PrecISE: Precision Medicine in Severe Asthma: An adaptive platform trial with biomarker ascertainment
| Autor: | M. Alison Marquis, Victor E. Ortega, Serpil C. Erzurum, Mario Castro, Steve N. Georas, Stanley J. Szefler, Rajesh Kumar, Wendy C. Moore, Fernando D. Martinez, Eric A. Hoffman, James N. Moy, Praveen Akuthota, Ronina A. Covar, Leonard B. Bacharier, Steven R. White, Benjamin Gaston, Kristie R. Ross, Michael R. Kosorok, Loren C. Denlinger, Anastasia Ivanova, Yuan Ji, Sonia Jain, Tara F. Carr, Monica Kraft, Patricia Noel, David T. Mauger, Nicholas J. Kenyon, Pandurangan Vijayanand, Nizar N. Jarjour, Laura E. Crotty Alexander, Amir A. Zeki, W. Gerald Teague, Lewis J. Smith, Wanda K. O'Neal, Lisa M. LaVange, Juan Carlos Cardet, Rosalind J. Wright, Sally E. Wenzel, Andrew H. Liu, Angeles Cinelli, Abigail F. Tulchinsky, Eugene R. Bleecker, Wanda Phipatanakul, Emily DiMango, Julia Bach, Michael E. Wechsler, Elliot Israel, Fernando Holguin, Mark C. Liu, Suzy A.A. Comhair, Daniel J. Jackson, Jerry A. Krishnan, Michael C. Peters, Ngoc P. Ly, John V. Fahy, David B. Peden, Merritt L. Fajt |
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| Rok vydání: | 2020 |
| Předmět: |
Severe asthma
medicine.medical_specialty Allergy Exacerbation precision medicine Clinical Trials and Supportive Activities Immunology Psychological intervention 01 natural sciences Article 010104 statistics & probability 03 medical and health sciences 0302 clinical medicine Clinical Research medicine adaptive design Immunology and Allergy Humans 030212 general & internal medicine 0101 mathematics Precision Medicine Intensive care medicine Lung Asthma Randomized Controlled Trials as Topic Protocol (science) therapy business.industry master protocol Clinical study design biomarkers clinical trial medicine.disease Precision medicine Clinical trial Good Health and Well Being platform trial Research Design Respiratory Biomarker (medicine) business Biomarkers Biotechnology |
| Zdroj: | J Allergy Clin Immunol The Journal of allergy and clinical immunology, vol 147, iss 5 |
| ISSN: | 1097-6825 |
| Popis: | 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. Alarge 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. |
| Databáze: | OpenAIRE |
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