Statistical & practical aspects of the design/analysis of Multi-Arm Multi-Stage Platform Trials

This workshop is designed for individuals interested in gaining a deeper understanding of the design and analysis of multi-arm multi-stage (MAMS) platform trials, including those who are new to the field.

There will be an opportunity for delegates to send questions in advance so that they can be addressed at the workshop.

This workshop aims to help participants:

• Understand the motivation behind these designs
• Learn how to choose the design parameters and stopping boundaries, both for lack-of-benefit and efficacy
• Learn how to deal with overwhelming efficacy
• Learn about stopping randomisation to research arms
• Learn how to add a new research arm, and how to control Type I and II error rates in both pre-planned and unplanned addition of a new arm
• Learn about MAMS designs in which arms are ranked and selectively chosen to continue
• Learn how the programs written in Stata software can be used to design MAMS trials with time-to-event and binary outcomes

MAMS platform trials

Typically, in these protocols, randomisation is stopped to insufficiently active treatment arms at interim stages and new research arms can be added during the course of the trial. MRC Clinical Trials Unit at UCL is a leader not only in the design, but also in implementation and analysis of such trials.  The MAMS approach is one of the few adaptive designs being deployed in a number of trials and across a range of disease in the phase III setting, including STAMPEDE (prostate cancer), CompARE (TB), TRUNCATE-TB (TB), RAMPART (renal cancer), and ROSSINI-II (wound surgery) .

Structure of the workshop

This half-day workshop consists of two main sessions. The first session of the workshop provides an overview of the design issues involved in MAMS platform protocols. The second session focuses on the implementation of the statistical aspects of such trials and provides guidelines on the design and analysis of such trials. It will also explore further design issues such as adding new research arms, and designs in which research arms are ranked and selectively chosen to continue.

Software and examples

In both sessions, the methods are explored using real platform trial examples, and using programs written in Stata software - i.e. the nstage suite in Stata for trials with binary and time-to-event outcomes, which can be usefully partnered with artpep for time-to-event projections.

The workshop concludes with a discussion session when participants have the opportunity to discuss specific design and analysis questions with the panel. For this reason, we encourage those planning to attend the workshop to submit their questions to the above email in advance, any time after registration

The panel will consist of Dr Babak Choodari-Oskooei and Prof Max Parmar.

Fees

This course is free for UCL members of staff within ICTM (Cancer Research UK & UCL Cancer Trials Centre, Comprehensive Clinical Trials Unit at UCL, Medical Research Council Clinical Trials Unit at UCL and PRIMENT Clinical Trials Unit) although places are limited.  Please contact ictm.cpd@ucl.ac.uk for a validation code to make the registration payment. 

• £107 - Low- and Middle- Income Countries (LMIC)
• £214 - Academics / attendees from not-for-profit organisations
• £257 - Attendees from for-profit organisations