A Single-arm, Open Label, Single-dose Clinical Study to Evaluate the Safety, Tolerability and Efficacy of BBM-F101 Injection in the Treatment of Pediatric Fabry Disease
试验速览
- 阶段
- 早期 1 期
- 状态
- 招募中
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Incidence of dose limited toxicity
研究概览
简要总结
This is a single-arm, open label, single-dose clinical study to evaluate the safety, tolerability and efficacy of BBM-F101 injection in the pediatric Fabry disease participants up to 52 weeks after infusion, and the long-term safety and efficacy of BBM-F101 injection up to 5 years after infusion.
BBM-F101 injection is an adeno-associated virus (AAV) gene therapy product for the treatment of pediatric Fabry disease.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 7 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •The participant's legal guardian fully understands the objectives, nature, methods and potential risks of the study and signs a written informed consent; If the participant is >= 8 years old, the participant must also agree to participate in the study and sign a written informed consent;
- •Decreased α-Gal A (α-galactosidase A) and confirmed diagnosis of Fabry Disease by genetic testing;
- •Males or females aged ≥7 years and <18 years old;
- •Acceptable eGFR (estimated Glomerular Filtration Rate) result in screening period;
- •Participants had at least one of the clinical manifestations for Fabry disease;
- •Acceptable capsid antibody titers;
- •Acceptable anti α-Gal A antibody titers;
- •Acceptable laboratory values;
- •Participant's legal guardian and participant with good cooperation and compliance;
- •Use of reliable contraception methods during the study for adolescence.
排除标准
- •Positive for hepatitis B surface antigen (HBsAg) or hepatitis B virus DNA (HBV-DNA), positive for hepatitis C virus RNA (HCV-RNA), positive for HIV or syphilis;
- •Have potential liver diseases;
- •Heart failure and severe arrhythmias;
- •Severe allergic reactions for enzyme replacement drugs or other medications;
- •Acute/chronic infections;
- •End-stage renal disease;
- •Have a vaccination history within 30 days prior to screening, or have a vaccination plan during the screening period and the main study period;
- •Have received gene therapy or used other investigational drugs within four weeks prior to dosing;
- •Other conditions that make the participant not eligible for the study according to the investigator.
结局指标
主要结局
Incidence of dose limited toxicity
时间窗: 12 weeks
The incidence of dose limited toxicity (DLT) events as determined by the safety review committee (SRC) within DLT observation period following the BBM-F101 injection
Incidence of adverse events and serious adverse events
时间窗: 52 weeks
The incidence of adverse events (AE) and serious adverse events (SAE) within 52 weeks following the BBM-F101 injection
次要结局
未报告次要终点