A clinical trial to evaluate the long-term safety and durability of efficacy of BI 3720931, an inhaled lentiviral vector gene therapy, after single dose administration in a previous clinical trial in people with cystic fibrosis rolled-over from a previous clinical trial with BI 3720931 (LenticlairTM-ON).
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 12
- 试验地点
- 6
- 主要终点
- Occurrence of treatment-emergent delayed AEs defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, until 15 years from enrolment
研究概览
简要总结
The primary objective is to investigate long-term safety in trial participants treated with at least one dose of BI 3720931.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Long term follow-up
- 盲法
- None
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •All trial participants who participated in a previous BI sponsored trial with BI 3720931 and have received at least one dose of BI 3720931, or matching placebo, are eligible for participation in extension trial 1504-0003 and should be enrolled.
- •Signed and dated written informed consent in accordance with International Council for Harmonisation (ICH)-Good Clinical Practice (GCP) and local legislation prior to admission to the trial.
排除标准
- •There are no exclusion criteria for enrolment into the trial.
结局指标
主要结局
Occurrence of treatment-emergent delayed AEs defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, until 15 years from enrolment
Occurrence of treatment-emergent delayed AEs defined as new malignancies, new serious neurologic disorders, new serious rheumatologic or autoimmune disorders, new serious hematologic disorders, or new serious potentially product-related infections, until 15 years from enrolment
次要结局
- Time to loss of efficacy defined as the drop to below 5% (absolute) above baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp)
- Time to first PEX from dosing
- Occurrence of AEs up to 2 years after enrolment
- Occurrence of serious adverse events (SAEs) throughout the study (up to 15 years)
- Occurrence of replication-competent lentivirus (RCL) throughout the study (up to 15 years)
研究者
CT Disclosure & Data Transparency
Scientific
Boehringer Ingelheim International GmbH