NCT05793307进行中（未招募）1 期
A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Infantile-onset Pompe Disease
GeneCradle Inc6 个研究点 分布在 1 个国家目标入组 16 人开始时间: 2023年6月2日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 16
- 试验地点
- 6
- 主要终点
- Safety and tolerability over time
研究概览
简要总结
This study is being conducted to evaluate the safety and effectiveness of GC301 adeno-associated virus vector expressing codon-optimized human acid alpha-glucosidase (GAA) as potential gene therapy for Pompe disease. Patients diagnosed with infantile-onset Pompe disease who are younger than 6 months old will be studied.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 6 Months（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age < 6 months
- •Patient has diagnosis of infantile onset Pompe disease
- •The patient's legal guardian(s) must be able to understand the purpose and risks of the study and voluntarily provide signed and dated informed consent prior to any study-related procedures being performed.
排除标准
- •Left ventricle ejection fraction (LVEF) < 40%;
- •Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
- •Patient who has received enzyme replacement therapy (ERT) more than twice;
- •Patient who has respiratory dysfunction before enrollment, including the blood oxygen (O2) saturation level < 90%, or the partial pressure of carbon dioxide (PCO2) in venous blood > 55 mmHg, or PCO2 in arterial blood > 40 mmHg;
- •Patient who has laboratory abnormalities of: creatinine > Upper Limit of Normal (ULN), hemoglobin < 90 g/L;
- •Patient with congenital organ absence;
- •Patient with a history of glucocorticoid allergy;
- •Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
- •Patient who has participated in a previous gene therapy research trial;
- •Patient who has any concurrent clinically significant major disease or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
结局指标
主要结局
Safety and tolerability over time
时间窗: 52 weeks
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
Proportion of patients treated with GC301 who are alive
时间窗: 52 weeks
次要结局
- Proportion of patients treated w/ GC301 who were alive and free of ventilator support(52 weeks)
- Changes from baseline Left Ventricular Mass (LVM) annd LVMI (LVM index)(26 and 52 weeks)
- Changes from baseline creatine kinase (CK), CK-MB, Troponin I, B-Type Natriuretic Peptide (BNP)(26 and 52 weeks)
研究者
研究点 (6)
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