SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols

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作者
Ursino, Moreno [1 ,2 ,3 ,4 ]
Villacampa, Guillermo [5 ,6 ,7 ]
Rekowski, Jan [5 ]
Dimairo, Munyaradzi [8 ]
Solovyeva, Olga [5 ]
Ashby, Deborah [9 ]
Berlin, Jordan [10 ]
Boix, Oliver [11 ]
Calvert, Melanie [11 ,12 ,13 ,14 ,15 ,16 ]
Chan, An-Wen [15 ,17 ]
Coschi, Courtney H. [16 ,18 ]
Evans, Thomas R. Jeffry [17 ,19 ]
Garrett-Mayer, Elizabeth [18 ,20 ]
Golub, Robert M. [19 ,21 ]
Guo, Christina [20 ,21 ,22 ,23 ]
Hayward, Kathryn S. [22 ,23 ,24 ,25 ,26 ]
Hopewell, Sally [27 ]
Isaacs, John D. [28 ,29 ]
Ivy, S. Percy [30 ]
Jaki, Thomas [31 ,32 ]
Kholmanskikh, Olga [33 ]
Kightley, Andrew [34 ]
Lee, Shing [35 ]
Liu, Rong [36 ]
Mander, Adrian [37 ]
Marshall, Lynley, V [20 ,21 ,22 ,23 ]
Matcham, James [38 ]
Patel, Dhrusti [5 ]
Peck, Richard [39 ,40 ]
Rantell, Khadija Rerhou [41 ]
Richards, Dawn P. [42 ]
Rouhifard, Mahtab [5 ]
Seymour, Lesley [16 ,18 ]
Tanaka, Yoshiya [43 ]
Weir, Christopher J. [44 ]
de Bono, Johann [22 ,23 ]
Yap, Christina [5 ]
机构
[1] INSERM, ReCAP F CRIN, F-5400 Nancy, France
[2] Univ Paris Cite, Univ Hosp Ctr Robert Debre, Unit Clin Epidemiol, Paris, France
[3] Univ Paris Cite, Sorbonne Univ, Ctr Rech Cordeliers, INSERM, Paris, France
[4] Ctr Inria, HeKA Team, Paris, France
[5] Inst Canc Res, Clin Trials & Stat Unit, London SM2 5NG, England
[6] Vall dHebron Inst Oncol VHIO, Stat Unit, Barcelona, Spain
[7] SOLTI Breast Canc Res Grp, Barcelona, Spain
[8] Univ Sheffield, Sheffield Ctr Hlth & Related Res, Div Populat Hlth, Sheffield, England
[9] Imperial Coll London, St Marys Hosp, Sch Publ Hlth, London, England
[10] Vanderbilt Ingram Canc Ctr, Nashville, TN USA
[11] Bayer, Berlin, Germany
[12] Univ Birmingham, Inst Appl Hlth Res, Ctr Patient Reported Outcomes Res, Birmingham, England
[13] Univ Birmingham, Birmingham Hlth Partners Ctr Regulatory Sci & Inno, Birmingham, England
[14] Univ Birmingham, Natl Inst Hlth & Care Res Appl Res Collaborat West, Birmingham, England
[15] Univ Birmingham, Natl Inst Hlth & Care Res, Blood & Transplant Res Unit Precis Transplant & Ce, Birmingham, England
[16] Univ Hosp NHS Fdn Trust, Birmingham Biomed Res Ctr, Natl Inst Hlth & Care Res, NIHR Birmingham Biomed Res Ctr,Inst Translat Med, Birmingham, England
[17] Univ Toronto, Womens Coll Res Inst, Dept Med, Toronto, ON, Canada
[18] Canadian Canc Trials Grp, Kingston, ON, Canada
[19] Univ Glasgow, Inst Canc Sci, CR UK Beatson Inst, Glasgow, Scotland
[20] Amer Soc Clin Oncol, Ctr Res & Analyt, Alexandria, VA USA
[21] Northwestern Univ Feinberg, Sch Med, Dept Med, Chicago, IL USA
[22] Inst Canc Res, London, England
[23] Royal Marsden NHS Fdn Trust, London, England
[24] Univ Melbourne, Dept Physiotherapy, Melbourne, Vic, Australia
[25] Univ Melbourne, Dept Med, Melbourne, Vic, Australia
[26] Univ Melbourne, Florey Inst Neurosci & Mental Hlth, Melbourne, Vic, Australia
[27] Univ Oxford, Oxford Clin Res Unit, NDORMS, Oxford, England
[28] Newcastle Univ, Translat & Clin Res Inst, Newcastle Upon Tyne, England
[29] Newcastle Tyne Hosp NHS Fdn Trust, Freeman Hosp, NIHR Newcastle Biomed Res Ctr, Newcastle Upon Tyne, England
[30] NIH, Div Canc Treatment & Diag, Canc Therapy Evaluat Program, Invest Drug Branch, Bethesda, MD 20892 USA
[31] Univ Cambridge, MRC Biostat Unit, Cambridge, England
[32] Univ Regensburg, Computat Stat Grp, Regensburg, Germany
[33] Fed Agcy Med & Hlth Prod, Brussels, Belgium
[34] Patient & Publ Involvement & Engagement PPIE Lead, Lichfield, England
[35] Columbia Univ, Mailman Sch Publ Hlth, New York, NY USA
[36] Regeneron, New York, NY USA
[37] Cardiff Univ, Ctr Trials Res, Cardiff, Wales
[38] Cytel Australia, Strateg Consulting, Perth, WA, Australia
[39] Univ Liverpool, Dept Pharmacol & Therapeut, Liverpool, England
[40] Hoffmann La Roche AG, Basel, Switzerland
[41] Med & Healthcare Prod Regulatory Agcy, London, England
[42] Clin Trials Ontario, MaRS Ctr, Toronto, ON, Canada
[43] Univ Occupat & Environm Hlth, Dept Internal Med 1, Kitakyushu, Japan
[44] Univ Edinburgh, Usher Inst, Edinburgh Clin Trials Unit, Edinburgh, Scotland
关键词
Early phase trials; Phase I; Dose-finding; Dose escalation/de-escalation; Protocol guidance; SPIRIT-DEFINE; SPIRIT; ADAPTIVE DESIGNS; 1ST-IN-HUMAN; TRENDS;
D O I
10.1016/j.eclinm.2024.102988
中图分类号
R5 [内科学];
学科分类号
1002 ; 100201 ;
摘要
Transparent and accurate reporting in early phase dose-finding (EPDF) clinical trials is crucial for informing subsequent larger trials. The SPIRIT statement, designed for trial protocol content, does not adequately cover the distinctive features of EPDF trials. Recent fi ndings indicate that the protocol contents in past EPDF trials frequently lacked completeness and clarity. To address this gap, the international consensus-driven SPIRIT-DEFINE checklist was developed through a robust methodological framework for guideline development, with the aim to improve completeness and clarity in EPDF trial protocols. The checklist builds on the SPIRIT statement, adding 17 new items and modifying 15 existing ones. The SPIRIT-DEFINE explanation and elaboration (E&E) document provides comprehensive information to enhance understanding and usability of the SPIRIT-DEFINE checklist when writing an EPDF trial protocol. Each new or modified checklist item is accompanied by a detailed description, its rationale with supportive evidence, and examples of good reporting curated from EPDF trial protocols covering a range of therapeutic areas and interventions. We recommend utilising this paper alongside the SPIRIT statement, and any relevant extensions, to enhance the development and review of EPDF trial protocols. By facilitating adoption of the SPIRIT-DEFINE statement for EPDF trials, this E&E document can promote enhancement of methodological rigour, patient safety, transparency, and facilitate the generation of high-quality, reproducible evidence that will strengthen the foundation of early phase research and ultimately improve patient outcomes. Funding This work is a further extension of the SPIRIT-DEFINE study, which obtained no external funding. The principal investigator (CY) used internal staff resources, together with additional resources from external partners, to conduct this study. The SPIRIT-DEFINE study is a component of the DEFINE project, which also developed the MRC/NIHR funded CONSORT-DEFINE guidance. ICR-CTSU receives programmatic infrastructure funding from Cancer Research UK (C1491/A25351; CTUQQR-Dec22/100004), which has contributed to accelerating the advancement and successful completion of this work. Copyright (c) 2024 The Author(s). Published by Elsevier Ltd. This is an open access article under the CC BY-NC license (http://creativecommons.org/licenses/by-nc/4.0/).
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