A Phase 1b Study of the NLRP3 Inhibitor VENT-02 in Patients With Mild to Moderate Parkinson's Disease

A Randomized, Double-Blind, Placebo-Controlled, Phase 1b Study of the NLRP3 Inhibitor VENT-02 in Patients With Mild to Moderate Parkinson's Disease

This trial is a randomized, double-blind, placebo-controlled Phase 1b study evaluating the safety/tolerability, PK, and pharmacodynamics of VENT 02, administered orally at 1 dose level twice daily (BID) over 28 days in patients with mild to moderate Parkinson's disease.

The study includes a screening period, a 28-day double-blind treatment period, and a 7-day follow-up period after last dose.

Approximately 30 patients will be randomized into 1 of the 2 treatment arms.

Study Overview

• Status: Terminated
• Conditions: Parkinson Disease
• Intervention / Treatment: Drug: VENT-02; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 29
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• United States
  • California
    • Fountain Valley, California, United States, 92708
      • Investigative Site
    • San Jose, California, United States, 95124
      • Investigative Site
    • West Hills, California, United States, 91307
      • Investigative Site
  • Colorado
    • Englewood, Colorado, United States, 80113
      • Investigative Site
  • Florida
    • Aventura, Florida, United States, 33180
      • Investigative Site
    • Boca Raton, Florida, United States, 33486
      • Investigative Site
    • Coral Gables, Florida, United States, 33134
      • Investigative Site
    • Coral Springs, Florida, United States, 33067
      • Investigative Site
    • Cutler Bay, Florida, United States, 33189
      • Investigative Site
    • Daytona Beach, Florida, United States, 32117
      • Investigative Site
    • Doral, Florida, United States, 33178
      • Investigative Site
    • Jacksonville, Florida, United States, 32256
      • Investigative Site
    • Maitland, Florida, United States, 32751
      • Investigative Site
    • Miami, Florida, United States, 33135
      • Investigative Site
    • Miami, Florida, United States, 33165
      • Investigative Site
    • Winter Park, Florida, United States, 32789
      • Investigative Site
  • Maine
    • Scarborough, Maine, United States, 04074
      • Investigative Site
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
      • Investigative Site
  • Pennsylvania
    • Abington, Pennsylvania, United States, 19001
      • Investigative Site
  • Tennessee
    • Memphis, Tennessee, United States, 38157
      • Investigative Site
  • Texas
    • Cypress, Texas, United States, 77429
      • Investigative Site
    • Round Rock, Texas, United States, 78681
      • Investigative Site
  • Washington
    • Spokane, Washington, United States, 99202
      • Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 45 to 90 years of age, inclusive.
• Body weight and body mass index (BMI) within the range of 100 to 265 pounds (45 to 120 kg) and 18 to 34 kg/m2 (inclusive), respectively.
• A diagnosis of idiopathic PD according to United Kingdom (UK) Brain Bank or Movement Disorder Society (MDS) criteria with bradykinesia and ≥ 1 additional cardinal sign of PD (e.g., resting tremor, rigidity).
• A diagnosis of PD for ≤ 7 years at Screening.
• A modified Hoehn & Yahr stage 1 to 2.5.
• If presently receiving treatment for PD, must be on a stable dose for ≥ 2 weeks prior to dosing, with no expected change in this regimen for the duration of the study.
• If presently taking any non-PD concomitant medications, must be on a stable dose for ≥ 2 weeks prior to dosing.

Exclusion Criteria:

• Any clinically significant abnormality at Screening
• A positive serology for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (anti-HCV), or human immunodeficiency virus antibodies (anti-HIV) 1/2 at Screening.
• A significant neurological disease affecting the central nervous system, other than PD, that may affect cognition or ability to complete the study, including but not limited to dementias, severe and repetitive past (up to 5 years) head trauma, normal pressure hydrocephalus, or epilepsy or recurrent seizures (except febrile childhood seizures), as determined by the investigator.
• Current serious or unstable illnesses, that, in the investigator's opinion, could compromise patient safety and ability to comply with study procedures, or has a life expectancy of < 24 months.
• A history of suicidal ideation or previous suicide attempt in the 12 months prior to Screening or is clinically judged by the investigator to be at serious risk for suicide as assessed by medical history, examination, or the C-SSRS.
• A contraindication (e.g., current use of anticoagulants) that would prohibit a lumbar puncture (LP).
• Currently enrolled in any other interventional clinical trial involving an investigational product or any other type of medical research judged not to be scientifically or medically compatible with this study, as assessed by the investigator.
• Has participated in a clinical trial involving an investigational product within 30 days or 5 half-lives (whichever is longer) prior to dosing.
• Has levodopa-induced dyskinesias lasting for > 25% of waking day or dyskinesias interfering with many daily activities.
• Has dysphagia to the extent that it would affect the patient's ability to swallow the investigational medicinal product (IMP).
• Has a parkinsonian syndrome, including atypical parkinsonism.
• Is a known carrier (i.e., confirmed by historical medical documentation) of familial PD genes.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Orally administered capsules
Experimental: VENT-02 Dose 1	Drug: VENT-02; Orally administered capsules

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of treatment-emergent adverse events (TEAEs)	Number of treatment-emergent adverse events (TEAEs) with VENT-02 treatment compared to placebo	Baseline to Follow-up Visit (Day 35)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Concentrations of biomarkers	Change from baseline in concentrations of biomarkers of inflammation and neurodegeneration in plasma and CSF	Baseline to Day 28
Concentrations of VENT-02 in plasma and CSF	PK as measured by concentrations of VENT-02 in plasma and CSF	Baseline to Day 28

Collaborators and Investigators

• Sponsor: Ventus Therapeutics U.S., Inc.

Study record dates

Study Major Dates

• Study Start (Actual): March 5, 2025
• Primary Completion (Actual): October 9, 2025
• Study Completion (Actual): October 9, 2025

Study Registration Dates

• First Submitted: February 6, 2025
• First Submitted That Met QC Criteria: February 6, 2025
• First Posted (Actual): February 12, 2025

Study Record Updates

• Last Update Posted (Estimated): October 29, 2025
• Last Update Submitted That Met QC Criteria: October 27, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: VENT-02-102

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No