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

Anastasia Ivanova; Elliot Israel; Lisa M. LaVange; Michael C. Peters; Loren C. Denlinger; Wendy C. Moore; Leonard B. Bacharier; M. Alison Marquis; Nathan M. Gotman; Michael R. Kosorok; Chalmer Tomlinson; David T. Mauger; Steve N. Georas; Rosalind J. Wright; Patricia Noel; Gary L. Rosner; Praveen Akuthota; Dean Billheimer; Eugene R. Bleecker; Juan Carlos Cardet; Mario Castro; Emily A. DiMango; Serpil C. Erzurum; John V. Fahy; Merritt L. Fajt; Benjamin M. Gaston; Fernando Holguin; Sonia Jain; Nicholas J. Kenyon; Jerry A. Krishnan; Monica Kraft; Rajesh Kumar; Mark C. Liu; Ngoc P. Ly; James N. Moy; Wanda Phipatanakul; Kristie Ross; Lewis J. Smith; Stanley J. Szefler; W. Gerald Teague; Michael E. Wechsler; Sally E. Wenzel; Steven R. White

doi:10.1080/10543406.2020.1821705

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

Anastasia Ivanova
, Elliot Israel
, Lisa M. LaVange
, Michael C. Peters
, Loren C. Denlinger
, Wendy C. Moore
, Leonard B. Bacharier
, M. Alison Marquis
, Nathan M. Gotman
, Michael R. Kosorok
, Chalmer Tomlinson
, David T. Mauger
, Steve N. Georas
, Rosalind J. Wright
, Patricia Noel
, Gary L. Rosner
, Praveen Akuthota
, Dean Billheimer
, Eugene R. Bleecker
, Juan Carlos Cardet

Mario Castro, Emily A. DiMango, Serpil C. Erzurum, John V. Fahy, Merritt L. Fajt, Benjamin M. Gaston, Fernando Holguin, Sonia Jain, Nicholas J. Kenyon, Jerry A. Krishnan, Monica Kraft, Rajesh Kumar, Mark C. Liu, Ngoc P. Ly, James N. Moy, Wanda Phipatanakul, Kristie Ross, Lewis J. Smith, Stanley J. Szefler, W. Gerald Teague, Michael E. Wechsler, Sally E. Wenzel, Steven R. White

Medicine

Research output: Contribution to journal › Article › peer-review

14 Scopus citations

Abstract

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.

Original language	English (US)
Pages (from-to)	1026-1037
Number of pages	12
Journal	Journal of Biopharmaceutical Statistics
Volume	30
Issue number	6
DOIs	https://doi.org/10.1080/10543406.2020.1821705
State	Published - 2020

Keywords

Master protocol
adaptive enrichment
compex
covariate adaptive randomization
platform trial
severe asthma

ASJC Scopus subject areas

Statistics and Probability
Pharmacology
Pharmacology (medical)

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10.1080/10543406.2020.1821705

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Ivanova, A., Israel, E., LaVange, L. M., Peters, M. C., Denlinger, L. C., Moore, W. C., Bacharier, L. B., Marquis, M. A., Gotman, N. M., Kosorok, M. R., Tomlinson, C., Mauger, D. T., Georas, S. N., Wright, R. J., Noel, P., Rosner, G. L., Akuthota, P., Billheimer, D., Bleecker, E. R., ... White, S. R. (2020). The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations. Journal of Biopharmaceutical Statistics, 30(6), 1026-1037. https://doi.org/10.1080/10543406.2020.1821705

Ivanova, A, Israel, E, LaVange, LM, Peters, MC, Denlinger, LC, Moore, WC, Bacharier, LB, Marquis, MA, Gotman, NM, Kosorok, MR, Tomlinson, C, Mauger, DT, Georas, SN, Wright, RJ, Noel, P, Rosner, GL, Akuthota, P, Billheimer, D , Bleecker, ER, Cardet, JC, Castro, M, DiMango, EA, Erzurum, SC, Fahy, JV, Fajt, ML, Gaston, BM, Holguin, F, Jain, S, Kenyon, NJ, Krishnan, JA, Kraft, M, Kumar, R, Liu, MC, Ly, NP, Moy, JN, Phipatanakul, W, Ross, K, Smith, LJ, Szefler, SJ, Teague, WG, Wechsler, ME, Wenzel, SE & White, SR 2020, 'The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations', Journal of Biopharmaceutical Statistics, vol. 30, no. 6, pp. 1026-1037. https://doi.org/10.1080/10543406.2020.1821705

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abstract = "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{\textquoteright} 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.",

keywords = "Master protocol, adaptive enrichment, compex, covariate adaptive randomization, platform trial, severe asthma",

author = "Anastasia Ivanova and Elliot Israel and LaVange, \{Lisa M.\} and Peters, \{Michael C.\} and Denlinger, \{Loren C.\} and Moore, \{Wendy C.\} and Bacharier, \{Leonard B.\} and Marquis, \{M. Alison\} and Gotman, \{Nathan M.\} and Kosorok, \{Michael R.\} and Chalmer Tomlinson and Mauger, \{David T.\} and Georas, \{Steve N.\} and Wright, \{Rosalind J.\} and Patricia Noel and Rosner, \{Gary L.\} and Praveen Akuthota and Dean Billheimer and Bleecker, \{Eugene R.\} and Cardet, \{Juan Carlos\} and Mario Castro and DiMango, \{Emily A.\} and Erzurum, \{Serpil C.\} and Fahy, \{John V.\} and Fajt, \{Merritt L.\} and Gaston, \{Benjamin M.\} and Fernando Holguin and Sonia Jain and Kenyon, \{Nicholas J.\} and Krishnan, \{Jerry A.\} and Monica Kraft and Rajesh Kumar and Liu, \{Mark C.\} and Ly, \{Ngoc P.\} and Moy, \{James N.\} and Wanda Phipatanakul and Kristie Ross and Smith, \{Lewis J.\} and Szefler, \{Stanley J.\} and Teague, \{W. Gerald\} and Wechsler, \{Michael E.\} and Wenzel, \{Sally E.\} and White, \{Steven R.\}",

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AU - Peters, Michael C.

AU - Denlinger, Loren C.

AU - Moore, Wendy C.

AU - Bacharier, Leonard B.

AU - Marquis, M. Alison

AU - Gotman, Nathan M.

AU - Kosorok, Michael R.

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AU - Georas, Steve N.

AU - Wright, Rosalind J.

AU - Noel, Patricia

AU - Rosner, Gary L.

AU - Akuthota, Praveen

AU - Billheimer, Dean

AU - Bleecker, Eugene R.

AU - Cardet, Juan Carlos

AU - Castro, Mario

AU - DiMango, Emily A.

AU - Erzurum, Serpil C.

AU - Fahy, John V.

AU - Fajt, Merritt L.

AU - Gaston, Benjamin M.

AU - Holguin, Fernando

AU - Jain, Sonia

AU - Kenyon, Nicholas J.

AU - Krishnan, Jerry A.

AU - Kraft, Monica

AU - Kumar, Rajesh

AU - Liu, Mark C.

AU - Ly, Ngoc P.

AU - Moy, James N.

AU - Phipatanakul, Wanda

AU - Ross, Kristie

AU - Smith, Lewis J.

AU - Szefler, Stanley J.

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AU - Wechsler, Michael E.

AU - Wenzel, Sally E.

AU - White, Steven R.

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KW - Master protocol

KW - adaptive enrichment

KW - compex

KW - covariate adaptive randomization

KW - platform trial

KW - severe asthma

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

Abstract

Keywords

ASJC Scopus subject areas

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