Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic S… (NCT07533591) | Clinical Trial Compass
Not Yet RecruitingEarly Phase 1
Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
China6 participantsStarted 2026-05
Plain-language summary
The investigators propose a gene therapy strategy for Parkinson's disease - a chemogenetic inhibition technique to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's patients. The investigators design and construct a therapeutic injection agent called STP-001, through an efficient adeno-associated virus capsid (AAV), a neuronal promoter (hSyn), and a chemogenetic effector element (hM4Di). Then, the drug was accurately injected into the bilateral subthalamic nuclei through stereotactic surgery. After the surgery, combined with clozapine, the abnormal activity of the subthalamic nucleus was precisely intervened to improve the core motor symptoms of Parkinson's disease.
Who can participate
Age range
40 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:
* Age range: 40 - 70 years (inclusive), gender not restricted;
* Diagnosed with primary Parkinson's disease, with H\&Y grade ranging from 4 to 5;
* Disease duration of at least 5 years;
* Response to levodopa treatment lasting for at least 12 months;
* Score of the third part of the MDS-UPDRS Part III ≥ 35;
* Improvement rate of the Madopar challenge test ≥ 30%;
* Stable clinical symptoms and drug treatment for at least 4 weeks before screening;
* Capable of understanding and voluntarily signing the informed consent form;
* Participants agree to postpone any neurological surgery, including deep brain stimulation, until the completion of 12-month follow-up;
* The efficacy of dopamine drugs has significantly decreased, or there are obvious motor complications, or severe drug side effects;
* There are no acute adverse reactions to the prescribed olanzapine dose;
* Good compliance and willing to complete all the follow-up as stipulated in this protocol.
Exclusion Criteria:
* Has a past history of brain surgery for Parkinson's disease, such as deep brain stimulation, or other abnormal brain imaging findings;
* Hamilton Depression Scale score ≥ 20;
* Hamilton Anxiety Scale score ≥ 14
* Has a history of brain injury or central nervous system infection;
* MoCA cognitive impairment score \< 26 points, and MMSE dementia rating scale cognitive impairment score ≤ 20 points;
* Focal neurological deficits;
* Evidence of major medical or mental illness, such as dement…
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
The incidence rates of adverse events and serious adverse events within 3 months after injecting STP-001 into the bilateral subthalamic nuclei.
Timeframe: 3 months after injecting STP-001
Trial details
NCT IDNCT07533591
SponsorSecond Affiliated Hospital, School of Medicine, Zhejiang University