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A Long-term Study to Monitor the Health Status of People With Cystic Fibrosis Who Took Part in a Previous Study With BI 3720931 (Lenticlair™-ON)

Phase 1
Not yet recruiting
Conditions
Cystic Fibrosis
Registration Number
NCT06962852
Lead Sponsor
Boehringer Ingelheim
Brief 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.

Detailed Description

Not available

Recruitment & Eligibility

Status
NOT_YET_RECRUITING
Sex
All
Target Recruitment
285
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Primary Outcome Measures
NameTimeMethod
Occurrence of treatment-emergent delayed adverse events (AEs)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

Secondary Outcome Measures
NameTimeMethod
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 dosingup to 15 years
Occurrence of AEs up to 2 years after enrolmentup to 2 years
Occurrence of serious adverse events (SAEs) throughout the studyup to 15 years
Occurrence of replication-competent lentivirus (RCL) throughout the studyup to 15 years

Trial Locations

Locations (10)

Istituto G. Gaslini

🇮🇹

Genova, Italy

HOP Necker

🇫🇷

Paris, France

Osp. Pediatrico Bambin Gesù

🇮🇹

Roma, Italy

HOP Gui de Chauliac

🇫🇷

Montpellier, France

Universitair Medisch Centrum Utrecht

🇳🇱

Utrecht, Netherlands

Queen Elizabeth University Hospital

🇬🇧

Glasgow, United Kingdom

Hospital Vall d'Hebron

🇪🇸

Barcelona, Spain

University Hospital Llandough

🇬🇧

Cardiff, United Kingdom

Royal Brompton Hospital

🇬🇧

London, United Kingdom

Wythenshawe Hospital

🇬🇧

Manchester, United Kingdom

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