Multicenter Cohort Long-term Follow-up Study of Participants From Phase III Clinical Trials of Donaperminogene Seltoplasmid Injection for Critical Limb Ischemia
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 542
- 试验地点
- 5
- 主要终点
- Incidence of adverse events (AEs) and serious adverse events (SAEs)
研究概览
简要总结
The goal of this observational study is to learn about the long-term effects of Donaperminogene Seltoplasmid Injection (NL003) in participants who have been received drug NL003 or placebo at least one dose from the parent phase III clinical study to treat their critical limb ischemia (CLI). The main questions it aims to answer are:
- First, what medical problems do participants have after taking drug NL003 to treat CLI?
- Second, does drug NL003 make CLI participants live longer without serious problems (amputations or target vessel revascularizations) ?
Participants who have already received drug NL003 for CLI will complete online surveys about their health conditions. This study will continue until at least 36 months after the participant's first dose.
详细描述
This study is a multicenter, open-label, long-term follow-up cohort study. A remote follow-up system is recommended as a tool for the study, however, in-person visits or phone calls at the research center will be conducted if remote follow-up is not feasible. The study will retrospectively collect data from the last visit in the parent Phase III study and prospectively gather data following informed consent. It will continue until at least 36 months after the first dose administered to the last enrolled participant.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 20 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Participants who have previously enrolled in Phase III clinical trials of recombinant human hepatocyte growth factor plasmid injection (Donaperminogene Seltoplasmid Injection) for the treatment of critical limb ischemia (including ulcers and rest pain) and have received at least one dose of the treatment.
排除标准
- •Participants who refused to provide written informed consent;
- •Participants who refused to cooperate with the retrospective or prospective data collection.
研究组 & 干预措施
Participants exposed to placebo from Phase III Clinical Trials
干预措施: Placebo (Drug)
Participants exposed to drug NL003 from Phase III Clinical Trials
干预措施: NL003 (Drug)
结局指标
主要结局
Incidence of adverse events (AEs) and serious adverse events (SAEs)
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Incidence of benign and malignant tumors
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Amputation-free survival or target vessel revascularization or neovascularization therapies (including stem cell or gene therapy) composite endpoint
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Amputation-free survival or target vessel revascularization or neovascularization therapies (including stem cell or gene therapy) composite endpoint is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb, death due to any cause, or target vessel revascularization and neovascularization therapies (including stem cell or gene therapy) of the trial limb, whichever occurs first.
Incidence of significant vision loss, blindness, or other obvious visual abnormalities
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Incidence of major cardiovascular and cerebrovascular events
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Pregnancy status and its outcomes
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Amputation-free survival or target vessel revascularization composite endpoint
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Amputation-free survival or target vessel revascularization composite endpoint is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb, death due to any cause, or target vessel revascularization\* of the trial limb, whichever occurs first.\*Target vessel revascularization: bypass grafting, endovascular revascularization, thrombectomy, or thrombolysis of the trial limb.
Amputation-free survival of the trial limb
时间窗: Every 3±1 months after enrollment, continuing until at least 36 months after the first dose
Amputation-free survival of the trial limb is one of the key endpoints for efficacy assessment. The time from the first dose administration to the occurrence of amputation above the ankle of the trial limb or death due to any cause, whichever occurs first.
次要结局
- Rehospitalization rate for worsening ischemic symptoms of the trial limb(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Incidence of death due to acute myocardial infarction or stroke(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- All-cause mortality rate, time to death(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Major amputation rate of the trial limb (amputation plane above the ankle)(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Minor amputation rate of the trial limb (below the ankle, including toe amputation)(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Incidence of target vessel revascularization of the trial limb(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Time to amputation of the trial limb(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Level of amputation of the trial limb (above the knee, knee-ankle amputation, below the ankle amputation/toe amputation)(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Change in Rutherford classification of the trial limb compared to baseline at first dose(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Change in rest pain of the trial limb assessed by Numeric Rating Scale (NRS) compared to baseline at first dose(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)
- Change in ulceration/necrosis of the trial limb compared to baseline at first dose(Every 3±1 months after enrollment, continuing until at least 36 months after the first dose)