A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People … (NCT06515002) | Clinical Trial Compass
TerminatedPhase 1/2
A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
Stopped: Sponsor decision
France, Italy, Netherlands5 participantsStarted 2024-11-04
Plain-language 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).
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key 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
* Stable CF disease with no pulmonary exacerbation 4 weeks prior to the screening visit and during the screening period and stable drug- and non-drug therapy for CF in the 4 weeks prior to dosing Further inclusion criteria apply.
Key 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
* Trial participants r…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration
Timeframe: up to 24 weeks
2
Phase II: Absolute change from baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp) at Week 8 after drug administration