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. 2020 Nov 1;30(6):1026-1037.
doi: 10.1080/10543406.2020.1821705. Epub 2020 Sep 17.

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

Anastasia Ivanova  1 , Elliot Israel  2 , Lisa M LaVange  1 , Michael C Peters  3 , Loren C Denlinger  4 , Wendy C Moore  5 , Leonard B Bacharier  6 , M Alison Marquis  1 , Nathan M Gotman  1 , Michael R Kosorok  1 , Chalmer Tomlinson  1 , David T Mauger  7 , Steve N Georas  8 , Rosalind J Wright  9 , Patricia Noel  10 , Gary L Rosner  11 , Praveen Akuthota  12 , Dean Billheimer  13 , Eugene R Bleecker  13 , Juan Carlos Cardet  14 , Mario Castro  15 , Emily A DiMango  16 , Serpil C Erzurum  17 , John V Fahy  3 , Merritt L Fajt  18 , Benjamin M Gaston  19 , Fernando Holguin  20 , Sonia Jain  20 , Nicholas J Kenyon  21 , Jerry A Krishnan  22 , Monica Kraft  13 , Rajesh Kumar  23 , Mark C Liu  24 , Ngoc P Ly  2 , James N Moy  25 , Wanda Phipatanakul  26 , Kristie Ross  27 , Lewis J Smith  28 , Stanley J Szefler  29 , W Gerald Teague  30 , Michael E Wechsler  31 , Sally E Wenzel  18 , Steven R White  32
Affiliations

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

Anastasia Ivanova et al. J Biopharm Stat. .

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.

Keywords: Master protocol; adaptive enrichment; compex; covariate adaptive randomization; platform trial; severe asthma.

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Figures

Figure 1.
Figure 1.
An example of a multiple-period crossover allocation in the PrecISE study. A subject is first randomized to a two-period, T:P or P:T, crossover sequence with an oral intervention and its matching placebo. In period 3, the subject is randomized to receive a placebo matching a dietary supplement intervention. In period 4, the subject is randomized to receive another oral intervention (non-placebo) and in period 5, to an intervention administered as an injection (non-placebo).
See this image and copyright information in PMC

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