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)

NCT06962852 | Active Not Recruiting Phase 1 DMC

• 3 other identifiers: 1504-00032023-504909-37-00U1111-1291-2932
• Study Type: interventional
• Target: 5
• Locations: 5 countries
• Sites: 5

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Occurrence of treatment-emergent delayed adverse events (AEs)

  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

  up to 15 years

Secondary Outcomes (5)

• 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

Study Arms (1)

All trial participants

EXPERIMENTAL

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.

Genetic: BI 3720931

Interventions

BI 3720931 GENETIC

BI 3720931

All trial participants

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

Sponsors & Collaborators

• Boehringer Ingelheim: lead

Study Sites (5)

Hôpital Gui de Chauliac

Montpellier, 34295, France

Location

Osp. Pediatrico Bambin Gesù

Roma, 00165, Italy

Location

Universitair Medisch Centrum Utrecht

Utrecht, 3584 CX, Netherlands

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Royal Brompton Hospital

London, SW3 6JY, United Kingdom

Location

Related Links

• Related Info

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The trial is intended to assess 2 different objectives: long-term safety over 15 years and durability of efficacy over at least 2 years of BI 3720931 after single dose administration in a previous clinical trial in trial participants with cystic fibrosis (CF).

Study Record Dates

• First Submitted: April 30, 2025
• First Posted: May 8, 2025
• Study Start: May 20, 2025
• Primary Completion (Estimated): October 29, 2040
• Study Completion (Estimated): October 29, 2040
• Last Updated: April 14, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1); IT (1); FR (1); NL (1); GB (1)