Actively Recruiting

Phase 1
Age: 18Years - 85Years
All Genders
NCT05184569

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

Led by Peter Ljubenkov, MD · Updated on 2025-10-15

64

Participants Needed

5

Research Sites

232 weeks

Total Duration

On this page

Sponsors

P

Peter Ljubenkov, MD

Lead Sponsor

N

National Institutes of Health (NIH)

Collaborating Sponsor

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 18Years - 85Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Between 18 and 85 years of age (inclusive) at screening
  • Meets 2011 consensus criteria for semantic variant primary progressive aphasia (svPPA)
  • Screening MRI consistent with svPPA without large strokes or severe white matter disease (Fazekas Grade 2 or less)
  • Clinical Dementia Rating plus NACC FTLD global score of 1 or less at screening
  • Stable dose for 2 months prior to screening of FDA-approved Alzheimer's or psychotropic medications
  • Stable dose for 30 days prior to screening of other allowed medications
  • Has a reliable study partner who spends at least 5 hours per week and agrees to accompany to visits
  • Agrees to two lumbar punctures
  • Signed informed consent from participant and study partner
  • Women of childbearing potential must agree to abstain from sex or use two methods of contraception including a barrier method during screening, treatment, and 30 days after last dose
  • Males must agree to abstain from sex with women of childbearing potential or use adequate contraception during treatment and 90 days after last dose
  • Able to swallow pills whole without crushing or chewing
Not Eligible

You will not qualify if you...

  • Clinical diagnosis of probable Alzheimer's disease or biomarker evidence of Alzheimer's disease
  • Diagnosis of other frontotemporal lobar degeneration syndromes except svPPA, including logopenic or non-fluent primary progressive aphasia and most behavioral variant frontotemporal dementia cases
  • Other medical conditions likely to cause cognitive or behavioral deficits (e.g., uncontrolled seizure, stroke, vascular dementia, substance abuse, Lewy body disease)
  • Uncontrolled thyroid disease or abnormal thyroid lab values at screening
  • Serious autoimmune disease or immunocompromised state
  • Significant cardiovascular, hematologic, renal, or liver disease or abnormal labs at screening
  • Gastrointestinal diseases or surgery affecting drug absorption or excretion
  • Recent myocardial infarction, heart failure hospitalization, unstable angina, or syncope within 1 year
  • Major psychiatric illness or untreated depression posing safety risk
  • Low neutrophil or platelet counts or abnormal kidney or liver function labs at screening
  • Clinically significant screening or baseline findings posing safety risk
  • Abnormal ECG findings including prolonged QTc or arrhythmias
  • Renal impairment with low estimated glomerular filtration rate or high serum creatinine
  • Hemoglobin A1C greater than 7.5% at screening
  • Recent significant infections within 4 weeks prior to screening
  • Known HIV infection
  • Major surgery or blood transfusion within 4 weeks prior to screening
  • History of stem cell treatment
  • Contraindications to MRI or lumbar puncture or inability to tolerate these procedures
  • Unlikely to comply with dosing or evaluations
  • Prior treatment with Verdiperstat
  • Use of investigational drugs, systemic corticosteroids, or strong CYP1A2 inhibitors within 30 days or 5 half-lives before screening
  • Known allergy to study drug ingredients
  • Pregnant or lactating women or positive pregnancy test
  • Cancer within 5 years except basal cell carcinoma
  • Known genetic mutations associated with FTLD without TDP-43 inclusions

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 5 locations

1

UCSF Memory and Aging Center

San Francisco, California, United States, 94158

Actively Recruiting

2

Northwestern University

Chicago, Illinois, United States, 60611

Actively Recruiting

3

Mayo Clinic

Rochester, Minnesota, United States, 55905

Actively Recruiting

4

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

Actively Recruiting

5

Houston Methodist Hospital - Nantz National Alzheimer Center

Houston, Texas, United States, 77030

Actively Recruiting

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Research Team

K

Karin Snowberg, MA

CONTACT

W

Whitney Walker, MS, CNS, RN

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

QUADRUPLE

Allocation

RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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