Parkinson's Disease (PD)Early Phase 1CGTPDNCT07533591
Clinical Study of Chemogenetic Gene Therapy With AAV for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
A Early Phase 1 study testing gene therapy in people with Parkinson's Disease (PD), run by Second Affiliated Hospital, Zhejiang University, School of Medicine at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 40 to 70 years
- Conditions the study is for
- Parkinson's Disease (PD)
- What is being tested?
- gene therapy
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- May 2026to Apr 2029
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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%;
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- 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.
Who cannot join
- 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;
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- 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;
- Received other gene or cell therapy drugs;
- 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;
- Clinically obvious or laboratory-detected infections;
- Chronic immunosuppressive therapy, including chronic steroids, immunotherapy, cytotoxic therapy and chemotherapy;
- Coagulation disorders or inability to temporarily stop any anticoagulant or antiplatelet treatment before surgery;
- Severe liver disease (AST/ALT ≥ 2.5 times the upper limit of normal (ULN));
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Second Affiliated Hospital, School of Medicine,Zhejiang University
Zhejiang, Province
Jiali Pu, +86-13989468062, Jialipu@zju.edu.cn
Yang Ruan, 18858736151, 11818236@zju.edu.cn
About this study
Official title: 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.
Read the detailed description
This is a single-arm, open-label preliminary safety assessment study design. Six patients with iPD will be recruited from the Department of Neurology of the Second Affiliated Hospital of Zhejiang University School of Medicine. After signing the informed consent form, these 6 participants will be divided into 2 dose groups according to the dose escalation principle and receive the STP-001 injection solution. The virus 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. The participants will take clozapine orally every day, with doses of 1/32, 1/16 and 1/8 tablets respectively, taken in the morning and noon, and each dose will be repeated for 3 days, totaling 9 days. If there is no disease progression during this period, the participants will continue to take the 1/8 dose of the drug twice a day as the maintenance dose and undergo monitoring for 12 months.
- Conditions
- Parkinson's Disease (PD)
- Being tested
- gene therapy
- Keywords
- clozapine, Chemical Genetic Gene Therapy, Subthalamic nucleus, Parkinson's Disease
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
gene therapyGenetic
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.
| Study group | Role | What they receive |
|---|---|---|
| STP-001 group | Experimental | This is a single-arm, open-label preliminary safety assessment study design. Six patients with iPD will be recruited from the Department of Neurology of the Second Affiliated Hospital of Zhejiang University School of Medicine. After signing the informed consent form, these 6 participants will be divided into 2 dose groups according to the dose escalation principle and receive the STP-001 injection solution. The virus 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 inject Receives: Genetic: gene therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence rates of adverse events and serious adverse events within 3 months after injecting STP-001 into the bilateral subthalamic nuclei.
Adverse events and serious adverse events will be evaluated through physical examinations, electrocardiogram tests and laboratory tests to assess their safety and tolerability, including routine blood tests, routine urine tests, imaging studies, etc. The main aspects include the occurrence of CRS and GvHD (≥ grade II), as well as injection-induced dyskinesia (GIDs), new-onset allogeneic tumors after injection, and death.
Measured: 3 months after injecting STP-001
Secondary outcomes (9)
The incidence rates of adverse events and serious adverse events within 12 months after STP-001 was injected into the bilateral subthalamic nuclei.
Incidence of homologous tumor occurrence: Compare the results of head MRI scans before and after the operation, at 1, 3, 6, and 12 months; as well as the results of head CT scans before and 12 months after the operation. If there are new tumors (if any), perform histological examination to determine the pathological type.
Incidence and severity of adverse events (AE) and serious adverse events (SAE): Include safety indicators such as vital signs, physical examination, laboratory tests, immunological tests, and tumor marker tests within 12 months after the operation. Evaluate injection-induced dyskinesia within 12 months after the operation using a scale.
Measured: 12 months after STP-001
The differences of the MDS-Unified Parkinson's Disease Rating Scale before and after treatment
Compare the changes in the Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) from baseline to post- treatment in all of the 6 participants.Minimum value: 0；Maximum value: 260；Higher scores mean the worse outcome (greater severity of Parkinson's disease symptoms).
Measured: 12 months after STP-001 injection
The differences of the Clinical Global Impression scale-improvement before and after treatment
Compare the changes in the Clinical Global Impression scale-improvement from baseline to post-treatment in all of the 6 participants.Minimum value: 1;Maximum value: 7.Higher scores mean the worse outcome (greater illness severity).
Measured: 12 months after STP-001 injection
The differences of the Patient Global Impression improvement scale before and after treatment
Compare the changes in the Patient Global Impression improvement scale from baseline to post-treatment in all of the 6 participants. Minimum value: 1;Maximum value: 7.Higher scores mean worse outcome (greater illness severity).
Measured: 12 months after STP-001injection
The differences of the Non-Motor Symptoms Questionnaire before and after treatment
Compare the changes in the Non-Motor Symptoms Questionnaire from baseline to post-treatment in all of the 6 participants.Minimum score of 0, Maximum score of 30.Higher scores indicate a worse outcome, reflecting a greater burden of non-motor symptoms.
Measured: 12 months after STP-001 injection
The differences of anti-PD drug usage before and after treatment
Compare the changes in anti-PD drug use, including daily oral levodopa or a levodopa-equivalent dose, from baseline to post-treatment in 6 participants.
Measured: 12 months after STP-001 injection
The differences of the Parkinson's Disease Questionnaire before and after treatment
Compare the changes in the Parkinson's Disease Questionnaire from baseline to post-treatment in all of the 6 participants.
Measured: 12 months after STP-001 injection
The differences of the Hoehn & Yahr (H&Y) stage before and after treatment
Compare the changes in the Hoehn \& Yahr (H\&Y) stage from baseline to post-treatment in all of the 6 participants.
Measured: 12 months after STP-001 injection
The changes of the DAT PET CT before and after treatment
Compare the changes in the DAT PET CT examination, from baseline to post-treatment in 6 participants.
Measured: 12 months after STP-001 injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2026
- Study startMay 2026 (estimated)
- Primary completionApr 2029 (estimated)
- Study completionApr 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson's Disease (PD)Studies of the same condition, open and under way.
- More trials run by Second Affiliated Hospital, Zhejiang University, School of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jiali Pu, Principal investigator, Second Affiliated Hospital, Zhejiang University, School of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07533591
- Sponsor's protocol ID
- 2026-0355
- Acronym
- CGTPD
- Status last verified
- Apr 2026
- First submitted
- 9 Apr 2026
- Last updated
- 24 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: As this study involves participants' personal health information, in strict compliance with the ethical approval terms and the Personal Information Protection Law, the original IPD will not be publicly shared. The data is currently stored on the secure server , and can be provided to eligible researchers under the condition of adhering to the data protection protocol. Researchers interested in conducting confirmatory or collaborative analyses can contact the corresponding author to submit a proposal.
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study of Chemogenetic Gene Therapy With Adeno-Associated Virus for Parkinson's Disease Using Stereotactic Surgery in the Subthalamic Nucleus
- ClinicalTrials.gov ID
- NCT07533591
- Acronym / sponsor ID
- CGTPD / 2026-0355
- Conditions
- Parkinson's Disease (PD)
- Keywords
- clozapine, Chemical Genetic Gene Therapy, Subthalamic nucleus, Parkinson's Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Second Affiliated Hospital, Zhejiang University, School of MedicineUniversity, hospital or other
- Study start
- May 2026 (estimated)
- Primary completion
- Apr 2029 (estimated)
- Study completion
- Apr 2029 (estimated)
- First posted
- 16 Apr 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a single-arm, open-label preliminary safety assessment study design. Six patients with iPD will be recruited from the Department of Neurology of the Second Affiliated Hospital of Zhejiang University School of Medicine. After signing the informed consent form, these 6 participants will be divided into 2 dose groups according to the dose escalation principle and receive the STP-001 injection solution. The virus 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. The participants will take clozapine orally every day, with doses of 1/32, 1/16 and 1/8 tablets respectively, taken in the morning and noon, and each dose will be repeated for 3 days, totaling 9 days. If there is no disease progression during this period, the participants will continue to take the 1/8 dose of the drug twice a day as the maintenance dose and undergo monitoring for 12 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| STP-001 group | Experimental | Genetic: gene therapyThis is a single-arm, open-label preliminary safety assessment study design. Six patients with iPD will be recruited from the Department of Neurology of the Second Affiliated Hospital of Zhejiang University School of Medicine. After signing the informed consent form, these 6 participants will be divided into 2 dose groups according to the dose escalation principle and receive the STP-001 injection solution. The virus 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 inject |
Who may qualify
- Ages
- 40 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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;
Key exclusion criteria (17 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| gene therapySix 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. | Genetic | STP-001 group |
Primary outcome
The incidence rates of adverse events and serious adverse events within 3 months after injecting STP-001 into the bilateral subthalamic nuclei.
Time frame: 3 months after injecting STP-001
Adverse events and serious adverse events will be evaluated through physical examinations, electrocardiogram tests and laboratory tests to assess their safety and tolerability, including routine blood tests, routine urine tests, imaging studies, etc. The main aspects include the occurrence of CRS and GvHD (≥ grade II), as well as injection-induced dyskinesia (GIDs), new-onset allogeneic tumors after injection, and death.
9 secondary outcomes
The incidence rates of adverse events and serious adverse events within 12 months after STP-001 was injected into the bilateral subthalamic nuclei.
Time frame: 12 months after STP-001
The differences of the MDS-Unified Parkinson's Disease Rating Scale before and after treatment
Time frame: 12 months after STP-001 injection
The differences of the Clinical Global Impression scale-improvement before and after treatment
Time frame: 12 months after STP-001 injection
The differences of the Patient Global Impression improvement scale before and after treatment
Time frame: 12 months after STP-001injection
The differences of the Non-Motor Symptoms Questionnaire before and after treatment
Time frame: 12 months after STP-001 injection
The differences of anti-PD drug usage before and after treatment
Time frame: 12 months after STP-001 injection
The differences of the Parkinson's Disease Questionnaire before and after treatment
Time frame: 12 months after STP-001 injection
The differences of the Hoehn & Yahr (H&Y) stage before and after treatment
Time frame: 12 months after STP-001 injection
The changes of the DAT PET CT before and after treatment
Time frame: 12 months after STP-001 injection
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2026.
- Individual participant dataSharing planned: No. As this study involves participants' personal health information, in strict compliance with the ethical approval terms and the Personal Information Protection Law, the original IPD will not be publicly shared. The data is currently stored on the secure server , and can be provided to eligible researchers under the condition of adhering to the data protection protocol. Researchers interested in conducting confirmatory or collaborative analyses can contact the corresponding author to submit a proposal.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jiali Pu principal investigator
Second Affiliated Hospital, Zhejiang University, School of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing gene therapy in people with Parkinson's Disease (PD), run by Second Affiliated Hospital, Zhejiang University, School of Medicine at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jiali Pu (+86-13989468062, Jialipu@zju.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence rates of adverse events and serious adverse events within 3 months after injecting STP-001 into the bilateral subthalamic nuclei. (time frame: 3 months after injecting STP-001). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026.