Post-treatment Long-term Follow-up Study of ADVM-043 Gene Therapy in Alpha-1 Antitrypsin Deficiency
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 6
- 试验地点
- 2
- 主要终点
- Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
研究概览
简要总结
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
详细描述
ADVM-043-03 was a multi-center, observational study without administration of the ADVM-043 investigational product. Subjects who participated in the ADVANCE study were enrolled into this LTFU study for continued safety monitoring for a 2-year period.
研究设计
- 研究类型
- Observational
- 观察模型
- Other
- 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
- •The subject has A1AT deficiency and has previously received ADVM-043 gene therapy
排除标准
- •The subject is unwilling or unable to participate in all required study evaluations in the long-term follow-up protocol
- •The subject is participating in another investigational treatment protocol
- •The subject is unable to understand the purpose and risks of the study or cannot provide a signed and dated informed consent form (ICF)
结局指标
主要结局
Number of Participants With Adverse Events of Special Interest and of Serious Adverse Events Related to ADVM-043
时间窗: 2 years from start of long-term follow-up study ADVM-043-03 (NCT03804021) (2 years from completion of the parent study (ADVM-043-01, NCT02168686)
All adverse events of special interest and all serious adverse events related to ADVM-043
次要结局
未报告次要终点