C4TR-POLG

Coalition for trial readiness in POLG

01.10.2023 bis 30.09.2026

Studying treatments for ultra-rare forms of mitochondrial disease is challenging. Participant pools are small and restricted by rigid inclusion and exclusion criteria. There is often incomplete understanding of genotype–phenotype relationships and natural history to inform trial endpoint development. Tackling these disparities is recognised as a long-standing, complex conundrum. Leveraging value in multistakeholder innovative networks is urgently needed. C4TR-POLG will capitalise on existing pockets of world-class excellence in POLG- related mitochondrial disease and ataxia and will explore the appropriateness and scalability of emerging outcome measures and potential barriers to their adoption. This programme of work will build capacity and develop new collaborative ways of working together to generate richer, more relevant data to accelerate global POLG-related research, connectivity, and improved patient care.