Veri-T is a phase 1, randomized, double-blind, placebo-controlled study of the safety, tolerability, preliminary efficacy and pharmcokinetics and pharmacodynamics of oral verdiperstat in patients with Semantic Variant Primary Progressive Aphasia (svPPA) Due to Frontotemporal Lobar Degeneration With TDP-43 Pathology (FTLD-TDP).
This study is an NIH R01 investigator-sponsor study, the PI and holder of IND 157, 370 is Peter Ljubenkov, MD, at UCSF. The UCSF IRB will serve as the IRB of Record.
This is a multicenter study with all sites located in the US. About 64 patients will participate. The study includes about 12 visits over about 8 months. The screening period is held over 6 weeks followed by 24 weeks of study medication. There is one follow-up visit 4 weeks after completing study medication. Study tests and procedures include physical and neruological exams, neurognitive tests, questionnaires, blood and urine laboratory tests including pregnancy tests for females of child-bearing potential, ECGs, MRIs and two lumbar punctures.
Participants will be randomized 3:1 to 300 mg extended-release verdiperstant or placebo. Participants will take one tablet daily for the first week, two tablets daily for the second week and two tablets twice daily during weeks 3-24.