@article{90bcd710e4da4c0690fedcf8a6f61935,
title = "The precision interventions for severe and/or exacerbation-prone asthma (PrecISE) adaptive platform trial: statistical considerations",
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.",
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.}",
note = "Funding Information: This trial was funded by the National Heart, Lung, and Blood Institute (NHLBI) of the National Institutes of Health (NIH). The request for funding called for a Phase 2 proof-of-concept clinical trial to assess a number of novel interventions in biomarker-defined subgroups of patients with severe asthma and obtain information on the utility of the biomarkers in identifying patients with a higher likelihood to respond to a specific treatment. Rather than propose more standard, non-adaptive, biomarker-stratified, parallel group designs, applicants were encouraged to propose adaptive trial designs that made use of accumulating data on biomarker-outcome relationships to modify design features during the trial. Funding Information: The PrecISE study is funded by the National Heart, Lung, and Blood Institute, National Institutes of Health, grant U24 HL138998. The study also gratefully acknowledges receiving contributed product from Vitaeris, owned and operated by CSL group (clazakizumab), Vitaflo (MCT), Sun Pharma (imatinib), OM Pharma, a Vifor Pharma Group Company (OM-85, Broncho-Vaxom), Incyte (itacitinib), Laurel Venture (cavosonstat) and GlaxoSmithKline (Advair Diskus and Ventolin). The authors thank anonymous reviewers for their helpful comments. Publisher Copyright: {\textcopyright} 2020 Taylor & Francis Group, LLC.",
year = "2020",
doi = "10.1080/10543406.2020.1821705",
language = "English (US)",
volume = "30",
pages = "1026--1037",
journal = "Journal of Biopharmaceutical Statistics",
issn = "1054-3406",
publisher = "Taylor and Francis Ltd.",
number = "6",
}