A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patien… (NCT07414290) | Clinical Trial Compass
Not Yet RecruitingPhase 1/2
A Trial to Evaluate Safety and Efficacy of a Product Named VGN-R08b in Parkinson's Disease Patients With GBA1 Mutations
China17 participantsStarted 2026-05-25
Plain-language summary
A Phase I/II Clinical Study to Evaluate the Tolerability, Safety, and Efficacy of VGN-R08b Intra-cerebroventricular injection in Parkinson's Disease Patients with GBA1 Mutations
Who can participate
Age range
30 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Male or female, aged 30 to 70 years (inclusive) at the time of signing the informed consent form.
. Documented GBA1-mutant Parkinson's disease, confirmed by medical history: meeting the International Parkinson and Movement Disorder Society (MDS) diagnostic criteria for idiopathic Parkinson's disease, with the presence of at least one pathogenic GBA1 gene mutation (confirmed by investigator interpretation).
. Glucocerebrosidase (GCase) enzyme activity below the normal range, as measured from past or screening dried blood spot tests.
. Hoehn-Yahr stage of 3 to 4 in the "OFF" state, an MDS-UPDRS Part III (motor examination) score ≥33 points in the "OFF" state, and the ability to walk without relying on a walker or wheelchair.
. Montreal Cognitive Assessment (MoCA) score meeting the following criteria: \>13 (for ≤6 years of education), \>15 (for 7-12 years of education), or \>16 (for \>12 years of education) .
. On an optimized levodopa regimen at screening (defined as a regimen optimized with at least a combination of levodopa preparations plus a dopamine agonist or MAO-B inhibitor, with levodopa administered ≥3 times per day and at a total daily dose of ≥300 mg), yet still experiencing suboptimal symptom control or significant "wearing-off" (as evidenced by a diary documenting a daily "OFF" time of ≥2.5 hours for three consecutive days during screening).
. Stable Parkinson's disease symptoms and stable optimized anti-Parkinson's medication regimen for ≥4 weeks prior to screening; patients with GD-PD receiving Gaucher disease (GD) therapy must have been on stable enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 3 months prior to screening.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Adverse Events (AEs), Serious Adverse Events (SAEs)Vital signs
. Men and women of childbearing potential must agree to consistently and correctly use a highly effective method of contraception from the screening period until at least 1 year after dosing.
Exclusion criteria
. Patients with atypical or secondary parkinsonian syndromes, including but not limited to those caused by trauma, brain tumors, infections, cerebrovascular diseases, or other neurological disorders; or symptoms confirmed by the investigator to be induced by drugs, chemicals, or toxins; or those with other serious neurological conditions deemed by the investigator to significantly compromise the safety and efficacy evaluation of the investigational drug.
. Patients with active infections (including viral infections such as HBV, HCV, or syphilis) or a history of severe infections within 12 weeks prior to screening (e.g., pneumonia, sepsis, or central nervous system infections such as meningitis or encephalitis).
. Patients with severe liver disease, severe immunodeficiency, or autoimmune diseases within 6 months prior to screening, or those requiring long-term immunosuppressive therapy.
. Patients with poorly controlled diabetes or hypertension, judged by the investigator as unsuitable for dosing or likely to substantially impact the efficacy and safety analysis of the investigational drug.
. Patients with a history of stroke or transient ischemic attack (TIA), unstable angina, myocardial infarction, chronic heart failure (NYHA Class III or IV), or clinically significant conduction abnormalities (e.g., unstable atrial fibrillation) within 1 year prior to screening.
. Patients with a history of epileptic seizures or unexplained coma, deemed unsuitable by the investigator for participation in the trial.
. Patients with a history of severe allergic reactions, or hypersensitivity to any inactive ingredient of the investigational drug or to immunosuppressants required by the trial protocol.
. Patients with contraindications to corticosteroids or sirolimus, including but not limited to osteoporosis with vertebral fractures within 1 year prior to screening, poorly controlled hyperlipidemia or hypercholesterolemia, renal insufficiency, or interstitial lung disease.