An Exploratory Clinical Study of UX-DA001 in Subjects With Idiopathic Parkinson's Disease

An Exploratory Clinical Study to Evaluate UX-DA001 Injection (Human Midbrain Dopaminergic Progenitor Cells Injection) in Subjects With Idiopathic Parkinson's Disease

This clinical study is designed to explore the safety and tolerability of UX-DA001. It will also explore if UX-DA001 works to improve motor function in subjects with Parkinson's disease. UX-DA001 manufactured from participant's own cells will differentiate into mature dopaminergic neurons after being transplanted into the brain of the participant.

This study is an open-label, multi-center, dose-escalation and dose-expansion exploratory clinical study to evaluate the safety, tolerability, and potential efficacy of UX-DA001 Injection at different dose levels implanted in subjects with idiopathic PD.

Each subject receives only one dose of UX-DA001 for implantation into the putamen bilaterally using stereotactic neurosurgery under general anesthesia. Safety and tolerability of UX-DA001 and its effect on Parkinson's disease symptoms are assessed for 2 years post-treatment.

Inclusion Criteria:

1. The subjects or their legally acceptable representative understand and comply with the study procedures, agree to participate in the clinical trial, and sign the ICF;
2. Aged between 50-75 years old, male or female;
3. Subjects diagnosed with idiopathic PD, with a medical history of 5-20 years;
4. Having received standard anti-PD treatment and been given optimal anti-PD treatment under the guidance of the investigator, but the efficacy has significantly declined;
5. Good response to levodopa medications; the LCT shows that the maximum improvement rate of the UPDRS part III score exceeds 30%;
6. The modified H&Y scale of clinical "OFF" period is ≥ Stage 3 and ≤ Stage 4;
7. Taking a stable dosage of anti-PD medications for at least 4 weeks;
8. Good physical condition or stable concomitant diseases;
9. With reliable caregivers who can cooperate to complete the assessment items;
10. Subjects with good compliance.

Exclusion Criteria:

1. PD Subjects in whom previous genetic testing has found a GBA gene mutation or PD Subjects whom the investigator considers unsuitable for participation in this clinical study due to other gene mutations;
2. Subjects with the atypical Parkinson's syndrome or secondary Parkinson's syndrome;
3. Subjects with HIV, HBV, HCV, treponema pallidum (TP) infection, or other active infections;
4. Subjects infected with HTLV, EBV or CMV whose infection renders their blood cells unsuitable for cell product preparation;
5. Subjects with a known hereditary disorder;
6. Subjects with any history of malignancy;
7. Subjects with other serious systemic diseases or functional disorders;
8. Accompanied by other serious central nervous system diseases or serious cognitive and mental disorders;
9. Subjects who are currently receiving or have previously received cell therapy or other medicine effecting safety and efficacy assessement;
10. Subjects whose prior head CT/MRI examinations indicate brain injury, or Subjects with imaging abnormalities in the striatum and other brain regions leading to a significant increase in surgical risk, or Subjects who have previously undergone brain surgery;
11. Subjects with clinically significant abnormal results in coagulation function, or Subjects who have been using anticoagulants for a long time and cannot discontinue use;
12. Subjects with a history of severe allergy or hypersensitivity reactions, or a known history of hypersensitivity, or a history of intolerance to the investigational cellular drug or its excipients;
13. Subjects who have undergone other surgeries within the past six months that the investigator deems may affect this trial, or Subjects who cannot tolerate general anesthesia or stereotactic surgery;
14. Subjects with contraindications to MRI and PET scans;
15. Subjects with a history of alcoholism or drug abuse;
16. Women during pregnancy or lactation;
17. Subjects who have participated in other interventional clinical trials or similar clinical trials within the past 3 months;
18. Subjects with other conditions that are not suitable for participation in the clinical study as judged by the investigator.