Project: 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.

Acronym JPNDWG00486 (Reference Number: JPNDWG00486)
Duration 01/01/2014 - 01/01/2015
Project Topic Neurodegenerative disease
Website visit project website
Network JPND
Call A Call for Proposals for Working Groups to Inform Cohort Studies in Neurodegenerative Disease Research

Project partner

Number Name Role Country
1 University College London Coordinator United Kingdom