NCT05184569

Veri-T: A Trial of Verdiperstat in Patients With svPPA Due to TDP-43 Pathology

Phase 1, Randomized, Double-Blind, Placebo-Controlled, Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy Study of Oral Verdiperstat (BHV-3241) in Patients With Semantic Variant Primary Progressive Aphasia (svPPA) Due to TDP-43 Pathology

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 64 (estimated)
• Sponsor: Peter Ljubenkov, MD · Academic / Other
• Sex: All
• Age: 18 Years – 85 Years
• Healthy volunteers: Not accepted

Summary

The purpose of the study is to test the safety and tolerability of twice daily Verdiperstat in patients with semantic variant primary progressive aphasia (svPPA) due to frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP). Three-fourths of the participants will receive Verdiperstat and one-fourth will receive Placebo during the 24-week treatment duration.

Detailed description

This is a Phase 1, randomized, double-blind, placebo-controlled study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of Verdiperstat in patients with semantic variant primary progressive aphasia (svPPA) due to frontotemporal lobar degeneration with TDP-43 pathology (FTLD-TDP). Approximately 64 subjects will be randomized 3:1 to active drug or placebo. Study drug will be administered orally bid (two Verdiperstat tablets bid or two placebo tablets bid (for a total daily dose of 600mg daily, following a one-week titration period of 1 tablet daily). The study will test the effects of Verdiperstat on cerebrospinal fluid (CSF) proteins, brain magnetic resonance imaging (MRI), and cognitive (thinking, memory and language) tests in subjects with svPPA due to FTLD-TDP. This study uses placebo which looks like the experimental drug but does not have any active drug in it.

Conditions

• Semantic Dementia

Interventions

Type	Name	Description
DRUG	Verdiperstat	Oral, extended release (ER) tablet

Timeline

• Start date: 2022-04-14
• Primary completion: 2026-06-30
• Completion: 2026-09-30
• First posted: 2022-01-11
• Last updated: 2025-10-15

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study