Risk and Modifying factors in Fronto Temporal Dementia
Award Number
MR/L501542/1Award Type
FellowshipProgramme
Research GrantStatus / Stage
ActiveDates
3 June 2014 -2 June 2017
Duration (calculated)
02 years 11 monthsFunder(s)
MRC (UKRI)Funding Amount
£461,582.83Funder/Grant study page
MRC UKRIContracted Centre
University College LondonPrincipal Investigator
Professor Hardy, JohnPI Contact
j.hardy@ucl.ac.ukPI ORCID
0000-0002-3122-0423WHO Catergories
Improving clinical trialsDisease Type
CPEC Review Info
| Reference ID | 271 |
|---|---|
| Researcher | Reside Team |
| Published | 12/06/2023 |
Data
| Award Number | MR/L501542/1 |
|---|---|
| Status / Stage | Active |
| Start Date | 20140603 |
| End Date | 20170602 |
| Duration (calculated) | 02 years 11 months |
| Funder/Grant study page | MRC UKRI |
| Contracted Centre | University College London |
| Funding Amount | £461,582.83 |
Abstract
Frontotemporal dementia (FTD) is a common cause of young onset dementia. The only known risk factors for FTD at present are genetic with three genes accounting for the majority of familial FTD, called progranulin, tau and chromosome 9 open reading frame 72. There are now promising avenues for treatment of these disorders but we still do not know when drugs should be started or how we should measure the response to treatment. This study plans to investigate people who have genetic FTD, including both people who have developed symptoms and also people who have a risk of developing symptoms in the future because they carry the abnormal genetic mutation. This allows a window into the earliest changes in the disease process. 30 study subjects from families with genetic FTD will have psychology testing, brain imaging, blood tests and spinal fluid collection annually at three time-points in order to investigate the patterns of change in these different tests at different stages of the disorder. Brain imaging will include not only magnetic resonance imaging but also the novel technique of tau positron emission tomography which may be able to identify the presence of tau pathology in the brains of people with tau mutations for the first time during life. The study subjects will be part of a larger multicentre study called the Genetic Frontotemporal dementia Initiative (GENFI) and data will be available for analysis from over 300 subjects who take part in that initiative. The key outcomes of the study will be to develop markers which help identify the disease at its earliest stage as well as markers that allow the progression of the disease to be tracked. These markers can then be used in future clinical trials of drugs in genetic FTD.
Aims
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.