Parkinson Disease
Conditions
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.
Interventions
DRUGVENT-02
Orally administered capsules
DRUGPlacebo
Orally administered capsules
Sponsors
Ventus Therapeutics U.S., Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
45 Years to 90 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | Baseline to Follow-up Visit (Day 35) | Number of treatment-emergent adverse events (TEAEs) with VENT-02 treatment compared to placebo |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Concentrations of biomarkers | Baseline to Day 28 | Change from baseline in concentrations of biomarkers of inflammation and neurodegeneration in plasma and CSF |
| Concentrations of VENT-02 in plasma and CSF | Baseline to Day 28 | PK as measured by concentrations of VENT-02 in plasma and CSF |
Countries
United States
Outcome results
None listed