Developing a methodological framework for trials in presymptomatic neurodegenerative disease - the Presymptomatic Neurodegeneration Initiative (PreNI)

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Multicentre presymptomatic cohort studies are underway in a number of differentneurodegenerative diseases and will be central to trials of disease modifying therapiesas they provide the opportunity for early intervention. Although each disorder has its ownunique issues in relation to such trials, there are shared themes across the differentdiseases that would benefit from greater discussion and interaction between experts inthe field. This working group will bring together experts in presymptomatic studies ofgenetic Alzheimer's disease, frontotemporal dementia, Huntington's disease,Parkinson's disease, prion disease, motor neuron disease and the spinocerebellarataxias. The groups will identify the key shared themes including trial design, the use ofsurrogate endpoints, regulatory perspectives, academic-industry partnerships,recruitment and registers, ethical considerations, and support for participants in trials.Through a series of face-to-face workshops and webex meetings the group will movefrom sharing individual disease-based perspectives to producing cross-diseaseguidelines for trials in presymptomatic neurodegenerative disease.