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

Launched by SHANGHAI UNIXELL BIOTECHNOLOGY CO., LTD · Jan 11, 2025

Keywords

ClinConnect Summary

This study is an open-label, single-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.

Gender

ALL

Eligibility criteria

• 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 5-15 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 with HTLV, EBV, CMV infections that lead to blood samples unsuitable for the preparation of cell products;
• • 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.