A long-term study to monitor the health status of people with cystic fibrosis who took part in a previous study with BI 3720931 (LenticlairTM-ON)

Phase 1

Conditions: Cystic fibrosis
MedDRA version: 20.0Level: PTClassification code: 10011763Term: Cystic fibrosis lung Class: 100000004850
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: CTIS2023-504909-37-00

Lead Sponsor: Boehringer Ingelheim International GmbH

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 27

Inclusion Criteria

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.

Exclusion Criteria

There are no exclusion criteria for enrolment into the trial.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective is to investigate long-term safety in trial participants treated with at least one dose of BI 3720931.;Secondary Objective: The secondary objective of the trial is to investigate the duration of clinical efficacy in trial participants after initial dosing of BI 3720931 over at least 2 years.;Primary end point(s): 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

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):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);Secondary end point(s):Time to first PEX from dosing;Secondary end point(s):Occurrence of AEs up to 2 years after enrolment;Secondary end point(s):Occurrence of serious adverse events (SAEs) throughout the study (up to 15 years);Secondary end point(s):Occurrence of replication-competent lentivirus (RCL) throughout the study (up to 15 years)