Gene therapy injected into a deep brain area for Parkinson's disease
Tests a gene therapy given by brain surgery in people with Parkinson's disease.
Plain-language title generated from the registry record
Registry title: Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
Key facts
- Where
- Zhejiang, ChinaSee the site
- Phase
- Early phase 1
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 40 and 70 years old?
Source: NCT07533591, who can take part
Where in China
- Second Affiliated Hospital, School of Medicine,Zhejiang UniversityZhejiang, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Second Affiliated Hospital, Zhejiang University, School of Medicine
- Trial code
- NCT07533591
- Record updated
- 24 April 2026
- Original description
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.Read full original descriptionShow less
Clinical Study of Chemogenetic Gene Therapy With Adeno-Associated Virus for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
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.
Inclusion: 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: Received other gene or cell therapy drugs; Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy and chemotherapy; Has a past history of brain surgery for Parkinson's disease, such as deep brain stimulation, or other abnormal brain imaging findings; 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; Evidence of major medical or mental illness, such as dementia, psychosis, history of drug abuse or severe depression; Atypical Parkinson's disease or secondary Parkinson's disease caused by factors such as trauma, brain tumor, infection, cerebrovascular disease, other neurological diseases or drugs, chemicals or toxins; Has a history of malignant tumors, but not including treated carcinoma in situ, or medical conditions that are poorly controlled and may increase the risk of surgery; Has a history of stroke, or obvious cardiovascular diseases, diabetes; Coagulation disorders or inability to temporarily stop any anticoagulant or antiplatelet treatment before surgery; Clinically obvious or laboratory-detected infections; Severe liver disease (AST/ALT ≥ 2.5 times the upper limit of normal (ULN)); Hamilton Depression Scale score ≥ 20; Hamilton Anxiety Scale score ≥ 14; Focal neurological deficits; Any factor that may prevent the subject from following the study protocol, including medical or social factors, including geographical inconvenience; Any other situation that the investigator deems unsuitable for participation in this study.