NCT06705192

Study in Asymptomatic GRN-FTD Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001

Open-label Multiple Dosing Study in Asymptomatic GRN-frontotemporal Dementia Patients to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VES001.

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 6 (actual)
• Sponsor: Mads Kjolby · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

This is an open-label, multiple dosing study in asymptomatic GRN-FTD carriers to investigate the safety, tolerability, PK, and PD of VES001. The study follows a MAD design within a single cohort, investigating 2 distinct dose levels (Dose 1: 360 mg and Dose 2: 900 mg), consecutively over a 3-month period. A total of 6 participants will be recruited over a fixed enrolment period of 6 months.

Conditions

• Dementia
• Frontotemporal Dementia
• Asymptomatic Condition

Interventions

Type	Name	Description
DRUG	VES001	VES001 is an oral, blood brain barrier ligand of sortilin. VES001 will be administered orally as a solid within a gelatine capsule without excipients; the capsule strength will be 180 mg.

Timeline

• Start date: 2024-12-12
• Primary completion: 2025-08-05
• Completion: 2025-08-05
• First posted: 2024-11-26
• Last updated: 2025-08-24

Locations

2 sites across 2 countries: Netherlands, United Kingdom