Phase 1/2, Open Label & Double Blind Randomized Placebo-controlled Study to Assess the Feasibility of BGC101 (EnEPC) in the Treatment of Peripheral Arterial Disease (PAD) With Critical Limb Ischemia (CLI)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 40
- 试验地点
- 5
- 主要终点
- Safety (Incidence of adverse events)
研究概览
简要总结
Evaluate the feasibility of an autologous cell preparation composed of a mixture of cells enriched for endothelial progenitor cells (EnEPCs) and multipotent adult hematopoietic stem/progenitor cells (HSPC) (BGC101), in the treatment of patients suffering from peripheral arterial disease (PAD) with critical limb ischemia (CLI) who have not responded to optimal pharmacological treatment or control of risk factors and/or had a revascularization failure, and do not have the option of further revascularization treatment.
详细描述
BGC101 is designed to treat peripheral vascular disease in patients suffering from Critical Leg Ischemia (CLI) also referred to as chronic limb threatening ischemia (CLTI).
This part of the study is designed as a placebo double-blind randomized controlled trial (CRT) assessing the safety and efficacy of BGC101 in 45 eligible subjects in 2 Arms: Arm A: BGC101 treatment and Arm B: Placebo treatment. The Arm A:Arm B ratio is 2:1 A single dose treatment of the personalized cells by intramuscular injections into the affected leg takes less than 10 minutes.
Cells from a standard blood draw (with no pre-treatment, bone marrow aspiration, mobilization or apheresis) are transformed, within a day, into the investigational medicinal product BGC101.
BGC101, intended for autologous use, is a 'ready-to-use' cell suspension in prefilled syringes.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Double (Participant, Outcomes Assessor)
盲法说明
the study will be double-blind for treatment allocation. Both BGC101 and placebo are supplied fresh in 5 mL ready-to use-syringes. Both BGC101 and placebo will be prepared by an unblinded trained personnel (external to the study staff members) who will not take part in the treatment and / or assessment of the patient. The patient will also be blinded and the investigator will delegate the unblinded and blinded staff in order to perform study procedures in a double-blind manner
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Placebo
Intramuscular injection of control medium only
干预措施: Control medium (Biological)
BGC101
Intramuscular injection of BGC101 (autologous EnEPC preparation)
干预措施: BGC101 (autologous EnEPC preparation) (Biological)
结局指标
主要结局
Safety (Incidence of adverse events)
时间窗: 12 Months
* Incidence and proportion of incidence between treatment arms of adverse events of specific interest (AESI) and injection-related AE * Incidence of serious adverse events (SAEs) including SAEs related or probably related to the treatment * Vital signs, physical examination, and electrocardiogram (ECG) * Safety laboratory values of hematology, blood chemistry, and urinalysis * Local tolerability (injection site reaction)
Efficacy (Improvement of indication signs)
时间窗: 12 Months
* Major amputation (below or above the knee) rate at Month 12 * Major amputation-free survival (AFS) rate at Month 12
次要结局
未报告次要终点