Let us introduce you to the CARE PREP Acceleration Award!

The goal of this 1-year project is to develop a grant application for an international, precision medicine platform trial for cavernoma.

CARE PREP is one of the five Acceleration Awards that were funded by the National Institute for Health and Care Research Efficacy and Mechanism Evaluation programme in August 2022.

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During the past couple of weeks, we have started our collaboration on the international PREcision medicine Platform trial design. The Steering Committee and Patient Advisory Group (PAG) met for the first time in September. CAUK representative David White, Alliance to Cure Cavernous Malformation representatives Connie Lee & Carlos Casaus, Cavernoma Portugal representative João Próspero Luis and Cavernöst Angiom Sverige (CASE) representative Jana Bergholtz are part of the PAG for this grant.

Illustration from:
Park et. al (2020) An overview of platform trials with a checklist for clinical readers. Journal of Clinical Epidemiology. Vol. 125. pp 1-8. 

The aim of the project is to design a clinical trial using a “platform” clinical trial design. In this design, there are multiple treatment arms that share a placebo arm. Pre-planned interim evaluations allow ineffective treatments to be dropped early and new treatments to be added as replacements. This design speeds trials and requires smaller enrollment overall because of the shared placebo arm. The medicines to be tested have not been selected; such decisions will be made during the planning process. Platform trials are cutting edge and have not yet been used for many diseases. This is primarily because of the amount of cooperation required between international research groups and the amount of funding required.