Extension Study Investigating the Long-Term Safety of Degarelix One-Month Depots in Patients With Prostate Cancer: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Extension Study Investigating the Long-Term Safety of Degarelix One-Month Depots in Patients With Prostate Cancer (NCT00117286), a non-randomized phase 2, phase 3 study. The primary outcome is Participants With Markedly Abnormal Change in Vital Signs and Body Weight.

2. Clinical Question

3. Trial Design

The registry describes an open-label, multicenter extension study of degarelix in participants with prostate cancer. It is listed as phase 2 and phase 3, with non-randomized allocation, no masking, and a parallel model. Enrollment was 57. The study began in 2005-03, primary completion was 2009-09, and its status is completed.

The extension, FE200486 CS14A, followed the main study FE200486 CS14. Participants who completed the 60 mg degarelix arm in the main study continued that dose into the extension; participants who completed the 80 mg arm likewise continued their dose. A protocol amendment in March 2006 changed the dosage to 160 mg for all participants. The descriptions specify concentrations of 20 mg/mL for the original doses and 40 mg/mL for the amended dose.

The brief summary says participants were to be treated until they were discontinued or withdrew from the study, or until a marketing authorization for degarelix had been obtained. The study was terminated when all ongoing participants had been treated for at least 5 years. These statements describe the planned continuation and the stated reason for termination; the registry record does not give individual treatment durations or explain how many participants discontinued or withdrew.

Because allocation was not randomized and there was no masking, the two dose-history groups should not be read as randomized treatment arms. The record does not report a concurrent untreated or active control group. It also does not report the study protocol’s rationale for the group structure, a sample-size justification, or a detailed statistical analysis plan.

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: 127 participants started and 87 participants completed the main CS14 study. Of these, 57 participants were recruited into the extension study CS14A and 34 participants signed the informed consent for dose shift.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: The data include data from participants participating in both the main study (FE200486 CS14) and the extension study FE200486 CS14A.

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 Participants With Markedly Abnormal Change in Vital Signs and Body Weight, measured over 5 years.

The primary outcome was the number of participants with markedly abnormal changes in vital signs or body weight. It covered systolic and diastolic blood pressure, pulse, and body weight, comparing measurements at the end of the trial with baseline. The outcome description restricts the count to participants whose baseline value was normal and whose post-baseline value was markedly abnormal. The stated timeframe was 5 years.

Counting participants with an event is a binary-outcome approach: for each person, the outcome is whether the specified event occurred. In general, such an endpoint can make safety monitoring interpretable as a count of people who crossed a defined threshold, rather than as an average change that could conceal different patterns among participants. That is a general explanation of the measure, not evidence about how the threshold was selected in this study.

The endpoint combines several kinds of measurements. The registry record does not specify the numerical thresholds defining “markedly abnormal,” give results for the individual components, or report the number of participants meeting the endpoint. It therefore does not permit interpretation of the observed frequency or comparison of component-specific patterns.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry describes the primary outcome as a count of participants who met a binary event definition. For each participant, the relevant classification is whether a normal baseline measurement was followed by a markedly abnormal post-baseline value in a listed vital sign or in body weight. This outcome structure differs from a continuous outcome, such as the mean change in a measurement. The record does not report the threshold values or the numerical endpoint results.

What does a participant count convey?

In general, a count conveys how many people met an outcome definition in a stated population. To interpret that count as a frequency, readers also need the number of people eligible for assessment and information about missing measurements. A count alone does not indicate how large a measurement change was, how long it persisted, or whether multiple measurements in one person contributed to the event definition. Those details are not reported for this endpoint in the registry record.

The outcome is a composite in the broad sense that it includes several measurement types: systolic and diastolic blood pressure, pulse, and body weight. In general, a composite can summarize multiple components in a single endpoint, but a combined result may not show which component contributed most. The registry record does not provide component-level results or the rule for combining components when a participant meets more than one criterion.

What can a confidence interval add?

In general, a confidence interval expresses the uncertainty around an estimate under the assumptions of the analysis. It can show which values are reasonably compatible with the observed data and method. A narrow interval generally conveys greater precision than a wide interval, while neither an interval nor a p-value establishes that a difference is clinically important. No confidence interval or endpoint estimate is reported in the registry record, so the precision of the primary outcome cannot be evaluated.

What do the group labels mean?

The group labels represent the dose participants completed in FE200486 CS14, followed by the extension’s dose change. Participants in the 60 mg-to-160 mg group continued 60 mg before the amendment, and participants in the 80 mg-to-160 mg group continued 80 mg before the amendment; the amendment changed the dose to 160 mg for all participants. These are dose histories, not two unchanging treatment conditions over the full follow-up.

In general, randomization is used to balance measured and unmeasured characteristics across treatment groups on average. This study is explicitly non-randomized, so that protection does not apply. The registry’s skip note also cautions that the arms are not a like-for-like control comparison. Any group-level contrast would need careful interpretation, and the record does not report an adjusted estimate or a method intended to account for group differences.

What do the planning assumptions mean?

The registry’s planning assumptions describe an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The effect to detect is expressed as a fraction of the observed effect. In general, a planning illustration asks how many participants per arm might be needed under stated assumptions; it is not a report of how many participants were actually observed or an estimate of the probability that a study result is true.

Any sample-size illustration is a planning exercise, not observed power. Its interpretation depends on the assumed effect and analysis method. The registry record does not report the study’s own sample-size justification or establish that these planning assumptions were used to design the trial. No numerical planning outputs are stated in the record, so no specific sample-size requirement can be interpreted.

Over-dispersion is a general concern when event-count data vary more than a simple statistical model expects. In such settings, standard errors from an overly restrictive model can be too small, making uncertainty look understated. The primary outcome is described as a participant count for a binary event, but the registry record does not report a model for that endpoint or an over-dispersion assessment. It would therefore be inappropriate to claim that a particular count model or correction was used.

The record lists no analyses. That does not establish that investigators performed no analyses; it means that analysis results and details are not reported in the registry record. In particular, it gives no method for handling multiple endpoints, missing measurements, repeated observations, or adjustment for baseline differences.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists two outcome measures but no analyses. It does not report a multiplicity plan, such as whether one outcome was designated as the sole basis for formal inference or whether results were adjusted for examining multiple outcomes. In general, examining several outcomes can increase the chance of a nominally notable result occurring by chance. The appropriate interpretation depends on the prespecified analysis plan, which is not reported in the registry record.

The primary outcome description says the population includes participants from both the main study and the extension, but it does not provide the number analyzed for this endpoint or the number with missing measurements. The record does not report how missing measurements, withdrawal, discontinuation, or incomplete follow-up were handled in the endpoint analysis. It also does not describe imputation, exclusions, or sensitivity analyses.

In general, missing data can affect an estimate when the likelihood of missingness is related to participants’ outcomes or other characteristics. The direction and size of any effect depend on the reasons for missingness and the analysis assumptions. Without counts and methods for missing endpoint data, those issues cannot be assessed for this study.

The record does not report an intention-to-treat, per-protocol, or other named analysis population for the primary endpoint. Its description of the endpoint population includes data from both studies, but that is not enough to determine exactly who contributed to each reported result. No endpoint-specific inferential results are available in the registry record.

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: pneumonia (10.0%), hiatus hernia (6.7%), coronary artery disease (3.7%), vertigo (3.7%), inguinal hernia, obstructive (3.7%).

The serious adverse-event figures are separate from the primary endpoint on markedly abnormal vital signs and body weight. For Degarelix (60 mg to 160 mg), the record lists 30 participants at risk and 11 affected by serious adverse events. For Degarelix (80 mg to 160 mg), it lists 27 at risk and 7 affected. The corresponding figures for other adverse events are 30 at risk and 29 affected in the first group, and 27 at risk and 26 affected in the second group.

The registry also lists serious adverse-event terms with percentages: Pneumonia, 10.0%; Hiatus hernia, 6.7%; Coronary artery disease, 3.7%; Vertigo, 3.7%; Inguinal hernia, obstructive, 3.7%; and Nausea, 3.7%. The record does not explain the denominators or provide further detail about these terms in the registry record, so the percentages should be read as registry-listed figures rather than reinterpreted as rates for a different population or period.

These group counts do not establish a comparative treatment effect. Allocation was non-randomized, the groups followed different prior doses, and all participants’ dosage changed under the amendment. Deaths are not reported in the registry record.

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

The record describes an extension study intended to investigate long-term safety and tolerability of degarelix in participants with prostate cancer. Its primary outcome focused on markedly abnormal changes in vital signs and body weight relative to baseline over a stated timeframe of 5 years. That focus is distinct from demonstrating comparative efficacy or establishing that one dose history is safer than another.

The numerical results for the primary endpoint are not reported in the registry record. Without the endpoint counts, denominators, and uncertainty estimates, readers cannot determine how frequently the specified changes occurred or how precisely any group difference was estimated. The serious adverse-event counts provide safety information, but they concern a different outcome and do not fill that gap.

The two groups were not randomized controls. Participants entered the extension after completing different dose arms in the main study, and the dose for all study participants changed to 160 mg after the March 2006 amendment. Thus, the group names describe treatment histories, not sustained exposure to two distinct doses throughout the extension. A difference between groups would not, by itself, demonstrate a causal effect of dose.

In general, a safety count is evidence about the events as defined and recorded in a study population; it is not automatically a complete account of all possible harms or a prediction for every person. Interpretation also depends on follow-up, event definitions, outcome ascertainment, and missing data. Several of those details are not reported in the registry record.

The posted information can support an account of the study design and the adverse-event figures explicitly listed. It does not support a conclusion about the unreported primary endpoint results, a randomized comparison, or medical advice. It also does not establish that degarelix does or does not produce a particular clinical outcome.

13. Why This Trial Matters Statistically

Statistically, the study illustrates the distinction between an extension cohort and a randomized controlled comparison. Its participants came from a prior study, and the registry identifies the extension’s allocation as non-randomized. This structure is relevant when considering group differences: statistical adjustment, even if reported, would not turn a non-randomized comparison into a randomized one.

The protocol amendment also matters for interpretation. The two group labels preserve the prior 60 mg and 80 mg dose histories, but the record says the dose changed to 160 mg for all participants in March 2006. Long-term group summaries would therefore describe histories that include a shared later dose, not a direct comparison of continuous exposure to the original doses.

The primary endpoint uses a threshold-based count across vital signs and body weight. In general, such endpoints are useful for describing the occurrence of specified changes, but interpretation depends on the threshold definition, denominator, and handling of multiple measurements. The record does not report the threshold details or endpoint results, which makes those issues especially important when judging what can be learned.

The study’s intended long follow-up is reflected in its stated 5-year timeframe and the reason given for termination: ongoing participants had been treated for at least 5 years. However, the registry record does not give individual follow-up durations or the primary outcome counts. The safety question is clear, while the available record does not permit assessment of the primary outcome’s frequency or precision.

Finally, the record distinguishes serious adverse events from other adverse events and lists several serious-event terms. These figures offer a descriptive view of recorded safety events, but the non-randomized design and absence of an appropriate control comparison limit causal interpretation. The example underscores why design, endpoint definition, and reporting completeness must be considered alongside event counts.

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 principal limitation for interpreting the primary outcome is that its numerical results are not reported in the registry record. The record does not state the number of participants meeting the endpoint, endpoint-specific denominators, confidence intervals, or p-values. It also does not give the thresholds used to define a markedly abnormal measurement or break the result down by blood pressure, pulse, and body weight.

The non-randomized, open-label design limits comparisons between the two dose-history groups. Participants entered the extension after completing different arms of the main study, and a March 2006 amendment changed the dosage to 160 mg for all participants. The groups therefore should not be treated as randomized controls or as two fixed-dose groups across the full study.

Because the study was an extension, the participants described were those who completed the main study and continued into FE200486 CS14A. The record does not report how this continuation affected the characteristics of the population available for follow-up. It also does not give individual follow-up duration or the number of participants who discontinued or withdrew, although the brief summary says treatment was to continue until discontinuation, withdrawal, or marketing authorization.

Missing-data methods, analysis populations, multiplicity procedures, and a detailed statistical plan are not reported. The registry lists no analyses, which leaves no reported inferential method to assess. The serious adverse-event figures are descriptive and do not provide a randomized basis for comparing the groups. These limitations restrict conclusions to the study description and the specific safety counts reported.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Extension Study Investigating the Long-Term Safety of Degarelix One-Month Depots in Patients With Prostate Cancer — NCT00117286.
• Registry record last updated 2025-12-18; status last verified 2010-09.
• Results first posted 2010-10-21.
• 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

NCT00117286 is a completed, open-label, multicenter, non-randomized extension study of degarelix in participants with prostate cancer. It followed FE200486 CS14 and included dose-history groups labeled Degarelix (60 mg to 160 mg) and Degarelix (80 mg to 160 mg). A March 2006 amendment changed the dosage to 160 mg for all participants. Enrollment was 57; the study began in 2005-03 and had primary completion in 2009-09.

The primary outcome was the number of participants with markedly abnormal post-baseline changes in blood pressure, pulse, or body weight among those with normal baseline values, with a stated timeframe of 5 years. Its numerical results and statistical analyses are not reported in the registry record. Serious adverse-event counts are reported for both groups, but the non-randomized design does not support treating them as a controlled comparison. The study was terminated when all ongoing participants had been treated for at least 5 years.