Summary:
VES001 is an experimental treatment that was designed to restore normal levels of the progranulin protein (PGRN) in the brain. The study will collect data to evaluate how safe VES001 is, as well as how well the body handles it (pharmacokinetics), what it does in the body (pharmacodynamics), and whether people can handle taking it without too many side effects (tolerability). In this phase 1 study, all participants will receive the experimental treatment.
Who is eligible?
- Men and women aged 18 to 75 years who are asymptomatic carriers of a GRN variant
- Body mass index between 18 and 32 (kg/m2), with minimum weight of 50 kg
- Using birth control during the study and for at least 90 days after the last dose
- Does not smoke more than 10 cigarettes per day and agrees not to smoke during the study period
- Does not have any known neurological disease, head trauma or loss of consciousness
- Does not have a history of cancer within the last 5 years, abnormal lab tests for infection diseases (Hepatitis B, Hepatitis C, and/or HIV), or any medical condition that could interfere with the study
- Does not have an abnormal ECG, abnormal heart rate or heart rhythm (e.g. atrial fibrillation), personal or family history of long QT syndrome or sudden death, untreated high or low blood pressure
- Is not currently pregnant, breastfeeding or planning to become pregnant
- Does not have severe allergic reactions to medication
- Has not donated blood or had significant blood loss recently
- Does not consume excessive caffeine (more than 8 cups per day)
- Has not participated in other research studies involving study treatment or devices
Interested individuals meeting the above requirements should contact the study coordinator for additional requirements and exclusions if interested in participating.
What will happen during the study?
Healthy participants who carry a GRN variant will receive 2 doses of the experimental treatment over the 16 week participation period. During the study period, there will be 8 visits. Blood, urine will be collected throughout the study period. In addition, spinal fluid will be collected through a spinal tap at three visits. Additional evaluations including ECG and neurological exams will be conducted as well.
What are they measuring to evaluate the experimental treatment?
- Side effects and reactions to the experimental treatment
- Changes in vital signs (pulse, blood pressure)
- Changes in electrocardiogram (ECG)
- Changes in physical and neurological examination findings
- Changes in Suicide Severity Rating Scale
- Changes in the concentration of the experimental treatment and other biomarkers in blood samples and spinal fluid
How long will participants be involved in the study?
Study participants will be monitored for 16 weeks.
