Real Life Evaluation of Lupron in the Management of Prostate Cancer: A Canadian Post Marketing Observational Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Real Life Evaluation of Lupron in the Management of Prostate Cancer: A Canadian Post Marketing Observational Study (NCT01386684), a registered phase not stated study. The primary outcome is Progression-free Survival (PFS) Defined as the Time From Patient Recruitment to Biochemical Progression Based on Doubling of Prostate Specific Antigen (PSA) Velocity or PSA > 5.0.

2. Clinical Question

The study asks a descriptive, real-world question about progression-free survival (PFS) among people with prostate cancer who were treated with Lupron. The primary endpoint is defined from patient recruitment to biochemical progression based on doubling of prostate-specific antigen (PSA) velocity or PSA > 5.0, objective tumor progression using RECIST criteria, or death. The endpoint title emphasizes biochemical progression, while the fuller description also includes objective tumor progression and death. The registry does not report the detailed operational rules used to combine these events.

This is not a randomized comparison of Lupron with another treatment or with no treatment. As a result, the central question is not whether Lupron outperforms a control. Instead, the registry describes a treated cohort and identifies outcomes intended to characterize its clinical course. Without a comparator, differences attributable to treatment cannot be separated from differences related to disease severity, other care, or participant characteristics.

The record also names outcomes involving castration-resistant prostate cancer, testosterone, PSA, and quality of life measured with the FACT-G questionnaire. Those outcomes broaden the description of the study’s intended assessments, but the registry record does not report their numerical results. The PFS estimate and its standard error are likewise not reported in the registry record.

3. Trial Design

The registry describes the study as a Canadian post-marketing observational evaluation of Lupron in the management of prostate cancer. Its listed study group consists of patients with prostate cancer who were receiving treatment with leuprolide acetate. Enrollment is 552. The study is marked completed; its listed start date is 2011-06-28 and its primary completion date is 2016-09-08.

The design is observational rather than randomized. Allocation is not stated, and no separate control group is described. The registry record specifically cautions that the arms are not a like-for-like control comparison. Accordingly, the results should be read as descriptions of participants receiving treatment, not as a controlled estimate of the effect of Lupron compared with an alternative.

The primary outcome is PFS, with a stated timeframe of 36 months. The registry says its distribution was estimated using Kaplan-Meier methodology and that a point estimate and standard error are provided. However, the numerical estimate and standard error are not reported in the registry record. The outcome description defines progression using biochemical criteria, objective tumor progression by RECIST criteria, or death. How the event criteria were implemented, and how follow-up time was handled, are not reported in the registry record.

The record lists 36 outcome measures, including secondary assessments of castration-resistant prostate cancer, serum testosterone, PSA, and FACT-G total score. It lists no analyses. This inventory does not establish which assessments were completed or which analyses were performed outside the registry record. The protocol, the rationale for the enrollment target, and any changes to the planned design are not reported in the registry record.

4. Participant Flow and Accounting

The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.

Overall Study

Reasons for not completing this period:

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 Progression-free Survival (PFS) Defined as the Time From Patient Recruitment to Biochemical Progression Based on Doubling of Prostate Specific Antigen (PSA) Velocity or PSA > 5.0, measured over 36 months.

The primary endpoint is PFS, expressed in months, over a stated timeframe of 36 months. It measures the time from patient recruitment until a qualifying event: biochemical progression based on doubling of PSA velocity or PSA > 5.0, objective tumor progression according to RECIST criteria, or death. The endpoint title highlights the biochemical progression criteria; the fuller description includes the additional event types. The precise rules for determining event dates and applying the criteria are not reported in the registry record.

Time-to-event endpoints are useful when the outcome concerns how long it takes for an event to occur and participants may have different lengths of follow-up. In general, such methods can use information from participants who have not experienced the event by the end of observation, provided their follow-up is appropriately handled. The registry states that Kaplan-Meier methodology was used to estimate the PFS distribution. It does not report the censoring rules or the numbers of participants at risk at particular times.

The registry describes the summary as a mean with a standard error. A mean provides a central summary of the measured time, while a standard error describes the statistical precision of an estimate. The numerical point estimate and standard error are not reported in the registry record, so the endpoint’s magnitude and precision cannot be assessed from the record. Nor is a confidence interval reported. The endpoint definition alone does not establish how many participants experienced progression or how their PFS times were distributed.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

How does the registered PFS method work?

The registry states that the PFS distribution was estimated using Kaplan-Meier methodology. In general, Kaplan-Meier estimation describes the probability of remaining free of an event over time while accommodating participants whose event status is not observed through the end of follow-up. Those participants may contribute information until their last known follow-up, under the method’s assumptions. This general explanation does not establish how the study handled follow-up or censoring; those details are not reported in the registry record.

The event in this study is a composite of qualifying progression and death. The full description includes biochemical progression based on doubling of PSA velocity or PSA > 5.0, objective tumor progression under RECIST criteria, or death. When several event types are combined, the time-to-event estimate reflects the first qualifying event under the study’s rules. The registry record does not report event counts by type, how the PSA velocity criterion was calculated, or how event dates were adjudicated.

What does the mean and standard error describe?

The registered summary is a mean PFS with a standard error. In general, the mean is a measure of central tendency, and the standard error characterizes the sampling uncertainty of an estimated quantity. A smaller standard error would indicate greater precision under the model used, while a larger one would indicate less precision. Neither the mean estimate nor its standard error is reported in the registry record. The record does not explain how the mean was derived from the Kaplan-Meier distribution or whether a restricted mean over a specified interval was used.

A confidence interval is a range constructed by a statistical procedure to express uncertainty about an estimate. It can help readers judge both the plausible magnitude of an effect and its precision. No confidence interval is reported in the registry record. A standard error alone, without the estimate and without details of the inferential procedure, does not allow the size or precision of the PFS result to be evaluated.

What does an observational design allow?

In general, observational studies describe outcomes in groups defined by care received rather than assigning treatment randomly. They can document clinical experience in the population studied, but comparisons may be affected by differences between people who receive different care. In this record, only a treated group is described and no control group is specified. There is therefore no like-for-like treatment contrast to interpret. Adjustment methods, if any, are not reported in the registry record, and none should be assumed.

The registry’s secondary outcomes cover several types of measurements. Castration-resistant prostate cancer is listed as both a participant count and a time-to-event outcome. Testosterone and PSA are listed as values and as categories at each visit, with additional outcomes for time to testosterone increase over castrate levels. FACT-G total score is listed as a change from baseline to each visit. These outcomes differ in scale and meaning: counts describe how many participants meet a condition, time-to-event measures describe timing, categories group measurements into stated ranges, and change scores compare a follow-up measure with baseline. The record does not report the numerical findings or analysis procedures for these outcomes.

How should planning calculations be read?

The sample-size calculations are planning illustrations based on the observed effect, not the trial’s own design or evidence of an achieved power level. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. For time-to-event calculations, the stated quantity is the total number of events; for other calculations, it is participants per arm. These are general planning conventions, not descriptions of the single treated group in the registry design.

Such calculations can illustrate how a hypothetical comparison’s required sample size varies with the effect chosen for detection and the statistical assumptions. They do not show that the observational study planned or enrolled two randomized arms. The actual sample-size illustration values, the effect estimate used as its basis, and a sample-size justification for the study are not reported in the registry record. No interpretation of observed power is warranted from a planning calculation.

What cannot be checked from the registered methods?

The record does not report the detailed statistical analysis plan, analysis populations, missing-data methods, censoring rules, model diagnostics, or whether any interim analyses occurred. It also does not report multiplicity adjustment or a hierarchy for the many outcomes. Without these details and the numerical results, readers cannot independently assess the implementation of the stated methods or the robustness of the estimates. General descriptions of Kaplan-Meier estimation and uncertainty explain the method’s purpose; they do not fill in methods that are absent from the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("Patients With Prostate Cancer")
n   <- c(552)
m   <- c(20.76)
sp  <- c(0.59)
sd  <- sp * sqrt(n)      # standard errors converted to SDs
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  Patients With Prostate Cancer  552  20.760  13.862  19.601  21.919

11. Multiplicity, Missing Data and Analysis Populations

The registry lists 36 outcome measures, spanning PFS, castration-resistant prostate cancer, testosterone, PSA, and FACT-G scores. With many outcomes and repeated measurements at each visit, a study may conduct multiple statistical tests or estimates. In general, repeated testing can increase the chance that at least one result appears notable by chance. Whether and how to address that issue depends on the planned hypotheses, testing hierarchy, and analysis strategy. Multiplicity adjustment, a prespecified hierarchy, and the statistical plan are not reported in the registry record.

The registry lists no analyses. This means the record does not provide an analysis inventory from which to evaluate which outcomes were analyzed, how many comparisons were made, or whether selective reporting occurred. It does not establish that no analyses were conducted outside the registry record. Numerical results for the primary and secondary outcomes are not reported in the registry record.

Enrollment is 552, but the record does not state how many participants were included in each outcome analysis, how many had usable baseline or follow-up measurements, or how missing observations were handled. The analysis populations and missing-data methods are not reported in the registry record. For a time-to-event endpoint, censoring and loss to follow-up can affect what is estimable; the relevant rules and follow-up completeness are not reported in the registry record. For repeated measurements, the handling of missed visits and incomplete questionnaires is also not reported.

These omissions limit assessment of both precision and potential bias. The enrollment count should not be treated as the number contributing to every endpoint. Without outcome-specific denominators and missingness information, the completeness of the evidence for each assessment cannot be judged from the registry record.

12. Safety Results

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

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: prostate cancer metastatic (2.5%), fall (2.0%), dyspnoea (2.0%), metastases to bone (1.8%), prostate cancer (1.8%).

For the group titled Patients With Prostate Cancer, the registry lists 552 participants at risk for serious adverse events and 132 participants affected. It lists 552 at risk for other adverse events and 77 affected. These are counts recorded for the study group, not a comparison between treatment arms. The registry does not report deaths in the adverse-event summary.

The listed serious-event terms and percentages include PROSTATE CANCER METASTATIC, 2.5%; FALL, 2.0%; DYSPNOEA, 2.0%; METASTASES TO BONE, 1.8%; PROSTATE CANCER, 1.8%; and CARDIAC FAILURE CONGESTIVE, 1.6%. These are the serious-event terms identified in the record; the percentages do not establish causation. The registry does not report the denominator or calculation details for each listed term, nor does it establish whether the same participant could contribute to more than one term.

In general, adverse-event summaries describe events recorded during a study and should be distinguished from events judged to be caused by treatment. The registry record does not provide a causal attribution assessment in these figures. It also does not report event timing, duration, severity grading beyond the serious/non-serious distinction, or participant-level overlap. These limits matter when interpreting counts: they describe recorded safety events in the cohort, but do not by themselves establish comparative risk or treatment causation.

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

The registry record describes a completed Canadian post-marketing observational study of Lupron in people with prostate cancer. It identifies enrollment of 552 and a primary PFS outcome defined from recruitment to specified biochemical progression, objective tumor progression, or death. It states that Kaplan-Meier methodology was used and that the intended summary was a point estimate with a standard error. The numerical PFS estimate and its standard error are not reported in the registry record.

Therefore, the record does not establish the observed length of PFS, the precision of that estimate, or the frequency of the separate event types. It also does not report results for the secondary outcomes involving castration-resistant prostate cancer, testosterone, PSA, or FACT-G score. The registered outcome list indicates what the study intended to assess, not what the measurements showed.

The design does not support a direct treatment-versus-control conclusion: no comparator is described, allocation is not stated, and the study is observational. A descriptive result in a treated group cannot isolate the effect of treatment from other influences. No adjusted comparison or inferential result is reported in the registry record. The safety counts and listed serious-event terms are similarly descriptive; the record does not establish that the events were caused by Lupron.

These limitations do not make the registry information meaningless. It records the study’s broad purpose, population, endpoint definition, stated time-to-event method, and safety counts. But the absence of numerical outcome results and key analysis details leaves important questions unanswered. The registry record does not report the analysis populations, missing-data handling, censoring rules, confidence intervals, or multiplicity procedures. Readers should distinguish the existence of a registered assessment from evidence about its result, and distinguish a cohort description from a controlled estimate of treatment effect.

14. Why This Trial Matters Statistically

This study is statistically informative as an example of the difference between a clearly named outcome and a fully assessable result. The registry defines PFS, gives a timeframe of 36 months, and identifies Kaplan-Meier methodology. Yet the numerical estimate and standard error are not reported in the registry record. Without those values, readers cannot evaluate the magnitude or precision of the primary outcome, even though the endpoint and nominal method are described.

It also illustrates why design matters when interpreting outcome summaries. The study concerns people receiving Lupron in routine practice, but it does not describe a comparator group or randomized allocation. A cohort’s outcome can characterize observed experience, while a treatment effect requires a defensible comparison that accounts for what would have happened under an alternative. That contrast is absent from the registered design.

The broad range of listed secondary outcomes also highlights the importance of transparent analysis plans. Repeated measures and multiple outcome types can create many possible summaries. The registry lists 36 outcome measures but does not report a statistical plan, multiplicity approach, analysis populations, or numerical results. Those details are needed to understand which questions were answered and how stable the findings might be.

Finally, the safety summary demonstrates the distinction between event counts and causal conclusions. Counts and term-specific percentages describe recorded adverse events, but they do not by themselves show that treatment caused those events or indicate comparative risk. Clear denominators, timing, attribution, and comparison groups would be needed for stronger interpretation; those details are not reported in the registry record.

15. 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.

Several trial-specific limitations restrict interpretation. The study is observational and the registry describes only a group of patients with prostate cancer receiving leuprolide acetate. No comparator group is described, and allocation is not stated. The record itself cautions that the arms are not a like-for-like control comparison. As a result, the registry cannot support a randomized or controlled treatment-effect interpretation.

The primary endpoint definition is not fully consistent in emphasis across its title and description. The title foregrounds biochemical progression based on PSA velocity or PSA > 5.0, while the fuller description also includes objective tumor progression by RECIST criteria or death. The precise event rules, adjudication procedures, and handling of event dates are not reported in the registry record. This limits reproducibility of the endpoint definition.

Most importantly, the numerical PFS estimate and standard error are not reported in the registry record, and the listed secondary outcome results are also absent. The record does not report confidence intervals, outcome-specific denominators, analysis populations, or the number of participants contributing to each assessment. Enrollment of 552 cannot be assumed to equal the analysis population for every outcome.

Important analytic details are not reported in the registry record, including censoring rules, missing-data methods, multiplicity procedures, model checks, and whether interim analyses occurred. The sample-size justification is also not reported. Safety interpretation is limited by the absence of reported attribution, event timing, and term-specific denominator details. These gaps mean that neither the magnitude and precision of the clinical outcomes nor the robustness of their analysis can be judged from the registry record alone.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Real Life Evaluation of Lupron in the Management of Prostate Cancer: A Canadian Post Marketing Observational Study — NCT01386684.
• Registry record last updated 2018-12-13; status last verified 2017-08.
• Results first posted 2018-04-02.
• 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.

19. Record Summary

NCT01386684 is a completed Canadian post-marketing observational study of Lupron in prostate cancer. Enrollment is 552; the listed start date is 2011-06-28 and primary completion date is 2016-09-08. The registry describes one group of patients receiving leuprolide acetate and no comparator.

The primary outcome is PFS over a stated timeframe of 36 months, estimated using Kaplan-Meier methodology and summarized as a mean with a standard error. The endpoint includes biochemical progression, objective tumor progression, or death. The numerical PFS result and standard error are not reported in the registry record. Secondary outcomes cover castration-resistant prostate cancer, testosterone, PSA, and FACT-G score, but their numerical results are not reported in the registry record.

The record lists 132 participants affected by serious adverse events and 77 affected by other adverse events, with 552 at risk for each category. The registry lists no analyses and does not report key analysis details, including missing-data handling, analysis populations, or multiplicity procedures.