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

NCT06822517 | Terminated Phase 1 FDA Drug

• 1 other identifier: VENT-02-102
• Study Type: interventional
• Target: 29
• Locations: 1 country
• Sites: 23

Brief Summary

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.

Conditions

Parkinson Disease

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Concentrations of biomarkers

  Baseline to Day 28

• Concentrations of VENT-02 in plasma and CSF

  Baseline to Day 28

Study Arms (2)

VENT-02 Dose 1

EXPERIMENTAL

Drug: VENT-02

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

VENT-02 DRUG

Orally administered capsules

VENT-02 Dose 1

Placebo DRUG

Orally administered capsules

Placebo

Eligibility Criteria

• Age: 45 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

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

Study Sites (23)

Investigative Site

Fountain Valley, California, 92708, United States

Location

Investigative Site

San Jose, California, 95124, United States

Location

Investigative Site

West Hills, California, 91307, United States

Location

Investigative Site

Englewood, Colorado, 80113, United States

Location

Investigative Site

Aventura, Florida, 33180, United States

Location

Investigative Site

Boca Raton, Florida, 33486, United States

Location

Investigative Site

Coral Gables, Florida, 33134, United States

Location

Investigative Site

Coral Springs, Florida, 33067, United States

Location

Investigative Site

Cutler Bay, Florida, 33189, United States

Location

Investigative Site

Daytona Beach, Florida, 32117, United States

Location

Investigative Site

Doral, Florida, 33178, United States

Location

Investigative Site

Jacksonville, Florida, 32256, United States

Location

Investigative Site

Maitland, Florida, 32751, United States

Location

Investigative Site

Miami, Florida, 33135, United States

Location

Investigative Site

Miami, Florida, 33165, United States

Location

Investigative Site

Winter Park, Florida, 32789, United States

Location

Investigative Site

Scarborough, Maine, 04074, United States

Location

Investigative Site

Farmington Hills, Michigan, 48334, United States

Location

Investigative Site

Abington, Pennsylvania, 19001, United States

Location

Investigative Site

Memphis, Tennessee, 38157, United States

Location

Investigative Site

Cypress, Texas, 77429, United States

Location

Investigative Site

Round Rock, Texas, 78681, United States

Location

Investigative Site

Spokane, Washington, 99202, United States

Location

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 6, 2025
• First Posted: February 12, 2025
• Study Start: March 5, 2025
• Primary Completion: October 9, 2025
• Study Completion: October 9, 2025
• Last Updated: October 29, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (23)