Long-Term Safety and Tolerability of Degarelix One-Month Dosing Regimen in Korean Patients: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Long-Term Safety and Tolerability of Degarelix One-Month Dosing Regimen in Korean Patients (NCT01215513), a na phase 3 study. The primary outcome is Number of Participants With Markedly Abnormal Values in Safety Laboratory Variables.

2. Clinical Question

3. Trial Design

This was a phase 3, open-label, multi-centre, single-group extension trial of degarelix in Korean patients with prostate cancer requiring androgen ablation therapy. Its stated purpose was to investigate long-term safety and tolerability. The registry lists the trial as completed, with enrollment of 127. The recorded study dates are 2010-09 for start and 2012-04 for primary completion.

Open-label means that masking was not used; the registry records masking as none. Allocation is listed as NA, consistent with the single-group design. There was no randomized comparator, so the study does not support a like-for-like comparison of degarelix against another treatment or against no treatment. Any changes observed over time cannot, from this design alone, be separated from other possible influences.

The primary outcome covers safety laboratory variables from baseline (day 0) to end of treatment, up to day 364. The registry says the descriptive safety population was the CS42 and CS42A safety analysis set, but does not explain the relationship of those sets to enrollment. The specific dosing schedule beyond the one-month regimen description, visit schedule, treatment discontinuation rules, and detailed eligibility criteria are not reported in the registry record.

The registry also lists serum levels of prostate specific antigen (PSA) over time as a secondary outcome. It records four outcome measures overall, but further details of the other measures are not reported in the registry record. The record lists zero analyses. Consequently, it does not document inferential testing or a comparative analysis for the primary safety question.

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:

Analysis population for the primary outcome, as defined in the registry: Descriptive statistics provided a view of the 1-year safety of degarelix (CS42 and CS42A safety analysis set).

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.

Population: The 156 participants included in the CS42 and CS42A saftey analysis set are included in this baseline analysis population.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants With Markedly Abnormal Values in Safety Laboratory Variables, measured over From baseline (day 0) to end of treatment (up to day 364).

The primary endpoint is the number of participants with markedly abnormal values in safety laboratory variables over the period from baseline (day 0) to end of treatment, up to day 364. The registry explains that the figures present participants who had markedly abnormal levels, and that only laboratory variables with at least one participant with an abnormal value are presented; more variables were included in the study. ULN means upper limit of normal.

This kind of endpoint is used to describe laboratory safety during treatment. It focuses on whether participants crossed a threshold for marked abnormality, rather than on the average laboratory value or the size of a change from baseline. In general, counts can make the occurrence of such findings easy to interpret, while the underlying definitions and assessment rules determine what qualifies as markedly abnormal. Those rules are not reported in the registry record.

The record identifies the primary population as the CS42 and CS42A safety analysis set and describes the safety statistics as descriptive. It does not report the primary endpoint count, the laboratory variables’ individual counts, or the denominators for those results. The numerical primary outcome result is therefore not reported in the registry record, and the frequency of markedly abnormal laboratory findings cannot be assessed from the posted primary outcome information.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The outcome type matters for interpretation. The primary endpoint counts participants with markedly abnormal laboratory values. In general, this is a participant-level event outcome: a participant is counted if the specified condition is met. It differs from a continuous outcome, such as a measured laboratory value, where analysis might focus on a mean or a change in the mean. The registry states that the primary outcome is reported as a number of participants and describes the analysis as descriptive.

What does a descriptive count tell the reader?

A count tells how many participants meet the endpoint definition in the analysis population. To interpret that count as a frequency, the denominator and the rules for determining eligibility for analysis also matter. The primary outcome description names the CS42 and CS42A safety analysis set, but the numerical endpoint count and its denominator are not reported in the registry record. It is therefore not possible to derive or assess a frequency from the primary outcome information. No percentage should be inferred from the record’s description alone.

In general, a confidence interval describes a range of values compatible with the data under a statistical model, and indicates the precision of an estimate. It does not make a non-randomized design comparative, and it does not remove uncertainty about how an endpoint was defined or who was included. Confidence intervals for the primary endpoint are not reported in the registry record. The record also lists zero analyses, so it does not document a model-based estimate or a hypothesis test for this outcome.

Why does the single-group design matter?

The allocation is recorded as NA and the model as single group. With no concurrent control, the trial can describe findings among participants receiving degarelix but cannot estimate a treatment contrast against another group. In general, a before-and-after description can show what was recorded over the observation period, but change over time may also reflect factors other than treatment. A randomized comparison is designed to address some of those interpretive problems; this trial did not use one.

The study was open-label, with masking recorded as none. In general, lack of masking can affect how outcomes are assessed or reported, particularly when judgments are required. Laboratory measurements may be more objective than subjective outcomes, but the registry does not report enough detail about the laboratory assessment process to characterize its susceptibility to assessment effects. This is a general consideration, not evidence that assessment was biased in this trial.

How should planning illustrations be understood?

The planning assumptions describe an illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. These assumptions describe a hypothetical planning framework, not the trial’s own single-group design. They do not establish that the trial was designed with those assumptions, and they are not observed power. Sample-size planning illustrations are prospective calculations about a hypothetical future study; they are not additional participants or outcomes observed in this trial. No numerical planning results are reported in the registry record used for this analysis.

In general, a normal approximation simplifies the probability model used for a planning calculation. Its suitability depends on the outcome and the expected data pattern. For count outcomes, over-dispersion means that observed variability exceeds what a simple model would expect; if present, it can affect uncertainty estimates and planning. The registry record does not report an over-dispersion assessment or a statistical model for the primary endpoint. No assumption about either should be treated as part of the trial’s documented analysis.

The listed serious and other adverse event figures are also descriptive. Counts and percentages communicate the recorded event experience, but without a concurrent comparator they do not quantify a causal treatment effect. The registry does not report confidence intervals for these figures, nor does it describe an inferential method for comparing event rates. The serious-event term percentages should therefore be interpreted as registry summaries, not as a comparative risk estimate.

10. Multiplicity, Missing Data and Analysis Populations

The registry lists four outcome measures and identifies serum levels of prostate specific antigen (PSA) over time as a secondary outcome. It also identifies the laboratory safety measure as primary. The record lists zero analyses, and no multiplicity procedure is reported in the registry record. It is therefore not possible to tell whether several outcomes, time points, or laboratory variables were formally tested or whether any adjustment was planned. Because the primary outcome is described as descriptive, there is no documented family of hypothesis tests to interpret.

Multiplicity is a general concern when many outcomes or repeated tests are considered: the chance of finding an apparently notable result can increase when many tests are performed. Adjustments or a prespecified testing hierarchy can address that issue in some settings, but neither is described in the registry record. This does not establish that multiplicity affected the reported findings; it means the record does not document how it was handled.

Missing-data methods are not reported in the registry record. The record does not state how missing laboratory measurements, missed visits, withdrawal, or incomplete follow-up were handled. It also does not provide a missingness summary for the primary outcome. Without those details, the effect of incomplete observations on the descriptive safety results cannot be assessed.

The primary population is described as the CS42 and CS42A safety analysis set, but inclusion rules and the relationship between that set and enrollment are not reported in the registry record. The registry reports enrollment of 127, while the adverse-event figures list 156 at risk; an explanation of that difference is not reported. No assumptions about the population definitions, data linkage, or counting method should be made from these figures alone.

11. 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 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: spinal compression fracture (1.3%), angina pectoris (0.6%), angina unstable (0.6%), atrial fibrillation (0.6%), coronary artery occlusion (0.6%).

The registry reports 156 at risk and 25 affected for serious adverse events in the Degarelix group. For other adverse events, it reports 156 at risk and 88 affected. These are separate reported categories; the figures should not be added together or treated as a count of unique participants. The record does not explain how the at-risk figures relate to the enrollment of 127, so their relationship should not be inferred.

The serious adverse event terms listed are Spinal compression fracture (1.3%), Angina pectoris (0.6%), Angina unstable (0.6%), Atrial fibrillation (0.6%), Coronary artery occlusion (0.6%), and Myocardial ischaemia (0.6%). These are the terms and percentages the registry reports. The record does not provide event-level narratives, attribution details, or information needed to determine whether terms refer to distinct participants or events.

The deaths field is null, so a death count is not reported in the registry record; null should not be interpreted as zero. As a single-group study, the trial does not provide a concurrent control rate for contextual comparison. These summaries describe recorded events and cannot establish that degarelix caused them.

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

This trial’s design and outcome focus are relevant to interpreting its purpose: it was an open-label, single-group extension trial intended to describe long-term safety and tolerability of degarelix in Korean patients with prostate cancer. The primary endpoint was the number of participants with markedly abnormal safety laboratory values over treatment, from baseline (day 0) to end of treatment, up to day 364.

The primary numerical result is not reported in the registry record. This means the posted primary outcome information does not establish how many participants had markedly abnormal laboratory values, which variables accounted for findings, or how precise any estimate was. The endpoint description is not a substitute for those data. The registry’s adverse-event figures and serious-event terms offer separate descriptive information, but do not fill in the missing primary endpoint result.

The design also limits what can be concluded. Without a concurrent comparator, the trial cannot show whether the observed event experience differs from what would have occurred under another treatment or without treatment. The open-label design is another feature to consider when evaluating outcome assessment. Neither feature proves that the reported summaries are inaccurate; they define the scope of conclusions the design can support.

More broadly, descriptive safety data can characterize what was recorded among participants in a study, while causal claims require a design and analysis that can support comparison and address relevant sources of bias. The record does not report a comparative analysis, confidence intervals for the primary endpoint, or details of missing-data handling. Interpretation should therefore remain with the documented design and outcomes rather than extending to unreported effects.

The record lists serum PSA levels over time as a secondary outcome, but its numerical results are not reported in the registry record. No conclusion about that outcome can be drawn from its listing alone. The study’s stated safety focus and the available registry summaries do not establish efficacy or a treatment benefit.

13. Why This Trial Matters Statistically

This trial illustrates an important distinction between a safety question and a comparative treatment question. A single-group extension can describe laboratory abnormalities and adverse events recorded during treatment. It cannot, by itself, separate treatment-associated changes from the background course of illness, other care, or other influences. The registry’s allocation and model fields make this distinction central to interpreting the results.

It also shows why endpoint definitions, denominators, and analysis populations matter. The primary measure is a count of participants with markedly abnormal laboratory values, but its numerical result and denominator are not reported in the registry record. The safety population is named as the CS42 and CS42A safety analysis set, while the adverse-event summaries use at-risk figures whose relationship to enrollment is not explained. Those omissions limit assessment of both frequency and precision.

Safety summaries are not interchangeable. The primary endpoint concerns marked laboratory abnormalities, while the separately reported adverse-event figures concern serious and other adverse events. Each speaks to a different type of observation, and the record does not provide enough detail to merge them or infer causes. The listed serious-event terms and percentages are descriptive entries, not comparative estimates.

Finally, planning calculations should not be confused with study findings. The stated planning assumptions describe an illustration for a two-arm design, while this trial is single-group. Such an illustration is not observed power and does not change the design or supply missing outcome results. This is a useful reminder that a calculation’s assumptions must match the question and design before its interpretation can be meaningful.

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 primary limitation for interpretation is that the numerical result for the primary laboratory endpoint is not reported in the registry record. The record gives the endpoint definition and timeframe but not its count, denominator, or variable-level findings. Consequently, the frequency and precision of markedly abnormal laboratory values cannot be assessed from the primary outcome information.

The trial had a single group, with no randomized or concurrent comparator. Its findings are therefore descriptive and do not estimate a comparative treatment effect. The open-label design may also be relevant to how outcomes were assessed, although details of laboratory measurement, adjudication, and monitoring are not reported in the registry record.

The registry identifies the CS42 and CS42A safety analysis set but does not explain its membership or relationship to enrollment. It reports enrollment of 127, while the adverse event figures list 156 at risk; why these figures differ is not reported in the registry record. The death count is also not reported. Missing-data handling, participant follow-up completeness, and event attribution are not described.

The record lists four outcome measures but reports zero analyses. It does not describe a statistical analysis plan, multiplicity handling, confidence intervals, or methods for missing observations. Although the primary endpoint description says that only laboratory variables with at least one participant with an abnormal value are presented and that more variables were included, the available information does not permit an assessment of all laboratory findings. These limitations constrain interpretation of the registry record, rather than establishing that unreported analyses or findings were absent from the broader study materials.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Long-Term Safety and Tolerability of Degarelix One-Month Dosing Regimen in Korean Patients — NCT01215513.
• Registry record last updated 2013-05-22; status last verified 2013-04.
• Results first posted 2013-05-22.
• 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 record describes NCT01215513 as a completed phase 3, open-label, multi-centre, single-group extension trial of degarelix in Korean patients with prostate cancer requiring androgen ablation therapy. Enrollment is listed as 127. The study start is recorded as 2010-09 and primary completion as 2012-04.

The primary outcome is the number of participants with markedly abnormal safety laboratory values from baseline (day 0) to end of treatment, up to day 364. Its numerical result is not reported in the registry record. The record lists PSA levels over time as a secondary outcome and records four outcome measures overall, with zero analyses.

Adverse-event summaries report 156 at risk and 25 affected for serious events, and 156 at risk and 88 affected for other events. The relationship of those figures to enrollment is not explained. The record is descriptive and has no concurrent comparator, so it does not establish a comparative treatment effect.