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

Who is this study for? Patients with semantic variant primary progressive aphasia due to TDP-43 pathology
What treatments are being studied? Verdiperstat
Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 85
Healthy Volunteers: f
View:

• Between 18 and 85 years of age (inclusive) at the initial screening visit;

• Meets 2011 consensus criteria for svPPA (Gorno-Tempini et al. 2011);

• 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);

• CDR® plus NACC FTLD (Miyagawa et al. 2020) global score at screening ≤1;

• The following medications are allowed, but must be stable for 2 months prior to the initial screening visit:

∙ Food and Drug Administration (FDA)-approved Alzheimer's disease (AD) medications;

‣ FDA-approved psychotropic medications;

• Other medications (except those listed under exclusion criteria) are allowed as long as the dose is stable for 30 days prior to the initial screening visit;

• Has a reliable study partner who agrees to accompany the participant to visits, and spends at least 5 hours per week with the participant;

• Agrees to 2 LPs;

• Signed and dated written informed consent obtained from the participant and the participant's study partner in accordance with local Institutional Review Board (IRB) regulations;

⁃ WOCBP must agree to abstain from sex or use highly effective birth control that includes two methods of contraception (one of which must be a barrier method) for the duration of the screening period, the RDBPC treatment period, and for 30 days after the last dose of study drug (active or placebo);

⁃ Males must agree to abstain from sex with WOCBP or use an adequate method of contraception for the duration of the RDBPC treatment period and for 90 days after the last dose of study drug (active or placebo);

⁃ Able to swallow pills whole without crushing or chewing.

Locations
United States
California
UCSF Memory and Aging Center
RECRUITING
San Francisco
Illinois
Northwestern University
RECRUITING
Chicago
Minnesota
Mayo Clinic
RECRUITING
Rochester
Pennsylvania
University of Pennsylvania
RECRUITING
Philadelphia
Texas
Houston Methodist Hospital - Nantz National Alzheimer Center
RECRUITING
Houston
Contact Information
Primary
Karin Snowberg, MA
Karin.Snowberg@ucsf.edu
415-476-8845
Backup
Whitney Walker, MS, CNS, RN
Whitney.Walker@ucsf.edu
415-353-7532
Time Frame
Start Date: 2022-04-14
Estimated Completion Date: 2026-09-30
Participants
Target number of participants: 64
Treatments
Experimental: Verdiperstat
Verdiperstat 2 tablets twice daily (600mg total daily) by mouth for 24 weeks.
Placebo_comparator: Placebo
Placebo 2 tablets twice daily by mouth for 24 weeks.
Sponsors
Collaborators: Alzheimer's Association, National Institute on Aging (NIA), National Institutes of Health (NIH)
Leads: Peter Ljubenkov, MD

This content was sourced from clinicaltrials.gov