Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2017 Oct;14(5):451-461.
doi: 10.1177/1740774517725697. Epub 2017 Aug 22.

Testing many treatments within a single protocol over 10 years at MRC Clinical Trials Unit at UCL: Multi-arm, multi-stage platform, umbrella and basket protocols

Mahesh Kb Parmar  1 , Matthew R Sydes  1 , Fay H Cafferty  1 , Babak Choodari-Oskooei  1 , Ruth E Langley  1 , Louise Brown  1 , Patrick Pj Phillips  1 , Melissa R Spears  1 , Sam Rowley  1 , Richard Kaplan  1 , Nicholas D James  2 , Timothy Maughan  3 , Nicholas Paton  1   4 , Patrick J Royston  1
Affiliations

Testing many treatments within a single protocol over 10 years at MRC Clinical Trials Unit at UCL: Multi-arm, multi-stage platform, umbrella and basket protocols

Mahesh Kb Parmar et al. Clin Trials. 2017 Oct.

Abstract

There is real need to change how we do some of our clinical trials, as currently the testing and development process is too slow, too costly and too failure-prone often we find that a new treatment is no better than the current standard. Much of the focus on the development and testing pathway has been in improving the design of phase I and II trials. In this article, we present examples of new methods for improving the design of phase III trials (and the necessary lead up to them) as they are the most time-consuming and expensive part of the pathway. Key to all these methods is the aim to test many treatments and/or pose many therapeutic questions within one protocol.

Keywords: Multi-arm; basket; multi-stage; platform; randomised trials; umbrella.

PubMed Disclaimer

Figures

Figure 1
Figure 1. STAMPEDE Protocol (a) at initiation (figure produced in Oct-2005) and (b) adapted protocol from 2005 to 2024 (figure produced in Sep-2016)
Figure 2
Figure 2. Protocol Schema for TRUNCATE TB
Figure 3
Figure 3. Protocol Schema for FOCUS4
Figure 4
Figure 4. Protocol Schema for Add-Aspirin
See this image and copyright information in PMC

References

    1. Djulbegovic B, Kumar A, Glasziou P, Miladinovic B, Chalmers I. Trial unpredictability yields predictable therapy gains. Nature. 2013;500(7463):395–6. doi: 10.1038/500395a. - DOI - PMC - PubMed
    1. Berry DA. The Brave New World of clinical cancer research: Adaptive biomarker-driven trials integrating clinical practice with clinical research. Mol Oncol. 2015;9(5):951–9. - PMC - PubMed
    1. Liu S, Lee JJ. An overview of the design and conduct of the BATTLE trials. Chin Clin Oncol. 2015;4(3):33. - PubMed
    1. O'Quigley J, Conaway M. Continual Reassessment and Related Dose-Finding Designs. Statistical Science. 2010;25(2):202–216. - PMC - PubMed
    1. Parmar MKB, Carpenter J, Sydes MR. More multiarm randomised trials of superiority are needed. 2014;384(9940):283–284. - PubMed

LinkOut - more resources