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.Summary of Results
We thank all of the participants who took part in this clinical study. Without participant’s support, advances in treatments for medical conditions would not be possible. This document provides summary of results of a clinical study for general audience. It is important to know what was learned from the results of a clinical study.
This summary only indicates the results of a single clinical study. Other clinical studies of the same indication or same study medicine(s) may show different results. Please do not take any health or treatment related decisions based on the results of this clinical study. If you have any questions about this study results, please talk to your doctor. For more information, please visit the links provided on last page of this summary.Thank you!
1. Why was this study needed?
Frontotemporal dementia (FTD) is a serious brain disease that often starts earlier than Alzheimer’s disease, commonly in people in their 50s or 60s. It can affect behaviour causing impulsive or inappropriate actions, repetitive or compulsive behaviours, and problems in planning, organising, or making decisions. Some people also develop language problems, such as speaking less, repeating words, or eventually being unable to speak. FTD may develop because of a change (mutation) in a gene called GRN. The GRN gene helps the body make a protein called progranulin (PGRN). PGRN helps protect brain cells, reduces inflammation and supports cells clear waste. People who have a change in the GRN gene usually make much less PGRN than normal. This can increase inflammation, buildup of harmful proteins, and damage brain cells contributing to the development of FTD.
Currently, there are no treatments that can slow down or prevent FTD. Available medicines only help manage symptoms and do not treat the main cause of the disease. This creates a need for new treatments that can increase PGRN levels and may help protect the brain. VES001 is a new drug being developed for treating people with FTD. It works by blocking a protein called sortilin, which helps increase levels of PGRN, in the brain. It is taken by mouth and can reach the brain. By raising PGRN levels, VES001 may help reduce inflammation, protect brain cells, and slow brain cell loss in people with FTD.
In this study, researchers wanted to find out if VES001 is safe and if it can increase PGRN levels in FTD patients who carry a GRN mutation but do not yet have symptoms.
Throughout this summary, the term “study medicine” will be used to refer to VES001.2. When and where was this study done?
This study started in December 2024 and ended in August 2025. Each participant was part of this study for up to 156 days. The study took place in 1 site in Netherlands and 1 site in United Kingdom.
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