FTD Trials
Structure of the progranulin protein
Genetic FTD: an intervention study in people with FTD due to a known GRANULIN mutation
This exciting new study is being led in Australia by Prof Amy Brodtmann and the Eastern Clinical Research Unit for Alector. There are sites in Sydney (A/Prof Rebekah Ahmed) and Perth (Dr Carolyn Orr). People in this study MUST have a specific genetic mutation in the granulin (also known as progranulin, or GRN) gene. Genetic testing will be available to people with FTD who may be interested in study participation. Trial participation is only available to people with this mutation.
The study will use a compound called AL001, a human monoclonal antibody designed to modulate progranulin. Progranulin is a regulator of immune activity in the brain, with genetic links to multiple neuro-degenerative disorders, including FTD, Alzheimer’s disease, and Parkinson’s disease. Recruitment in Melbourne was expected to start in early August but have been put on hold temporarily due to COVID-19. Contact Claire Mccarthy to register interest, but please note that this will not be available for most people with FTD – only for those with the gene.