PrecISE: Precision Medicine in Severe Asthma: An adaptive platform trial with biomarker ascertainment

Israel, Elliot; Denlinger, Loren C.; Bacharier, Leonard B.; LaVange, Lisa M.; Moore, Wendy C.; Peters, Michael C.; Georas, Steve N.; Wright, Rosalind J.; Mauger, David T.; Noël, Patricia; Akuthota, Praveen; Bach, Julia; Bleecker, Eugene R.; Cardet, Juan Carlos; Carr, Tara F.; Castro, Mario; Cinelli, Angeles; Comhair, Suzy; Covar, Ronina; Alexander, Laura E. Crotty; DiMango, Emily; Erzurum, Serpil C.; Fahy, John V.; Fajt, Merritt L.; Gaston, Benjamin; Hoffman, Eric A.; Holguín, Fernando; Jackson, Daniel J.; Jain, Sonia; Jarjour, Nizar N.; Ji, Yuan; Kenyon, Nicholas J.; Kosorok, Michael R.; Kraft, Monica; Krishnan, Jerry A.; Kumar, Rajesh; Liu, Andrew H.; Liu, Mark C.; Ly, Ngoc P.; Marquis, Miriam; Martínez, Fernando D.; Moy, James N.; O’Neal, Wanda K.; Ortega, Victor E.; Peden, David B.; Phipatanakul, Wanda; Ross, Kristie; Smith, Lewis J.; Szefler, Stanley J.; Teague, W. Gerald; Tulchinsky, Abigail; Vijayanand, Pandurangan; Wechsler, Michael E.; Wenzel, Sally E.; White, Steven R.; Zeki, Amir A.; Ivanova, Anastasia

doi:10.1016/j.jaci.2021.01.037

Cited by 32 publications

(27 citation statements)

References 6 publications

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“…The I-SPY2 and PrecISE trials are examples of adaptive platform trials ( table ). 79 , 80 I-SPY2 has been designed to evaluate neoadjuvant therapies for high-risk, early stage breast cancer, 12 while therapies for severe and exacerbation-prone asthma are being assessed in PrecISE. 81 Both are signal-finding phase 2 trials that aim to rapidly evaluate multiple candidate therapies for their probability of success in phase 3 trials.…”

Section: Clinical Trials To Advance Precision Medicinementioning

confidence: 99%

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Advancing precision medicine for acute respiratory distress syndrome

Beitler

Thompson

Baron

et al. 2022

The Lancet Respiratory Medicine

121

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Acute respiratory distress syndrome (ARDS) is a heterogeneous clinical syndrome. Understanding of the complex pathways involved in lung injury pathogenesis, resolution, and repair has grown considerably in recent decades. Nevertheless, to date, only therapies targeting ventilation-induced lung injury have consistently proven beneficial, and despite these gains, ARDS morbidity and mortality remain high. Many candidate therapies with promise in preclinical studies have been ineffective in human trials, probably at least in part due to clinical and biological heterogeneity that modifies treatment responsiveness in human ARDS. A precision medicine approach to ARDS seeks to better account for this heterogeneity by matching therapies to subgroups of patients that are anticipated to be most likely to benefit, which initially might be identified in part by assessing for heterogeneity of treatment effect in clinical trials. In October 2019, the US National Heart, Lung, and Blood Institute convened a workshop of multidisciplinary experts to explore research opportunities and challenges for accelerating precision medicine in ARDS. Topics of discussion included the rationale and challenges for a precision medicine approach in ARDS, the roles of preclinical ARDS models in precision medicine, essential features of cohort studies to advance precision medicine, and novel approaches to clinical trials to support development and validation of a precision medicine strategy. In this Position Paper, we summarise workshop discussions, recommendations, and unresolved questions for advancing precision medicine in ARDS. Although the workshop took place before the COVID-19 pandemic began, the pandemic has highlighted the urgent need for precision therapies for ARDS as the global scientific community grapples with many of the key concepts, innovations, and challenges discussed at this workshop.

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Section: Clinical Trials To Advance Precision Medicinementioning

confidence: 99%

“…Innovative trial designs might help to accelerate precision therapeutic discovery, in part by embracing disease I-SPY2 trial 79 for breast cancer PrecISE trial 80 for severe asthma…”

Section: Lessons From Other Fields: Innovative Trial Designsmentioning

confidence: 99%

Advancing precision medicine for acute respiratory distress syndrome

Beitler

Thompson

Baron

et al. 2022

The Lancet Respiratory Medicine

121

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“…Our hybrid approach combining phases ii and iii allows reducing the number of placebos and increasing the number of experimental therapies each participant may receive. This makes the study more attractive to potential participants (Israel et al 2021). Under the hybrid approach, the placebo data are shared across interventions for analysis (see Section 6).…”

Section: Crossover Designmentioning

confidence: 99%

“…The protocol was finalized in early 2019, and the PrecISE study (ct.gov number is NCT04129931) began enrollment in December 2019. An overview of the study design, including the selection of interventions and clinical and other scientific considerations in designing the study, is provided elsewhere (Israel, et al, 2021). In this paper we describe the statistical considerations that were critical to designing the PrecISE study.…”

Section: Introductionmentioning

confidence: 99%

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

Ivanova

Israel

LaVange

et al. 2020

Journal of Biopharmaceutical Statistics

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“…Recently, systemic interleukin-6 (IL-6), an obesity related cytokine, has gained recognition as a potential biomarker for the early signs of metabolic dysfunction in children with asthma and an associated risk of asthma morbidity in both children and adults (5)(6)(7). Anti-IL-6 therapy is currently being evaluated as a potential asthma treatment (8,9). Mechanistically, data suggest that IL-6 is directly linked to airway inflammation (10)(11)(12).…”

mentioning

confidence: 99%