Building an international precision medicine platform trial for the acute respiratory distress syndrome (ARDS)

The acute respiratory distress syndrome (ARDS) is a heterogeneous clinical syndrome characterised by acute hypoxaemic respiratory failure. The “syndromic” definition of ARDS ignores the significant clinical and biological heterogeneity within ARDS. A novel precision medicine approach to identifying pharmacological treatments for ARDS, targeting ARDS phenotypes which are linked to the host biological response, is urgently required.

The objective of this application is to support the development of an international precision medicine platform trial aiming to efficiently test several pharmacological interventions in biological phenotypes in ARDS. This will be achieved by undertaking a series of interlinked work packages which will aim to:

1) define the best approach to phenotyping patients and prioritising initial interventions to be tested. 2) develop the study design and draft platform trial core (master) protocol and analysis plan.

3) undertake collaborative work with international regulators, sponsors and funders to define their individual requirements in the delivery of a UK led platform trial. 4) incorporate learning from existing platform trials. 5) build research capacity for clinical trial delivery. 6) engage with industry to develop pathways for testing novel agents and for sustainable funding.

7) develop a framework to embed biobanking globally within the platform trial. 8) ensure the patient and public voice is incorporated in every aspect of the trial design and execution. This project will be delivered over a 12 month period by a world class team. This project will establish the infrastructure to deliver an international precision medicine platform trial for ARDS.

Dissemination will take multiple routes including publication in international journals, presentation at relevant national and international conferences, communication with participant and family networks as well as through social media and the lay press. Learning from this project will be shared as a template for future precision medicine trials in critical care.