A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 8
- 试验地点
- 6
- 主要终点
- Number of incidents of new and unexpected adverse events and serious adverse events.
研究概览
简要总结
This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease. This study is intended to follow those subjects for up to 5 years since they received treatment to look for any long-term safety concerns. There is no investigational drug or therapy provided as part of this study.
详细描述
Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic metabolic disorder characterized by absent or severely reduced capacity to catabolize circulating LDL particles by the hepatic LDL receptor. As a consequence, HoFH subjects present abnormal total plasma cholesterol (LDL-C) levels, resulting in severe atherosclerosis often leading to early onset of cardiovascular disease. Early initiation of aggressive treatment for these patients is therefore essential. Unfortunately, despite existing therapies, treated LDL-C levels could remain well above acceptable levels. Thus, the functional replacement of the defective LDLR via AAV-based liver-directed gene therapy, RGX-501, may be a viable approach to treat this disease and improve response to current lipid-lowering treatments.
This is a prospective, observational study to evaluate the long-term safety and efficacy after a single administration of RGX-501. Eligible participants are those who previously have enrolled in a clinical study and received a single intravenous infusion of RGX-501.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
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
- •To be eligible to participate in this study, a participant must have previously received RGX-501 in a separate parent trial, and the participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent after the nature of the study has been explained, prior to any research-related procedures.
排除标准
- 未提供
研究组 & 干预措施
RGX-501
Study participants who have received RGX-501 gene therapy in a separate parent trial
干预措施: Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy (Drug)
结局指标
主要结局
Number of incidents of new and unexpected adverse events and serious adverse events.
时间窗: Up to 5 years after receiving treatment with RGX-501
The number of times a new and unexpected adverse event and/or serious adverse event is reported.
次要结局
- Absolute total cholesterol, LDL-C, very low density lipoprotein cholesterol (VLDL-C), high density lipoprotein cholesterol (HDL-C), calculated non-HDL-C, triglycerides (TG), and lipoprotein a (Lp(a)) over the study duration(Up to 5 years after receiving treatment with RGX-501)
- Usage of lipid-lowering therapies over time(Up to 5 years after receiving treatment with RGX-501)
- The absolute LDL-C level in mg/dL by beta quantification(Year 3 after receiving treatment with RGX-501)