A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001 in Healthy Participants
- Registration Number
- NCT06226064
- Lead Sponsor
- Vesper Biotechnologies ApS
- Brief 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).
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 78
Inclusion Criteria
Not provided
Exclusion Criteria
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description VES001 (Healthy Participants) VES001 Part A: Ascending single doses and Part B: Multiple ascending dose (seven days of treatment), for healthy volunteers. Placebo (Healthy Participants) Placebo Part A: Ascending single doses and Part B: Multiple ascending dose (seven days of treatment), for healthy volunteers.
- Primary Outcome Measures
Name Time Method Change from baseline in vital sign measurement: Systolic blood pressure (mmHg) and Diastolic blood pressure (mmHg). Part A: 21 weeks. Part B: 13 weeks. Incidence of clinically significant abnormalities in safety laboratory values. Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Pulse Rate (bpm). Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter Heart Rate (HR). Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter beats per minute (bpm) Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter PR Interval Part A: 21 weeks. Part B: 13 weeks. Incidence, severity, and seriousness of treatment-emergent adverse events (TEAEs). Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter QRS Interval Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter QT Interval. Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter QTcB (calculated using Bazzet method). Part A: 21 weeks. Part B: 13 weeks. Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS; Parts B). Part A: 21 weeks. Part B: 13 weeks. Change from baseline in vital sign measurement: Electrocardiogram parameter QTcF, (calculated using Fredericia's method). Part A: 21 weeks. Part B: 13 weeks. Incidence of clinically significant abnormalities in physical/neurological examination findings. Part A: 21 weeks. Part B: 13 weeks.
- Secondary Outcome Measures
Name Time Method Plasma PK parameter: Area under the concentration-time curve from time zero to infinity AUCinf(%extrapolated). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Area under the concentration-time from time zero to time of last measurable concentration (AUClast). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Area under the concentration-time curve from time zero to infinity (AUCinf). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Apparent total clearance following extravascular administration (CL/F). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Maximum concentration (Cmax). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Absorption lag time (tlag). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Time to reach maximum concentration (tmax). Part A: 21 weeks. Part B: 13 weeks. Plasma PK parameter: Terminal elimination half-life (t1/2). Part A: 21 weeks. Part B: 13 weeks. Concentration of VES001 in plasma/CSF ratio in the highest two dose level cohorts in Part A. Part A: 21 weeks. Part B: 13 weeks. Concentration of VES001 in plasma/CSF ratio in all dose level cohorts in Part B. Part A: 21 weeks. Part B: 13 weeks. Comparison of the plasma PK of VES001 following a single oral dose in the fed and fasted state in Part A. Part A: 21 weeks. Part B: 13 weeks. Refer to the PK parameters listed above.
Plasma PK parameter: Apparent volume of distribution during the terminal elimination phase after extravascular administration (Vz/F). Part A: 21 weeks. Part B: 13 weeks. Concentration of VES001 in CSF in the highest two dose level cohorts in Part A. Part A: 21 weeks. Part B: 13 weeks. Concentration of VES001 in CSF in all dose level cohorts in Part B. Part A: 21 weeks. Part B: 13 weeks.
Trial Locations
- Locations (1)
Center for Human Drug Research
🇳🇱Leiden, Netherlands