Clinical trial NCT06962852: 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)
Official title: 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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Cystic Fibrosis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2025-05-08
Enrollment: 285
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
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).
Primary purpose: OTHER

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Occurrence of treatment-emergent delayed adverse events (AEs)
timeFrame: up to 15 years
Secondary outcomes:
measure: 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)
timeFrame: up to 15 years
measure: Time to first pulmonary exacerbation (PEX) from last dosing
timeFrame: up to 15 years
measure: Occurrence of AEs up to 2 years after enrolment
timeFrame: up to 2 years
measure: Occurrence of serious adverse events (SAEs) throughout the study
timeFrame: up to 15 years
measure: Occurrence of replication-competent lentivirus (RCL) throughout the study
timeFrame: up to 15 years
Other outcomes:
Locations:
city: Montpellier
country: France
facility: HOP Gui de Chauliac
city: Paris
country: France
facility: HOP Necker
city: Genova
country: Italy
facility: Istituto G. Gaslini
city: Roma
country: Italy
facility: Osp. Pediatrico Bambin Gesù
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron
city: Cardiff
country: United Kingdom
facility: University Hospital Llandough
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
city: London
country: United Kingdom
facility: Royal Brompton Hospital
city: Manchester
country: United Kingdom
facility: Wythenshawe Hospital
Study officials:
References: