Journal article
PrecISE: Precision Medicine in Severe Asthma: An adaptive platform trial with biomarker ascertainment
Journal of allergy and clinical immunology, Vol.147(5), pp.1594-1601
05/2021
DOI: 10.1016/j.jaci.2021.01.037
PMCID: PMC8113113
PMID: 33667479
Abstract
Severe asthma accounts for almost half the cost associated with asthma. Severe asthma is driven by heterogeneous molecular mechanisms. Conventional clinical trial design often lacks the power and efficiency to target subgroups with specific pathobiological mechanisms. Furthermore, the validation and approval of new asthma therapies is a lengthy process. A large proportion of that time is taken by clinical trials to validate asthma interventions. The National Institutes of Health Precision Medicine in Severe and/or Exacerbation Prone Asthma (PrecISE) program was established with the goal of designing and executing a trial that uses adaptive design techniques to rapidly evaluate novel interventions in biomarker-defined subgroups of severe asthma, while seeking to refine these biomarker subgroups, and to identify early markers of response to therapy. The novel trial design is an adaptive platform trial conducted under a single master protocol that incorporates precision medicine components. Furthermore, it includes innovative applications of futility analysis, cross-over design with use of shared placebo groups, and early futility analysis to permit more rapid identification of effective interventions. The development and rationale behind the study design are described. The interventions chosen for the initial investigation and the criteria used to identify these interventions are enumerated. The biomarker-based adaptive design and analytic scheme are detailed as well as special considerations involved in the final trial design.
Details
- Title: Subtitle
- PrecISE: Precision Medicine in Severe Asthma: An adaptive platform trial with biomarker ascertainment
- Creators
- Elliot Israel - Harvard UniversityLoren C. Denlinger - University of Wisconsin–MadisonLeonard B. Bacharier - Vanderbilt UniversityLisa M. LaVange - University of North Carolina at Chapel HillWendy C. Moore - Wake Forest UniversityMichael C. Peters - University of California, San FranciscoSteve N. Georas - University of Rochester Medical CenterRosalind J. Wright - Icahn School of Medicine at Mount SinaiDavid T. Mauger - Pennsylvania State UniversityPatricia Noel - National Institutes of HealthPraveen Akuthota - University of California San DiegoJulia Bach - University of Wisconsin–MadisonEugene R. Bleecker - University of ArizonaJuan Carlos Cardet - University of South FloridaTara F. Carr - University of ArizonaMario Castro - University of KansasAngeles Cinelli - Brigham and Women's HospitalSuzy A.A. Comhair - Cleveland ClinicRonina A. Covar - National Jewish Health, Denver, ColoLaura Crotty Alexander - Pulmonary Division, Department of Medicine, University of California–San Diego, La Jolla, CalifEmily A. DiMango - Columbia University Irving Medical CenterSerpil C. Erzurum - Cleveland ClinicJohn V. Fahy - University of California, San FranciscoMerritt L. Fajt - University of PittsburghBenjamin M. Gaston - Indiana University – Purdue University IndianapolisEric A. Hoffman - University of IowaFernando Holguin - University of Colorado DenverDaniel J. Jackson - University of Wisconsin–MadisonSonia Jain - University of California San DiegoNizar N. Jarjour - University of Wisconsin–MadisonYuan Ji - University of ChicagoNicholas J. Kenyon - University of California, DavisMichael R. Kosorok - University of North Carolina at Chapel HillMonica Kraft - University of ArizonaJerry A. Krishnan - University of Illinois ChicagoRajesh Kumar - Lurie Children's HospitalAndrew H. Liu - University of Colorado DenverMark C. Liu - Johns Hopkins UniversityNgoc P. Ly - University of California, San FranciscoM. Alison Marquis - University of North Carolina at Chapel HillFernando D. Martinez - University of ArizonaJames N. Moy - Rush University Medical CenterWanda K. O’Neal - Center for Environmental Medicine, Asthma, and Lung Biology, University of North Carolina, Chapel Hill, NCVictor E. Ortega - Wake Forest UniversityDavid B. Peden - University of North Carolina at Chapel HillWanda Phipatanakul - Boston Children's HospitalKristie Ross - Boston Children's HospitalLewis J. Smith - Northwestern UniversityStanley J. Szefler - University of Colorado DenverW. Gerald Teague - University of VirginiaAbigail F. Tulchinsky - Brigham and Women's HospitalPandurangan Vijayanand - La Jolla Institute For Allergy & ImmunologyMichael E. Wechsler - University of Colorado DenverSally E. Wenzel - University of PittsburghSteven R. White - University of ChicagoAmir A. Zeki - University of California, DavisAnastasia Ivanova - University of North Carolina at Chapel Hill
- Resource Type
- Journal article
- Publication Details
- Journal of allergy and clinical immunology, Vol.147(5), pp.1594-1601
- DOI
- 10.1016/j.jaci.2021.01.037
- PMID
- 33667479
- PMCID
- PMC8113113
- NLM abbreviation
- J Allergy Clin Immunol
- ISSN
- 0091-6749
- eISSN
- 1097-6825
- Publisher
- Elsevier Inc
- Grant note
- UL1TR002451; UL1TR000427; UL1TR002366; UL1TR002389; UL1TR002489; UL1TR001857; UL1TR001442; UL1TR001872 / National Institute of Health Clinical & Translational Science Award William W. and Judith H. Busse Professor of Allergy & Asthma Research U24 HL138998, 1UG1HL139054, 1UG1HL139098, 1UG1HL139106, 1UG1HL139117, 1UG1HL139118, 1UG1HL139119, 1UG1HL139123, 1UG1HL139124, 1UG1HL139125; 1UG1HL139126 / National Institutes of Health Gloria M. and Anthony C. Simboli Distinguished Chair in Asthma Research 5U24TR001608 / Duke/Vanderbilt Trial Innovation Center National Heart, Lung, and Blood Institute
- Language
- English
- Date published
- 05/2021
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Radiology; Internal Medicine
- Record Identifier
- 9984318690102771
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