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

NCT06515002 | Terminated Phase 1 DMC

• 4 other identifiers: 1504-00012023-503281-23-00U1111-1291-08001504.1
• Study Type: interventional
• Target: 5
• Locations: 5 countries
• Sites: 5

Brief Summary

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).

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (2)

• Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration

  up to 24 weeks

• Phase II: Absolute change from baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp) at Week 8 after drug administration

  at baseline, at week 8

Secondary Outcomes (6)

• Phase I: Occurrence of treatment response defined as change from baseline ≥5% in FEV1pp, comparing the mean of 3 pre-treatment FEV1pp measured in the screening period with the mean of 3 post-treatment FEV1pp-values at Weeks 4, 6, and 8

  up to 12 weeks

• Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration

  at baseline, at week 24

• Phase I: Occurrence of any dose limiting toxicities (DLTs) up to Week 24 after drug administration

  up to 24 weeks

• Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration

  at baseline, at week 24

• Phase II: Occurrence of any serious adverse events (SAEs) up to Week 24 after drug administration

  up to 24 weeks

Study Arms (6)

Phase I: Dose group 1 (low dose)

EXPERIMENTAL

Genetic: BI 3720931; Device: Inhaler

Phase I: Dose group 2 (medium dose)

EXPERIMENTAL

Genetic: BI 3720931; Device: Inhaler

Phase I: Dose group 3 (high dose)

EXPERIMENTAL

Genetic: BI 3720931; Device: Inhaler

Phase II: Dose group 1

EXPERIMENTAL

Genetic: BI 3720931; Device: Inhaler

Phase II: Dose group 2

EXPERIMENTAL

Genetic: BI 3720931; Device: Inhaler

Phase II: Placebo group

PLACEBO COMPARATOR

Drug: Placebo; Device: Inhaler

Interventions

BI 3720931 GENETIC

BI 3720931

Phase I: Dose group 1 (low dose); Phase I: Dose group 2 (medium dose); Phase I: Dose group 3 (high dose); Phase II: Dose group 1; Phase II: Dose group 2

Placebo DRUG

Placebo

Phase II: Placebo group

Inhaler DEVICE

Inhaler for application of BI 3720931 and placebo

Phase I: Dose group 1 (low dose); Phase I: Dose group 2 (medium dose); Phase I: Dose group 3 (high dose); Phase II: Dose group 1; Phase II: Dose group 2; Phase II: Placebo group

Eligibility Criteria

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

You may qualify if:

• 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

You may not qualify if:

• 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

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

Interventions

Nebulizers and Vaporizers

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

Intervention Hierarchy (Ancestors)

Equipment and Supplies

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Phase I: open-label Phase II: double-blind
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 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.

Study Record Dates

• First Submitted: July 18, 2024
• First Posted: July 23, 2024
• Study Start: November 4, 2024
• Primary Completion: February 3, 2026
• Study Completion: February 3, 2026
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

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