drugset / Trial / NCT07533591
Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
Phase 0
Not yet recruiting
6 enrolled
Second Affiliated Hospital, School of Medicine, Zhejiang University
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2026-05
- Primary completion
- 2029-04
- Completion
- 2029-04
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | STP-001 | Gene therapy (AAV / viral vector) | 1e+12 unknown | Other |
| Subject | STP-001 | Gene therapy (AAV / viral vector) | 3e+12 unknown | Other |
| Background | Clozapine | Other / unclassified | 0.125 unknown | Oral |