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

Autor: Israel E; Department of Medicine, Divisions of Pulmonary & Critical Care Medicine & Allergy & Immunology, Brigham & Women's Hospital, Harvard Medical School, Boston, Mass. Electronic address: eisrael@partners.org., Denlinger LC; Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, Wis., Bacharier LB; Vanderbilt University School of Medicine, Nashville, Tenn., LaVange LM; Department of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC., Moore WC; Wake Forest University School of Medicine, Winston-Salem, NC., Peters MC; University of California, San Francisco School of Medicine, San Francisco, Calif., Georas SN; Division of Pulmonary and Critical Care Medicine, Department of Medicine, University of Rochester Medical Center, Rochester, NY., Wright RJ; Icahn School of Medicine at Mount Sinai, New York, NY., Mauger DT; Pennsylvania State University School of Medicine, Hershey, Pa., Noel P; Division of Lung Diseases, National Heart, Lung and Blood Institute, National Institutes of Health, Bethesda, Md., Akuthota P; Pulmonary Division, Department of Medicine, University of California-San Diego, La Jolla, Calif., Bach J; Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, Wis., Bleecker ER; Asthma and Airway Disease Research Center, University of Arizona, Tucson, Ariz., Cardet JC; Allergy and Immunology, University of South Florida, Tampa, Fla., Carr TF; Asthma and Airway Disease Research Center, University of Arizona, Tucson, Ariz., Castro M; University of Kansas School of Medicine, Kansas City, Kan., Cinelli A; Brigham and Women's Hospital, Boston, Mass., Comhair SAA; Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio., Covar RA; National Jewish Health, Denver, Colo., Alexander LC; Pulmonary Division, Department of Medicine, University of California-San Diego, La Jolla, Calif., DiMango EA; Columbia University Medical Center, New York, NY., Erzurum SC; Lerner Research Institute, Cleveland Clinic, Cleveland, Ohio., Fahy JV; University of California, San Francisco School of Medicine, San Francisco, Calif., Fajt ML; University of Pittsburgh Asthma Institute, University of Pittsburgh, Pittsburgh, Pa., Gaston BM; Wells Center for Pediatric Research, Indiana University, Indianapolis, Ind., Hoffman EA; Department of Radiology, University of Iowa, Iowa City, Iowa., Holguin F; University of Colorado School of Medicine, Aurora, Colo., Jackson DJ; Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, Wis., Jain S; Pulmonary Division, Department of Medicine, University of California-San Diego, La Jolla, Calif., Jarjour NN; Department of Medicine, University of Wisconsin School of Medicine and Public Health, Madison, Wis., Ji Y; Department of Health Studies, University of Chicago, Chicago, Ill., Kenyon NJ; Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Internal Medicine, University of California Davis School of Medicine, Davis, Calif., Kosorok MR; Department of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC., Kraft M; Asthma and Airway Disease Research Center, University of Arizona, Tucson, Ariz., Krishnan JA; Division of Pulmonary, Critical Care, Sleep, and Allergy, Department of Medicine, University of Illinois at Chicago, Chicago, Ill., Kumar R; Lurie Children's Hospital, Chicago, Ill., Liu AH; University of Colorado School of Medicine, Aurora, Colo; Children's Hospital Colorado, Aurora, Colo., Liu MC; Pulmonary and Critical Care Medicine, Department of Medicine, the Johns Hopkins University, Baltimore, Md., Ly NP; University of California, San Francisco School of Medicine, San Francisco, Calif., Marquis MA; Department of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC., Martinez FD; Asthma and Airway Disease Research Center, University of Arizona, Tucson, Ariz., Moy JN; Rush University Medical Center, Chicago, Ill., O'Neal WK; Center for Environmental Medicine, Asthma, and Lung Biology, University of North Carolina, Chapel Hill, NC., Ortega VE; Wake Forest University School of Medicine, Winston-Salem, NC., Peden DB; Marsico Lung Institute, UNC CF Research Center, University of North Carolina, Chapel Hill, NC., Phipatanakul W; Boston Children's Hospital and Harvard Medical School, Boston, Mass., Ross K; UH Rainbow Babies and Children's Hospitals, Cleveland, Ohio., Smith LJ; Northwestern University, Chicago, Ill., Szefler SJ; University of Colorado School of Medicine, Aurora, Colo; Children's Hospital Colorado, Aurora, Colo., Teague WG; University of Virginia School of Medicine, Charlottesville, Va., Tulchinsky AF; Brigham and Women's Hospital, Boston, Mass., Vijayanand P; La Jolla Institute for Immunology, La Jolla, Calif., Wechsler ME; National Jewish Health, Denver, Colo; University of Colorado School of Medicine, Aurora, Colo., Wenzel SE; University of Pittsburgh Asthma Institute, University of Pittsburgh, Pittsburgh, Pa., White SR; Section of Pulmonary and Critical Care Medicine, Department of Medicine, University of Chicago, Chicago, Ill., Zeki AA; Division of Pulmonary, Critical Care, and Sleep Medicine, Department of Internal Medicine, University of California Davis School of Medicine, Davis, Calif., Ivanova A; Department of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC.
Jazyk: angličtina
Zdroj: The Journal of allergy and clinical immunology [J Allergy Clin Immunol] 2021 May; Vol. 147 (5), pp. 1594-1601. Date of Electronic Publication: 2021 Mar 02.
DOI: 10.1016/j.jaci.2021.01.037
Abstrakt: 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.
(Copyright © 2021 American Academy of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.)
Databáze: MEDLINE
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