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 (Lenticlair™-ON)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 5
- 试验地点
- 9
- 主要终点
- Occurrence of treatment-emergent delayed adverse events (AEs)
研究概览
简要总结
This study is for people with cystic fibrosis who took part in a previous study with a medicine called BI 3720931. The main purpose of this study is to monitor the long-term health of participants who were treated with BI 3720931 in the previous study. Participants in this study do not receive additional treatment with BI 3720931.
Participants who previously took BI 3720931 are in this study for 15 years. Participants who previously took placebo are in the study only until it is disclosed that they were in the placebo group, after which they stop. Participants who previously took BI 3720931 visit the study site about 20 times. Participants visit once every 3 months during the first 2 years.
After that, they visit once a year. During study visits, doctors check the health of the participants and note any health problems that could have been caused by BI 3720931. They also do lung function tests to see if BI 3720931 helps people with cystic fibrosis in the long term.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Other
- 盲法
- None
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
- •Trial participants who participated in a previous BI sponsored trial with BI 3720931 and have received any dose of BI 3720931, or matching placebo
- •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
- •There are no
排除标准
- •for enrolment into the trial.
研究组 & 干预措施
All trial participants
All trial participants who have received at least one dose of BI 3720931 or matching placebo in a previous trial with BI 3720931.
Trial participants who received placebo in the previous trial do not need to be followed for 15 years; once the treatment in the previous trial has been unblinded, their participation in this trial is then terminated.
干预措施: BI 3720931 (Genetic)
结局指标
主要结局
Occurrence of treatment-emergent delayed adverse events (AEs)
时间窗: up to 15 years
Treatment-emergent delayed AEs are 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 first pulmonary exacerbation (PEX) from last dosing(up to 15 years)
- Occurrence of AEs up to 2 years after enrolment(up to 2 years)
- 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)
- 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)(up to 15 years)