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

Shanghai UniXell Biotechnology Co., Ltd

Status and phase: Invitation-only; Phase 1

Conditions: Parkinson Disease, Idiopathic

Treatments: Biological: UX-DA001

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT06778265

UX-DA001-101

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 12 estimated patients

Sex: All

Ages: 50 to 75 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

UX-DA001

Experimental group

Treatment:

Biological: UX-DA001