The UK GENetic Frontotemporal dementia Initiative
Study Code / Acronym
UK GENFIAward Number
MR/M023664/1Programme
Research GrantStatus / Stage
ActiveDates
1 May 2015 -30 April 2020
Duration (calculated)
04 years 11 monthsFunder(s)
MRC (UKRI)Funding Amount
£2,613,582.38Funder/Grant study page
MRC UKRIContracted Centre
University College LondonContracted Centre Webpage
Principal Investigator
Professor Rossor, Martin NeilWHO Catergories
Development of BiomarkersImproving clinical trials
Understanding Underlying Disease
Disease Type
Frontotemporal Dementia (FTD)CPEC Review Info
| Reference ID | 267 |
|---|---|
| Researcher | Reside Team |
| Published | 12/06/2023 |
Data
| Study Code / Acronym | UK GENFI |
|---|---|
| Award Number | MR/M023664/1 |
| Status / Stage | Active |
| Start Date | 20150501 |
| End Date | 20200430 |
| Duration (calculated) | 04 years 11 months |
| Funder/Grant study page | MRC UKRI |
| Contracted Centre | University College London |
| Contracted Centre Webpage | |
| Funding Amount | £2,613,582.38 |
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 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. There are currently no treatments that can delay the onset or prevent the progression of genetic FTD but there are now potential disease-modifying therapies in development. However, there are still no biomarkers of genetic FTD that can confidently predict when disease-modifying therapy should be initiated or how the response to it should be monitored. By studying participants who carry a mutation and are thus destined to develop the disease we can explore the entire disease pathway, using mutation negative first-degree relatives as controls. A pilot study has created a common methodological platform to study genetic FTD. This follow-on study aims to capitalize on the work of the pilot, extending both the platform and expertise generated to a UK-wide study of genetic FTD. Specifically, we will establish a UK-wide familial FTD cohort of 200 participants which will (1) lead to greater understanding of the biological underpinning of the cognitive and behavioural deficits seen in FTD; (2) permit large-scale cohesive and integrated longitudinal biomarker studies of familial FTD with the specific remit of informing clinical trial design; and (3) provide a “readiness” cohort of patients for disease-modifying drug trials. The experimental plan includes longitudinal clinical and neuropsychological assessments, neuroimaging and both blood and spinal fluid sampling, with the key outcomes including improved understanding regarding the selective vulnerability of the specific neural systems affected in FTD, robust biomarkers of disease onset and progression, and estimation of sample sizes required for trials.
Aims
This follow-on study aims to capitalize on the work of the pilot, extending both the platform and expertise generated to a UK-wide study of genetic FTD. Specifically, we will establish a UK-wide familial FTD cohort of 200 participants which will (1) lead to greater understanding of the biological underpinning of the cognitive and behavioural deficits seen in FTD; (2) permit large-scale cohesive and integrated longitudinal biomarker studies of familial FTD with the specific remit of informing clinical trial design; and (3) provide a “readiness” cohort of patients for disease-modifying drug trials. The experimental plan includes longitudinal clinical and neuropsychological assessments, neuroimaging and both blood and spinal fluid sampling, with the key outcomes including improved understanding regarding the selective vulnerability of the specific neural systems affected in FTD, robust biomarkers of disease onset and progression, and estimation of sample sizes required for trials.