Clinical trial NCT06822517: A Phase 1b Study of the NLRP3 Inhibitor VENT-02 in Patients With Mild to Moderate Parkinson's Disease
Official title: A Randomized, Double-Blind, Placebo-Controlled, Phase 1b Study of the NLRP3 Inhibitor VENT-02 in Patients With Mild to Moderate Parkinson's Disease
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Parkinson Disease
Lead sponsor: Ventus Therapeutics U.S., Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-12
Enrollment: 30
Sex: ALL
Minimum age (years): 45
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs)
timeFrame: Baseline to Follow-up Visit (Day 35)
Secondary outcomes:
measure: Concentrations of biomarkers
timeFrame: Baseline to Day 28
measure: Concentrations of VENT-02 in plasma and CSF
timeFrame: Baseline to Day 28
Other outcomes:
Locations:
city: Fountain Valley
country: United States
facility: Investigative Site
state: California
city: Los Alamitos
country: United States
facility: Investigative Site
state: California
city: San Jose
country: United States
facility: Investigative Site
state: California
city: West Hills
country: United States
facility: Investigative Site
state: California
city: Englewood
country: United States
facility: Investigative Site
state: Colorado
city: Aventura
country: United States
facility: Investigative Site
state: Florida
city: Boca Raton
country: United States
facility: Investigative Site
state: Florida
city: Coral Gables
country: United States
facility: Investigative Site
state: Florida
city: Coral Springs
country: United States
facility: Investigative Site
state: Florida
city: Cutler Bay
country: United States
facility: Investigative Site
state: Florida
city: Daytona Beach
country: United States
facility: Investigative Site
state: Florida
city: Doral
country: United States
facility: Investigative Site
state: Florida
city: Jacksonville
country: United States
facility: Investigative Site
state: Florida
city: Maitland
country: United States
facility: Investigative Site
state: Florida
city: Miami
country: United States
facility: Investigative Site
state: Florida
city: Miami
country: United States
facility: Investigative Site
state: Florida
city: Winter Park
country: United States
facility: Investigative Site
state: Florida
city: Farmington Hills
country: United States
facility: Investigative Site
state: Michigan
city: Abington
country: United States
facility: Investigative Site
state: Pennsylvania
city: Memphis
country: United States
facility: Investigative Site
state: Tennessee
city: Cypress
country: United States
facility: Investigative Site
state: Texas
city: Round Rock
country: United States
facility: Investigative Site
state: Texas
city: Spokane
country: United States
facility: Investigative Site
state: Washington
Study officials:
References: