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

ConditionSemantic Dementia

Trial statusRecruiting

Trial phasePhase 1

Trial typeInterventional

Biological sexAll

Age18-85

SponsorPeter Ljubenkov, MD

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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;

Disqualifiers

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);

Trial design

Design model

Parallel

Treatments tested in this trial

Verdiperstat
Drug
Oral, extended release (ER) tablet

Treatment groups

64 Participants

are divided into 2 treatment groups

Group A: VerdiperstatExperimental treatment 1 intervention

Group B: PlaceboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

Incidence of Treatment-Emergent Adverse Events

Assess adverse events during 6 months administration of Verdiperstat or Placebo

Time frame

24 Weeks

Secondary outcomes

Changes in Pharmacokinetic properties of Verdiperstat in Cerebrospinal Fluid (CSF)

Measure steady-state cerebrospinal fluid concentrations of Verdiperstat and its metabolites

Time frame

24 Weeks

Changes in Pharmacokinetic properties of Verdiperstat in Plasma

Measure steady-state plasma concentrations of Verdiperstat and its metabolites

Time frame

24 Weeks

Other outcomes

Changes in Pharmacodynamic (PD) properties of Verdiperstat in Plasma

Measure plasma myeloperoxidase (MPO) activity

Time frame

24 Weeks

Changes in Pharmacodynamic properties of CSF biomarkers of neurofilament light chain protein

Measure CSF concentrations of neurofilament light chain protein (NfL) pg/ml

Time frame

24 Weeks

Change in brain volume on brain MRI

Measure of global and regional volumes of interest (such as whole brain and temporal lobes)

Time frame

24 Weeks

Change in structural and functional connectivity on brain MRI

Connectivity between brain regions measured using diffusion tensor MRI and resting state functional MRI

Time frame

24 Weeks

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Peter Ljubenkov, MD

Lead sponsor

University of California, San Francisco

Sponsor institution

National Institutes of Health (NIH)

Collaborator

Alzheimer's Association

Collaborator

National Institute on Aging (NIA)

Collaborator

Source ClinicalTrials.gov

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