Second Workshop of the NIHR Statistics Group’s Early Phase Trials Research Section

Category: Early phase clinical trials

Location: Rooms 2&3, Biomedical Research Center, 16th Floor, Guy's Hospital Great Maze Pond London SE1 9RT | Date: 16 February 2018

The second workshop of the NIHR Statistics Early Phase Trials group is being held on the 16th February 2018 in central London at Guy’s Hospital.

There will be two sessions.

  1. The morning session will be focused on statistical methodology.
  2. The afternoon session will be a collaborative dialogue between statisticians and clinicians working in this area.

Full agenda below.

Please use this link to register for both morning and afternoon sessions, this would typically be for statisticians but clinicians and researchers are welcome.

If you are a clinician and wish to attend the afternoon session only or are unable to attend but would like to submit a question please use this link. We are keen to know your professional background, the more information you can provide the better we can make the meeting


Morning Session

10:0-10:30           Registration and Coffee

10.30           Welcome – Simon Bond Addenbrookes Hospital, Cambridge University Hospitals

10:30-11:30        Early Phase Trials Group Outputs to date

Victoria Cornelius Imperial College London

Sharon Love University of Oxford, Slides

Graham Wheeler University College London 

Content: We aim to motivate wider use of model-based designs for dose-finding. We have identified perceived barriers and we suggest required changes. We will discuss the continual reassessment method, CRM, in detail giving suggestions on design parameters.

Objectives: During this session you will hear reasons for the lack of use of model based designs and learn some ways round them. With respect to the CRM, you should leave with an idea of one way to parameterise a trial.

11:30 – 13:00     Rare Diseases

Simon Skene University College London, Slides

Graham Wheeler University College London, Slides

Content: Trials of advanced therapies in rare diseases often have very small sample sizes. This presents several challenges, including how best to design the study. Attendees will be presented with a real trial, and discuss how best to design the study given various constraints.

Objectives: From this, we will develop new work on how best to design future trials that overcome key problems in this area.”

13:00- 13:30 Lunch

Afternoon Session

13:30- 14:00       Practical application of dose-response models

Phillip Pallmann University of Cardiff, Slides

Content: we will demonstrate the functionality of MoDEsT, a free and easy-to-use web tool for designing and evaluating dose-escalation studies, and illustrate the practical workflow when designing and conducting a phase I dose-escalation study with a previously published data example.

Objectives: Enabling clinicians to fully comprehend model-based designs and apply them with confidence, and to try out various design options and watch the changes become visible in real time. Thus tasks that currently discourage people from using model-based designs, like having to formalise prior beliefs, will appear much less daunting.

14:00-14:30        Time-to-toxicity-event trials

Jane Holmes University of Oxford, Slides

Content: We will give a brief introduction to the CRM modification TiTE-CRM and give an example of applying this design to a real trial. We will discuss some of the hurdles we faced and how we dealt with them.

Objectives:During this session we will introduce one of the modifications to the CRM design, the TiTE-CRM which is useful in situations where the toxicity period is long ( eg for radiotherapy).  Via a real trial example, you will learn of some of the items to consider when using this design.

14:30 -15:30       Five thoughts on early phase trials, Q&A Session

Sharon Love University of Oxford, Slides

Simon Bond Addenbrookes Hospital, Cambridge University Hospitals

Content:  Have you run model based designs and had to change the way you work on the fly? We give 5 ways in which early phase CRM design trials are different. Forewarned is forearmed!

Objectives: During this session you will find out about some practicalities of running early phase CRM and CRM modification design trials

15:30 – 16:00     Round-up of workshop outputs and action points

Graham Wheeler University College London

Victoria Cornelius Imperial College London

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