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High-Risk Stage III Melanoma Phase 3 Randomized NCT00636168

EORTC 18071: Complete Statistical Analysis of Ipilimumab in High-Risk Stage III Melanoma

An independent statistical review of the randomized phase 3 EORTC 18071 trial evaluating ipilimumab versus placebo to prevent recurrence after complete resection of high-risk stage III melanoma.

Trial status: COMPLETED  ·  Enrollment: 1211  ·  Start: June 30, 2008  ·  Primary completion: July 26, 2013
Scope of this record

This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record.

1. Trial at a Glance

EORTC 18071 was a randomized, double-blind, phase 3 parallel-group trial evaluating ipilimumab versus placebo in participants with high-risk stage III melanoma after complete resection. The registry reports 1211 enrolled participants and two treatment arms.

1211
Enrolled
Phase 3 trial
2
Arms
Ipilimumab vs placebo
0.75
Primary RFS HR
95% CI 0.64–0.90
0.0013
Primary RFS P-value
Two-sided analysis
FeatureEORTC 18071
Trial nameEORTC 18071
ClinicalTrials.gov identifierNCT00636168
PhasePhase 3
ConditionHigh Risk Stage III Melanoma
AllocationRandomized
Design modelParallel
MaskingDouble
Primary purposeTreatment
Enrollment1211
InterventionsIpilimumab; placebo
Lead sponsorBristol-Myers Squibb
Sponsor typeIndustry
StatusCompleted

2. Clinical Question

The central statistical question was whether treatment with ipilimumab, compared with placebo, improved recurrence-related outcomes after complete resection in participants with high-risk stage III melanoma.

Population

Participants with high-risk stage III melanoma following complete resection.

Intervention

Ipilimumab, with the registry identifying the treatment group as ipilimumab 10 mg/kg in the posted statistical analyses.

Comparator

Placebo.

Primary question

Does ipilimumab improve recurrence-free survival and related recurrence outcomes compared with placebo?

3. Trial Design

01
Randomize 1211 participants
02
Two arms Ipilimumab vs placebo
03
Double-blind Masked treatment assignment
04
Follow Recurrence, death, disease assessment
05
Analyze RFS, DMFS, and OS
ARM A · IPILIMUMAB

Ipilimumab 10 mg/kg

  • Ipilimumab
  • Registry statistical analyses identify the comparison dose as 10 mg/kg
  • Evaluated against placebo in the randomized population
ARM B · PLACEBO

Placebo

  • Placebo
  • Served as the randomized comparator
  • Included in the same double-blind parallel design

The registry describes the allocation as randomized, the design model as parallel, and the masking as double. These design features are important statistically because randomization establishes the basis for comparing treatment groups, while double masking is intended to reduce the influence of treatment knowledge on trial conduct and assessment.

4. Trial Timing and Registry Status

June 30, 2008

Trial start

The registry lists June 30, 2008 as the study start date.

July 26, 2013

Primary completion

The registry lists July 26, 2013 as the primary completion date.

Completed

Current registry status

The trial is listed as completed.

5. Analysis Population and Stratification

The primary recurrence-free survival analysis was conducted in the intent-to-treat population: all randomized participants were analyzed in the treatment arm to which they were allocated by randomization.

Analysis featureRegistry-supported specification
Primary efficacy populationIntent-to-treat population: all randomized participants, analyzed according to randomized treatment assignment
Primary comparisonIpilimumab 10 mg/kg vs placebo
Primary time-to-event methodLog-rank test
Effect measureHazard ratio
Model for primary HRCox proportional-hazards model
StratificationStage at randomization

The primary Cox model was stratified by stage at randomization using four categories: IIIa, IIIb, IIIc with 1–3 positive lymph nodes, and IIIc with ≥4 positive lymph nodes. Treatment was the single covariate in the model.

Why ITT matters: analyzing participants according to their randomized assignment preserves the comparison created by randomization. It also means that the primary efficacy estimate is a comparison of randomized strategies rather than a comparison restricted to participants who remained on treatment.

6. Endpoints

Registered endpointTypeTime frame
Recurrence Free Survival (RFS) Per Independent Review Committee (IRC) in the Intent to Treat (ITT) Population Time-to-event Date of randomization to first date of recurrence or death or last available disease assessment with RFS data up to 5 years
Number of Participants With Recurrence or Death as Per Independent Review Committee (IRC) in the Intent to Treat (ITT) Population Binary Date of randomization to first date of recurrence or death or last available disease assessment with RFS data up to 5 years
Recurrence-Free Survival (RFS) Rates Per IRC at 1 Year, 2 Years, and 3 Years in the ITT Population Time-to-event At years 1, 2, and 3

How recurrence was defined

The registry defines recurrence as the appearance of one or more new melanoma lesions, including local, regional, or distant metastasis. CT and MRI were mandatory to establish recurrence. A participant who died without a reported recurrence was considered to have recurred on the date of death.

Disease was assessed at randomization and every 12 weeks (±2 weeks) for 3 years according to the registry definition of the recurrence endpoint.

How yearly RFS rates were estimated

The registered yearly RFS rates were estimated using the Kaplan-Meier product-limit method. The registry specifies corresponding log-log transformed 95% confidence intervals for these time-specific estimates.

Endpoint distinction: the three registered primary endpoints are related but are not interchangeable. RFS is a time-to-event endpoint, recurrence/death is a binary event count over the specified follow-up framework, and the 1-, 2-, and 3-year RFS rates are time-specific survival estimates.

7. Statistical Methodology

Kaplan-Meier estimation

RFS is a time-to-event endpoint, so the Kaplan-Meier product-limit estimator is appropriate for describing the probability of remaining recurrence-free over time while accounting for right-censoring.

Kaplan-Meier survival function
S(t) = ∏ti ≤ t (1 − di/ni)

Here, di is the number of events at event time ti, while ni is the number of participants at risk immediately before that time.

The important feature is that participants who have not yet experienced recurrence or death can contribute information until their last usable disease assessment. They do not simply disappear from the analysis at the first point at which follow-up ends.

Log-rank test

The registry reports a log-rank test as the primary comparison method. A log-rank test compares the observed and expected numbers of events between randomized groups across the observed follow-up period.

Conceptual comparison
Observed events  vs.  Expected events under the null hypothesis

The test addresses whether the time-to-event experience differs between treatment groups. It does not itself quantify the magnitude of the difference; the hazard ratio supplies that complementary effect measure.

Stratified analysis

The primary analysis was stratified by stage at randomization. Stratification allows the comparison to account for the prespecified stage categories while estimating the treatment effect across the randomized population.

Cox proportional-hazards model

The primary hazard ratio and its 95% confidence interval were estimated with a Cox proportional-hazards model. The model was stratified by stage and included treatment as the single covariate.

Hazard-ratio interpretation
HR = hazard under ipilimumab ÷ hazard under placebo

An HR below 1 indicates a lower estimated instantaneous event hazard in the ipilimumab group relative to placebo under the fitted model. It is not a direct percentage of patients who avoid recurrence.

Analysis timing

The primary RFS analysis was performed after 528 RFS events per IRC were reported. The registry identifies the analysis as using two-sided 95% confidence intervals.

8. Primary Results: Recurrence-Free Survival

The posted primary statistical analysis compares ipilimumab 10 mg/kg with placebo for recurrence-free survival in the ITT population. The analysis used a log-rank test, with the treatment hazard ratio estimated from a stratified Cox proportional-hazards model.

Recurrence-free survival hazard ratio

0.75

95% CI: 0.64–0.90   ·   P = 0.0013

Comparison: ipilimumab 10 mg/kg vs placebo

Primary endpointEffect estimate95% CIP-valueMethod
Recurrence Free Survival (RFS) Per IRC in the ITT Population HR 0.75 0.64–0.90 0.0013 Stratified log-rank test; stratified Cox model for HR
Clinical Biostats interpretation

An HR of 0.75 means that the estimated instantaneous rate of recurrence or death was approximately 25% lower in the ipilimumab group than in the placebo group under the fitted proportional-hazards model. The 25% figure is a relative hazard interpretation, not a statement that 25% of participants were protected from recurrence.

The estimate does not mean that every participant experienced a 25% reduction in individual risk, nor does it mean that recurrence was prevented in exactly 25% of participants. A hazard ratio summarizes relative event rates over follow-up within the statistical model.

The two-sided 95% confidence interval of 0.64–0.90 describes uncertainty around the estimated hazard ratio under the analysis framework. It is not a range containing the effects experienced by individual patients. Because the interval lies below 1, the estimated treatment effect is consistently in the direction of lower recurrence/death hazard across the interval.

The P-value of 0.0013 measures the strength of evidence against the relevant null hypothesis under the specified testing framework; it does not measure the size of the treatment effect. The HR and its confidence interval are needed to understand magnitude and precision.

The analysis also depends on the Cox proportional-hazards framework. If the proportional-hazards assumption were substantially violated, a single hazard ratio could provide an incomplete description of how treatment effects evolve over time. The ClinicalTrials.gov record does not provide a formal assessment of that assumption.

Why the primary analysis uses both a test and an effect estimate

The log-rank test and Cox model answer complementary questions. The log-rank test evaluates evidence that the event-time distributions differ, whereas the hazard ratio quantifies the relative event rate under the Cox model. Reporting both prevents a very small P-value from being mistaken for a measure of treatment magnitude.

9. Secondary Results: Distant Metastasis-Free Survival

The registry also posts a secondary analysis of distant metastasis-free survival (DMFS) in the ITT population. The analysis compares ipilimumab 10 mg/kg with placebo from June 2008 to January 2016, approximately 90 months.

Distant metastasis-free survival hazard ratio

0.76

95% CI: 0.64–0.92   ·   P = 0.0024

Confidence interval: 95.8%, two-sided

Secondary endpointEffect estimateCIP-valueAnalysis
Distant Metastasis-Free Survival (DMFS) Per IRC in the ITT Population HR 0.76 95.8% CI 0.64–0.92 0.0024 Stratified two-sided log-rank test; stratified analysis by stage
Statistical interpretation

The DMFS HR of 0.76 corresponds to an approximately 24% lower estimated instantaneous hazard of the DMFS event in the ipilimumab group relative to placebo under the reported hazard-ratio framework.

The 95.8% confidence interval of 0.64–0.92 quantifies uncertainty around that estimate. It does not mean that individual treatment effects fall between 0.64 and 0.92.

The P-value of 0.0024 provides evidence against the null hypothesis within the reported testing framework, but it should not be interpreted as a 0.24% probability that the null hypothesis is true, nor as a measure of the clinical magnitude of the effect.

The registry notes that medians and associated two-sided 95% confidence intervals were calculated using the method of Brookmeyer and Crowley. No median values are included in the ClinicalTrials.gov record, so they are not reproduced here.

10. Secondary Results: Overall Survival

Overall survival was also analyzed in the ITT population over the period from June 2008 to January 2016, approximately 90 months. The registry reports a stratified log-rank comparison and a hazard ratio for ipilimumab 10 mg/kg versus placebo.

Overall survival hazard ratio

0.72

95% CI: 0.58–0.88   ·   P = 0.0013

Confidence interval: 95.1%, two-sided

Secondary endpointEffect estimateCIP-valueAnalysis
Overall Survival in the ITT Population HR 0.72 95.1% CI 0.58–0.88 0.0013 Stratified two-sided log-rank test; stratified analysis by stage
Clinical Biostats interpretation

An OS HR of 0.72 means that the estimated instantaneous hazard of death was approximately 28% lower in the ipilimumab group than in the placebo group under the reported Cox model.

This does not mean that 28% of participants survived because of treatment, that an individual patient's probability of death fell by exactly 28%, or that survival time for every participant increased by a fixed percentage.

The 95.1% confidence interval of 0.58–0.88 gives the statistical uncertainty around the estimated hazard ratio. The width of the interval is important: the point estimate alone does not communicate precision.

The P-value of 0.0013 concerns the statistical evidence against the null hypothesis. P-values are not effect-size measures and should be interpreted alongside the hazard ratio and confidence interval.

As with the RFS and DMFS analyses, interpretation of a single Cox hazard ratio depends on the proportional-hazards framework. The ClinicalTrials.gov record does not report a separate diagnostic assessment of proportional hazards.

11. Comparing the Time-to-Event Results

EndpointRoleHRConfidence intervalP-value
Recurrence-free survival Primary 0.75 95% CI 0.64–0.90 0.0013
Distant metastasis-free survival Secondary 0.76 95.8% CI 0.64–0.92 0.0024
Overall survival Secondary 0.72 95.1% CI 0.58–0.88 0.0013

All three posted hazard-ratio estimates are below 1. That common direction is useful descriptively, but the endpoints answer different clinical questions. RFS incorporates recurrence or death, DMFS concerns distant metastatic events, and OS concerns death from any cause.

The estimates should therefore not be averaged, ranked, or treated as interchangeable measures of one underlying effect. Their different event definitions and follow-up frameworks mean that each hazard ratio belongs to its own endpoint analysis.

12. Recurrence-Free Survival Rates at 1, 2, and 3 Years

The registry lists recurrence-free survival rates at 1 year, 2 years, and 3 years as a primary endpoint. These rates were estimated separately for each treatment group using the Kaplan-Meier product-limit method, with log-log transformed 95% confidence intervals.

Primary endpointEstimation methodTime pointsConfidence interval method
RFS rates per IRC in the ITT population Kaplan-Meier product-limit method 1 year, 2 years, 3 years Log-log transformed 95% confidence intervals for median RFS were computed by the Brookmeyer and Crowley method using log-log transformation. stratified 2-sided log-rank test
Registry reporting boundary: the ClinicalTrials.gov record identifies these time-specific RFS rates as a registered and posted outcome, but do not provide the numerical treatment-group rates. They are therefore not reproduced or inferred here.

13. Statistical Methods Explained

Why was a log-rank test used?

RFS, DMFS, and OS are time-to-event endpoints. A log-rank test is designed to compare event-time experience between two groups while accounting for the timing of events and censoring. It is therefore more informative for these endpoints than a simple comparison of event proportions at one arbitrary time point.

What does an RFS hazard ratio of 0.75 mean?

Under the reported Cox model, an HR of 0.75 indicates an estimated instantaneous recurrence-or-death hazard that is 75% of the corresponding hazard in the placebo group. Expressed as a relative reduction, that is approximately 25%. It is not a 25-percentage-point difference in recurrence-free survival.

Why was the analysis stratified by stage?

Stage at randomization was used to define four strata: IIIa, IIIb, IIIc with 1–3 positive lymph nodes, and IIIc with ≥4 positive lymph nodes. Stratified analysis allows the treatment comparison to account for these prespecified stage categories rather than treating them as if their distributions were irrelevant to the survival comparison.

What does the confidence interval tell us?

A confidence interval describes uncertainty around an estimated treatment effect under the statistical model and sampling framework. For the primary RFS HR, the interval extends from 0.64 to 0.90. It does not describe the range of outcomes an individual patient might experience.

Why is the P-value not the treatment effect?

The P-value is evidence against a null hypothesis under the specified analysis. It is affected by the amount of information in the study as well as by the observed separation between groups. The hazard ratio describes relative effect magnitude, while its confidence interval communicates precision.

Why is ITT important in this trial?

The primary analysis used all randomized participants according to their randomized assignment. This preserves the treatment comparison generated by randomization and avoids redefining the efficacy population based on later treatment behavior.

Why are RFS, DMFS, and OS not interchangeable?

They use different event definitions. RFS includes recurrence or death, DMFS focuses on distant metastasis-free survival, and OS measures death from any cause. Even when their hazard ratios point in the same direction, the estimates describe different endpoints and should be interpreted separately.

14. Confidence Intervals and Statistical Precision

RFS

HR 0.75 with a 95% CI of 0.64–0.90. The interval provides the uncertainty range reported for the primary treatment-effect estimate.

DMFS

HR 0.76 with a 95.8% CI of 0.64–0.92. The registry reports a confidence level different from the primary RFS analysis.

Overall survival

HR 0.72 with a 95.1% CI of 0.58–0.88. The interval is wider than the RFS interval in absolute HR units.

What precision means

Precision concerns uncertainty around the estimated population effect. It does not tell us how variable treatment response is between individual patients.

It is important not to compare the confidence levels mechanically. The primary RFS analysis uses a 95% confidence interval, whereas the posted DMFS and OS analyses report 95.8% and 95.1% confidence intervals, respectively. Those differences should be retained rather than silently converted to a common confidence level.

15. The Role of Censoring in RFS Analysis

Time-to-event analyses frequently include participants who have not experienced the endpoint by the time their usable follow-up ends. Such participants are censored rather than treated as if they had experienced the event.

For RFS, the registered definition uses the time from randomization to first recurrence or death, or to the last available disease assessment with RFS data. This structure allows the Kaplan-Meier estimator and Cox model to use information from participants with different lengths of follow-up.

Censoring is not the same as cure. A censored participant has not contributed an observed recurrence or death by the relevant follow-up point. The statistical analysis does not establish that the participant will remain recurrence-free indefinitely.

16. The Cox Model and Its Assumption

The primary RFS hazard ratio was estimated using a Cox proportional-hazards model. The key interpretive feature is that the model summarizes the relative hazard through a single treatment-effect parameter.

Model concept
h(t | treatment) = h0(t) × exp(β treatment)

The hazard ratio is represented by exp(β). In a proportional-hazards model, the treatment effect is assumed to act multiplicatively on the hazard over time.

The registry supplies the HR estimate and model specification but does not report a separate proportional-hazards diagnostic in the ClinicalTrials.gov record. Therefore, the HR should be understood as the reported model-based summary rather than as proof that the proportional-hazards assumption is exactly true at every point in follow-up.

17. Analysis Population: Why Randomization Still Matters After Follow-Up Begins

Randomization protects the initial treatment comparison by assigning participants to groups without using later outcomes to determine assignment. The ITT analysis then retains those randomized assignments throughout the primary efficacy analysis.

ApproachQuestion it answers
ITT efficacy analysis What was the outcome associated with being randomized to ipilimumab rather than placebo?
Per-protocol or treatment-adherent analysis What happened among participants meeting additional treatment-adherence criteria?

The EORTC 18071 primary analysis reported here is explicitly ITT. That distinction is important because post-randomization treatment behavior should not be used to redefine the principal randomized comparison.

18. Safety Results

The registry provides serious adverse-event counts by treatment arm. The affected and at-risk counts are:

Treatment armParticipants with serious adverse eventsParticipants at risk
Ipilimumab 10 mg/kg 257 471
Placebo 128 474
Serious adverse events: affected / at risk
Ipilimumab 10 mg/kg
257 / 471
Placebo
128 / 474

The visual bars above use the affected-to-at-risk fractions represented by the registry counts. The page does not substitute a newly calculated percentage for the registry-reported counts; the underlying data are shown directly as affected participants over participants at risk.

Safety interpretation: these serious-adverse-event data are distinct from the efficacy analyses. They describe affected participants among those at risk and should not be combined with the RFS, DMFS, or OS hazard ratios to create a single summary measure.

19. What the Primary Hazard Ratio Does — and Does Not — Mean

Effect size

The primary RFS HR of 0.75 corresponds to an approximately 25% lower estimated instantaneous hazard of recurrence or death in the ipilimumab group relative to placebo under the reported Cox model.

Not an individual risk reduction

The HR does not mean that every participant had exactly a 25% lower probability of recurrence, nor does it mean that 25% of participants avoided recurrence because of treatment.

Precision

The 95% CI of 0.64–0.90 communicates uncertainty around the estimated HR. A confidence interval is not a prediction interval for individual patients.

P-value

The P-value of 0.0013 addresses evidence against the null hypothesis under the specified analysis. It does not quantify how large or clinically important the effect is.

20. Primary and Secondary Evidence in Context

Evidence layerEndpointWhat it contributes
Primary RFS Direct randomized comparison of recurrence-free survival using the ITT population.
Primary Recurrence or death Registered binary characterization of participants experiencing recurrence or death during the specified RFS framework.
Primary 1-, 2-, and 3-year RFS rates Time-specific Kaplan-Meier estimates of recurrence-free probability.
Secondary DMFS Time-to-event assessment focused on distant metastatic outcomes.
Secondary OS Time-to-event assessment based on death from any cause.

This hierarchy matters because an individual endpoint's P-value should not be interpreted without considering its role in the trial. Primary endpoints are the principal prespecified efficacy questions, while secondary endpoints provide additional evidence addressing related but distinct outcomes.

21. Multiplicity and Multiple Endpoints

The ClinicalTrials.gov record identifies 3 primary endpoints and 12 posted outcome measures. They also identify 1 primary- endpoint statistical analysis among the posted statistical analyses.

Registry featureReported valueStatistical implication
Primary endpoints 3 More than one primary endpoint creates a multiplicity consideration when interpreting the overall confirmatory evidence.
Outcome measures posted 12 The registry contains multiple efficacy and safety outcomes beyond the primary statistical analysis.
Statistical analyses posted 3 The registry contains three posted formal statistical analyses.
Primary-endpoint analyses 1 The ClinicalTrials.gov record identifies one posted statistical analysis as primary.

The ClinicalTrials.gov record does not describe an alpha-allocation procedure, gatekeeping hierarchy, Hochberg procedure, Bonferroni adjustment, or other formal multiplicity-control method. Accordingly, this page does not infer one.

Interpretive caution: three primary endpoints mean that the overall inferential structure is more complicated than a single endpoint with a single hypothesis test. Without the protocol or statistical analysis plan, the exact familywise-error strategy cannot be reconstructed from the registry fields alone.

22. Interim Analysis and Other Design Topics

Design topicWhat the ClinicalTrials.gov record supports
Non-inferiority marginNot reported in the ClinicalTrials.gov record; the registered hypothesis type is superiority.
CrossoverNot reported in the ClinicalTrials.gov record.
Factorial designNot reported; the design model is parallel.
Multiplicity procedureNot reported in the ClinicalTrials.gov record.
Interim analysisNot reported in the ClinicalTrials.gov record.
Missing-data/imputation procedureNot reported in the ClinicalTrials.gov record.
Bayesian methodsNot reported; the posted methods are frequentist log-rank/Cox survival analyses.
StratificationSupported: primary Cox analysis was stratified by stage at randomization.

These distinctions are important because a statistical-analysis page should not infer protocol features merely because they are common in phase 3 oncology trials. Only the design characteristics supported by the ClinicalTrials.gov record is described as trial-specific facts here.

23. Why the Log-Rank Test and Cox Model Work Together

The log-rank test and Cox model are often presented together because they perform different statistical jobs.

Log-rank test

Provides a formal comparison of the survival experience between treatment groups across follow-up.

Cox model

Provides a model-based estimate of the relative hazard and its confidence interval.

Kaplan-Meier

Describes the estimated event-free probability over time and supports time-specific RFS rates.

ITT population

Maintains the randomized treatment comparison for the primary efficacy analysis.

In EORTC 18071, these components form a coherent time-to-event analysis framework: Kaplan-Meier methods describe recurrence-free survival, the log-rank test compares randomized groups, and the stratified Cox model summarizes the treatment effect through the hazard ratio.

24. Interpreting the Three Posted Hazard Ratios

The three formal analyses reported in the ClinicalTrials.gov record all use the same broad survival-analysis family and compare ipilimumab 10 mg/kg with placebo in the ITT population.

EndpointHRRelative hazard interpretation
RFS 0.75 Approximately 25% lower estimated instantaneous recurrence/death hazard
DMFS 0.76 Approximately 24% lower estimated instantaneous event hazard
OS 0.72 Approximately 28% lower estimated instantaneous death hazard

These derived percentage interpretations follow directly from the reported hazard ratios and are useful for teaching. They should not be confused with absolute risk reductions, relative risks, or differences in survival probabilities at a fixed time point.

25. Limitations and Interpretation Issues

26. Why This Trial Matters Statistically

EORTC 18071 is a useful teaching example because it combines randomized treatment allocation, double masking, multiple time-to-event endpoints, ITT analysis, stratified survival methods, Kaplan-Meier estimation, log-rank testing, and Cox hazard-ratio estimation.

ConceptHow it appears in EORTC 18071
Randomization Participants were randomized between ipilimumab and placebo in a parallel-group design.
Double blinding The trial is registered as double masked.
ITT analysis The primary RFS analysis included all randomized participants according to randomized assignment.
Kaplan-Meier estimation Used to estimate yearly RFS rates at 1, 2, and 3 years.
Log-rank test Used for the primary RFS comparison and posted secondary time-to-event analyses.
Hazard ratio Used to quantify relative treatment effects for RFS, DMFS, and OS.
Cox model Used to estimate the primary RFS HR and its confidence interval.
Stratified analysis Primary Cox analysis was stratified by stage at randomization.
Multiple endpoints Three primary endpoints are registered, requiring careful attention to the inferential hierarchy.
Safety analysis Serious adverse events are reported by randomized treatment arm with affected and at-risk counts.

27. Statistical Concepts in This Trial

Learn more about the methods used in this trial:

28. Related Statistical Calculators

29. Sources

Continue with the Clinical Biostats methods pathway

Explore the statistical concepts behind randomized trials, survival analysis, hazard ratios, confidence intervals, and time-to-event endpoints.

30. Record Summary

EORTC 18071 provides a compact example of a randomized phase 3 time-to-event analysis. The ClinicalTrials.gov record describes a double-blind, parallel, randomized trial enrolling 1211 participants with high-risk stage III melanoma and comparing ipilimumab 10 mg/kg with placebo. The primary RFS analysis used the ITT population, a stratified log-rank test, and a stratified Cox proportional-hazards model. The reported RFS HR was 0.75 with a two-sided 95% CI of 0.64–0.90 and a P-value of 0.0013.

The registry also reports secondary DMFS and OS analyses. DMFS had an HR of 0.76 with a 95.8% CI of 0.64–0.92 and P = 0.0024. OS had an HR of 0.72 with a 95.1% CI of 0.58–0.88 and P = 0.0013. These estimates all describe different time-to-event endpoints and should therefore be interpreted individually rather than collapsed into one overall treatment statistic.

The most important statistical lesson is that the hazard ratio, confidence interval, P-value, Kaplan-Meier estimator, log-rank test, and ITT principle each answer different questions. Together they provide a more complete description of the randomized evidence than any single number could provide.

Clinical Biostats methodology: This page distinguishes registry-reported results from statistical interpretation. Numbers not present in the ClinicalTrials.gov record has not been reconstructed, rounded, inferred, or imported from other sources.