Developing an evidence base for trials in genetic frontotemporal dementia – measures of disease onset and progression
Award Number
MR/M008525/1Programme
Research GrantStatus / Stage
ActiveDates
1 August 2015 -31 July 2019
Duration (calculated)
03 years 11 monthsFunder(s)
MRC (UKRI)Funding Amount
£880,122.06Funder/Grant study page
MRC UKRIContracted Centre
University College LondonPrincipal Investigator
Dr Rohrer, Jonathan DanielPI Contact
jonathan.rohrer@nhs.netPI ORCID
0000-0002-6155-8417WHO Catergories
Development of BiomarkersUnderstanding Underlying Disease
Disease Type
Frontotemporal Dementia (FTD)CPEC Review Info
| Reference ID | 265 |
|---|---|
| Researcher | Reside Team |
| Published | 12/06/2023 |
Data
| Award Number | MR/M008525/1 |
|---|---|
| Status / Stage | Active |
| Start Date | 20150801 |
| End Date | 20190731 |
| Duration (calculated) | 03 years 11 months |
| Funder/Grant study page | MRC UKRI |
| Contracted Centre | University College London |
| Funding Amount | £880,122.06 |
Abstract
Frontotemporal dementia (FTD) is a common cause of young onset dementia, approximately equal in frequency to Alzheimer’s disease in people below the age of 65. Its effect particularly on people of working age with young families represents a major health and economic burden on society. The only known risk factors for FTD are genetic, and around a third of FTD is familial. The aim of the study is to understand the ordering and temporal evolution of imaging and fluid biomarker change in genetic FTD and their clinical associations, forming an evidence base for future clinical trials. In particular, through comprehensive longitudinal phenotyping, the key objectives are to: characterise the earliest neuropsychological and behavioural changes; identify the earliest anatomical changes that occur and how these change over time; evaluate how measures of structural and functional connectivity change over time; examine the role of tau PET imaging as a marker of tau pathology; and correlate changes in CSF and serum markers with changes in neuroimaging. The experimental plan uses cross-sectional and longitudinal measures of behaviour, cognition, neuroimaging and both CSF and serum markers to elucidate the patterns of biomarker change in genetic FTD with the overarching hypothesis being that biomarkers can aid in the identification of disease and tracking of disease progression. The study aims to be the foundation for clinical trials in genetic FTD with the key outcomes being identification of markers of disease onset including those including those indicative of optimal time to start disease-modifying therapy and markers of disease progression that can be used as outcome measures in clinical trials, as well as estimations of the sample sizes necessary for those trials.
Aims
The aim of the study is to understand the ordering and temporal evolution of imaging and fluid biomarker change in genetic FTD and their clinical associations, forming an evidence base for future clinical trials. In particular, through comprehensive longitudinal phenotyping, the key objectives are to: characterise the earliest neuropsychological and behavioural changes; identify the earliest anatomical changes that occur and how these change over time; evaluate how measures of structural and functional connectivity change over time; examine the role of tau PET imaging as a marker of tau pathology; and correlate changes in CSF and serum markers with changes in neuroimaging. The experimental plan uses cross-sectional and longitudinal measures of behaviour, cognition, neuroimaging and both CSF and serum markers to elucidate the patterns of biomarker change in genetic FTD with the overarching hypothesis being that biomarkers can aid in the identification of disease and tracking of disease progression. The study aims to be the foundation for clinical trials in genetic FTD with the key outcomes being identification of markers of disease onset including those including those indicative of optimal time to start disease-modifying therapy and markers of disease progression that can be used as outcome measures in clinical trials, as well as estimations of the sample sizes necessary for those trials.