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Acute Coronary Syndrome Phase 2 Randomized NCT02293395

GEMINI-ACS-1: Complete Statistical Analysis of Rivaroxaban in Acute Coronary Syndrome

An independent statistical analysis of the randomized, double-blind phase 2 GEMINI-ACS-1 trial comparing rivaroxaban with acetylsalicylic acid in addition to P2Y12 inhibitor therapy in participants with acute coronary syndrome.

Trial start: 2015-04-20  ·  Primary completion: 2016-10-14  ·  Enrollment: 3037
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

GEMINI-ACS-1 was a randomized, double-blind, parallel phase 2 trial evaluating the safety of rivaroxaban versus acetylsalicylic acid when used in addition to either clopidogrel or ticagrelor therapy in participants with acute coronary syndrome.

3037
Enrollment
Participants
4
Arms
Parallel design
1.09
Hazard Ratio
95% CI 0.8–1.5
0.584
P-value
Two-sided
FeatureGEMINI-ACS-1
Trial nameGEMINI-ACS-1
NCT IDNCT02293395
Therapeutic areaCardiovascular
ConditionAcute Coronary Syndrome
PhasePhase 2
StatusCompleted
Enrollment3037
AllocationRandomized
Design modelParallel
MaskingDouble
Primary purposeTreatment
Lead sponsorJanssen Research & Development, LLC
Sponsor typeIndustry

2. Clinical Question

The registered trial question was whether rivaroxaban, used in addition to P2Y12 inhibitor therapy, could be compared with acetylsalicylic acid used in addition to P2Y12 inhibitor therapy with respect to the occurrence of non coronary artery bypass graft-related Thrombolysis in Myocardial Infarction (TIMI) clinically significant bleeding events in participants with acute coronary syndrome.

Population

Participants with acute coronary syndrome.

Intervention

Rivaroxaban in addition to P2Y12 inhibitor therapy.

Comparator

Acetylsalicylic acid in addition to P2Y12 inhibitor therapy.

Primary question

How do rivaroxaban and acetylsalicylic acid compare for the registered bleeding endpoint over follow-up of up to 390 days?

3. Trial Design

01
Randomize3037 participants
02
Double-blindMasked treatment allocation
03
ParallelFour trial arms
04
FollowUp to 390 days
05
AnalyzeTime-to-event endpoint
ANTITHROMBOTIC COMPARISON

Rivaroxaban

  • Rivaroxaban 2.5 mg twice daily (BID)
  • Used in addition to P2Y12 inhibitor therapy
  • 1510 participants were at risk for the reported serious-adverse-event measure
ANTIPLATELET COMPARISON

Acetylsalicylic acid

  • Acetylsalicylic acid 100 mg once daily (OD)
  • Used in addition to P2Y12 inhibitor therapy
  • 1506 participants were at risk for the reported serious-adverse-event measure

The trial also included clopidogrel and ticagrelor as interventions. Importantly, the registry states that randomization was not performed for use of the P2Y12 inhibitor. Consequently, comparisons across clopidogrel and ticagrelor strata are not randomized treatment comparisons.

Design caveat: The registry specifically states that randomization was not performed for use of P2Y12 inhibitor and that strata comparisons between bleeding and efficacy endpoints are confounded and were not planned. This is important when interpreting the four-arm description: the randomized comparison reported here is rivaroxaban versus acetylsalicylic acid, rather than a randomized factorial comparison of all combinations of the listed interventions.

4. Trial Timeline

2015-04-20

Study start

The registered study start date was 2015-04-20.

2016-10-14

Primary completion

The registered primary completion date was 2016-10-14.

Completed

Registry status

The study is listed as completed, with results posted on ClinicalTrials.gov.

5. Primary Endpoint

EndpointRegistry definition / time frameStatistical analysis
Number of Participants With Non Coronary Artery Bypass Graft-Related (Non CABG-related) Thrombolysis in Myocardial Infarction (TIMI) Clinically Significant Bleeding Events From start of study treatment until follow-up (up to 390 days) Log-rank test; hazard ratio

The registry classifies the primary endpoint as binary in its registered endpoint information, while the posted statistical analysis treats the outcome as a time-to-event endpoint and uses a log-rank test with a hazard ratio. The latter is the analysis framework directly represented by the posted statistical analysis.

What counted as clinically significant bleeding?

The registry defines non CABG-related TIMI clinically significant bleeding events as the sum of non CABG-related TIMI major bleeding events, TIMI minor bleeding events, and TIMI bleeding events requiring medical attention.

The registered definition identifies major bleeding as including symptomatic intracranial bleeding and clinically overt signs of hemorrhage with a hemoglobin drop of greater than or equal to 5 gram per deciliter (g/dl), among other criteria contained in the registry definition.

Endpoint interpretation: This is a clinically defined bleeding outcome rather than a simple count of any bleeding episode. Because the posted statistical analysis uses time-to-event methods, the relevant comparison concerns the timing of clinically significant bleeding events during the specified follow-up, together with censoring of participants who do not experience the event during their observed period.

6. Statistical Methodology

Log-rank test

The posted analysis used the log-rank test to compare the time-to-event experience between the rivaroxaban and acetylsalicylic acid groups. The log-rank test is designed for comparing survival or event-time distributions when some participants are censored before experiencing the event.

Conceptual comparison
H0: the event-time distributions are the same between randomized treatment groups

The log-rank test evaluates whether the observed pattern of events over follow-up differs between groups, accounting for the timing of events rather than reducing the endpoint to a simple yes/no proportion at a single time point.

Hazard ratio

The effect measure reported for the primary analysis was the hazard ratio (HR). The estimated hazard ratio for rivaroxaban versus acetylsalicylic acid was 1.09.

Interpretation of the reported effect measure
HR = 1.09

An HR of 1 would correspond to equal estimated hazards under the model. An HR of 1.09 corresponds to an estimated hazard that is 9% higher in the rivaroxaban group relative to the acetylsalicylic acid group, within the model and analysis framework used for the comparison.

Confidence interval

The reported 95% confidence interval was 0.8 to 1.5. A confidence interval gives a measure of statistical precision around the estimated hazard ratio. It is not a range containing 95% of individual patient outcomes.

P-value

The reported two-sided p-value was 0.584. In the context of the registered superiority hypothesis, this is the probability measure produced by the specified statistical test under the null hypothesis; it is not a measure of the size, clinical importance, or probability of the observed effect.

Analysis population

The registry describes the analysis population as including all randomized participants who had received at least one dose of study agent and had events occurring between randomization and the last dose of the study agent plus the applicable follow-up described in the registry analysis record.

Analysis-population caution: This population description is not identical to a simple statement that every randomized participant was analyzed regardless of treatment exposure. The registry specifically includes the requirement that participants had received at least one dose of study agent. That distinction should be retained when interpreting the posted analysis.

7. Primary Result

Non CABG-related TIMI Clinically Significant Bleeding Events

Hazard ratio for clinically significant bleeding

1.09

95% CI: 0.8–1.5   ·   P = 0.584

Rivaroxaban 2.5 mg BID vs acetylsalicylic acid 100 mg OD

Primary endpointComparisonMethodEffect estimate95% CIP-value
Number of Participants With Non CABG-related TIMI Clinically Significant Bleeding Events Rivaroxaban 2.5 mg BID vs Acetylsalicylic Acid 100 mg OD Log-rank HR 1.09 0.8–1.5 0.584
Clinical Biostats interpretation

The estimated hazard ratio of 1.09 means that the fitted relative event rate for non CABG-related TIMI clinically significant bleeding was estimated to be 9% higher with rivaroxaban than with acetylsalicylic acid in this randomized comparison.

That estimate does not mean that 9% more participants experienced bleeding, nor does it mean that an individual participant had a 9% higher probability of bleeding. A hazard ratio is a relative time-to-event measure, not an absolute risk difference or risk ratio.

The 95% CI of 0.8–1.5 indicates substantial uncertainty around the point estimate. The interval includes 1, so the data represented by this analysis are compatible with a range of relative hazard values on both sides of equal hazard.

The p-value of 0.584 does not quantify the magnitude of the treatment difference. It describes the statistical evidence against the null hypothesis under the specified two-sided log-rank testing framework. It should therefore be read alongside the HR and confidence interval rather than as a standalone measure of effect.

Because the endpoint was analyzed as a time-to-event outcome, interpretation also depends on the event timing, censoring, and the assumptions underlying the hazard-based representation. A single HR summarizes a complex event-time process and should not automatically be interpreted as a constant difference in risk at every point in follow-up.

Educational note: a Kaplan-Meier curve is not reconstructed here because the ClinicalTrials.gov record contains the hazard ratio, confidence interval, p-value, and endpoint definition but do not provide the underlying individual event and censoring times needed to construct a valid curve.

8. Safety Results

The ClinicalTrials.gov record reports serious adverse events by arm for the primary randomized treatment comparison.

Serious adverse-event measureAffected / at risk
Rivaroxaban 2.5 mg Twice Daily (BID)125 / 1510
Acetylsalicylic Acid 100 mg Once Daily138 / 1506

These counts describe affected participants relative to the participants at risk in the corresponding groups. They should not be substituted for the primary bleeding endpoint: serious adverse events and non CABG-related TIMI clinically significant bleeding are distinct outcome concepts in the ClinicalTrials.gov record.

Rivaroxaban

125 affected participants among 1510 participants at risk for the reported serious-adverse-event measure.

Acetylsalicylic acid

138 affected participants among 1506 participants at risk for the reported serious-adverse-event measure.

Do not conflate safety measures: The serious-adverse-event counts above are not the same endpoint as the formally analyzed non CABG-related TIMI clinically significant bleeding endpoint. The ClinicalTrials.gov record provides a formal time-to-event analysis for the latter but do not provide a formal statistical comparison of these serious-adverse-event counts.

9. Statistical Methods Explained

Why was a log-rank test used?

The primary outcome was analyzed as a time-to-event endpoint, with follow-up extending from the start of study treatment until follow-up of up to 390 days. A log-rank test is suited to comparing event-time distributions between randomized groups when participants may have different observed follow-up times because of censoring.

What does a hazard ratio of 1.09 mean?

An HR of 1.09 means that the estimated hazard in the rivaroxaban group was 1.09 times the estimated hazard in the acetylsalicylic acid group under the analysis model. Equivalently, it represents an estimated 9% higher hazard. It does not directly give the percentage of participants who experienced bleeding.

Why does the confidence interval matter?

The point estimate is only one estimate of the treatment contrast. The 95% CI of 0.8–1.5 shows the uncertainty around that estimate. Because the interval spans 1, the registry-reported analysis does not establish a statistically distinguishable difference from equal hazard under the two-sided superiority framework.

Why does the p-value not measure effect size?

The p-value of 0.584 describes the statistical evidence against the null hypothesis under the specified test. It does not tell us that the treatment effect is "58.4%" or that there is a 58.4% probability that the treatments are equivalent. Effect magnitude is described by the HR, while its uncertainty is described by the confidence interval.

Why is randomization important here?

Randomization is the design feature that supports a causal comparison between the randomized treatment groups, subject to the trial's eligibility criteria, conduct, analysis population, and other limitations. It is different from the nonrandomized choice of P2Y12 inhibitor described in the registry.

Why should clopidogrel and ticagrelor not be compared as if they were randomized arms?

The registry explicitly states that randomization was not performed for use of the P2Y12 inhibitor. Therefore, differences between participants receiving clopidogrel and ticagrelor can be confounded by factors associated with how that therapy was selected. The registry also states that such strata comparisons were confounded and were not planned.

What does "double-blind" contribute statistically?

Double masking reduces the opportunity for knowledge of treatment assignment to influence trial conduct and assessment. In a trial involving a clinically defined bleeding endpoint, maintaining masking can help reduce differential assessment or management related to knowledge of assigned treatment.

10. Confidence Intervals and the Superiority Question

The registered hypothesis type for the posted primary analysis is superiority. The reported hazard ratio of 1.09 is therefore interpreted as an estimate of the relative event hazard under the superiority comparison.

Primary estimate
HR = 1.09    95% CI = 0.8–1.5

The point estimate lies above 1, but the confidence interval extends below and above 1. The registry-reported p-value is 0.584 from a two-sided log-rank analysis.

This distinction is important. A point estimate above 1 is not, by itself, evidence of a statistically established difference. Conversely, a confidence interval that includes 1 does not prove that the two treatments have exactly identical effects. It indicates that the data are compatible with equal hazard as well as with values on either side of 1 within the interval.

11. Time-to-Event Analysis in GEMINI-ACS-1

The primary endpoint combines two pieces of information: whether a clinically significant bleeding event occurred and when it occurred during the observed follow-up. That makes the posted analysis different from simply calculating the percentage of participants who experienced an event by a fixed calendar date.

Event timing

The analysis retains information about when clinically significant bleeding events occurred during follow-up.

Censoring

Participants who do not experience the event during their observed period can contribute information before their censoring time.

Log-rank comparison

The test compares the event-time experience between the two randomized treatment groups.

Hazard ratio

The HR summarizes the relative hazard between the rivaroxaban and acetylsalicylic acid groups.

Why not reduce the result to a single percentage?

A fixed-time percentage can be useful, but it can discard information about event timing and follow-up. The posted GEMINI-ACS-1 analysis instead uses a survival-analysis framework, which is consistent with a trial in which participants may have different amounts of observed follow-up.

12. Randomization and the P2Y12 Inhibitor Issue

the ClinicalTrials.gov record identifies the overall allocation as randomized and the design as parallel. At the same time, the registry provides an important qualification: randomization was not performed for use of the P2Y12 inhibitor.

FeatureWhat the ClinicalTrials.gov record supports
Overall allocationRandomized
Design modelParallel
MaskingDouble
Rivaroxaban vs acetylsalicylic acidRandomized treatment comparison represented in the posted primary analysis
P2Y12 inhibitor selectionNot randomized
Clopidogrel vs ticagrelor strataConfounded comparison; registry states strata comparisons were not planned

This distinction is central to interpreting the trial's four listed interventions. The presence of four intervention names does not mean that every pairwise contrast among them represents a randomized causal comparison.

Statistical principle: Randomization supports causal interpretation for the treatment assignment that was randomized. It does not automatically make every post-randomization, nonrandomized, or stratification-based comparison causal.

13. What the Primary Result Does — and Does Not — Mean

What the HR means

The HR of 1.09 is a relative time-to-event estimate comparing rivaroxaban 2.5 mg BID with acetylsalicylic acid 100 mg OD for the registered non CABG-related TIMI clinically significant bleeding endpoint.

What the HR does not mean

It does not mean that 9% of participants experienced bleeding, that the absolute bleeding probability increased by 9 percentage points, or that every participant had exactly the same relative change in risk.

What the confidence interval says

The 95% CI of 0.8–1.5 indicates uncertainty around the estimated HR. It includes values below 1, equal to 1, and above 1, so the point estimate should not be treated as a precise determination of the direction or magnitude of the underlying treatment effect.

What the p-value says

The two-sided p-value of 0.584 is a test statistic summary under the specified null hypothesis. It does not measure clinical relevance, equivalence, or the probability that the null hypothesis is true.

14. Safety and Efficacy Are Different Statistical Questions

GEMINI-ACS-1 illustrates why a trial can have more than one important statistical question even when the primary endpoint is focused on safety.

QuestionSupplied dataStatistical interpretation
Primary bleeding endpointHR 1.09; 95% CI 0.8–1.5; P = 0.584Formal time-to-event comparison using a log-rank test
Serious adverse events125/1510 vs 138/1506Counts by arm are reported; no formal comparison is reported

The distinction matters because the primary endpoint has an explicitly posted inferential analysis, whereas the registry-reported serious-adverse-event information consists of affected and at-risk counts. It would be inappropriate to attach the primary endpoint's HR or p-value to the serious-adverse-event counts.

15. Limitations

16. Why This Trial Matters Statistically

GEMINI-ACS-1 is a useful teaching case because it shows how a randomized trial can combine a safety-focused endpoint, survival-analysis methodology, masked treatment assignment, and an important distinction between randomized and nonrandomized treatment factors.

ConceptHow it appears in GEMINI-ACS-1
RandomizationThe trial allocation is randomized for the treatment comparison represented in the primary analysis.
BlindingThe trial is double-masked.
Parallel designThe registry identifies a parallel design model.
Time-to-event endpointThe posted primary analysis treats the bleeding endpoint as a time-to-event outcome.
Kaplan-Meier frameworkThe primary endpoint is analyzed in a survival-analysis framework, for which Kaplan-Meier estimation is the standard descriptive companion.
Log-rank testThe registry-posted statistical method is a log-rank test.
Hazard ratioThe treatment effect is reported as an HR of 1.09.
Confidence intervalThe 95% CI is 0.8–1.5.
P-valueThe reported two-sided p-value is 0.584.
Superiority testingThe posted hypothesis type is superiority.
Nonrandomized factorP2Y12 inhibitor use was not randomized, limiting interpretation of strata comparisons.
GeneralizabilityThe registry identifies a homogeneous population with limited participation of non-Caucasian participants.

17. A Practical Reading of the Statistical Result

A disciplined interpretation of the primary result can be organized into four layers.

1. Effect estimate

The HR was 1.09 for rivaroxaban versus acetylsalicylic acid.

2. Precision

The 95% CI was 0.8–1.5, indicating uncertainty around the point estimate.

3. Hypothesis test

The two-sided log-rank p-value was 0.584 under the registered superiority framework.

4. Design context

The comparison was randomized and double-blind, while P2Y12 inhibitor use was not randomized.

This four-part reading is more informative than simply reporting that the p-value was above or below a conventional threshold. It keeps the estimated effect, its uncertainty, the inferential test, and the design context connected.

18. How to Read the Serious-Adverse-Event Counts

The ClinicalTrials.gov record reports 125 affected participants among 1510 at risk in the rivaroxaban group and 138 among 1506 at risk in the acetylsalicylic acid group.

Reported safety data
Rivaroxaban: 125 / 1510    ·    Acetylsalicylic acid: 138 / 1506

These are descriptive affected/at-risk counts. The ClinicalTrials.gov record does not provide a formal hypothesis test, confidence interval, or hazard ratio for this serious-adverse-event measure.

It is therefore appropriate to report the counts as reported in the registry, while avoiding the appearance that they represent the formal statistical analysis of the primary endpoint. The primary analysis is specifically the log-rank comparison of non CABG-related TIMI clinically significant bleeding events.

19. Statistical Interpretation vs Clinical Interpretation

Statistical interpretation

The posted analysis estimated an HR of 1.09 with a 95% CI of 0.8–1.5 and a two-sided p-value of 0.584 using a log-rank test.

Clinical interpretation

The ClinicalTrials.gov record describes the relative time-to-event comparison and selected serious-adverse-event counts, but they do not provide enough additional outcome information to construct a broader clinical benefit-risk assessment.

The distinction is deliberate. Statistical evidence describes the observed randomized comparison under a specified analysis. A broader clinical interpretation would require considering the full set of efficacy, safety, follow-up, and patient-level outcomes, and those additional results are not included in the ClinicalTrials.gov record.

20. What Is Not Supported by the Supplied Data

The ClinicalTrials.gov record does not provide results for additional endpoints beyond the posted primary analysis and the reported serious-adverse-event counts. Accordingly, this page does not introduce median event times, subgroup effect estimates, Kaplan-Meier percentages, additional p-values, additional confidence intervals, or other outcome measures from outside sources.

Data boundary: The primary analysis in the ClinicalTrials.gov record contains one formal statistical comparison. The absence of an additional numerical result in this page is intentional: numbers are not inferred from publications, memory, or external trial reports when they are not contained in the ClinicalTrials.gov record.

21. Related Tutorials

Learn more about the methods used in this trial:

22. Related Calculators

23. Sources

Continue through the Clinical Biostats statistical pathway

Connect this trial's randomized design, time-to-event endpoint, hazard ratio, confidence interval, and log-rank analysis to deeper statistical tutorials and calculation tools.

24. Record Summary

GEMINI-ACS-1 provides a useful example of how a randomized, double-blind phase 2 trial can be analyzed when its primary endpoint is represented as a time-to-event outcome. The registry analysis compares rivaroxaban 2.5 mg twice daily with acetylsalicylic acid 100 mg once daily using a log-rank test and reports a hazard ratio of 1.09, a two-sided 95% confidence interval of 0.8–1.5, and a p-value of 0.584. The most appropriate reading of that result keeps the effect estimate, its uncertainty, and the hypothesis test together rather than reducing the analysis to the p-value alone.

The trial also illustrates an important design principle: overall randomization does not make every treatment factor randomized. The registry states that P2Y12 inhibitor use was not randomized and that comparisons between clopidogrel and ticagrelor strata were confounded and not planned. Finally, the limited participation of non-Caucasian participants is an explicit limitation on generalizability.

Clinical Biostats methodology: A trial-results page should distinguish reported evidence from statistical interpretation. For GEMINI-ACS-1, the formal primary analysis is the posted log-rank comparison of the registered non CABG-related TIMI clinically significant bleeding endpoint. Other the ClinicalTrials.gov record is presented descriptively rather than being assigned an analysis that the registry data do not report.