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

Multicentre presymptomatic cohort studies are underway in a number of different
neurodegenerative diseases and will be central to trials of disease modifying therapies
as they provide the opportunity for early intervention. Although each disorder has its own
unique issues in relation to such trials, there are shared themes across the different
diseases that would benefit from greater discussion and interaction between experts in
the field. This working group will bring together experts in presymptomatic studies of
genetic Alzheimer's disease, frontotemporal dementia, Huntington's disease,
Parkinson's disease, prion disease, motor neuron disease and the spinocerebellar
ataxias. The groups will identify the key shared themes including trial design, the use of
surrogate 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 move
from sharing individual disease-based perspectives to producing cross-disease
guidelines for trials in presymptomatic neurodegenerative disease.