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CARE PREP: New cutting edge research trial for cavernoma

Cavernous malformations: A Randomised Efficacy PREcision medicine Platform trial (CARE PREP)

We are delighted to announce that a consortium, led by CAUK medical advisor Professor Rustam Al-Shahi Salman and comprising of a number of leading clinicians and patient advocacy organisation leaders – including David White of CAUK – has been awarded a grant to initiate the planning of an international ‘platform trial’ to test multiple potential cavernoma medications at a time.

This new research study, funded by a special NIHR programme, is an international project, involving the UK, the US and Europe. For the trial to succeed, a significant patient, carer and public input is required; thus, the trial’s Patient and Public Advisory Group (PAG) consists of CAUK’s David White, Connie Lee of Alliance to Cure (US), Jana Bergholtz of the European Cavernoma Association (ECA), Carlos Casaus from the Alliance to Cure, and João Próspero Luís of Cavernoma Portugal.

The project

The aim of the CARE PREP project is to design an application for funding for a type of clinical trial – called a ‘platform trial’ – that will determine the effectiveness of several potential drugs for the treatment of cavernoma. At the end of the project year, the planned application will be submitted to NIHR for funding.

What is a platform trial?

Platform trials are an advanced type of randomised clinical trial (RCT) that allow for concurrent evaluations of multiple treatments. These types of trials are an efficient way to compare several interventions to a shared placebo at once.

The aim of the CARE PREP platform trial is to evaluate the effectiveness of several potential drugs for the treatment of cavernoma at the same time.

The planning process

Since platform trials are cutting edge, complex and have not yet been used for many diseases, the consortium will use this planning year to determine the details of what exactly is required for success, and to ascertain whether such a trial is feasible with cavernoma.

The design

The drugs to be tested will be confirmed during the planning process, though the trial plans to start with 3 drugs that are used for other conditions: a betablocker, a statin, and aspirin.

The trial will be designed to study drug effects on patients grouped according to gene changes or CCM location (known as ‘stratification’), which will tell us whether these drugs can be targeted at patients (known as ‘precision medicine’). We will identify additional drugs and seek collaboration from commercial partners with drugs that might benefit CCM; one commercial partner is collaborating already.

This is a really exciting opportunity for cavernoma to become an example of how platform trials can be used to effectively and efficiently test multiple treatments.

It would not be possible to execute a platform clinical trial of this nature and complexity without the collaboration and cooperation of an international scientific and research community. We are grateful to be part of a wonderful international cavernoma community that consistently works together to advance treatment and care for cavernoma.