A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants (SORT-IN-1) [sponsored by Vesper Biotechnologies ApS]

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, participants are randomized to receive either the experimental treatment or a placebo (something that looks like the experimental treatment but has no experimental medicine in it). The study is ‘double blind’ which means that participants will not know if they received VES001 or a placebo and the study doctor will not know either. Only the researchers analyzing the data will have that information. There are two parts to this phase 1 study. Part 1A will include healthy volunteers only. Part 1B will include healthy volunteers who know they have a GRN variant (asymptomatic GRN carriers).

Who is eligible?

• Men and women aged 18 to 55 years.
• Body mass index between 18 and 32 (kg/m²)
• Using birth control during the study and for at least 90 days after the last dose
• Does not smoke and agrees not to smoke during the study period
• Do 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
• 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

For part 1B only—participants need to be healthy with a known pathogenic progranulin gene (GRN) variant 

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?

This study will randomly assign participants to receive the experimental treatment or the placebo group where participants will receive a placebo (something that looks like the experimental treatment but has no medicine in it). Both the experimental treatment and placebo are oral medications, taken by mouth. Among healthy participants who do not have a GRN variant (group 1A) each participants will receive a single dose, though doses will vary between participants. Among healthy participants who carry a GRN variant (group 1B), participants will receive multiple doses over 7 days (experimental treatment or placebo) where the dose in the experimental treatment increases over the 7 days.

​​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 in group 1A (healthy no GRN variant) will be monitored for 21 weeks; participants in group 1B (healthy with a known disease causing GRN variant) will be monitored for 13 weeks.