CompletedPhase 1

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

Netherlands, United Kingdom6 participantsStarted 2024-12-12

Plain-language 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.

Who can participate

Age range18 Years – 75 Years

SexALL

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Exclusion criteria

✕. QTcF \> 450 or \< 300 msec for males and QTcF \> 470 or \< 300 msec for females
✕. Notable resting bradycardia (HR \< 45 bpm) or tachycardia (HR \> 100 bpm)
✕. Personal or family history of congenital long QT syndrome or sudden death attributed to underlying heart disease
✕. ECG with QRS and/or T-wave judged to be unfavorable for a consistently accurate QT measurement (e.g., neuromuscular artefact that cannot be readily eliminated, arrhythmias, indistinct QRS onset, low amplitude T-wave, merged T- and U-waves, prominent U-waves)
✕. a local infection at the site of the LP
✕. \< 50 × 10E3/μL platelet count at screening
✕. clinically significant coagulation abnormality
✕. significant active bleeding, treatment with an anticoagulant, or treatment with 2 or more antiplatelet agents

What they're measuring

Change in PGRN levels in CSF at Day 28 and Day 84 compared to baseline

Timeframe: 16 weeks

Change in PGRN levels in plasma at Day 28 and Day 84 compared to baseline

Timeframe: 16 weeks

Trial details

NCT IDNCT06705192

SponsorMads Kjolby

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-08-05

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