The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations

被引：10

作者：

Ivanova, Anastasia ^{[1
]}

Israel, Elliot ^{[1
]}

LaVange, Lisa M. ^{[1
]}

Peters, Michael C. ^{[1
]}

Denlinger, Loren C. ^{[1
]}

Moore, Wendy C. ^{[1
]}

Bacharier, Leonard B. ^{[1
]}

Marquis, M. Alison ^{[1
]}

Gotman, Nathan M. ^{[1
]}

Kosorok, Michael R. ^{[1
]}

Tomlinson, Chalmer ^{[1
]}

Mauger, David T. ^{[1
]}

Georas, Steve N. ^{[1
]}

Wright, Rosalind J. ^{[1
]}

Noel, Patricia ^{[1
]}

Rosner, Gary L. ^{[1
]}

Akuthota, Praveen ^{[1
]}

Billheimer, Dean ^{[1
]}

Bleecker, Eugene R. ^{[1
]}

Cardet, Juan Carlos ^{[1
]}

Castro, Mario ^{[1
]}

DiMango, Emily A. ^{[1
]}

Erzurum, Serpil C. ^{[1
]}

Fahy, John V. ^{[1
]}

Fajt, Merritt L. ^{[1
]}

Gaston, Benjamin M. ^{[1
]}

Holguin, Fernando ^{[1
]}

Jain, Sonia ^{[1
]}

Kenyon, Nicholas J. ^{[1
]}

Krishnan, Jerry A. ^{[1
]}

Kraft, Monica ^{[1
]}

Kumar, Rajesh ^{[1
]}

Liu, Mark C. ^{[1
]}

Ly, Ngoc P. ^{[1
]}

Moy, James N. ^{[1
]}

Phipatanakul, Wanda ^{[1
]}

Ross, Kristie ^{[1
]}

Smith, Lewis J. ^{[1
]}

Szefler, Stanley J. ^{[1
]}

Teague, W. Gerald ^{[1
]}

Wechsler, Michael E. ^{[1
]}

Wenzel, Sally E. ^{[1
]}

White, Steven R. ^{[1
]}

机构：

[1] Univ N Carolina, Dept Biostat, Chapel Hill, NC 27599 USA

来源：

JOURNAL OF BIOPHARMACEUTICAL STATISTICS | 2020年 / 30卷 / 06期

关键词：

Master protocol; platform trial; covariate adaptive randomization; adaptive enrichment; compex; severe asthma; BENRALIZUMAB; SUBGROUP; RECEPTOR;

D O I：

10.1080/10543406.2020.1821705

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

The Precision Interventions for Severe and/or Exacerbation-prone Asthma (PrecISE) study is an adaptive platform trial designed to investigate novel interventions to severe asthma. The study is conducted under a master protocol and utilizes a crossover design with each participant receiving up to five interventions and at least one placebo. Treatment assignments are based on the patients' biomarker profiles and precision health methods are incorporated into the interim and final analyses. We describe key elements of the PrecISE study including the multistage adaptive enrichment strategy, early stopping of an intervention for futility, power calculations, and the primary analysis strategy.

引用

页码：1026 / 1037

页数：12

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