Parkinson Disease (PD)Early Phase 1NCT07085195
A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease
A Early Phase 1 study testing gene therapy and clozapine in people with Parkinson Disease (PD) and Gene Therapy, run by Ruijin Hospital at 1 site in China.
Trial summary
The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus… Read more about this study
- Who is it for?
- Ages 40 to 65 years
- Conditions the study is for
- Parkinson Disease (PD), Gene Therapy, Safety and Efficacy
- What is being tested?
- gene therapy and clozapine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteChina
- Study dates
- 1 Aug 2025to 30 Dec 2025
- 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 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants who meet all the following criteria can be included in this clinical study:
- Clinically diagnosed with idiopathic Parkinson's Disease [in accordance with the Diagnostic Criteria for Parkinson's Disease in China published in 2016, or in accordance with the Diagnostic Criteria for Primary PD of the International Parkinson's and Movement Disorders Society (MDS) in 2015];
- Subjects aged 40-65 years old (including the boundary value), regardless of gender;
- Medical history of disease ≥5 years;
- Hoehn-Yahr staging scale for "off" period is 2.5-4;
- Regular use of dopaminergic drugs before the screening period, including taking levodopa for at least 4 weeks;
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- MDS-UPDRS III score >35 in the "off" period, and the improvement rate of acute levodopa stress test ≥30%;
- AAV neutralizing antibody titer ≤1:2000;
- The subject agrees to postpone other neurosurgery during the main study phase (including deep brain stimulation, except for sudden life-threatening conditions requiring neurosurgery during the trial);
- No acute adverse reactions to the scheduled therapeutic dose of clozapine (1-month observation period);
- The subject agrees not to participate in other therapeutic intervention studies during the trial;
- The subject agrees not to be vaccinated during the study phase;
- Use reliable contraceptive methods from the screening period to at least 8 weeks after drug infusion;
- The subject has good compliance and can follow up regularly. During the follow-up period, the PD patient diary can be accurately completed. Family members, guardians, or caregivers can help the subject fill in the patient diary;
- The subject fully understands the nature, purpose, methods, and possible adverse reactions of this clinical trial, voluntarily participates as a subject, and signs the informed consent form; if the subject is unable to read, the legal agent or impartial witness can read the informed consent form and other written materials and witness the informed consent.
Who cannot join
- If any of the following criteria are met, the patient shall be excluded from this study:
- Atypical or secondary Parkinson's syndrome (such as Parkinson's plus syndrome, hereditary Parkinson's syndrome, drug-induced Parkinson's syndrome);
- Contraindications to surgery or previous deep brain stimulation surgery, pallidotomy, extrapyramidal surgery, other brain surgeries, or other neurosurgery that the researcher determines will affect their participation in this trial;
- Previous cranial imaging suggests abnormal brain structure, cerebrovascular malformations, intracranial tumors, intracranial hemorrhage risk, brain trauma, and other abnormalities;
- Mini-Mental State Examination (MMSE) score <24 points;
- Montreal Cognitive Assessment (MoCA) score <26 points;
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- Mini-Mental State Questionnaire (PHQ-9) ≥16;
- Abnormal liver and kidney function: Aspartate aminotransferase (AST) or Alanine aminotransferase (ALT) > 1.5 times the upper limit of normal value, serum creatinine (Cr) >1.5 times the upper limit of normal value;
- Abnormal coagulation function or using anticoagulants;
- Infectious disease screening: Hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA) positive, hepatitis C virus RNA (HCV-RNA) positive, human immunodeficiency virus (HIV) positive, or syphilis serum positive;
- Undergoing antiviral treatment for hepatitis B and hepatitis C;
- Suffering from unstable or severe other systemic diseases: including active tuberculosis, cardiovascular system, respiratory system, Diseases of the digestive system, urinary system, mental and nervous system (such as epilepsy), blood system, immune system, etc., or abnormal laboratory test values, the investigator determines that the patient is not suitable for participating in this trial;
- Suffering from malignant tumors or having a history of malignant tumors;
- Having had severe allergic reactions, allergic to contrast agents, or being unable to accept surgical anesthesia;
- Participating in other clinical trials or having participated in other clinical trials within 3 months before the screening period;
- Having received gene therapy before being enrolled into this trial;
- Having received stem cell therapy within 6 months before the screening period;
- Having used other investigational drugs within 4 weeks before screening or within 5 half-lives of the investigational drug (whichever is longer), or drugs that the investigator determines affect this trial;
- Having received live vaccines within 2 months before the screening period or having a history of vaccination within 30 days before the screening period;
- Have a history of alcohol dependence or drug addiction and cannot stop drinking as advised by the doctor during the trial;
- Female subjects who are pregnant or breastfeeding;
- Severe motor complications after anti-PD drug treatment and no suitable alternative treatment options;
- Subjects who are assessed by the researchers as unsuitable for inclusion.
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
Department of neurology, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai
Jun Liu, professor, 15221303819, lj11128@rjh.com.cn
About this study
The investigators propose a gene therapy strategy using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.
Read the detailed description
This is a single-arm and open-label study design for initial safety assessment. 6 cases of patients with iPD will be recruited from the neurology department of Ruijin Hospital, Affiliated Shanghai Jiao Tong University, School of Medicine. After the informed consent is signed, six participants will be divided into three dose groups in the dose escalation principle to receive the STP-001 injection. The virus dose escalation principle is as follows:
After the first sentinel subject receives 1×10¹² vg virus vectors, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another subject will receive a 4×10¹² vg dose. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive a 4×10¹² vg dose. 4 weeks After receiving the STP-001 injection, when the participants have recovered, clozapine ramp-up will be performed, and the participants will be treated orally with clozapine. Clozapine is 25 mg per tablet, and the oral dose is 1/32, 1/16, and 1/8 tablet twice a day, in the morning and at noon, respectively, with each dose repeated for 3 days for a total of 9 days. If no DLT develops during this time, the participants will continue to take 1/8 pill orally twice a day as a maintenance dose, and they will be monitored for 12 months.
- Conditions
- Parkinson Disease (PD), Gene Therapy, Safety and Efficacy, Clozapine
- Being tested
- gene therapy, clozapine
- 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 three dose groups in the dose escalation principle. The dose escalation principle is as follows: After the first sentinel subject receives 1×10¹² vg virus vectors, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another participant will receive a 4×10¹² vg dose as a new sentinel subject. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive a 4×10¹² vg dose.
clozapineDrug
Clozapine ramp-up will be performed four weeks after neurosurgery, when the participants have recovered, and the participants will be treated orally with clozapine. Clozapine is 25mg per tablet, and the oral dose is 1/32, 1/16, and 1/8 tablet twice a day, in the morning and at noon, respectively, with each dose repeated for 3 days for a total of 9 days. If no DLT develops during this time, the participants will continue to take 1/8 pill orally twice a day as a maintenance dose, and they will be monitored for 12 months.
| Study group | Role | What they receive |
|---|---|---|
| all of 6 patients plan to receive chemogenetic gene therapy | Experimental | This is a single-arm and open-label study design for initial safety assessment. Six participants will be divided into three dose groups in the dose escalation principle. The dose escalation principle is as follows:
After the first sentinel subject receives 1×10¹² vg virus vector, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another subject will receive 4×10¹² vg dose. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive 4×10¹² vg dose. Receives: Genetic: gene therapy; Drug: clozapine |
What is being measured
The primary outcome is the main question the study is designed to answer.
1. The incidence of adverse events and serious adverse events within 12 months after bilateral subthalamic nucleus injection of STP-001, which is the adeno-associated virus vector carrying the target gene.
Adverse events and serious adverse events will be assessed by electrocardiogram and laboratory tests for safety and tolerability assessment, including routine blood tests, routine urine tests, and blood biochemistry.
Measured: Electrocardiogram and laboratory tests will be conducted at baseline and then prior to, during, and after the neurosurgery operation and 4, 12, 24, 36, and 48 weeks after administration of the intervention.
2. The changes in the titer levels of the binding and capsid-neutralizing antibodies against adeno-associated virus in serum within 12 months after bilateral subthalamic nucleus injection of STP-001.
The changes in the titer levels of the binding and capsid-neutralizing antibodies against adeno-associated virus in serum will be assessed for safety and tolerability assessment.
Measured: The titer levels of binding and capsid-neutralizing antibodies will be conducted at baseline, at the time after the neurosurgery operation, and at 12, 24, 36, and 48 weeks after administration of the intervention.
Secondary outcomes (19)
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: The anti-PD drug usage will be conducted at baseline, at the time after the neurosurgery operation, and at 12, 24, 36, and 48 weeks.
The differences of the MDS-Unified Parkinson's Disease Rating Scale before and after treatment
Compare the changes in the MDS-Unified Parkinson's Disease Rating Scale from baseline to post- treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
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.
Measured: 12, 24, 36, and 48 weeks
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
Measured: 12, 24, 36, and 48 weeks
The differences of the Gait and Falls questionnaires before and after treatment
Compare the changes in the Gait and Falls questionnaires from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
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.
Measured: 12, 24, 36, and 48 weeks
The differences of the Scales for Outcomes in Parkinson's Disease Autonomic before and after treatment
Compare the changes in the Scales for Outcomes in Parkinson's Disease Autonomic from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the REM Sleep Behavior Disorder Screening Questionnaire before and after treatment
Compare the changes in the REM Sleep Behavior Disorder Screening Questionnaire from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Sniffin' Sticks 16-item Test and Pittsburgh Sleep Quality Index before and after treatment
Compare the changes in the Sniffin' Sticks 16-item Test and Pittsburgh Sleep Quality Index from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Montreal Cognitive Assessment and Mini-Mental State Examination before and after treatment
Compare the changes in the Montreal Cognitive Assessment and Mini-Mental State Examination from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Mini-Mental State Examination before and after treatment
Compare the changes in the Mini-Mental State Examination from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Hamilton Depression Scale before and after treatment
Compare the changes in the Hamilton Depression Scale from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Hamilton Anxiety Scale before and after treatment
Compare the changes in the Hamilton Anxiety Scale from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Parkinson's Disease Sleep Scale-2 before and after treatment
Compare the changes in the Parkinson's Disease Sleep Scale-2 from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
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, 24, 36, and 48 weeks
The differences of the Tinetti Performance Oriented Mobility Assessment before and after treatment
Compare the changes in the Tinetti Performance Oriented Mobility Assessment from baseline to post-treatment in all of the 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Berg balance scale before and after treatment
Compare the changes in the Berg balance scale from baseline to post-treatment in 6 participants.
Measured: 12, 24, 36, and 48 weeks
The differences of the Webster Rating Scale before and after treatment
Compare the changes in the Webster Rating Scale from baseline to post-treatment in 6 participants.
Measured: 12, 24, 36, and 48 weeks
The changes of the MRI before and after treatment
Compare the changes in the MRI examination, including BOLD-MRI, DTI, QSM, and NM-MRI, from baseline to post-treatment in 6 participants.
Measured: 12, 24, 36, and 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Jul 2025
- Study start1 Aug 2025 (estimated)
- Primary completion30 Dec 2025 (estimated)
- Study completion30 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Parkinson Disease (PD)Studies of the same condition, open and under way.
- More trials run by Ruijin HospitalThe 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
- Lead sponsor
- Ruijin Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07085195
- Sponsor's protocol ID
- Chemogenetics
- Status last verified
- Jul 2025
- First submitted
- 15 Jul 2025
- Last updated
- 25 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease
- ClinicalTrials.gov ID
- NCT07085195
- Acronym / sponsor ID
- Chemogenetics
- Conditions
- Parkinson Disease (PD), Gene Therapy, Safety and Efficacy, Clozapine
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 6 participants (estimated)
- Lead sponsor
- Ruijin HospitalUniversity, hospital or other
- Study start
- 1 Aug 2025 (estimated)
- Primary completion
- 30 Dec 2025 (estimated)
- Study completion
- 30 Dec 2025 (estimated)
- First posted
- 25 Jul 2025 (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 using chemical genetic inhibition to intervene in the abnormal activity of the subthalamic nucleus in Parkinson's disease. The investigators design and construct a highly efficient therapeutic injection STP-001 (first drug), through the efficient adeno-associated virus capsid (AAV), neuronal promoter (hSyn), and chemical genetic effector element (hM4Di), and accurately inject the drug into the bilateral subthalamic nucleus, the core pathological nucleus of Parkinson's disease, through stereotactic technology. Combined with a very low dose of clozapine (the second drug), the abnormal activity of the subthalamic nucleus is precisely intervened to improve the core motor symptoms of Parkinson's disease.
Detailed description
This is a single-arm and open-label study design for initial safety assessment. 6 cases of patients with iPD will be recruited from the neurology department of Ruijin Hospital, Affiliated Shanghai Jiao Tong University, School of Medicine. After the informed consent is signed, six participants will be divided into three dose groups in the dose escalation principle to receive the STP-001 injection. The virus dose escalation principle is as follows:
After the first sentinel subject receives 1×10¹² vg virus vectors, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another subject will receive a 4×10¹² vg dose. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive a 4×10¹² vg dose. 4 weeks After receiving the STP-001 injection, when the participants have recovered, clozapine ramp-up will be performed, and the participants will be treated orally with clozapine. Clozapine is 25 mg per tablet, and the oral dose is 1/32, 1/16, and 1/8 tablet twice a day, in the morning and at noon, respectively, with each dose repeated for 3 days for a total of 9 days. If no DLT develops during this time, the participants will continue to take 1/8 pill orally twice a day as a maintenance dose, and they will be monitored 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 |
|---|---|---|
| all of 6 patients plan to receive chemogenetic gene therapy | Experimental | Genetic: gene therapy, Drug: clozapineThis is a single-arm and open-label study design for initial safety assessment. Six participants will be divided into three dose groups in the dose escalation principle. The dose escalation principle is as follows: After the first sentinel subject receives 1×10¹² vg virus vector, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another subject will receive 4×10¹² vg dose. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive 4×10¹² vg dose. |
Who may qualify
- Ages
- 40 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (15 listed)
- Clinically diagnosed with idiopathic Parkinson's Disease [in accordance with the Diagnostic Criteria for Parkinson's Disease in China published in 2016, or in accordance with the Diagnostic Criteria for Primary PD of the International Parkinson's and Movement Disorders Society (MDS) in 2015];
- Subjects aged 40-65 years old (including the boundary value), regardless of gender;
- Medical history of disease ≥5 years;
- Hoehn-Yahr staging scale for "off" period is 2.5-4;
- Regular use of dopaminergic drugs before the screening period, including taking levodopa for at least 4 weeks;
Key exclusion criteria (23 listed)
- Atypical or secondary Parkinson's syndrome (such as Parkinson's plus syndrome, hereditary Parkinson's syndrome, drug-induced Parkinson's syndrome);
- Contraindications to surgery or previous deep brain stimulation surgery, pallidotomy, extrapyramidal surgery, other brain surgeries, or other neurosurgery that the researcher determines will affect their participation in this trial;
- Previous cranial imaging suggests abnormal brain structure, cerebrovascular malformations, intracranial tumors, intracranial hemorrhage risk, brain trauma, and other abnormalities;
- Mini-Mental State Examination (MMSE) score <24 points;
- Montreal Cognitive Assessment (MoCA) score <26 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 three dose groups in the dose escalation principle. The dose escalation principle is as follows: After the first sentinel subject receives 1×10¹² vg virus vectors, the research team will evaluate the safety, tolerability, and efficacy of the current drug dose after 4 weeks. If this sentinel does not experience dose-limiting toxicity, another sentinel subject will receive a 2×10¹² vg dose; if this sentinel develops dose-limiting toxicity (DLT), this dose will be defined as an intolerable dose, and the Data Review Committee will determine whether to select a lower dose for exploration or terminate dose escalation based on the existing data. If this sentinel subject doesn't experience dose-limiting toxicity, another participant will receive a 4×10¹² vg dose as a new sentinel subject. If this sentinel subject doesn't experience DLT, all of the next three subjects will receive a 4×10¹² vg dose. | Genetic | all of 6 patients plan to receive chemogenetic gene therapy |
| clozapineClozapine ramp-up will be performed four weeks after neurosurgery, when the participants have recovered, and the participants will be treated orally with clozapine. Clozapine is 25mg per tablet, and the oral dose is 1/32, 1/16, and 1/8 tablet twice a day, in the morning and at noon, respectively, with each dose repeated for 3 days for a total of 9 days. If no DLT develops during this time, the participants will continue to take 1/8 pill orally twice a day as a maintenance dose, and they will be monitored for 12 months. | Drug | all of 6 patients plan to receive chemogenetic gene therapy |
Primary outcome
1. The incidence of adverse events and serious adverse events within 12 months after bilateral subthalamic nucleus injection of STP-001, which is the adeno-associated virus vector carrying the target gene.
Time frame: Electrocardiogram and laboratory tests will be conducted at baseline and then prior to, during, and after the neurosurgery operation and 4, 12, 24, 36, and 48 weeks after administration of the intervention.
Adverse events and serious adverse events will be assessed by electrocardiogram and laboratory tests for safety and tolerability assessment, including routine blood tests, routine urine tests, and blood biochemistry.
2. The changes in the titer levels of the binding and capsid-neutralizing antibodies against adeno-associated virus in serum within 12 months after bilateral subthalamic nucleus injection of STP-001.
Time frame: The titer levels of binding and capsid-neutralizing antibodies will be conducted at baseline, at the time after the neurosurgery operation, and at 12, 24, 36, and 48 weeks after administration of the intervention.
The changes in the titer levels of the binding and capsid-neutralizing antibodies against adeno-associated virus in serum will be assessed for safety and tolerability assessment.
19 secondary outcomes
The differences of anti-PD drug usage before and after treatment
Time frame: The anti-PD drug usage will be conducted at baseline, at the time after the neurosurgery operation, and at 12, 24, 36, and 48 weeks.
The differences of the MDS-Unified Parkinson's Disease Rating Scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Patient Global Impression improvement scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Clinical Global Impression scale-improvement before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Gait and Falls questionnaires before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Non-Motor Symptoms Questionnaire before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Scales for Outcomes in Parkinson's Disease Autonomic before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the REM Sleep Behavior Disorder Screening Questionnaire before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Sniffin' Sticks 16-item Test and Pittsburgh Sleep Quality Index before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Montreal Cognitive Assessment and Mini-Mental State Examination before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Mini-Mental State Examination before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Hamilton Depression Scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Hamilton Anxiety Scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Parkinson's Disease Sleep Scale-2 before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Parkinson's Disease Questionnaire before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Tinetti Performance Oriented Mobility Assessment before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Berg balance scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The differences of the Webster Rating Scale before and after treatment
Time frame: 12, 24, 36, and 48 weeks
The changes of the MRI before and after treatment
Time frame: 12, 24, 36, and 48 weeks
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 25 Jul 2025.
- Individual participant dataSharing planned: No
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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.
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 and clozapine in people with Parkinson Disease (PD) and Gene Therapy, run by Ruijin Hospital 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 15 inclusion and 23 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 25 Jul 2025. 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 Jun Liu, professor (15221303819, lj11128@rjh.com.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
1. The incidence of adverse events and serious adverse events within 12 months after bilateral subthalamic nucleus injection of STP-001, which is the adeno-associated virus vector carrying the target gene. (time frame: Electrocardiogram and laboratory tests will be conducted at baseline and then prior to, during, and after the neurosurgery operation and 4, 12, 24, 36, and 48 weeks after administration of the intervention.). The study lists 2 primary outcomes in all. 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 25 Jul 2025; AzTrials last refreshed its copy on 8 Nov 2026.