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

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.

Timeline

Start

2024-12-12

Primary completion

2025-08-05

Completion

2025-08-05

Drugs

Indications