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

Running, not enrolling · Phase 1 · Has a placebo group

Conditions studied: Semantic Dementia

In brief

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.

Key facts

Study ID: NCT05184569
Run by: Peter Ljubenkov, MD
People needed: 64
Starts: 2022-04-14
Expected to finish: 2027-01-31
Last updated by the study team: 2026-07-06

Who can join

Age: 18 and older, up to 85. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

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.

You may not qualify if…

A clinical diagnosis of probable AD (McKhann et al. 2011) or previous biomarker evidence of AD biology using amyloid positron emission tomography (PET) imaging, CSF amyloid beta (Aβ)/total tau (t-tau) ratio, CSF/plasma amyloid beta isoform with 40 amino acid residues (Aβ40)/amyloid beta isoform with 42 amino acid residues (Aβ42) ratio, or plasma phosphorylated tau [phosphorylated tau at residue 181 (p-tau181) and phosphorylated tau at residue 217 (p-tau217)] assessments;
A clinical diagnosis of a comorbid FTLD-associated clinical syndrome other than svPPA, including:
logopenic primary progressive aphasia (lvPPA; Gorno-Tempini et al. 2011);
non-fluent/agrammatic variant primary progressive aphasia (nfvPPA; Gorno-Tempini et al. 2011);
c. behavioral variant for frontotemporal dementia (bvFTD; Rascovsky et al. 2011). Patients who meet diagnostic criteria for svPPA (Gorno-Tempini et al. 2011) may still be included if they have a secondary diagnosis of bvFTD, so long as the PI can reasonably attribute their disinhibition, dietary changes, compulsions, and/or loss of empathy to anterior temporal lobe atrophy (Seeley et al. 2005) and MRI is consistent with right anterior atrophy (or left temporal atrophy in participants with suspected right hemispheric language dominance);
d. progressive supranuclear palsy (PSP; Höglinger et al. 2017); e. corticobasal syndrome (CBS; Armstrong et al. 2013);
Any other medical condition other than FTLD that is likely to account for cognitive or behavioral deficits (e.g., uncontrolled seizure disorder, stroke, vascular dementia, substance abuse or alcoholism, Lewy body disease);
History of uncontrolled thyroid disease or evidence thereof [i.e., abnormal free thyroxine (T4) levels and thyroid stimulating hormone (TSH) > 10 milli-international units (mIU)/liter (L) at screening (confirmed by repeat)];
Serious autoimmune disease, or ongoing immunocompromised state;
History of significant cardiovascular, hematologic, renal, or hepatic disease (or laboratory evidence thereof at screening);
History or presence of gastrointestinal (GI) or other disease known to interfere with absorption, distribution, metabolism, or excretion of drugs, or a history of surgery known to interfere with absorption or excretion of drugs (i.e., gastric bypass);
Within 1 year prior to initial screening visit or between screening and baseline (pre-dose Day 1), any of the following: myocardial infarction; hospitalization for congestive heart failure; hospitalization for, or symptoms of, unstable angina; or syncope;
History of major psychiatric illness or untreated depression that in the opinion of the PI would pose a safety risk or interfere with the appropriate interpretation of study data;
Neutrophil count <1,500/cubic millimeter (mm3), platelets <100,000/mm3, serum creatinine >1.5 x upper limit of normal (ULN), total bilirubin (TBL) >1.5 x ULN, alanine aminotransferase (ALT) >1.5 x ULN, aspartate aminotransferase (AST) >1.5 x ULN, or international normalized ratio (INR) >1.2 at screening (confirmed by repeat);
Evidence of any clinically significant findings on screening or baseline evaluations which, in the opinion of the PI would pose a safety risk or interfere with appropriate interpretation of study data;
Corrected QT interval by Fridericia (QTcF) ≥ 470 milliseconds (msec) or uncontrolled arrhythmia or frequent premature ventricular contractions (PVCs; >5/minute) or Mobitz Type II second or third degree atrioventricular (AV) block or left bundle branch block or right bundle branch block with a QRS duration ≥ 150 msec or intraventricular conduction defect with a QRS duration ≥ 150 msec or evidence of acute or sub-acute myocardial infarction or ischemia or other ECG findings at screening or baseline that, in the PI's opinion, would preclude participation in the study;
Pathologic renal findings at screening as defined by the presence of either of the following criteria:
Estimated glomerular filtration rate (eGFR) [determined by the Modification of Diet in Renal Disease (MDRD) Study equation] < 30 milliliter (mL)/minute/1.73 square meter (m2). The MDRD Study equation is as follows: eGFR (mL/minute/1.73 m2) = 175 x (Scr)-1.154 x (Age)-0.203 x (0.742 if female) x (1.212 if African American), where Scr = serum creatinine in mg/deciliter (dL) as measured by a method calibrated to an isotope dilution mass spectrometry (IDMS) reference method;
Serum creatinine ≥ 2.5 mg/dL;
Hemoglobin A1C >7.5% at screening (confirmed by repeat);
Current or recent history (within four weeks prior to initial screening visit) of a clinically significant bacterial, fungal, or mycobacterial infection;
Current clinically significant viral infection, including "known" positive status for human immunodeficiency virus (HIV) (i.e., based on prior testing; HIV testing will not be performed as part of the screening evaluations for this trial);
Major surgery within four weeks prior to initial screening visit;
Blood transfusion within 4 weeks of initial screening visit;
History of stem cell treatment;

Where it is running

UCSF Memory and Aging Center — San Francisco, California, United States
Northwestern University — Chicago, Illinois, United States
Mayo Clinic — Rochester, Minnesota, United States
University of Pennsylvania — Philadelphia, Pennsylvania, United States
Houston Methodist Hospital - Nantz National Alzheimer Center — Houston, Texas, United States

Full record on ClinicalTrials.gov

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