VENT-02 for Parkinson's Disease

Recruiting at 18 trial locations

Overseen By Sofina Rodas

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Ventus Therapeutics U.S., Inc.

Must not be taking: Anticoagulants

Disqualifiers: Hepatitis, HIV, Dementias, Epilepsy, others

Trial Summary

What is the purpose of this trial?

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.

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.

What safety data exists for VENT-02 or similar treatments for Parkinson's Disease?

Inhaled levodopa, a treatment similar to VENT-02, has been studied for safety in Parkinson's patients and is generally well tolerated, with common side effects like mild cough and nausea. Long-term safety studies show no significant changes in lung function, and gene therapy treatments for Parkinson's have also shown long-term safety with no serious adverse effects directly related to the treatment. ^{1 2 3 4 5}

How does the VENT-02 treatment for Parkinson's disease differ from other treatments?

The VENT-02 treatment for Parkinson's disease is unique because it may involve a novel approach or mechanism not detailed in the provided research, as the existing studies focus on device-aided therapies like deep brain stimulation and levodopa infusions, which are standard for advanced Parkinson's. Without specific details on VENT-02, its uniqueness could lie in its administration method, components, or a new mechanism of action. ^{6 7 8 9 10}

Eligibility Criteria

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 am between 45 and 90 years old.

My Parkinson's disease is in the early to mid-stage.

Exclusion Criteria

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

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 major brain conditions affecting my thinking or memory, other than Parkinson's Disease.

Trial Timeline

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)

Treatment Details

Interventions

• VENT-02 (NLRP3 Inhibitor)

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).

Participant Groups

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+

Findings from Research

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 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]

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]

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]

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]

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 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]

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]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Profile of inhaled levodopa and its potential in the treatment of Parkinson's disease: evidence to date. [2020]

2. Netherlands pubmed.ncbi.nlm.nih.gov

Motor and cognitive advantages persist 12 months after exenatide exposure in Parkinson's disease. [2018]

3. United States pubmed.ncbi.nlm.nih.gov

Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer. [2022]

4. New Zealand pubmed.ncbi.nlm.nih.gov

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

5. Germany pubmed.ncbi.nlm.nih.gov

[Monotherapy of Parkinson's disease with budipine. A double blind comparison with amantadine]. [2013]

6. United States pubmed.ncbi.nlm.nih.gov

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

7. United States 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. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Patient Preferences for Device-Aided Treatments Indicated for Advanced Parkinson Disease. [2018]

9. New Zealand pubmed.ncbi.nlm.nih.gov

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

10. England 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. [2020]