Group-Sequential Strategies in Clinical Trials with Multiple Co-Primary Outcomes

Hamasaki, Toshimitsu; Asakura, Koko; Evans, Scott; Sugimoto, Tomoyuki; Sozu, Takashi

doi:10.1080/19466315.2014.1003090

Cited by 28 publications

(26 citation statements)

References 28 publications

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“…() and Hamasaki et al. (), this could offer the opportunity of stopping measurement of an endpoint for which superiority has already been demonstrated. Stopping measurement may be desirable if the endpoint is very invasive or expensive (e.g., data from a liver biopsy or gastro‐fiberscope, or data from expensive imaging).…”

Section: Group‐sequential Designs For Efficacy and Futility Assessmentsmentioning

confidence: 99%

“…() and Hamasaki et al. (), the selection of a different boundary type has a minimal effect on the power and ASN in clinical trials with co‐primary endpoints including those with only efficacy assessments. Table illustrates the MSS and ASN with a combination of OF‐ and PC‐type boundaries for two endpoints (EP1 and EP2; a common combination between the efficacy and futility assessments).…”

Section: Two Practical Considerationsmentioning

confidence: 99%

“…The former framework is independently discussed by Cheng et al (2014) and evaluated in clinical trials with two co-primary endpoints. Hamasaki et al (2015) discussed more flexible decision-making frameworks, allowing the different time points of analyses among the endpoints. Jennison and Turnbull (1993), and Cook and Farewell (1994) discussed the decision-making frameworks associated with interim evaluation of efficacy and futility to monitor the efficacy and safety responses.…”

Section: Introductionmentioning

confidence: 99%

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Interim evaluation of efficacy or futility in group‐sequential trials with multiple co‐primary endpoints

Asakura¹,

Hamasaki²,

Evans³

2016

Biometrical J

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We discuss group-sequential designs in superiority clinical trials with multiple co-primary endpoints, i.e., when trials are designed to evaluate if the test intervention is superior to the control on all primary endpoints. We consider several decision-making frameworks for evaluating efficacy or futility, based on boundaries using group-sequential methodology. We incorporate the correlations among the endpoints into the calculations for futility boundaries and sample sizes as a function of other design parameters including mean differences, the number of analyses, and efficacy boundaries. We investigate the operating characteristics of the proposed decision-making frameworks in terms of efficacy/futility boundaries, power, the Type I error rate and sample sizes, while varying the number of analyses, the correlations among the endpoints, and the mean differences. We provide an example to illustrate the methods and discuss practical considerations when designing efficient group-sequential designs in clinical trials with co-primary endpoints.

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Section: Group‐sequential Designs For Efficacy and Futility Assessmentsmentioning

confidence: 99%

Section: Two Practical Considerationsmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Interim evaluation of efficacy or futility in group‐sequential trials with multiple co‐primary endpoints

Asakura¹,

Hamasaki²,

Evans³

2016

Biometrical J

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“…However, as seen in Section 2, the correlation between the two test statistics for the AS and the NI are determined by the allocation ratio and the fraction margin and the test statistics may be positively correlated in some situations (e.g., n E k : n R k : n P k = 1:1:1 and θ < 0.5). By analogy to work by Hamasaki et al (2015), the Type I error rate is inflated over the targeted significance level when the test statistics are positively correlated. Therefore use of adaptive Type I error allocation for the fraction approach should be carefully considered.…”

Section: A Further Extensionmentioning

confidence: 99%

Group-sequential three-arm noninferiority clinical trial designs

Ochiai

Hamasaki

Evans

et al. 2016

Journal of Biopharmaceutical Statistics

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We discuss group-sequential three-arm noninferiority clinical trial designs that include active and placebo controls for evaluating both assay sensitivity and noninferiority. We extend two existing approaches, the fixed margin and fraction approaches, into a group-sequential setting with two decision-making frameworks. We investigate the operating characteristics including power, Type I error rate, maximum and expected sample sizes, as design factors vary. In addition, we discuss sample size recalculation and its’ impact on the power and Type I error rate via a simulation study.

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“…Suppose that a maximum of L analyses are planned and the two endpoints are analyzed at the same interim timepoint. For more flexible group-sequential designs for clinical trials with co-primary endpoints, please see Asakura et al (2014, 2015), and Hamasaki et al (2015).…”

Section: An Extension To Group-sequential Designsmentioning

confidence: 99%

Sample Size Considerations in Clinical Trials When Comparing Two Interventions Using Multiple Co-Primary Binary Relative Risk Contrasts

Ando

Hamasaki

Evans

et al. 2015

Statistics in Biopharmaceutical Research

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The effects of interventions are multi-dimensional. Use of more than one primary endpoint offers an attractive design feature in clinical trials as they capture more complete characterization of the effects of an intervention and provide more informative intervention comparisons. For these reasons, multiple primary endpoints have become a common design feature in many disease areas such as oncology, infectious disease, and cardiovascular disease. More specifically in medical product development, multiple endpoints are utilized as co-primary to evaluate the effect of the new interventions. Although methodologies to address continuous co-primary endpoints are well-developed, methodologies for binary endpoints are limited. In this paper, we describe power and sample size determination for clinical trials with multiple correlated binary endpoints, when relative risks are evaluated as co-primary. We consider a scenario where the objective is to evaluate evidence for superiority of a test intervention compared with a control intervention, for all of the relative risks. We discuss the normal approximation methods for power and sample size calculations and evaluate how the required sample size, power and Type I error vary as a function of the correlations among the endpoints. Also we discuss a simple, but conservative procedure for appropriate sample size calculation. We then extend the methods allowing for interim monitoring using group-sequential methods.

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Group-Sequential Strategies in Clinical Trials with Multiple Co-Primary Outcomes

Cited by 28 publications

References 28 publications

Interim evaluation of efficacy or futility in group‐sequential trials with multiple co‐primary endpoints

Interim evaluation of efficacy or futility in group‐sequential trials with multiple co‐primary endpoints

Group-sequential three-arm noninferiority clinical trial designs

Sample Size Considerations in Clinical Trials When Comparing Two Interventions Using Multiple Co-Primary Binary Relative Risk Contrasts

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