Idiopathic Parkinson's Disease
Conditions
Interventions
Low dose group:UX-DA001 injection
High dose group:UX-DA001 injection
Sponsors
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
50 Years to 75 Years
Inclusion criteria
Inclusion criteria: 1.The patients or their legal representatives 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.Patients with iPD who meet the clinical diagnostic criteria revised by the Movement Disorder Society (MDS) (2015) or the 2016 Chinese Diagnostic Criteria for Parkinson's Disease, with a disease duration 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, i.e., end-of-dose motor symptoms have worsened, or "OFF" time during waking hours has lengthened, or there are drug-induced motor complications (symptom fluctuations or aggravation of dyskinesia), which affect the quality of life; 5.Good response to levodopa medications; the LCT shows that the maximum improvement rate of the MDS-UPDRS part III score exceeds 30%; 6.The modified H&Y scale of clinical "OFF" period is>= Stage 3 and = 2.0 × 10^9/L; 2)White blood cell count >=4.0 × 10^9/L; 3)Platelet count >= 100 × 10^9/L; 4)AST and ALT = 60 mL/min/1.73 m^2; 9.With effective caregivers who can cooperate to complete the assessment tasks of the study; 10.Patients with good compliance.
Exclusion criteria
Exclusion criteria: 1.PD patients in whom previous genetic testing has found a GBA gene mutation or PD patients whom the investigator considers unsuitable for participating in this study due to other gene mutations; 2.Patients with the atypical parkinsonism or secondary parkinsonism; 3.Patients with HIV, HBV, HCV, treponema pallidum (TP) infection, or known to have other active infections (such as active tuberculosis, etc.); 4.Patients with HTLV, EBV, CMV infections that lead to blood samples unsuitable for the preparation of cell products; 5.Patients with a known hereditary disorder; 6.Patients with any history of malignancy; 7.Patients with other serious systemic diseases or functional disorders: 1)Cardiovascular and cerebrovascular diseases, such as unstable angina, congestive heart failure (NYHA Grade II or above), severe arrhythmia, myocardial infarction, stroke or a history of transient ischemic attack, etc.; 2)Chronic respiratory disease, severe chronic obstruction pulmonary disease (COPD) (FEV1% 160 mmHg and/or diastolic pressure > 100 mmHg under the treatment of antihypertensive drug); 4)Diabetes mellitus with poor blood glucose control (Hemoglobin A1c > 9.0%, or FPG = 7.0 mmol/L); 8.Accompanied by other serious central nervous system diseases or serious cognitive and mental disorders: MMSE score 24 points); cognitive and behavioral examinations indicating dementia; history of schizophrenia and bipolar affective disorder; history of epileptic seizure, etc; 9.Patients who are currently receiving or have previously received the following treatments: 1) use of corticosteroids or cytotoxic drugs or immunosuppressive therapy within 3 months before screening; 2) patients who had used antipsychotic drugs within 3 months before screening, such as antidepressant drugs and anti-manic drugs; 3) Use of botulinum toxin or other medication for dystonia or muscle spasticity within 6 months before screening; 4) Use of antiepileptic drugs at the time of screening; 5) received cell therapy within 3 months before screening 10.Patients whose previous head CT/MRI examinations indicate brain injury such as traumatic brain injury, vascular malformation, hydrocephalus, brain tumor, etc.; or patients with imaging abnormalities in the striatum and other brain regions leading to a significant increase in surgical risk; or patients who have previously undergone brain surgery; 11.Patients with clinically significant abnormal results in coagulation function (prothrombin time >= 1.5 × ULN, thrombin time >= 1.5 × ULN), or patients who have been using anticoagulants for a long time and cannot interrupt usage; 12.Patients with a history of severe allergy or hypersensitivity reactions, or a known history of hypersensitivity, or a history of intolerance to the investigational product or excipients of it; 13.Patients with a history of the following surgeries (such as implantation of cochlear implants, cardiac pacemakers, cardiac defibrillators, stereotactic nucleus lesioning surgery, or prior implantation of similar products in unilateral or bilateral brain regions, etc.), or patients who have undergone other surgeries within the past six months that the investigator considers may have impact on this study, or patients who cannot tolerate general anesthesia or stereotactic surgery; 14.Patients with contraindications to MRI and PET scans; 15.Pa
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The incidence and severity of adverse events (AEs) related to surgery and/or investigational product;The incidence and severity of AEs/serious adverse events (SAEs); | — |
Secondary
| Measure | Time frame |
|---|---|
| Changes in 18F-FP-CIT uptake;Implantation and hyperplasia of transplanted cells;Changes in MDS-UPDRS score;Changes in total daily "OFF" time during waking hours;Changes in the sum of "ON" time without dyskinesia and "ON" time with non-troublesome dyskinesia;Changes in "ON" time with troublesome dyskinesia;Changes in daily levodopa equivalent dose (LED);Changes in the scores of non-motor symptom scales;Changes in modified H&Y scale; | — |
Countries
China
Contacts
Public ContactJun Liu
Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed