Verdiperstat for semantic variant primary progressive aphasia (svPPA) linked to TDP‑43
Veri-T: A phase 1 Placebo-Controlled Trial of Verdiperstat in Semantic Variant Primary Progressive Aphasia Due to Underlying FTLD-TDP
Adults with semantic variant primary progressive aphasia (svPPA) will receive Verdiperstat or placebo to find safe doses and look for early signs it affects disease markers.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11179299 on NIH RePORTER |
What this research studies
You would join a multi-site, double-blind, randomized trial where neither you nor the team knows who gets Verdiperstat or placebo. The study enrolls sequential cohorts to test two oral dose levels and watches for safety, tolerability, and early biological effects. Visits include clinical exams and biomarker tests guided by the ALLFTD research network to track any changes. The goal is to set up larger trials if the drug appears safe and shows target engagement.
Who could benefit from this research
Good fit: Adults diagnosed with semantic variant primary progressive aphasia (svPPA) consistent with underlying FTLD‑TDP who meet the study's medical and safety criteria are ideal candidates.
Not a fit: People whose language problems are due to other conditions, who are medically ineligible, or who cannot tolerate the study drug may not receive benefit from participating.
Why it matters
Potential benefit: If successful, Verdiperstat could reduce harmful inflammation linked to TDP‑43 and pave the way for treatments that slow svPPA and related TDP‑43 dementias.
How similar studies have performed: Preclinical studies support targeting myeloperoxidase (MPO) in TDP‑43 disease, but human clinical proof of benefit is limited and this is an early-phase clinical test.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Ljubenkov, Peter Alexander — University of California, San Francisco
- Study coordinator: Ljubenkov, Peter Alexander
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.