A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)

NCT ID: NCT06515002

Last Updated: 2025-11-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

36 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-11-04

Study Completion Date

2028-07-04

Brief Summary

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This study is open to adult men with cystic fibrosis and adult women with cystic fibrosis who cannot have children. People with cystic fibrosis can join if they are not eligible to receive cystic fibrosis transmembrane conductance regulator modulator therapy (CFTR-MT). The purpose of this study is to find out how well a medicine called BI 3720931 is tolerated and whether it improves lung function in people with cystic fibrosis. In this study, BI 3720931 is given to humans for the first time.

This study has two phases. In Phase 1, participants are put in one of 3 groups, one group after the other. Each group gets a different dose of BI 3720931. Group 1 starts with the lowest dose, followed by group 2 with the middle and group 3 with the high dose. In Phase 2, participants are put into 3 groups by chance, but at the same time. 2 groups get different doses of BI 3720931 selected based on results of Phase 1, and 1 group gets placebo. All study participants get only 1 dose of BI 3720931 or placebo and they use a special inhaler to take the study medicine. The placebo inhaler looks like the BI 3720931 inhaler but does not contain any medicine. During the study, participants continue taking their usual medicines.

Doctors closely monitor participants' health at the study site for the first 3 days after receiving BI 3720931. Participants visit their doctors regularly thereafter. The doctors check the health of the participants and note any health problems that could have been caused by BI 3720931. Study participants regularly do a standard lung function test to measure how well their lungs are working. Participants, in either Phase 1 or Phase 2, are in the study for 7 months. After completion of this study, participants will take part in a long-term follow-up study (1504-0003).

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

The open-label dose escalation part, Phase I, will study the safety and efficacy of single doses of three different dose levels of BI 3720931 in trial participants with cystic fibrosis (CF).

Phase II will be a double-blind, placebo-controlled, dose expansion part with 3 arms: first arm investigating Phase II dose 1, second arm investigating Phase II dose 2, and third arm for placebo.

An interim analysis of the safety and efficacy data from Phase I will be used for the decision making to move to Phase II and the selection of doses in Phase II.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
Phase I: open-label Phase II: double-blind

Study Groups

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Phase II: Dose group 1

Group Type EXPERIMENTAL

BI 3720931

Intervention Type GENETIC

BI 3720931

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Phase II: Dose group 2

Group Type EXPERIMENTAL

BI 3720931

Intervention Type GENETIC

BI 3720931

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Phase II: Placebo group

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Phase I: Dose group 1 (low dose)

Group Type EXPERIMENTAL

BI 3720931

Intervention Type GENETIC

BI 3720931

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Phase I: Dose group 2 (medium dose)

Group Type EXPERIMENTAL

BI 3720931

Intervention Type GENETIC

BI 3720931

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Phase I: Dose group 3 (high dose)

Group Type EXPERIMENTAL

BI 3720931

Intervention Type GENETIC

BI 3720931

Inhaler

Intervention Type DEVICE

Inhaler for application of BI 3720931 and placebo

Interventions

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BI 3720931

BI 3720931

Intervention Type GENETIC

Placebo

Placebo

Intervention Type DRUG

Inhaler

Inhaler for application of BI 3720931 and placebo

Intervention Type DEVICE

Eligibility Criteria

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Inclusion Criteria

* Cystic fibrosis (CF)-pulmonary phenotype and a confirmed diagnosis of CF:

* Positive sweat chloride ≥60 mmol/L by pilocarpine iontophoresis OR
* Genotype with 2 identifiable CF-causing mutations accompanied by one or more clinical features if sweat chloride testing is between 30 and 59 mmol/L
* Trial participants who are not eligible for treatment with cystic fibrosis transmembrane conductance regulator modulator therapy (CFTRmt) due to their genotype with 2 identified CFTR-mutations (including Class I CFTR gene mutations) and are also not expected to become eligible during the trial according to investigator´s opinion
* Trial participants able to perform acceptable spirometric maneuvers according to American Thoracic Society/European Respiratory Society 2019 standards
* Forced expiratory volume in 1 second, percent of predicted value (FEV1pp) ≥50% and ≤100% of predicted normal at Visit 1. Predicted value based on Global Lung Initiative lung function reference equations

Exclusion Criteria

* Trial participants not eligible for CFTRmt based on contraindications (e.g. liver failure) or who needed to withdraw CFTRmt due to intolerability are not appropriate candidates for this Phase I/II trial
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Boehringer Ingelheim

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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HOP Gui de Chauliac

Montpellier, , France

Site Status

HOP Necker

Paris, , France

Site Status

Istituto G. Gaslini

Genova, , Italy

Site Status

Osp. Pediatrico Bambin Gesù

Roma, , Italy

Site Status

Universitair Medisch Centrum Utrecht

Utrecht, , Netherlands

Site Status

Hospital Universitari Vall D Hebron

Barcelona, , Spain

Site Status

University Hospital Llandough

Cardiff, , United Kingdom

Site Status

Queen Elizabeth University Hospital

Glasgow, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

Wythenshawe Hospital

Manchester, , United Kingdom

Site Status

Countries

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France Italy Netherlands Spain United Kingdom

Related Links

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Other Identifiers

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2023-503281-23-00

Identifier Type: REGISTRY

Identifier Source: secondary_id

U1111-1291-0800

Identifier Type: REGISTRY

Identifier Source: secondary_id

1504.1

Identifier Type: OTHER

Identifier Source: secondary_id

1504-0001

Identifier Type: -

Identifier Source: org_study_id

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