A Phase 1 interventional study of ZR001 in Parkinson's Disease (PD) and Parkinsonian Disorders, sponsored by Qianfoshan Hospital. Not yet recruiting at 1 site in China. Open to participants aged 40 Years to 70 Years. Per ClinicalTrials.gov, last updated 2026-07-22.
Sponsored by Qianfoshan Hospital · Phase 1, Interventional, and Treatment
This study aims to evaluate the safety, tolerability, and preliminary efficacy of ZR001, a novel chemogenetic gene therapy product, in patients with moderately advanced Parkinson's disease (PD). ZR001 is administered via stereotactic injection into the bilateral substantia nigra, followed by postoperative ultra-low-dose clozapine to activate the transduced direct pathway, with the goal of improving motor symptoms of Parkinson's disease.
This is a first-in-human (FIH), open-label, single-arm, single-dose, dose-escalation study. The trial plans to enroll 6-8 patients with moderately advanced PD across three dose cohorts: 0.5E12, 1.0E12, and 2.0E12 vg/participant. All participants will receive a single bilateral stereotactic injection into the substantia nigra under robot-assisted guidance. To mitigate immunogenicity, prophylactic corticosteroids will be administered (intravenous methylprednisolone followed by a 12-week oral prednisone taper). Four weeks after injection, oral clozapine will be titrated over 9 days to a maintenance dose of 3.125 mg (1/8 tablet) twice daily, continued through Week 52. The study includes a 60-day screening period, an inpatient stay from Day 1 to Day 7, and outpatient follow-up visits through Week 52 at Weeks 4, 8, 12, 26, 39, and 52. After completion, participants will be invited to participate in a 5-year long-term follow-up study.
4,573 studies on the registry are indexed under Parkinson Disease; 1,086 are open to participants now.
This study's planned enrollment of 8 is below the median of 40 across 3,353 interventional studies indexed under Parkinson Disease.
Browse Parkinson Disease studies →Qianfoshan Hospital is the lead sponsor of 144 studies on the registry; 75 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants who meet all of the following criteria are eligible for enrollment:
Exclusion Criteria:
Participants who meet any of the following criteria will be excluded from this study:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The study will enroll up to 3 cohorts: low-dose, medium-dose, and high-dose ZR001 administration.
Genetic: ZR001
This is a prospective, open-label, single-arm, single-dose, dose-escalation, investigator-initiated trial. A sentinel-based 3+ design will evaluate three dose cohorts: 0.5E12, 1.0E12, and 2.0E12 vg/participant. One sentinel participant is enrolled per cohort; a Safety Review Committee assesses safety at 6-8 weeks post-injection before escalation. After completing escalation, remaining participants are enrolled at the selected dose for expansion. Total planned enrollment is 6-8 evaluable participants.
The incidence of adverse events and serious adverse events after ZR001 treatment
Adverse events and serious adverse events will be collected from dosing through Week 52; Including vital signs, clinical laboratory parameters, and physical examinations
Time frame: 52 weeks
The differences of the daily levodopa equivalent dose (LED) after ZR001 treatment
Compare total daily LED (collected from dosing through Week 52) with baseline; LED is calculated using standard conversion factors for all dopaminergic medications
Time frame: 52 weeks
The differences of the Clinical Global Impression - Severity (CGI-S) after ZR001 treatment
Compare the change in CGI-S scores from baseline to post-treatment; CGI-S is a 7-point-scale (1 = normal to 7 = among the most extremely ill)
Time frame: 52 weeks
The differences of the Clinical Global Impression - Improvement (CGI-I) after ZR001 treatment
Compare the change in CGI-I scores from baseline to post-treatment; CGI-I is a 7-point-scale (1 = very much improved to 7 = very much worse)
Time frame: 52 weeks
The differences of the MDS Unified Parkinson's Disease Rating Scale (MDS-UPDRS) after ZR001 treatment
Change from baseline in the total UPDRS score and subscores for Parts I-IV. The UPDRS is an internationally recognized rating scale for Parkinson's disease symptoms. It comprises 4 parts assessing non-motor and motor experiences of daily living and motor complications. The total score is the sum of all 4 part scores, ranging from 0 to 183 (Part I: 0-16, Part II: 0-52, Part III: 0-56, Part IV: 0-23), with higher scores indicating greater severity of Parkinson's disease symptoms. Part I assesses non-motor experiences of daily living, including neuropsychiatric symptoms such as cognition, mood, and behavior. Part II assesses motor experiences of daily living, including activities such as writing, dressing, personal hygiene, and turning in bed. Part III assesses motor function through clinician-scored examination, including facial expression, tremor, rigidity, bradykinesia, postural instability, and gait. Part IV assesses motor complications, including dyskinesia and motor fluctuations.
Time frame: 52 weeks
The differences of the Parkinson's Disease Questionnaire 39 (PDQ-39) after ZR001 treatment
Compare the change from baseline to Week 52 in PDQ-39 score; The PDQ-39 is a 39-item questionnaire, with each item scored from 0 to 4, where 0 = never and 4 = always or unable to do at all. Item scores are summed, higher scores indicating worse quality of life.
Time frame: 52 weeks
The differences of the Non-Motor Symptom Scale (NMSS) after ZR001 treatment
Change from baseline in the NMSS total score. The NMSS is a 30-item scale covering 9 dimensions assessing non-motor symptoms in Parkinson's disease. Each item evaluates both severity and frequency of each symptom. Severity is scored from 0 to 3, where 0 = none, 1 = mild (symptom present but causes slight discomfort or distress), 2 = moderate (symptom causes some distress), and 3 = severe (symptom causes great distress). Frequency is scored from 1 to 4, where 1 = rarely (less than once a week), 2 = often (once a week), 3 = frequently (several times a week), and 4 = very frequently (daily or continuously present). For each item, the final score is calculated as severity × frequency (product). The total score is the sum of all 30 item products, ranging from 0 to 360, with higher scores indicating greater severity and higher frequency of non-motor symptoms. Units are expressed as the score on the 0-360 scale. Assessments are performed at specified post-treatment timepoints.
Time frame: 52 weeks
The differences of the Parkinson's Disease Sleep Scale (PDSS) after ZR001 treatment
Change from baseline in the PDSS total score. The PDSS is a 15-item scale assessing common sleep disturbances in Parkinson's disease. Each item is scored from 0 to 10, with the total score calculated as the sum of all 15 items, ranging from 0 to 150. Higher scores indicate worse sleep quality.
Time frame: 52 weeks
The differences of the Pittsburgh Sleep Quality Index (PSQI) after ZR001 treatment
Change from baseline to Week 52 in PSQI global score; The PSQI is a standardized instrument developed by the Sleep Research Center at the University of Pittsburgh Medical Center for assessing sleep quality. It comprises 7 components: subjective sleep quality, sleep latency, sleep duration, habitual sleep efficiency, sleep disturbances, use of sleeping medication, and daytime dysfunction. Each component is scored, and the total score is ranging from 0 to 21. Higher scores indicate worse sleep quality, with a total score \>5 generally considered indicative of poor sleep quality.
Time frame: 52 weeks
The differences of the Montreal Cognitive Assessment (MoCA) after ZR001 treatment
Change from baseline to Week 52 in MoCA total score; The MoCA is a neuropsychological assessment tool developed by Nasreddine and colleagues in 2004 at McGill University, Canada. It covers 8 cognitive domains, including attention and concentration, executive functions, memory, language, and visuospatial skills, and comprises 11 testing tasks such as the Trail Making Test (alternating), cube copying, and delayed recall of memory. The total score is ranging from 0 to 30. A score of ≥26 is generally considered as normal, with lower scores indicating cognitive impairment.
Time frame: 52 weeks
The differences of the Mini Mental State Examination (MMSE) after ZR001 treatment
Change from baseline in the MMSE total score. The MMSE is a screening tool for cognitive and intellectual function impairment in the elderly, developed by Folstein and colleagues in 1975 in the United States. The scale comprises 5 cognitive domains: orientation, registration(memory), attention and calculation, recall, and language. Each item is scored 1 point for a correct answer, and 0 points for an incorrect answer or unknown. The total score ranges from 0 to 30, with higher scores indicating better cognitive function. The cut-off for dementia is adjusted by educational level: \<17 for illiterate individuals, \<20 for primary school education, and \<24 for middle school education or above are considered indicative of dementia. Severity reference: 27-30 = normal, 21-26 = mild, 10-20 = moderate, and 0-9 = sever
Time frame: 52 weeks
The differences of the Hamilton Depression Rating Scale (HAM-D) after ZR001 treatment
Change from baseline in the HAM-D total score. The HAM-D is a clinician-rated scale developed by Hamilton in 1960 for assessing the severity of depressive symptoms and evaluating treatment response. Assessments are performed through interview and observation. The total score is calculated as the sum of all item scores, ranging from 0 to 52, with higher scores indicating greater severity of depression. Clinical interpretation: total score \< 8 = normal; 8-20 = possible depression; 20-35 = definite depression; \> 35 = severe depression. A score of ≥ 20 is generally considered to indicate moderate to severe depressive symptoms.
Time frame: 52 weeks
The differences of the Hamilton Anxiety Scale (HAM-A) after ZR001 treatment
Change from baseline in the HAM-A total score. The HAM-A can be used to evaluate the severity of anxiety symptoms in patients with anxiety and depressive disorders, as well as to assess treatment response to various pharmacological and psychological interventions. All items are rated on a 5-point scale from 0 to 4, where 0 = absent, 1 = mild, 2 = moderate, 3 = severe, and 4 = very severe. The total score is calculated as the sum of all item scores, ranging from 0 to 56, with higher scores indicating greater severity of anxiety. Clinical interpretation: total score \< 7 = no anxiety; 7-13 = possible anxiety; 14-20 = definite anxiety; 21-28 = definite marked anxiety; ≥ 29 = severe anxiety. Additionally, scores of ≥ 17, ≥ 25, and ≥ 30 are commonly used as thresholds for mild, moderate, and severe anxiety respectively.
Time frame: 52 weeks
The differences of the viral shedding after ZR001 treatment
Detection of vector DNA by qPCR in blood, saliva, urine, and feces at baseline and at Day 7, Weeks 4, 8, 12, 26, 39, and 52 post-dose
Time frame: 52 weeks
The humoral and cellular immune responses to ZR001
Change from baseline to Week 52 in humoral and cellular immune responses to ZR001
Time frame: 52 weeks
The differences of the brain MRI performance after ZR001 treatment
Change from baseline to Week 52 in MRI parameters
Time frame: 52 weeks
The differences of the electromyography (EMG) tremor analysis after ZR001 treatment
Change from baseline to Week 52 in EMG tremor analysis
Time frame: 52 weeks
The differences of the orthostatic blood pressure regulation after ZR001 treatment
Change from baseline in orthostatic blood pressure response (systolic and diastolic) measured during supine to standing test
Time frame: 52 weeks
The differences of the polysomnography (PSG) parameters after ZR001 treatment
Change from baseline in polysomnography (PSG) parameters
Time frame: 52 weeks
Plan to share: No
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