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)

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)

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06962852

Enrollment

Registered

2025-05-08

Start date

2025-05-20

Completion date

2040-10-29

Last updated

2026-03-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis

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.

Interventions

GENETICBI 3720931

BI 3720931

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

OTHER

Masking

NONE

Intervention model description

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).

Eligibility

Sex/Gender

ALL

Age

18 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Trial participants who participated in a previous BI sponsored trial with BI 3720931 and have received any dose of BI 3720931, or matching placebo 2. 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

Exclusion criteria

for enrolment into the trial.

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame
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

Countries

France, Italy, Netherlands, Spain, United Kingdom

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Apr 1, 2026