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Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus

NCT07533591 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2026-04-24

No results posted yet for this study

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.

Conditions

  • Parkinson's Disease (PD)

Interventions

GENETIC

gene therapy

Six participants are planned to be divided into two dose groups in the dose escalation principle. The dose escalation principle is as follows: After 3 sentinel subjects receive 1×10¹² volts of the virus vector, the research team will assess the safety, tolerance and efficacy of the current drug dosage 3 months later. If the test subjects do not show dose-limiting toxicity and have some efficacy, the sample size will be expanded to 6 at the original dosage. If the test subjects do not show dose-limiting toxicity but have no obvious efficacy, the titer will be increased to 3×10¹² volts and three more participants will be recruited for the trial. Four weeks after the injection of STP-001, when the participants recover, clozapine dose escalation treatment will be carried out.

Sponsors & Collaborators

  • Second Affiliated Hospital, School of Medicine, Zhejiang University

    lead OTHER

Principal Investigators

  • Jiali Pu · Second Affiliated Hospital, School of Medicine, Zhejiang University

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
40 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-31
Primary Completion
2029-04-30
Completion
2029-04-30

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07533591 on ClinicalTrials.gov