of-life outcomes is also not reported in the registry record. The stated primary safety population should not be assumed to apply to those outcomes. A randomized design does not, by itself, specify how incomplete observations were handled or which participants contributed to each analysis. Since the registry lists 0 analyses and does not provide a primary comparison, the impact of missingness or multiplicity on the reported findings cannot be assessed from the record.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: gallbladder stone (1.6%), parapneumonic pleural effusion (1.5%), pneumonia (1.5%), syncope (1.5%).

The adverse-event entries list 66 participants at risk in the treatment arm and 64 in the control arm. Serious adverse events are reported for 3 in the treatment arm and 1 in the control arm. Other adverse events are reported for 49 and 47, respectively. The registry lists 0 deaths in each arm. These are descriptive counts; the record does not report a comparative statistical analysis or confidence interval for them.

The registry also lists serious-event terms with percentages: Gallbladder stone, 1.6%; Parapneumonic pleural effusion, 1.5%; Pneumonia, 1.5%; and Syncope, 1.5%. The percentages and terms describe the serious-event entries but do not, by themselves, show how the overall safety experience compares between arms. The registry record does not explain the denominator used for each listed percentage or provide event-level timing and attribution details.

Counts should be read in the context of the recorded at-risk population and the trial’s treatment and follow-up period. They are not estimates of risk beyond the study period, and they do not establish causation. The primary safety endpoint, biochemical recurrence, is distinct from these general adverse-event entries.

12. What the Result Does, and Does Not, Mean

The registry record establishes that the study was a completed, randomized, double-masked, parallel-group phase II trial of weekly testosterone cypionate versus placebo for 12 weeks in men with prostate cancer after radical prostatectomy. It specifies biochemical recurrence as the primary safety endpoint and defines recurrence using consecutive PSA measurements during treatment and follow-up. It also states who was included in the primary safety analysis population.

Those design details do not amount to an outcome finding. The registry lists 0 analyses, and the primary outcome is not reduced to a single comparison. It does not report the number of participants with biochemical recurrence by arm, an arm contrast, a confidence interval, or a p-value. The posted primary endpoint information therefore cannot establish the direction or magnitude of a difference, nor how precisely such a difference was estimated.

The adverse-event entries provide descriptive counts for serious and other adverse events, and the record lists no deaths in either arm. These entries do not demonstrate that one arm is safer or less safe: no statistical comparison or uncertainty estimate is reported, and the counts alone cannot distinguish a treatment effect from chance or other factors. The listed serious-event terms are not a substitute for a full comparative safety analysis.

The study summary describes an aim to assess symptoms of androgen deficiency and health-related quality of life, and the registry lists several relevant secondary outcomes. The record does not provide results for those outcomes in the information recorded. It is therefore not possible to use the registry entries to conclude whether the intervention changed symptoms, questionnaire scores, or other measures.

In short, the record supports a description of trial design, endpoint definition, analysis population rules, and selected adverse-event counts. It does not support an efficacy conclusion or a precise comparative safety conclusion. The completed status indicates the study is marked completed; it does not fill the gaps in the posted outcome analyses.

13. Why This Trial Matters Statistically

This trial raises a statistical question in which the target outcome is a defined safety event rather than a continuous symptom score: whether participants meet the registry’s biochemical recurrence criterion during the specified treatment and follow-up period. The definition matters because interpretation depends on the requirement for two or more consecutive serum PSA levels at or above 0.2 ng/mL. A count based on that rule answers a different question from an average change in PSA or a general description of recurrence.

The randomized, double-masked, parallel design provides a framework for comparing treatment and placebo, while the primary safety population definition makes clear that the primary analysis does not include participants who stopped before receiving study treatment. Such population choices determine what group a result describes and are important to assess alongside any estimated effect. In this record, the number included in the primary endpoint analysis and the endpoint results are not reported.

The registry also lists outcomes spanning several domains, from laboratory measures to urinary and sexual-function questionnaires. Statistically, that range makes clear why outcome definitions, analysis populations, missing-data handling, and multiplicity plans are important. Those elements help readers distinguish an intended primary question from supporting or exploratory questions. The registry record does not document these analysis details, so the listed outcomes cannot be treated as a complete account of the trial’s statistical conclusions.

The adverse-event counts illustrate another important distinction: descriptive frequencies are not the same as comparative estimates with uncertainty. Without an analysis and its precision, the counts do not resolve whether differences between arms are meaningful or compatible with chance. The trial’s statistical importance therefore lies not in an unreported conclusion, but in the need to connect its defined endpoints and design to fully reported analyses.

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 registry record does not report the primary endpoint event counts by arm or a comparative estimate. It lists 0 analyses and indicates that the primary outcome has several categories or time points and is not reduced to one comparison. As a result, the record does not provide the information needed to evaluate the observed frequency of biochemical recurrence, the arm difference, or its precision.

The number included in the primary safety analysis population is not reported. Although the population rule excludes participants who discontinued before receiving study treatment because of COVID-19 disruption or other reasons, the record does not state how many participants were excluded for this reason or how incomplete endpoint assessments after treatment began were handled. The efficacy analysis population is not reported either.

The registry does not report the statistical methods for the primary endpoint, the prespecified analysis plan, multiplicity handling, missing-data methods, interim analyses, or the trial’s sample-size justification. The record describes planning illustrations based on the observed effect, but does not establish that those illustrations were the trial’s design calculations. No primary endpoint confidence interval or p-value is available in the registry record.

The adverse-event entries are descriptive. The registry does not provide a comparative analysis, uncertainty estimates, event timing, attribution, or the denominator used for each serious-event percentage. These details limit interpretation of the safety entries. The listed secondary outcomes also lack posted analysis results in this record, so the study’s broader questions about symptoms and health-related quality of life cannot be assessed from the information recorded.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Improving Quality of Life of Prostate Cancer Survivors With Androgen Deficiency — NCT03716739.
• Registry record last updated 2026-07-16; status last verified 2025-01.
• Results first posted 2026-07-16.
• 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.

18. Record Summary

The registry identifies NCT03716739 as a completed phase II randomized, double-masked, parallel-group study sponsored by Dana-Farber Cancer Institute. The recorded enrollment is 136. The study compared weekly intramuscular testosterone cypionate 100 mg for 12 weeks with weekly intramuscular placebo for 12 weeks in men with prostate cancer who had undergone radical prostatectomy.

The primary safety endpoint is the number of participants with biochemical recurrence during the 12-week treatment and the 3-month follow-up, defined as two or more consecutive serum PSA levels of 0.2 ng/mL or higher. The primary safety population includes participants who received at least 1 study treatment administration; those who discontinued before treatment because of COVID-19 disruption or other reasons are excluded.

The registry lists 20 outcome measures and 0 analyses. It reports serious and other adverse-event counts by arm, but does not report a primary endpoint comparison or analysis. The study start is recorded as 2019-03-19 and primary completion as 2025-05-16.