Phase I/II Clinical Trial Evaluating the Safety, Tolerability and Preliminary Efficacy of UX-DA003 Injection in Patients With Parkinson's Disease
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 12
- 试验地点
- 1
- 主要终点
- Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
研究概览
简要总结
This clinical study is designed to explore the safety and tolerability of UX-DA003. It will also explore if UX-DA003 works to improve motor function in subjects with Parkinson's disease.
详细描述
This study is an open-label, multicenter, two-stage, Phase I/II clinical trial. Phase I study is a single-arm, open-label, dose-escalation clinical study aimed at evaluating the safety, tolerability, and preliminary efficacy of UX-DA003 transplantation therapy for PD patients across different dosage groups, as well as determining the recommended Phase II dose (RP2D). Eligible participants include PD patients who have experienced significant decline in response to standard anti-PD treatment and have not undergone DBS surgery. The study plans to establish two dosage. A total of 12 patients are planned for enrollment. Each patient will undergo a single dose of UX-DA003 for bilateral putamenal cell transplantation.
Phase II study is a single-arm, open-label, single-dose study aimed at further evaluating the efficacy and safety of UX-DA003 for the treatment of PD patients at the RP2D dose, providing evidence for Phase III confirmatory clinical studies. The enrollment population is the same as in the clinical Phase I. This phase includes only one dose group, with the dose being the recommended dose (RP2D) determined in the Phase I stage.
The trial visit schedule includes screening period (D-30~D-8), baseline period (D-7~D-1), surgery and postoperative observation period (D0~D28), and follow-up period (M3~M24).Patients who have completed the 24-month follow-up will enter the long-term follow-up period.
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
- 30 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged between 30 and 75 years, regardless of gender
- •Diagnosed with Parkinson's disease conforming to relevant clinical diagnostic standards 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 levodopa challenge test (LCT) shows that the maximum improvement rate of the UPDRS-III score exceeds 30%
- •The modified H&Y staging (Off period) of ≥2.5 and ≤4, with total daily "off" time >2 hours
- •Stable dose of anti-Parkinson' disease drugs for at least 4 weeks prior to screening
- •Good physical condition or stable concomitant diseases
排除标准
- •The subjects with atypical Parkinson's syndrome or secondary Parkinson's syndrome
- •Severe systemic diseases or functional impairments
- •Evidence of active infection or chronic infection, which may pose a risk to the subject
- •Severe concomitant central nervous system diseases or severe cognitive and psychiatric disorders
- •Subjects with a history of brain injuries, cerebral vascular malformations, hydrocephalus, brain tumors, or other brain damages as shown by previous Head CT/MRI scans, or subjects with abnormal brain imaging in the striatum or other brain regions that significantly increase surgical risks
- •Subjects who have participated in clinical trials for stem cell therapy or gene therapy for Parkinson's disease, or have participated in other interventional clinical trials within the last 3 months
- •Subjects with a history of severe allergies or hypersensitivity reactions, known hypersensitivity to the investigational drug or its ancillary materials, or intolerance to them
- •Pregnant or breastfeeding women
结局指标
主要结局
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
时间窗: Within 6 months after administration
次要结局
- Changes in 18F-FP-CIT uptake detected by positron emission tomography (PET)(Within 24 months after administration)
- Changes in MDS-UPDRS scores(Within 24 months after administration)
- Changes in modified H&Y staging(Within 24 months after administration)
- Changes in daily levodopa equivalent dose (LED)(Within 24 months after administration)
- Changes in average daily 'off' and 'on' duration while awake(Within 24 months after administration)