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)

Official Summary

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.

Study Design

• Study Type: INTERVENTIONAL
• Allocation: NA
• Model: SINGLE_GROUP
• Masking: NONE
• Enrollment: 5 participants

Interventions

• GENETIC: BI 3720931 — BI 3720931

Primary Outcomes

• Occurrence of treatment-emergent delayed adverse events (AEs) (up to 15 years)

Secondary Outcomes

• 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)
• 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)

Trial Locations

• Hôpital Gui de Chauliac, Montpellier, France
• Osp. Pediatrico Bambin Gesù, Roma, Italy
• Universitair Medisch Centrum Utrecht, Utrecht, Netherlands
• Hospital Universitari Vall d'Hebron, Barcelona, Spain
• Royal Brompton Hospital, London, United Kingdom

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