VES001 (SORT-IN-2)
Research type
Research Study
Full title
Open-label multiple dosing study in asymptomatic GRN-frontotemporal dementia patients to investigate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VES001
IRAS ID
1010574
Contact name
Ditte Mikkelsen
Contact email
Sponsor organisation
Vesper Bio Asp
Clinicaltrials.gov Identifier
Research summary
This study, sponsored by Vesper Bio AsP, will test a new medicine called VES001 for the treatment of frontotemporal dementia (FTD). One of the causes of FTD is related to a gene called Granulin which makes a protein called progranulin (PGRN) that is important for the growth, survival, protection and functioning of brain cells. When there is a problem in the Granulin gene, PGRN levels decrease and brain function declines, resulting in FTD. There is no approved treatment to cure FTD, just help to manage symptoms and improve quality of life. VES001 is designed to increase PGRN levels in the brain by targeting a receptor called sortilin and preventing the interaction between sortilin and PGRN. Researchers believe that VES001 may help to raise PGRN levels and slow down progression of FTD.
The main purpose of the study is to explore how VES001 interacts with the participant and produces its effects. The amount of PGRN in blood and cerebrospinal fluid (CSF) is measured. The second purpose is to find a safe and tolerable dose of VES001 for people with FTD by observing side effects.
A total of 6 people who carry the GRN-FTD gene but have no symptoms, will take part in the study at one site in Netherlands and one site in the United Kingdom (University College London NHS Foundation Trust). Participants will self administer two different doses of the study medicine, the first dose once one days 1-34 and the second dose twice on days 35 to 84. There is no washout between doses.
The participants will be in the study for about 156 days with 8 site visits and 4 telephone visits. Participants will need to provide blood and urine samples at most site visits and keep a diary to record when they take the study medicine every day. Participants will undergo a lumbar puncture 3 times, general health tests, physical examinations, electrocardiograms and possibly an MRI scan. Biomarkers in blood and CSF samples will be checked to see if the study medicine is effective in treating FTD.REC name
London - Brent Research Ethics Committee
REC reference
24/LO/0708
Date of REC Opinion
25 Oct 2024
REC opinion
Further Information Favourable Opinion