NCT06391736招募中1 期
A Multi-centered, Single Arm, Open Labeled, Study to Evaluate the Safety, Tolerability, and Efficacy of an Adeno-associated Virus Vector Expressing the Human Acid Alpha-glucosidase (GAA) Transgene Intravenous Injection in Patients With Late-onset Pompe Disease
GeneCradle Inc2 个研究点 分布在 1 个国家目标入组 33 人开始时间: 2024年4月19日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 33
- 试验地点
- 2
- 主要终点
- Percent Predicted Upright Forced Vital Capacity (FVC)(Phase 2)
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 late-onset Pompe disease （LOPD） who are ≥ 6 years 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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 6 years, males or females;
- •Patient has a diagnosis of LOPD;
- •Patient has upright FVC ≥ 30% of predicted normal value;
- •A 6MWT ≥ 40 meters, assistive device allowed;
- •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
- •Patient who has any history or concurrent clinical organic disease, including cardiovascular and liver diseases, respiratory system, nervous system disease, or any other condition that, in the opinion of the investigator, makes the subject unsuitable for participation in the study.
- •Patient who requires invasive mechanical ventilation, or rely on noninvasive non-non-invasive assisted ventilation when sitting upright;
- •Patient who is positive for human immunodeficiency (HIV) antibody, hepatitis B surface antigen, hepatitis C antibody, or treponema pallidum antibody;
- •Patient with a history of glucocorticoid allergy;
- •Patient who has a contraindication to study drug or to corticosteroids, or has demonstrated hypersensitivity to any of the components of the study drug;
- •Patient who has AAV9 neutralizing antibody titer ≥ 1:100；
- •Patient who has participated in a previous gene therapy research trial;
- •Pregnant or lactating female participants;
- •Patients who have fertility plans within 6 months from screening to the end of the study and are unwilling to take effective physical contraceptive measures (such as a condom, intrauterine device, contraceptive ring, ligation, abstinence, etc.) for contraception (including the subject's partner);
结局指标
主要结局
Percent Predicted Upright Forced Vital Capacity (FVC)(Phase 2)
时间窗: 52 weeks
Change from baseline in percentage of predicted FVC measured by pulmonary function testing
Number of Participants With Adverse Events
时间窗: 52 weeks
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
Dose-limiting toxicity (DLT) rate based on protocol-specific adverse events (Phase 1)
时间窗: within 30 days after treatment
次要结局
- Maximum Expiratory Pressure (MEP)(52 weeks)
- Muscle Status Testing - Quick Motor Function Test (QMFT) Measure(52 weeks)
- Quality of life evaluation: 12-item short form health survey (SF-12) for LOPD participants(52 weeks)
- The viral load of adeno-associated virus (AAV) vector(52 weeks)
- 6-Minute Walk Test(52 weeks)
- Maximum Inspiratory Pressure (MIP)(52 weeks)
- Occurrence of immune response against AAV capsid annd GAA transgene(52 weeks)
- Time needed for non-invasive ventilatory support(52 weeks)
研究者
研究点 (2)
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