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Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage

Blenkinsop, A; Choodari-Oskooei, B; (2019) Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage. The Stata Journal , 19 (4) pp. 782-802. 10.1177/1536867X19893616. Green open access

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Abstract

Royston et al.’s (2011, Trials 12: 81) multiarm, multistage (MAMS) framework for the design of randomized clinical trials uses intermediate outcomes to drop research arms early for lack of benefit at interim stages, increasing efficiency in multiarm designs. However, additionally permitting interim evaluation of efficacy on the primary outcome measure could increase adoption of the design and result in practical benefits, such as savings in patient numbers and cost, should any efficacious arm be identified early. The nstage command, which aids the design of MAMS trial designs, has been updated to support this methodological extension. Operating characteristics can now be calculated for a design with binding or nonbinding stopping rules for lack of benefit and with efficacy stopping boundaries. An additional option searches for a design that strongly controls the familywise error rate at the desired level. We illustrate how the new features can be used to design a trial with the drop-down menu, using the original comparisons from the MAMS trial STAMPEDE as an example. The new functionality of the command will serve a broader range of trial objectives and increase efficiency of the design and thus increase uptake of the MAMS design in practice.

Type: Article
Title: Multiarm, multistage randomized controlled trials with stopping boundaries for efficacy and lack of benefit: An update to nstage
Open access status: An open access version is available from UCL Discovery
DOI: 10.1177/1536867X19893616
Publisher version: https://doi.org/10.1177/1536867X19893616
Language: English
Additional information: This article is distributed under the terms of the Creative Commons Attribution 4.0 License (https://creativecommons.org/licenses/by/4.0/) which permits any use, reproduction and distribution of the work without further permission provided the original work is attributed as specified on the SAGE and Open Access pages (https://us.sagepub.com/en-us/nam/open-access-at-sage).
Keywords: st0175 2, nstage, nstagemenu, multiarm multistage, familywise error rate, efficacy stopping boundaries, adaptive designs
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Inst of Clinical Trials and Methodology > MRC Clinical Trials Unit at UCL
URI: https://discovery.ucl.ac.uk/id/eprint/10083247
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