ZR001 Chemogenetics Gene Therapy Study in Patients With Parkinson's Disease (NCT07718659) | Clinical Trial Compass
Not Yet RecruitingPhase 1
ZR001 Chemogenetics Gene Therapy Study in Patients With Parkinson's Disease
China8 participantsStarted 2026-12-01
Plain-language summary
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.
Who can participate
Age range
40 Years – 70 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥40 and ≤70 years (at the time of signing the informed consent), any gender.
. Diagnosed with Parkinson's disease according to the Diagnostic Criteria for Parkinson's Disease in China (2016 edition).
. Modified Hoehn \& Yahr stage between 2.5 and 4.
. History of Parkinson's disease for at least 5 years but less than 15 years.
. Moderate to severe MDS-UPDRS Part III score in the off period, and improvement rate ≥30% after acute levodopa stress test.
. Clinically stable symptoms and stable types and doses of anti-Parkinsonian medications within 3 months prior to enrollment.
. Agree to provide biological samples required for the study (e.g., blood, urine).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Since this is a Phase 1 trial focused specifically on measuring safety and side effects of ZR001, what do we currently know about the risks of this type of chemogenetics gene therapy, and how does that uncertainty compare to my current treatment options?
2This trial involves gene therapy delivered directly to treat Parkinson's — can you help me understand what 'chemogenetics' means in this context, what the procedure would actually involve, and whether that's something my overall health situation could handle?
3The trial is listed as 'not yet recruiting' — do you know when it might open, where it would be located, and whether it's worth waiting for versus pursuing other treatments now?
4Because this is a Phase 1 study primarily designed to detect adverse events rather than to prove the therapy works, what would be the realistic goal for me personally if I were to participate — is it potential benefit, contributing to research, or both?
5Are there standard Parkinson's treatments or other clinical trials already recruiting that you think I should try first before considering an early-phase gene therapy study like this one?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The incidence of adverse events and serious adverse events after ZR001 treatment
. Consent to hospitalization for intraparenchymal drug injection surgery.
Exclusion criteria
. Have participated in or are currently participating in other clinical studies of PD drugs or other AAV gene therapy or cell therapy.
. Presence of other severe psychiatric disorders (e.g., severe depression, schizophrenia, etc.).
. Previous adverse reactions to clozapine, such as agranulocytosis or severe neutropenia.
. Participants with known allergic constitution, including allergy or hypersensitivity to clozapine, prednisone acetate, other glucocorticoids, their excipients, or local anesthetics.
. History of alcohol or drug abuse within the past 2 years.
. Participants requiring invasive or non-invasive ventilatory support.
. Serum AAV binding antibody titer \>1:2000.
. Presence of clinically significant laboratory abnormalities as assessed by the investigator: alanine aminotransferase (ALT), aspartate aminotransferase (AST), gamma-glutamyl transferase (GGT), total bilirubin; creatinine, hemoglobin (Hb), prothrombin time (PT), activated partial thromboplastin time (APTT), fasting blood glucose, platelets (PLT).