An Exploratory Clinical Study to Evaluate UX-DA001 Injection (Human Midbrain Dopaminergic Progenitor Cells Injection) in Subjects With Idiopathic Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 12
- 试验地点
- 2
- 主要终点
- The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 50 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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;
- •Aged between 50-75 years old, male or female;
- •Subjects diagnosed with idiopathic PD, with a medical history of 5-20 years;
- •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;
- •Good response to levodopa medications; the LCT shows that the maximum improvement rate of the UPDRS part III score exceeds 30%;
- •The modified H&Y scale of clinical "OFF" period is ≥ Stage 3 and ≤ Stage 4;
- •Taking a stable dosage of anti-PD medications for at least 4 weeks;
- •Good physical condition or stable concomitant diseases;
- •With reliable caregivers who can cooperate to complete the assessment items;
- •Subjects with good compliance.
排除标准
- •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;
- •Subjects with the atypical Parkinson's syndrome or secondary Parkinson's syndrome;
- •Subjects with HIV, HBV, HCV, treponema pallidum (TP) infection, or other active infections;
- •Subjects infected with HTLV, EBV or CMV whose infection renders their blood cells unsuitable for cell product preparation;
- •Subjects with a known hereditary disorder;
- •Subjects with any history of malignancy;
- •Subjects with other serious systemic diseases or functional disorders;
- •Accompanied by other serious central nervous system diseases or serious cognitive and mental disorders;
- •Subjects who are currently receiving or have previously received cell therapy or other medicine effecting safety and efficacy assessement;
- •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;
- •Subjects with clinically significant abnormal results in coagulation function, or Subjects who have been using anticoagulants for a long time and cannot discontinue use;
- •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;
- •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;
- •Subjects with contraindications to MRI and PET scans;
- •Subjects with a history of alcoholism or drug abuse;
- •Women during pregnancy or lactation;
- •Subjects who have participated in other interventional clinical trials or similar clinical trials within the past 3 months;
- •Subjects with other conditions that are not suitable for participation in the clinical study as judged by the investigator.
研究组 & 干预措施
UX-DA001
干预措施: UX-DA001 (Biological)
结局指标
主要结局
The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product
时间窗: within 4 weeks post surgery
The incidence and and severity of adverse events (AEs) associated with surgery and/or investigational product during the surgical treatment period and the 4-week postoperative observation period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
The incidence and severity of AEs/serious adverse events (SAEs)
时间窗: From baseline to 2 years post surgery
The incidence and severity of AEs/serious adverse events (SAEs) during the study, including AEs and SAEs during the surgery treatment period, postoperative observation period, and follow-up period. Number of participants with treatment-related adverse events as assessed by CTCAE v5.0.
次要结局
- 18F-FP-CIT uptake using positron emission tomography (PET)(From baseline to 2 years post surgery)
- The situation of implantation and overgrowth of transplanted cells using cranial MRI(From baseline to 2 years post surgery)
- Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part III, from baseline.(From baseline to 2 years post surgery)
- Changes in Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) score, part II, from baseline.(From baseline to 2 years post surgery)
- Changes in modified modified Hoehn-Yahr (H&Y) scale from baseline(From baseline to 2 years post surgery)
- Changes in daily levodopa equivalent dose (LED) from baseline.(From baseline to 2 years post surgery)
- Changes in the scores of non-motor symptom scales (NMSS) from baseline.(From baseline to 2 years post surgery)