NCT05567627Unknown不适用
Single Arm, Multicenter, Open and Dose-escalation Clinical Study on Safety, Tolerance, and Efficacy of GC301, an AAV-Delivered Gene Transfer Therapy in Patients With Infantile-onset Pompe Disease
Seventh Medical Center of PLA General Hospital1 个研究点 分布在 1 个国家目标入组 6 人开始时间: 2022年8月1日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 发起方
- 入组人数
- 6
- 试验地点
- 1
- 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
- Single Group
- 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
- •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;
- •The patient must be no older than 6 months;
- •The patient must be diagnosed with infantile-onset Pompe disease.
排除标准
- •Class IV patient based on Modified Ross Heart Failure Classification for Children;
- •Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 3x upper limit of normal (ULN), alkaline phosphatase (ALP) > 2x ULN (with the exception of liver abnormalities related to Pompe disease);
- •Patient has severe organ dysfunction, such as liver and kidney failure (Liver failure: patients may have liver failure syndrome, including fatigue, severe gastrointestinal symptoms; clinical examination found prolonged prothrombin time, prothrombin activity less than 40%; Neuropsychiatric symptoms, such as restlessness, changes in personality and behavior, lethargy, coma, etc.; Toxic tympanic bowel, ascites, multiple organ dysfunction, etc.; hyperalbuminemia exceeding 171 μmol/L, hypoalbuminemia. Renal failure: creatinine exceeding 110 μmol/L, or glomerular filtration rate less than 100 mL/min), congenital/acquired encephalopathy, etc.;
- •Patient with congenital organ absence;
- •Patient with primary immunodeficiency;
- •Patient who is positive for human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
- •Patient with a history of glucocorticoid allergy；
- •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: Infusion to the end of study, average 1 year
Frequency of adverse events (AEs), serious adverse events (SAEs), and changes from baseline in relevant clinical laboratory tests
次要结局
- Changes from baseline creatine kinase (CK)(26 and 52 weeks)
- Changes from baseline Left Ventricular Mass (LVM)(26 and 52 weeks)
- Proportion of patients treated w/ GC301 who were alive and free of ventilator support at 12 months of age;(52 weeks)
研究者
研究点 (1)
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