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Carotid Stenosis Prevention Completed NCT02089217

CREST-2: Complete Statistical Analysis of Carotid Revascularization and Medical Management in Carotid Stenosis

An independent statistical review of CREST-2, a randomized clinical trial evaluating carotid endarterectomy, carotid stenting, and intensive medical management in patients with carotid stenosis.

Study start: December 9, 2014  ·  Primary completion: September 12, 2025  ·  Sponsor: Mayo Clinic
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

CREST-2 is a randomized, parallel-group clinical trial evaluating carotid revascularization and intensive medical management in patients with carotid stenosis. The registry identifies a four-arm design and a primary composite endpoint of stroke and death.

2,486
Enrollment
Randomized trial
4
Arms
Four intervention groups
4 years
Endpoint frame
Primary outcome
NA
Phase
Registry classification
FeatureCREST-2
Trial acronymCREST-2
ClinicalTrials.gov identifierNCT02089217
Study titleCarotid Revascularization and Medical Management for Asymptomatic Carotid Stenosis Trial
ConditionCarotid Stenosis
PhaseNA
StatusCOMPLETED
AllocationRANDOMIZED
Design modelPARALLEL
MaskingSINGLE
Primary purposePREVENTION
Enrollment2,486
Number of arms4
Lead sponsorMayo Clinic

2. Clinical Question

The clinical question is how carotid revascularization strategies and intensive medical management compare within a randomized trial of carotid stenosis. The registry identifies carotid endarterectomy, carotid stenting, and two intensive-medical-management approaches as the four interventions.

Population

Patients enrolled in the CREST-2 trial for the condition identified in the registry as carotid stenosis.

Revascularization interventions

Carotid endarterectomy (CEA) and carotid stenting (CAS).

Medical management

Intensive Medical Management - no CEA and Intensive Medical Management - no CAS.

Primary question

How do these randomized strategies relate to the registry-defined composite outcome of stroke and death through 4 years?

3. Trial Design

01
Randomize 2,486 participants
02
Four arms CEA, CAS, or intensive medical management strategies
03
Parallel design Parallel-group allocation
04
Follow Primary outcome through 4 years
05
Assess Stroke and death composite
ARM 1

Carotid endarterectomy

  • Intervention type: procedure
  • Registry intervention: Carotid endarterectomy (CEA)
ARM 2

Carotid stenting

  • Intervention type: device
  • Registry intervention: Carotid Stenting (CAS)
ARM 3

Intensive medical management

  • Registry intervention: Intensive Medical Management - no CEA
  • Intervention type: other
ARM 4

Intensive medical management

  • Registry intervention: Intensive Medical Management - no CAS
  • Intervention type: other
Four-arm structure matters statistically. CREST-2 is not a simple two-group randomized comparison. The registry identifies four arms, so the statistical interpretation depends on which randomized contrasts are specified for the primary question. A four-arm design can support several clinically distinct comparisons, but those comparisons must be defined before treating them as confirmatory hypotheses.

4. Trial Timeline

December 9, 2014

Study start

The registry lists December 9, 2014 as the study start date.

September 12, 2025

Primary completion

The registry lists September 12, 2025 as the primary completion date.

Completed

Current registry status

The study is listed as COMPLETED in the registry record.

5. Primary Endpoint

EndpointTime frameRegistry definition
Stroke and death 4 years The primary outcome is the composite of stroke plus death within 44 days after randomization and ipsilateral stroke thereafter up to 4 years.

This endpoint has an important time-dependent structure. The registry definition combines stroke plus death within the first 44 days after randomization with ipsilateral stroke after that early period, continuing through 4 years. The resulting endpoint is therefore not simply a count of all strokes and deaths occurring at any time during follow-up.

Endpoint structure
Composite event = stroke + death within 44 days after randomization, followed by ipsilateral stroke thereafter through 4 years

The definition establishes both the event components and the time periods in which they contribute to the primary outcome.

6. Statistical Methodology

Time-to-event analysis

The primary endpoint is defined over a follow-up period extending to 4 years and therefore naturally has a time-to-event structure. For an endpoint of this type, the analysis generally considers not only whether an event occurred, but also when the first qualifying event occurred and how long participants were observed without an event.

Common methods for a randomized time-to-event comparison include Kaplan-Meier estimation to describe event-free survival over time, a log-rank test to compare event-time distributions, and a Cox proportional-hazards model to estimate a hazard ratio. The specific statistical model and testing framework for CREST-2 are not reported in the ClinicalTrials.gov record.

Conceptual survival function
S(t) = P(T > t)

Here, S(t) represents the probability of remaining free of the defined primary event beyond time t. For a composite endpoint, the event is the first qualifying component specified by the endpoint definition.

Kaplan-Meier estimation

Kaplan-Meier methods are commonly used when participants have different lengths of observed follow-up or when some participants reach the end of observation without experiencing the event. The method uses the observed event times and the number of participants still at risk at each point in time.

Hazard ratio

If a Cox model is used, the hazard ratio would provide a relative comparison of the instantaneous event rates between two randomized groups under the model. A hazard ratio below 1 would indicate a lower estimated instantaneous event rate in the numerator group relative to the reference group; a hazard ratio above 1 would indicate a higher estimated rate.

Important distinction

A hazard ratio is not a risk ratio and is not an absolute difference in the probability of experiencing the composite endpoint by 4 years. A time-specific event probability and a hazard ratio describe different aspects of the treatment comparison and should not be treated as interchangeable.

Composite endpoint analysis

The primary endpoint combines clinically distinct events. In a composite endpoint, a participant is generally counted according to the first qualifying component that occurs under the endpoint definition. This makes the exact component definitions and timing rules central to interpretation.

For CREST-2, the registry's wording is particularly important because the definition changes after 44 days: stroke plus death contributes within 44 days after randomization, while ipsilateral stroke contributes thereafter up to 4 years.

7. Statistical Methods Explained

Why is the primary endpoint a composite?

A composite endpoint combines multiple clinically relevant outcomes into one statistical endpoint. This can allow the study to capture more events than a single component would produce, potentially increasing the amount of information available for a time-to-event comparison. The tradeoff is that the components can differ in clinical meaning and frequency, so interpretation should always return to the exact component definitions.

Why does the 44-day boundary matter?

The 44-day boundary is part of the registry-defined endpoint itself. Within the first 44 days after randomization, the endpoint includes stroke plus death. After that period, the endpoint is based on ipsilateral stroke through 4 years. An analysis that ignored this timing rule would be analysing a different endpoint.

Why is ipsilateral stroke different from any stroke?

Ipsilateral means occurring on the same side as the relevant carotid lesion or treated vascular territory. The registry specifically defines the later component as ipsilateral stroke, so later events are not interchangeable with a generic count of all strokes.

Why would Kaplan-Meier methods be useful?

The primary endpoint is evaluated through 4 years, so the timing of events matters. Kaplan-Meier estimation can show the proportion remaining free of the defined endpoint over follow-up while appropriately accounting for participants whose observations are censored before an event occurs.

What would a hazard ratio mean here?

If a Cox model were used, the hazard ratio would compare the estimated instantaneous rate of the first qualifying primary event between two randomized groups. It would not mean that the same percentage of participants avoided stroke or death, nor would it describe the absolute probability of being event-free at 4 years.

Why does the four-arm design require careful comparisons?

With four randomized arms, there can be multiple possible pairwise or grouped comparisons. Each additional confirmatory comparison can affect the overall type I error unless the testing strategy accounts for multiplicity. The ClinicalTrials.gov record does not specify the multiplicity strategy or confirmatory comparison hierarchy.

8. Planned Analysis

No formal statistical analyses are posted to ClinicalTrials.gov for the primary endpoint in the registry information available here. The registry identifies the endpoint that is to be measured, but it does not provide an effect estimate, confidence interval, p-value, event count, or posted statistical-analysis result.

Primary endpoint

Stroke and death

Time frame: 4 years

Composite of stroke plus death within 44 days after randomization and ipsilateral stroke thereafter up to 4 years.

For this type of endpoint, a conventional statistical analysis would generally begin with time-to-event summaries. Kaplan-Meier methods could describe the event-free probability over time, while a log-rank framework could compare randomized groups. A Cox proportional-hazards model could provide a hazard ratio and confidence interval for a prespecified comparison.

Because CREST-2 has four arms, the appropriate analysis also depends on the trial's prespecified comparison structure. If several hypotheses are tested, multiplicity control is important so that the probability of a false-positive confirmatory conclusion remains consistent with the study's statistical design.

No outcome estimate is posted in the registry information. The registry does not report a primary-endpoint hazard ratio, confidence interval, p-value, median event time, or number of primary events in the information used for this analysis.

9. Interpreting a Future Hazard Ratio

If a hazard ratio is subsequently reported for one of the prespecified CREST-2 comparisons, its interpretation should be tied to the exact randomized contrast and endpoint definition. For example, an HR of 0.70 would describe an estimated instantaneous event rate approximately 30% lower in the numerator group under the fitted model.

What the estimate would mean

A hazard ratio compares event rates over time. It would summarize a relative treatment effect for the defined composite endpoint rather than directly stating the absolute probability of stroke or death by 4 years.

What it would not mean

A hazard ratio would not mean that every participant has exactly the same proportional reduction in risk. It would also not mean that the percentage of participants who experience the event is reduced by the same numerical percentage.

Why the confidence interval matters

The confidence interval would quantify statistical uncertainty around the estimated hazard ratio under the specified model and sampling framework. A narrow interval would indicate greater statistical precision than a wide interval, while the interval's location relative to the null value of 1 would be relevant to the corresponding hypothesis test.

Why the p-value is not an effect-size measure

A p-value addresses the compatibility of the observed data with a specified null hypothesis under the statistical testing framework. It does not describe the magnitude of the treatment effect, its clinical importance, or the probability that a treatment is beneficial.

10. Analysis Population and Randomization

The registry identifies the allocation as RANDOMIZED and the design model as PARALLEL. These design features are important because randomization establishes the basis for comparing outcomes according to assigned intervention groups rather than according to treatments selected after enrollment.

Design featureRegistry informationStatistical relevance
Allocation RANDOMIZED Randomization provides the basis for treatment-group comparisons and helps balance measured and unmeasured prognostic factors in expectation.
Design model PARALLEL Participants are assigned to parallel intervention groups rather than being described as crossing between randomized treatment sequences.
Masking SINGLE The registry identifies single masking, although the ClinicalTrials.gov record does not specify which participants or study personnel were masked.
Primary purpose PREVENTION The registry classifies the trial's primary purpose as prevention.

11. Multiplicity and the Four-Arm Design

Multiplicity is especially important whenever a randomized trial contains more than two arms. Four arms create multiple possible comparisons, but the existence of multiple possible contrasts does not by itself establish that every contrast is a confirmatory hypothesis.

A prespecified statistical testing hierarchy can distinguish the primary comparison from secondary or exploratory comparisons. Alternatively, procedures such as multiplicity-adjusted testing can be used when several hypotheses are intended to support formal conclusions.

One primary comparison

If a single prespecified comparison is the confirmatory hypothesis, the interpretation of its type I error can remain focused on that comparison.

Several confirmatory comparisons

If multiple comparisons are intended to support confirmatory conclusions, the testing strategy should account for the multiplicity created by those hypotheses.

The ClinicalTrials.gov record does not report a multiplicity procedure or endpoint hierarchy. Consequently, the four-arm structure should be described as a design feature rather than treated as evidence of a particular statistical testing strategy.

12. Missing Data, Censoring, and Follow-Up

Time-to-event analysis distinguishes between participants who experience a qualifying event and participants whose event status is not observed through the end of their available follow-up. The latter observations are typically censored at the relevant time rather than treated as if the event had not occurred throughout the entire 4-year period.

For CREST-2, this distinction matters because the primary endpoint is defined over 4 years. A participant with incomplete follow-up contributes information for the period during which their event status is observed.

Censoring concept
Observed time = min(event time, censoring time)

The event indicator distinguishes whether the qualifying event occurred during the observed period. Appropriate censoring assumptions are an important part of any time-to-event analysis.

The ClinicalTrials.gov record does not specify a missing-data or imputation method. For a time-to-event endpoint, standard survival methods generally do not impute a numerical event time for every censored participant; instead, they use the observed follow-up information under their censoring assumptions.

13. Proportional-Hazards Considerations

If a Cox proportional-hazards model is used, the hazard ratio has its clearest conventional interpretation when the relative hazard is reasonably stable over time. When hazards change substantially over follow-up, a single hazard ratio can compress a more complicated time-varying pattern into one summary number.

This is especially relevant for a composite endpoint with an explicitly different definition during the first 44 days and after that period. The endpoint's time structure should therefore be considered alongside any model-based summary of treatment effect.

Model assumption: a Cox hazard ratio is a statistical summary, not a complete description of the event-time distributions. Kaplan-Meier curves or time-specific event probabilities can provide additional information about how the treatment groups evolve over follow-up.

14. Safety and Secondary Endpoints

The ClinicalTrials.gov record identifies one registered primary endpoint: stroke and death over 4 years. It does not provide secondary endpoint definitions, serious adverse-event counts by arm, adverse-event rates, or posted safety analyses.

Analysis domainRegistry information available here
Primary endpointStroke and death, 4 years
Secondary endpoint resultsNot reported in the ClinicalTrials.gov record
Serious adverse events by armNot reported in the ClinicalTrials.gov record
Primary endpoint statistical estimatesNot posted in the ClinicalTrials.gov record
Formal statistical analysesNo posted statistical analyses

Safety and efficacy should remain analytically distinct. A treatment-group comparison for the primary composite endpoint addresses the prespecified outcome question; it does not by itself summarize the complete safety profile of the interventions.

15. Design Features Not Reported in the Registry Information

Several statistical-design features are important in interpreting a large randomized clinical trial but are not specified in the information available for this record.

Design topicWhat is reportedWhat is not reported
Non-inferiority marginNo non-inferiority framework is identified in the ClinicalTrials.gov record.No numerical margin is reported.
CrossoverThe ClinicalTrials.gov record does not identify a crossover procedure.No crossover rate or crossover analysis is reported.
Factorial designThe design is identified as PARALLEL with four arms.No factorial allocation structure is reported.
MultiplicityFour randomized arms are reported.No multiplicity-control method is reported.
Interim analysisNo interim analysis is reported in the ClinicalTrials.gov record.No interim boundary or alpha-spending method is reported.
Missing-data methodThe primary endpoint has a 4-year time frame.No specific imputation or missing-data method is reported.
StratificationRandomized allocation is reported.No stratification factors are reported.
Bayesian methodsNo Bayesian analysis is reported.No prior distribution or Bayesian model is reported.

16. Limitations

17. Why This Trial Matters Statistically

CREST-2 is a useful statistical teaching case because its design illustrates several recurring issues in randomized clinical-trial analysis without reducing the study to a simple two-arm comparison.

ConceptHow it appears in CREST-2
RandomizationThe registry identifies a randomized allocation.
Parallel-group designThe design model is PARALLEL.
Four-arm designThe trial has four intervention arms.
Composite endpointThe primary outcome combines stroke and death during the first 44 days and ipsilateral stroke thereafter.
Time-to-event analysisThe primary endpoint is evaluated over a 4-year time frame.
CensoringTime-to-event methods provide a framework for participants with incomplete event observation.
Hazard ratioA potential model-based relative measure for comparing event rates between randomized groups.
MultiplicityFour randomized arms can create multiple possible treatment contrasts.
Endpoint definitionThe 44-day boundary changes which events qualify for the composite endpoint.
Prevention trialThe registry classifies the primary purpose as PREVENTION.

18. A Statistical Reading of the Primary Endpoint

The most important statistical feature of CREST-2 is not simply that the primary endpoint is a composite, but that it is a time-structured composite. During the first 44 days after randomization, both stroke and death contribute to the endpoint. After that period, the qualifying event is ipsilateral stroke through 4 years.

This structure has consequences for interpretation. A single overall treatment-effect estimate would combine information arising from different event definitions across follow-up. Therefore, the endpoint definition should accompany any reported hazard ratio, p-value, or survival estimate rather than being reduced to the abbreviated phrase "stroke and death."

Interpretation principle

The statistical estimand should always be connected to the exact event definition. A treatment effect for the CREST-2 primary endpoint is not automatically a treatment effect for all-cause mortality, all stroke, or ipsilateral stroke considered independently.

19. From Randomization to Statistical Inference

Randomization provides the foundation for causal comparison. Once participants are assigned to the four randomized groups, the primary statistical analysis can compare subsequent event times while preserving the randomized treatment assignment as the organizing principle.

The inferential sequence is conceptually straightforward:

01
Randomize Assign participants to intervention groups
02
Define Apply the exact composite endpoint
03
Observe Record event and censoring times
04
Model Compare event-time distributions
05
Interpret Combine relative and absolute measures

The strength of this framework comes from keeping the analysis tied to the randomized comparison and the prespecified endpoint. Statistical interpretation becomes less reliable when the endpoint, analysis population, treatment contrast, or testing framework changes without being explicitly identified.

20. Why This Endpoint Requires More Than a P-Value

A p-value alone cannot communicate the size or clinical meaning of a treatment effect. For a time-to-event composite such as the CREST-2 primary endpoint, a complete statistical report would ideally include an effect estimate, its confidence interval, the event-time distribution or time-specific event probabilities, and the exact randomized contrast being tested.

For example, if a future analysis reports a statistically significant hazard ratio, that result would still need to be interpreted alongside the absolute event probabilities and the component-specific event experience. Conversely, a non-significant result would not necessarily demonstrate equivalence between the strategies; the interpretation would depend on the confidence interval and the question the trial was designed to answer.

Relative effect

A hazard ratio summarizes the relative difference in event rates under a time-to-event model.

Absolute effect

A time-specific event probability describes the observed or estimated absolute probability of experiencing the defined endpoint by a particular time.

Precision

A confidence interval communicates uncertainty around the estimated treatment effect.

Evidence against the null

A p-value addresses the statistical-testing question but does not quantify the magnitude or clinical importance of an effect.

21. Sources

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22. Record Summary

CREST-2 is a completed randomized, parallel-group prevention trial with 2,486 participants and four intervention arms. Its registered primary endpoint is a 4-year composite of stroke plus death within 44 days after randomization and ipsilateral stroke thereafter through 4 years.

Statistically, the trial is best understood through the combination of its randomized four-arm structure and its time-dependent composite endpoint. A rigorous analysis requires the exact randomized contrast, the endpoint definition, appropriate handling of censoring, and a prespecified approach to multiple comparisons when more than one hypothesis is tested.

Clinical Biostats interpretation: The registry establishes the study design and primary endpoint, but no formal statistical analyses are posted in the information available for this record. The appropriate educational framework is therefore to understand how the registered endpoint would ordinarily be analysed and how future effect estimates, confidence intervals, and hypothesis tests should be interpreted without confusing a composite time-to-event measure with any single component outcome.