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SORT-IN-1
CompletedPhase 1A 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
Lead sponsor
Asset
VES001
Listed sites
1
Recruiting sites
-
Enrollment
78
actual
Study population
Frontotemporal dementia
Key I/E criterion
•Age 18-55
Primary endpoints
•Incidence, severity, and seriousness of treatment-emergent adverse events•Clinically significant abnormalities in safety laboratory values•Vital sign measurement
Footprint
Where this trial recruits
Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.
Identifiers
Registered as
Timeline
Milestones
Assets
Drug assets
Study populations
Who this study enrolls
Eligibility
Who can enroll
Inclusion criteria
Part A \& B:
1. Healthy men or women aged 18 to 55 years.
2. Body Mass Index between 18 and 32 kg/m2, with a minimum weight of 50 kg.
3. Effective contraception required during the study and for at least 90 days after their last dose.
4. Participants in group 3, where the food effect is being investigated, must be able to eat a high-fat meal within 30 minutes for breakfast
Exclusion criteria
Part A \& B:
1. Medical conditions or treatments that could interfere with the study.
2. History of any known neurologic disease, cognitive impairment, or a history of seizure, (significant) head trauma, or loss of consciousness.
3. History of active malignancy (active cancer cells or tumors) within the last 5 years.
4. Abnormal laboratory test results or infectious diseases (Hepatitis B, Hepatitis C, and/or HIV).
5. Recent medication or supplement use, unless allowed by the investigator.
6. Participation in other research studies involving study treatment or devices.
7. Positive tests for illegal drugs or alcohol at screening.
8. Heavy smoking or inability to abstain from smoking during the study.
9. Excessive consumption of caffeine (more than 8 cups per day).
10. History of severe allergic reactions to medication
11. Recent blood donation or significant blood loss.
12. Pregnancy, breastfeeding, or plans to become pregnant (for women).
Endpoints (27)
What's being measured
Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.
Coverage by outcome category
Fluid / digital biomarkers
4 endpointsConcentration of VES001 in CSF in the highest two dose level cohorts in Part A.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Concentration of VES001 in CSF in all dose level cohorts in Part B.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Concentration of VES001 in plasma/CSF ratio in the highest two dose level cohorts in Part A.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Concentration of VES001 in plasma/CSF ratio in all dose level cohorts in Part B.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Safety / tolerability / PK
14 endpointsIncidence, severity, and seriousness of treatment-emergent adverse events (TEAEs).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
event count, event
Incidence of clinically significant abnormalities in safety laboratory values.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
event count, event
Change from baseline in vital sign measurement: Pulse Rate (bpm).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Systolic blood pressure (mmHg) and Diastolic blood pressure (mmHg).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter Heart Rate (HR).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter beats per minute (bpm)
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter PR Interval
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter QRS Interval
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter QT Interval.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter QTcB (calculated using Bazzet method).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Change from baseline in vital sign measurement: Electrocardiogram parameter QTcF, (calculated using Fredericia's method).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, event
Plasma PK parameter: Maximum concentration (Cmax).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Plasma PK parameter: Time to reach maximum concentration (tmax).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
time to event, event
Plasma PK parameter: Terminal elimination half-life (t1/2).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Other (unclassified)
9 endpointsIncidence of clinically significant abnormalities in physical/neurological examination findings.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
event count, event
Change from baseline in Columbia-Suicide Severity Rating Scale (C-SSRS; Parts B).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
change from baseline, improvement
Plasma PK parameter: Area under the concentration-time curve from time zero to infinity (AUCinf).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Plasma PK parameter: Area under the concentration-time curve from time zero to infinity AUCinf(%extrapolated).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Plasma PK parameter: Area under the concentration-time from time zero to time of last measurable concentration (AUClast).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
concentration, descriptive
Plasma PK parameter: Apparent total clearance following extravascular administration (CL/F).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
ratio, descriptive
Plasma PK parameter: Absorption lag time (tlag).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
descriptive
Plasma PK parameter: Apparent volume of distribution during the terminal elimination phase after extravascular administration (Vz/F).
Time frame:Part A: 21 weeks. Part B: 13 weeks.
ratio, descriptive
Comparison of the plasma PK of VES001 following a single oral dose in the fed and fasted state in Part A.
Time frame:Part A: 21 weeks. Part B: 13 weeks.
descriptive
Provenance
Sources
Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.