VENT-02 for Parkinson's Disease

No longer recruiting at 22 trial locations

Overseen By Sofina Rodas

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Ventus Therapeutics U.S., Inc.

Must not be taking: Anticoagulants

Disqualifiers: Hepatitis, HIV, Dementias, Epilepsy, others

Compensation: Varies

Number of Visits: 1

Duration: 4 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new oral treatment called VENT-02 to determine its safety and tolerability for people with mild to moderate Parkinson's disease. Researchers aim to understand the drug's behavior in the body and its effects over a 28-day period. Participants will receive either VENT-02 or a placebo (a pill with no active drug) for comparison. Those who have had Parkinson's for seven years or less and experience symptoms like tremors or stiffness might be suitable candidates for this study. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.

Will I have to stop taking my current medications?

The trial requires that if you are currently taking medications for Parkinson's disease or any other condition, you must be on a stable dose for at least 2 weeks before starting the trial and should not expect to change this regimen during the study.

Is there any evidence suggesting that VENT-02 is likely to be safe for humans?

Research has shown that VENT-02 has been safe in past studies. In earlier research, patients tolerated it well, with no major side effects reported. Importantly, the studies suggested that the treatment did not cause any significant harm. While some minor side effects might occur, they have not been common or serious enough to cause concern.

This trial will further evaluate VENT-02's safety over 28 days in people with mild to moderate Parkinson's disease. The study is designed to carefully monitor any possible side effects. Participating in a clinical trial helps advance research that can lead to better treatments for everyone. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Parkinson's disease?

Unlike the standard treatments for Parkinson's disease, which often focus on managing symptoms with medications like levodopa or dopamine agonists, VENT-02 offers a novel approach. Researchers are excited about VENT-02 because it targets the underlying disease mechanisms differently, potentially slowing the progression of Parkinson's rather than just alleviating symptoms. This innovative mechanism of action sets VENT-02 apart from existing therapies and could offer a new avenue for managing this challenging condition.

What evidence suggests that VENT-02 might be an effective treatment for Parkinson's disease?

Research has shown that VENT-02 targets a protein complex called the NLRP3 inflammasome, which is involved in inflammation and may play a role in Parkinson's disease. Early trials found VENT-02 to be well-tolerated, with no serious side effects reported. The drug demonstrated strong effects in the body, effectively reaching and acting on its target. This suggests that VENT-02 could help reduce brain inflammation, potentially slowing the progression of Parkinson's disease symptoms. Although human data remains limited, its promising mechanism offers hope for effectiveness. Participants in this trial will receive either VENT-02 or a placebo to further evaluate its potential benefits. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for people with mild to moderate Parkinson's Disease who can safely take oral medication. The exact criteria for joining aren't listed, but typically participants must meet certain health standards and not be on conflicting medications.

Inclusion Criteria

I was diagnosed with Parkinson's disease less than 7 years ago.

I've been on a stable Parkinson's disease treatment for at least 2 weeks.

I am between 45 and 90 years old.

Exclusion Criteria

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

I do not have any serious illnesses that could affect my safety in the study.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive VENT-02 or placebo orally at 1 dose level twice daily over 28 days

4 weeks

Daily dosing

Follow-up

Participants are monitored for safety and effectiveness after treatment

1 week

1 visit (in-person)

What Are the Treatments Tested in This Trial?

Interventions

• VENT-02

Trial Overview The study tests VENT-02, a potential new treatment for Parkinson's. Participants will either receive VENT-02 or a placebo twice daily for 28 days. They won't know which one they're getting, and neither will the researchers (double-blind).

How Is the Trial Designed?

Group I: VENT-02 Dose 1 Experimental Treatment 1 Intervention

Group II: Placebo Placebo Group 1 Intervention

Who Is Running the Clinical Trial?

Ventus Therapeutics U.S., Inc.

Lead Sponsor

Trials

1

Recruited

30+

Published Research Related to This Trial

In a follow-up study of 20 patients with Parkinson's disease who had previously taken exenatide for 12 months, significant improvements in motor function and cognitive performance were observed even 12 months after stopping the medication, compared to a control group.

Patients who received exenatide showed a notable advantage in motor severity scores (5.6 points improvement) and cognitive function (5.3 points improvement) compared to controls, suggesting potential long-term benefits that warrant further investigation into exenatide as a disease-modifying treatment for Parkinson's disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Motor and cognitive advantages persist 12 months after exenatide exposure in Parkinson's disease. Aviles-Olmos, I., Dickson, J., Kefalopoulou, Z., et al.[2018]

Amantadine extended-release (ER) capsules significantly improve levodopa-induced dyskinesia (LID) in Parkinson's disease patients, as shown in phase III trials lasting 13 and 25 weeks, with benefits including increased ON time without troublesome dyskinesia.

The treatment is generally well tolerated, with most side effects being mild to moderate, although common adverse events include hallucinations and dizziness. Long-term safety and efficacy are still being evaluated in an ongoing study.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Amantadine Extended-Release (GOCOVRI™): A Review in Levodopa-Induced Dyskinesia in Parkinson's Disease. Paik, J., Keam, SJ.[2021]

CVT-301, a self-administered levodopa inhalation powder, significantly improved motor scores in patients with Parkinson's disease during off periods, showing a greater reduction in UPDRS scores compared to placebo (mean difference of -3.92).

The treatment was found to be safe and well tolerated, with only a small percentage of patients experiencing severe or serious adverse events, indicating a favorable safety profile for CVT-301.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Safety and efficacy of CVT-301 (levodopa inhalation powder) on motor function during off periods in patients with Parkinson's disease: a randomised, double-blind, placebo-controlled phase 3 trial. LeWitt, PA., Hauser, RA., Pahwa, R., et al.[2020]

Inhaled levodopa provides a rapid absorption and quicker relief of 'off' symptoms in Parkinson's disease compared to traditional oral formulations, making it a promising new treatment option.

Long-term safety studies (up to 1 year) show that inhaled levodopa is well tolerated with no significant adverse effects on lung function, although common side effects include mild cough and nausea.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Profile of inhaled levodopa and its potential in the treatment of Parkinson's disease: evidence to date. Patel, AB., Jimenez-Shahed, J.[2020]

In a study of 91 advanced Parkinson's disease patients, duodenal levodopa infusion was found to be an effective last-line treatment for motor complications, with over 90% of long-term users reporting improvements in motor fluctuations and quality of life.

Despite some technical issues, the treatment had few severe adverse events, suggesting it is a relatively safe option for patients who have not responded to other therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient profile, indications, efficacy and safety of duodenal levodopa infusion in advanced Parkinson's disease. Devos, D.[2022]

A long-term safety assessment of rAAV2-neurturin in 53 patients with advanced Parkinson's disease showed that the treatment is generally safe, with only a few non-serious adverse events reported, none of which were definitively linked to the therapy.

Over a follow-up period of up to 5 years, patients demonstrated stable or modestly improved motor function, indicating that rAAV2-neurturin not only has a favorable safety profile but may also provide therapeutic benefits in managing Parkinson's disease symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer. Marks, WJ., Baumann, TL., Bartus, RT.[2022]

In a study involving 53 patients with mild to moderate Parkinson's disease, both budipine and amantadine significantly improved Parkinsonian symptoms, but budipine showed a better effect on tremor after 12 weeks of treatment.

Budipine was associated with fewer adverse events compared to amantadine, suggesting it may be a safer option for patients, although both medications had similar overall efficacy in reducing symptoms.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Monotherapy of Parkinson's disease with budipine. A double blind comparison with amantadine]. Malsch, U., Bliesath, H., Böther, K., et al.[2013]

A 24-year-old patient with intractable posttraumatic Holmes tremor showed significant improvement after deep brain stimulation using two electrodes targeting both the VIM/VOP and VOA/VOP thalamic regions, indicating a potential new treatment approach.

The patient experienced tremor rebound with monotherapy targeting VIM/VOP, but activation of the second lead at VOA/VOP resulted in sustained tremor improvement at the 12-month follow-up, suggesting that dual lead stimulation may enhance treatment efficacy for difficult-to-control tremors.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ventralis intermedius plus ventralis oralis anterior and posterior deep brain stimulation for posttraumatic Holmes tremor: two leads may be better than one: technical note. Foote, KD., Okun, MS.[2019]

A survey of 401 patients with advanced Parkinson's disease revealed that improving cognitive function was prioritized over movement symptom control, indicating that patients value mental clarity highly in treatment decisions.

Patients preferred treatment via a portable infusion pump over deep brain stimulation (DBS), highlighting a preference for less invasive options, especially avoiding neurosurgery, which was deemed more important than avoiding other surgical procedures.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Patient Preferences for Device-Aided Treatments Indicated for Advanced Parkinson Disease. Marshall, T., Pugh, A., Fairchild, A., et al.[2018]

Levodopa/carbidopa enteral suspension (ES) has been shown to significantly improve motor fluctuations and quality of life in adults with advanced Parkinson's disease during short-term (12-week) treatment, with benefits sustained for up to 7 years.

The safety profile of levodopa/carbidopa ES is comparable to that of traditional oral formulations, with adverse events primarily related to aging and Parkinson's disease itself, indicating it is a well-tolerated treatment option for patients not adequately managed by other therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Levodopa/Carbidopa Enteral Suspension: A Review in Advanced Parkinson's Disease. Hoy, SM.[2020]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06822517

A Phase 1b Study of the NLRP3 Inhibitor VENT-02 in ... This trial is a randomized, double-blind, placebo-controlled Phase 1b study evaluating the safety/tolerability, PK, and pharmacodynamics of VENT 02, ...

2. ventustx.com ventustx.com/ventus-therapeutics-announces-results-from-phase-1-clinical-trial-of-vent-02-a-novel-orally-administered-brain-penetrant-nlrp3-inhibitor/

VENT-02 demonstrated robust pharmacodynamic ... In this Phase 1 clinical trial, VENT-02 was well-tolerated, with no dose-limiting toxicities or serious adverse events reported and only mild or ...

3. withpower.com withpower.com/trial/phase-2-parkinson-disease-1-2025-57708

VENT-02 for 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, ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC12409668/

Profile of NT-0527, a brain penetrant NLRP3 Inflammasome ... Two further NLRP3i, VTX3232 and VENT-02, have been advanced into human clinical trials for Parkinson's disease, but structures for these ...

5. apdaparkinson.org apdaparkinson.org/article/new-pd-treatments-clinical-trial-pipeline/

New Parkinson's Treatments in the Clinical Trial Pipeline VENT-02 (Ventus Therapeutics) is currently being studied in a Phase 1b study. NCT06822517; Dapansutrile (Olatec Therapeutics) is currently being ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11345644/

Screening NLRP3 drug candidates in clinical development This article reviews properties of these molecules and comments on current technologies to design and screen for potential candidates.

7. parkinsonsnewstoday.com parkinsonsnewstoday.com/news/first-patient-dosed-parkinsons-clinical-trial-ventus-vent-02/

First patient dosed in Parkinson's clinical trial of Ventus ... The study's main goal is to assess VENT-02's safety and tolerability. Secondary goals include changes in biomarkers of inflammation ...