Literature DB >> 28830236

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 Parmar1, Matthew R Sydes1, Fay H Cafferty1, Babak Choodari-Oskooei1, Ruth E Langley1, Louise Brown1, Patrick Pj Phillips1, Melissa R Spears1, Sam Rowley1, Richard Kaplan1, Nicholas D James2, Timothy Maughan3, Nicholas Paton1,4, Patrick J Royston1.   

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.

Entities:  

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

Mesh:

Year:  2017        PMID: 28830236      PMCID: PMC5700799          DOI: 10.1177/1740774517725697

Source DB:  PubMed          Journal:  Clin Trials        ISSN: 1740-7745            Impact factor:   2.486


  23 in total

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3.  Abiraterone for Prostate Cancer Not Previously Treated with Hormone Therapy.

Authors:  Nicholas D James; Johann S de Bono; Melissa R Spears; Noel W Clarke; Malcolm D Mason; David P Dearnaley; Alastair W S Ritchie; Claire L Amos; Clare Gilson; Rob J Jones; David Matheson; Robin Millman; Gerhardt Attard; Simon Chowdhury; William R Cross; Silke Gillessen; Christopher C Parker; J Martin Russell; Dominik R Berthold; Chris Brawley; Fawzi Adab; San Aung; Alison J Birtle; Jo Bowen; Susannah Brock; Prabir Chakraborti; Catherine Ferguson; Joanna Gale; Emma Gray; Mohan Hingorani; Peter J Hoskin; Jason F Lester; Zafar I Malik; Fiona McKinna; Neil McPhail; Julian Money-Kyrle; Joe O'Sullivan; Omi Parikh; Andrew Protheroe; Angus Robinson; Narayanan N Srihari; Carys Thomas; John Wagstaff; James Wylie; Anjali Zarkar; Mahesh K B Parmar; Matthew R Sydes
Journal:  N Engl J Med       Date:  2017-06-03       Impact factor: 91.245

4.  Medical research: Trial unpredictability yields predictable therapy gains.

Authors:  Benjamin Djulbegovic; Ambuj Kumar; Paul Glasziou; Branko Miladinovic; Iain Chalmers
Journal:  Nature       Date:  2013-08-22       Impact factor: 49.962

5.  Celecoxib plus hormone therapy versus hormone therapy alone for hormone-sensitive prostate cancer: first results from the STAMPEDE multiarm, multistage, randomised controlled trial.

Authors:  Nicholas D James; Matthew R Sydes; Malcolm D Mason; Noel W Clarke; John Anderson; David P Dearnaley; John Dwyer; Gordana Jovic; Alastair W S Ritchie; J Martin Russell; Karen Sanders; George N Thalmann; Gianfilippo Bertelli; Alison J Birtle; Joe M O'Sullivan; Andrew Protheroe; Denise Sheehan; Narayanan Srihari; Mahesh K B Parmar
Journal:  Lancet Oncol       Date:  2012-03-26       Impact factor: 41.316

6.  Addition of docetaxel, zoledronic acid, or both to first-line long-term hormone therapy in prostate cancer (STAMPEDE): survival results from an adaptive, multiarm, multistage, platform randomised controlled trial.

Authors:  Nicholas D James; Matthew R Sydes; Noel W Clarke; Malcolm D Mason; David P Dearnaley; Melissa R Spears; Alastair W S Ritchie; Christopher C Parker; J Martin Russell; Gerhardt Attard; Johann de Bono; William Cross; Rob J Jones; George Thalmann; Claire Amos; David Matheson; Robin Millman; Mymoona Alzouebi; Sharon Beesley; Alison J Birtle; Susannah Brock; Richard Cathomas; Prabir Chakraborti; Simon Chowdhury; Audrey Cook; Tony Elliott; Joanna Gale; Stephanie Gibbs; John D Graham; John Hetherington; Robert Hughes; Robert Laing; Fiona McKinna; Duncan B McLaren; Joe M O'Sullivan; Omi Parikh; Clive Peedell; Andrew Protheroe; Angus J Robinson; Narayanan Srihari; Rajaguru Srinivasan; John Staffurth; Santhanam Sundar; Shaun Tolan; David Tsang; John Wagstaff; Mahesh K B Parmar
Journal:  Lancet       Date:  2015-12-21       Impact factor: 79.321

Review 7.  Addition of docetaxel or bisphosphonates to standard of care in men with localised or metastatic, hormone-sensitive prostate cancer: a systematic review and meta-analyses of aggregate data.

Authors:  Claire L Vale; Sarah Burdett; Larysa H M Rydzewska; Laurence Albiges; Noel W Clarke; David Fisher; Karim Fizazi; Gwenaelle Gravis; Nicholas D James; Malcolm D Mason; Mahesh K B Parmar; Christopher J Sweeney; Matthew R Sydes; Bertrand Tombal; Jayne F Tierney
Journal:  Lancet Oncol       Date:  2015-12-21       Impact factor: 41.316

8.  ADD-ASPIRIN: A phase III, double-blind, placebo controlled, randomised trial assessing the effects of aspirin on disease recurrence and survival after primary therapy in common non-metastatic solid tumours.

Authors:  Christopher Coyle; Fay H Cafferty; Samuel Rowley; Mairead MacKenzie; Lindy Berkman; Sudeep Gupta; C S Pramesh; Duncan Gilbert; Howard Kynaston; David Cameron; Richard H Wilson; Alistair Ring; Ruth E Langley
Journal:  Contemp Clin Trials       Date:  2016-10-21       Impact factor: 2.226

9.  Evaluating many treatments and biomarkers in oncology: a new design.

Authors:  Richard Kaplan; Timothy Maughan; Angela Crook; David Fisher; Richard Wilson; Louise Brown; Mahesh Parmar
Journal:  J Clin Oncol       Date:  2013-11-18       Impact factor: 44.544

10.  Speeding up the evaluation of new agents in cancer.

Authors:  Mahesh K B Parmar; Friederike M-S Barthel; Matthew Sydes; Ruth Langley; Rick Kaplan; Elizabeth Eisenhauer; Mark Brady; Nicholas James; Michael A Bookman; Ann-Marie Swart; Wendi Qian; Patrick Royston
Journal:  J Natl Cancer Inst       Date:  2008-08-26       Impact factor: 13.506

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  28 in total

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Journal:  BMJ       Date:  2020-06-17

2.  Motor Neuron Disease Systematic Multi-Arm Adaptive Randomised Trial (MND-SMART): a multi-arm, multi-stage, adaptive, platform, phase III randomised, double-blind, placebo-controlled trial of repurposed drugs in motor neuron disease.

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3.  Trials for neurodegenerative diseases: time to innovate.

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4.  Three-month Prostate-specific Antigen Level After Androgen Deprivation Therapy Predicts Survival in Patients With Metastatic Castration-sensitive Prostate Cancer.

Authors:  Naohiro Fujimoto; Masaki Shiota; Takuo Matsukawa; Akinori Minato; Ikko Tomisaki; Rei Ohnishi; Masatoshi Eto
Journal:  In Vivo       Date:  2021 Mar-Apr       Impact factor: 2.155

5.  Multi-arm Clinical Trials - Teams within Teams.

Authors:  R E Langley; M D Mason
Journal:  Clin Oncol (R Coll Radiol)       Date:  2017-10-27       Impact factor: 4.126

Review 6.  Results of the Seventh Scientific Workshop of ECCO: Precision Medicine in IBD-Disease Outcome and Response to Therapy.

Authors:  Bram Verstockt; Nurulamin M Noor; Urko M Marigorta; Polychronis Pavlidis; Parakkal Deepak; Ryan C Ungaro
Journal:  J Crohns Colitis       Date:  2021-09-25       Impact factor: 9.071

7.  Adding abiraterone or docetaxel to long-term hormone therapy for prostate cancer: directly randomised data from the STAMPEDE multi-arm, multi-stage platform protocol.

Authors:  M R Sydes; M R Spears; M D Mason; N W Clarke; D P Dearnaley; J S de Bono; G Attard; S Chowdhury; W Cross; S Gillessen; Z I Malik; R Jones; C C Parker; A W S Ritchie; J M Russell; R Millman; D Matheson; C Amos; C Gilson; A Birtle; S Brock; L Capaldi; P Chakraborti; A Choudhury; L Evans; D Ford; J Gale; S Gibbs; D C Gilbert; R Hughes; D McLaren; J F Lester; A Nikapota; J O'Sullivan; O Parikh; C Peedell; A Protheroe; S M Rudman; R Shaffer; D Sheehan; M Simms; N Srihari; R Strebel; S Sundar; S Tolan; D Tsang; M Varughese; J Wagstaff; M K B Parmar; N D James
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8.  The adaptive designs CONSORT extension (ACE) statement: a checklist with explanation and elaboration guideline for reporting randomised trials that use an adaptive design.

Authors:  Munyaradzi Dimairo; Philip Pallmann; James Wason; Susan Todd; Thomas Jaki; Steven A Julious; Adrian P Mander; Christopher J Weir; Franz Koenig; Marc K Walton; Jon P Nicholl; Elizabeth Coates; Katie Biggs; Toshimitsu Hamasaki; Michael A Proschan; John A Scott; Yuki Ando; Daniel Hind; Douglas G Altman
Journal:  Trials       Date:  2020-06-17       Impact factor: 2.279

Review 9.  New strategies in ovarian cancer treatment.

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10.  Optimizing Clinical Trial Design to Maximize Evidence Generation in Pediatric HIV.

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