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
Peter Ljubenkov, MD
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.
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…
Eligibility
- Age range
- 18–85 years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Between 18 and 85 years of age (inclusive) at the initial screening visit; 2. Meets 2011 consensus criteria for svPPA (Gorno-Tempini et al. 2011); 3. MRI at screening is consistent with the underlying svPPA with no large strokes or severe white matter disease (Fazekas Grade ≤2; Fazekas et al. 1987); 4. CDR® plus NACC FTLD (Miyagawa et al. 2020) global score at screening ≤1; 5. The following medications are allowed, but must be stable for 2 months prior to the initial screening visit: 1. Food and Drug Administration (FDA)-approved Alzheimer's disease (AD) medications…
Interventions
- DrugVerdiperstat
Oral, extended release (ER) tablet
Locations (5)
- UCSF Memory and Aging CenterSan Francisco, California
- Northwestern UniversityChicago, Illinois
- Mayo ClinicRochester, Minnesota
- University of PennsylvaniaPhiladelphia, Pennsylvania
- Houston Methodist Hospital - Nantz National Alzheimer CenterHouston, Texas