Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases

被引:7
作者
Kawano-Dourado, Leticia [1 ,2 ,3 ,36 ]
Kulkarni, Tejaswini [4 ]
Ryerson, Christopher J. [5 ,6 ]
Rivera-Ortega, Pilar [7 ]
Baldi, Bruno Guedes [2 ]
Chaudhuri, Nazia [8 ]
Funke-Chambour, Manuela [9 ]
Hoffmann-Vold, Anna-Maria [10 ,11 ]
Johannson, Kerri A. [12 ]
Khor, Yet Hong [13 ,14 ]
Montesi, Sydney B. [15 ]
Piccari, Lucilla [16 ]
Prosch, Helmut [17 ]
Molina-Molina, Maria [18 ]
Sellares Torres, Jacobo [19 ]
Bauer-Ventura, Iazsmin [20 ]
Rajan, Sujeet [21 ]
Jacob, Joseph [22 ,23 ]
Richards, Duncan [24 ]
Spencer, Lisa G. [25 ]
Wendelberger, Barbara [26 ]
Jensen, Tom [26 ]
Quintana, Melanie [26 ]
Kreuter, Michael [27 ,28 ]
Gordon, Anthony C. [29 ]
Martinez, Fernando J. [30 ]
Kaminski, Naftali [31 ]
Cornelius, Victoria [32 ]
Lewis, Roger [33 ]
Adams, Wendy [34 ]
Jenkins, Gisli [35 ]
机构
[1] Hcor Hosp, Hcor Res Inst, Sao Paulo, Brazil
[2] Univ Sao Paulo, Heart Inst InCor, Pulm Div, Sao Paulo, Brazil
[3] MAGIC Evidence Ecosyst Fdn, Oslo, Norway
[4] Univ Alabama Birmingham, Heersink Sch Med, Birmingham, AL USA
[5] Univ British Columbia, Dept Med, Vancouver, BC, Canada
[6] Univ British Columbia, Ctr Heart Lung Innovat, Vancouver, BC, Canada
[7] Manchester Univ NHS Fdn Trust, Interstitial Lung Dis Unit, Resp Med, Manchester, England
[8] Univ Ulster, Sch Med, Dept Hlth & Life Sci, Londonderry, North Ireland
[9] Univ Hosp Bern, Dept Pulmonol Allergol & Clin Immunol, Inselspital, Bern, Switzerland
[10] Oslo Univ Hosp, Dept Rheumatol, Oslo, Norway
[11] Univ Hosp Zurich, Univ Zurich, Dept Rheumatol, Zurich, Switzerland
[12] Univ Calgary, Dept Med, Calgary, AB, Canada
[13] Monash Univ, Cent Clin Sch, Resp Res Alfred, Melbourne, Vic, Australia
[14] Austin Hlth, Dept Resp & Sleep Med, Heidelberg, Vic, Australia
[15] Massachusetts Gen Hosp, Div Pulm & Crit Care Med, Boston, MA USA
[16] Hosp Mar, Dept Pulmonol, Barcelona, Spain
[17] Med Univ Vienna, Dept Biomed Imaging & Image Guided Therapy, Vienna, Austria
[18] Hosp Univ Bellvitge, Inst Invest Biomed Bellvitge IDIBELL, Serv Pneumol, Grup Recerca Pneumol, Barcelona, Spain
[19] Hosp Clin Barcelona, Grup Treball Malalties Pulm Intersticials, Pneumol Serv, Barcelona, Spain
[20] Univ Chicago, Rheumatol Div, Pritzker Sch Med, Chicago, IL USA
[21] Bombay Hosp & Med Res Ctr, Inst Med Sci, Mumbai, Maharashtra, India
[22] UCL, Ctr Med Imaging & Comp, London, England
[23] UCL, Dept Resp Med, London, England
[24] Univ Oxford, Nuffield Dept Orthopaed Rheumatol & Musculoskeleta, Oxford, England
[25] Liverpool Univ Hosp NHS Fdn Trust Lib & Knowledge, Aintree Hosp, Liverpool Interstitial Lung Dis Serv, Liverpool, England
[26] Berry Consultants, Austin, TX USA
[27] Mainz Univ, Mainz Ctr Pulm Med, Dept Pulmol, Med Ctr, Mainz, Germany
[28] Marienhaus Clin Mainz, Dept Pulm Crit Care & Sleep Med, Mainz, Germany
[29] Imperial Coll London, Div Anaesthet Pain Med & Intens Care, London, England
[30] Weill Cornell Med, Dept Med, Div Pulm & Crit Care Med, New York, NY USA
[31] Yale Sch Med, Pulm Crit Care & Sleep Med, New Haven, CT USA
[32] Imperial Coll London, Sch Publ Hlth, London, England
[33] Berry Consultants, Los Angeles, CA USA
[34] Act Pulm Fibrosis Fdn, London, England
[35] Imperial Coll London, Natl Heart & Lung Inst, Margaret Turner Warwick Ctr Fibrosing Lung Dis, London, England
[36] Hcor Hosp, Hcor Res Inst, BR-04004030 Sao Paulo, Brazil
基金
美国国家卫生研究院;
关键词
Connective tissue disease associated lung disease; Hypersensitivity pneumonitis; Idiopathic pulmonary fibrosis; Interstitial Fibrosis; Rheumatoid lung disease; IDIOPATHIC PULMONARY-FIBROSIS; DOUBLE-BLIND; PIRFENIDONE; UPDATE;
D O I
10.1136/thorax-2023-221148
中图分类号
R56 [呼吸系及胸部疾病];
学科分类号
摘要
Background Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).Methods Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.Results The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.Conclusion By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.
引用
收藏
页码:788 / 795
页数:8
相关论文
共 37 条
[1]   Occupational and environmental risk factors for idiopathic pulmonary fibrosis in Australia: case-control study [J].
Abramson, Michael J. ;
Murambadoro, Tsitsi ;
Alif, Sheikh M. ;
Benke, Geza P. ;
Dharmage, Shyamali C. ;
Glaspole, Ian ;
Hopkins, Peter ;
Hoy, Ryan F. ;
Klebe, Sonja ;
Moodley, Yuben ;
Rawson, Shuli ;
Reynolds, Paul N. ;
Wolfe, Rory ;
Corte, Tamera J. ;
Walters, E. Haydn .
THORAX, 2020, 75 (10) :864-869
[2]   Adaptive platform trials: definition, design, conduct and reporting considerations The Adaptive Platform Trials Coalition [J].
Angus, Derek C. ;
Alexander, Brian M. ;
Berry, Scott ;
Buxton, Meredith ;
Lewis, Roger ;
Paoloni, Melissa ;
Webb, Steven A. R. ;
Arnold, Steven ;
Barker, Anna ;
Berry, Donald A. ;
Bonten, Marc J. M. ;
Brophy, Mary ;
Butler, Christopher ;
Cloughesy, Timothy F. ;
Derde, Lennie P. G. ;
Esserman, Laura J. ;
Ferguson, Ryan ;
Fiore, Louis ;
Gaffey, Sarah C. ;
Gaziano, J. Michael ;
Giusti, Kathy ;
Goossens, Herman ;
Heritier, Stephane ;
Hyman, Bradley ;
Krams, Michael ;
Larholt, Kay ;
LaVange, Lisa M. ;
Lavori, Philip ;
Lo, Andrew W. ;
London, Alex John ;
Manax, Victoria ;
McArthur, Colin ;
O'Neill, Genevieve ;
Parmigiani, Giovanni ;
Perlmutter, Jane ;
Petzold, Elizabeth A. ;
Ritchie, Craig ;
Rowan, Kathryn M. ;
Seymour, Christopher W. ;
Shapiro, Nathan, I ;
Simeone, Diane M. ;
Smith, Bradley ;
Spellberg, Bradley ;
Stern, Ariel Dora ;
Trippa, Lorenzo ;
Trusheim, Mark ;
Viele, Kert ;
Wen, Patrick Y. ;
Woodcock, Janet .
NATURE REVIEWS DRUG DISCOVERY, 2019, 18 (10) :797-807
[3]   Computed Tomography Honeycombing Identifies a Progressive Fibrotic Phenotype with Increased Mortality across Diverse Interstitial Lung Diseases [J].
Adegunsoye, Ayodeji ;
Oldham, Justin M. ;
Bellam, Shashi K. ;
Montner, Steven ;
Churpek, Matthew M. ;
Noth, Imre ;
Vij, Rekha ;
Strek, Mary E. ;
Chung, Jonathan H. .
ANNALS OF THE AMERICAN THORACIC SOCIETY, 2019, 16 (05) :580-588
[4]   The REMAP-CAP (Randomized Embedded Multifactorial Adaptive Platform for Community-acquired Pneumonia) Study Rationale and Design [J].
Angus, Derek C. ;
Berry, Scott ;
Lewis, Roger J. ;
Al-Beidh, Farah ;
Arabi, Yaseen ;
van Bentum-Puijk, Wilma ;
Bhimani, Zahra ;
Bonten, Marc ;
Broglio, Kristine ;
Brunkhorst, Frank ;
Cheng, Allen C. ;
Chiche, Jean-Daniel ;
De Jong, Menno ;
Detry, Michelle ;
Goossens, Herman ;
Gordon, Anthony ;
Green, Cameron ;
Higgins, Alisa M. ;
Hullegie, Sebastiaan J. ;
Kruger, Peter ;
Lamontagne, Francois ;
Litton, Edward ;
Marshall, John ;
McGlothlin, Anna ;
McGuinness, Shay ;
Mouncey, Paul ;
Murthy, Srinivas ;
Nichol, Alistair ;
O'Neill, Genevieve K. ;
Parke, Rachael ;
Parker, Jane ;
Rohde, Gernot ;
Rowan, Kathryn ;
Turner, Anne ;
Young, Paul ;
Derde, Lennie ;
McArthur, Colin ;
Webb, Steven A. .
ANNALS OF THE AMERICAN THORACIC SOCIETY, 2020, 17 (07) :879-891
[5]   The DIAN-TU Next Generation Alzheimer's prevention trial: Adaptive design and disease progression model [J].
Bateman, Randall J. ;
Benzinger, Tammie L. ;
Berry, Scott ;
Clifford, David B. ;
Duggan, Cynthia ;
Fagan, Anne M. ;
Fanning, Kathleen ;
Farlow, Martin R. ;
Hassenstab, Jason ;
McDade, Eric M. ;
Mills, Susan ;
Paumier, Katrina ;
Quintana, Melanie ;
Salloway, Stephen P. ;
Santacruz, Anna ;
Schneider, Lon S. ;
Wang, Guoqiao ;
Xiong, Chengjie .
ALZHEIMERS & DEMENTIA, 2017, 13 (01) :8-19
[6]   Pirfenidone in patients with progressive fibrotic interstitial lung diseases other than idiopathic pulmonary fibrosis (RELIEF): a double-blind, randomised, placebo-controlled, phase 2b trial [J].
Behr, Juergen ;
Prasse, Antje ;
Kreuter, Michael ;
Johow, Johannes ;
Rabe, Klaus F. ;
Bonella, Francesco ;
Bonnet, Reiner ;
Grohe, Christian ;
Held, Matthias ;
Wilkens, Heinrike ;
Hammerl, Peter ;
Koschel, Dirk ;
Blaas, Stefan ;
Wirtz, Hubert ;
Ficker, Joachim H. ;
Neumeister, Wolfgang ;
Schoenfeld, Nicolas ;
Claussen, Martin ;
Kneidinger, Nikolaus ;
Frankenberger, Marion ;
Hummler, Simone ;
Kahn, Nicolas ;
Tello, Silke ;
Freise, Julia ;
Welte, Tobias ;
Neuser, Petra ;
Guenther, Andreas .
LANCET RESPIRATORY MEDICINE, 2021, 9 (05) :476-486
[7]   Adaptive clinical trials in oncology [J].
Berry, Donald A. .
NATURE REVIEWS CLINICAL ONCOLOGY, 2012, 9 (04) :199-207
[8]   The Platform Trial An Efficient Strategy for Evaluating Multiple Treatments [J].
Berry, Scott M. ;
Connor, Jason T. ;
Lewis, Roger J. .
JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2015, 313 (16) :1619-1620
[9]   Adaptive Designs for Clinical Trials [J].
Bhatt, Deepak L. ;
Mehta, Cyrus .
NEW ENGLAND JOURNAL OF MEDICINE, 2016, 375 (01) :65-74
[10]   Comparison of Bayesian vs Frequentist Adaptive Trial Design in the Stroke Hyperglycemia Insulin Network Effort Trial [J].
Broglio, Kristine ;
Meurer, William J. ;
Durkalski, Valerie ;
Pauls, Qi ;
Connor, Jason ;
Berry, Donald ;
Lewis, Roger J. ;
Johnston, Karen C. ;
Barsan, William G. .
JAMA NETWORK OPEN, 2022, 5 (05) :E2211616