NCT06226064

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants

A Randomised, Double-blind, Placebo-controlled Single and Multiple Ascending Dose Study in Healthy Volunteers and Asymptomatic GRN Mutation Carriers to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 78 (actual)
• Sponsor: Vesper Biotechnologies ApS · Industry
• Sex: All
• Age: 18 Years – 55 Years
• Healthy volunteers: Accepted

Summary

This is a Phase 1, randomized, placebo-controlled, double-blind study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of single and multiple doses of VES001 in a two part followed by a multicenter, open-label Phase 1b study in asymptomatic GRN mutation carriers. Part A will evaluate the safety, tolerability, PK, and PD of single doses of VES001 in healthy volunteers. Part B will evaluate the safety, tolerability, PK, and PD of multiple doses of VES001 in healthy volunteers.

Detailed description

Part A will include six cohorts, with eight participants per cohort. Participants in each cohort will be randomised in a 6:2 ratio (VES001 vs. placebo). Part B will include three cohorts, with ten participants per cohort. Participants in each cohort will be randomised in a 8:2 ratio (VES001 vs. placebo).

Conditions

• Dementia, Frontotemporal

Interventions

Type	Name	Description
DRUG	VES001	VES001 is an oral, blood brain barrier penetrating ligand of sortilin.
DRUG	Placebo	A matching dosage form, indistinguishable from the active treatment will be used as the placebo treatment.

Timeline

• Start date: 2023-10-11
• Primary completion: 2024-07-26
• Completion: 2024-07-26
• First posted: 2024-01-26
• Last updated: 2024-08-22

Locations

1 site across 1 country: Netherlands