Cystic FibrosisPhase 1/2NCT06515002
A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
A Phase 1/2 study testing BI 3720931 and Inhaler in people with Cystic Fibrosis, run by Boehringer Ingelheim at 5 sites in 5 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- BI 3720931 and Inhaler
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 5 participants
- Where
- 5 sitesin 5 countries
- Study dates
- 4 Nov 2024to 3 Feb 2026
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 requiring chronic use of systemic corticosteroids or immunosuppressants to treat another condition Further exclusion criteria apply.
Trial locations and contacts
5 sites in 5 countries.
5 sites in 5 countries
About this study
Official title: A Seamless Phase I/II Trial With an Initial Open-label Dose Escalation Part and a Subsequent Randomised, Double-blind, Placebo-controlled Expansion Part to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of BI 3720931, an Inhaled Lentiviral Vector Gene Therapy, in Adult People With Cystic Fibrosis Who Are Ineligible for CFTR Modulators (Lenticlair 1)
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
- Being tested
- BI 3720931, Placebo, Inhaler
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BI 3720931Genetic
BI 3720931
PlaceboDrug
Placebo
InhalerDevice
Inhaler for application of BI 3720931 and placebo
| Study group | Role | What they receive |
|---|---|---|
| Phase I: Dose group 1 (low dose) | Experimental | Not described Receives: Genetic: BI 3720931; Device: Inhaler |
| Phase I: Dose group 2 (medium dose) | Experimental | Not described Receives: Genetic: BI 3720931; Device: Inhaler |
| Phase I: Dose group 3 (high dose) | Experimental | Not described Receives: Genetic: BI 3720931; Device: Inhaler |
| Phase II: Dose group 1 | Experimental | Not described Receives: Genetic: BI 3720931; Device: Inhaler |
| Phase II: Dose group 2 | Experimental | Not described Receives: Genetic: BI 3720931; Device: Inhaler |
| Phase II: Placebo group | Placebo comparator | Not described Receives: Drug: Placebo; Device: Inhaler |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration
Measured: 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
Measured: 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
Measured: up to 12 weeks
Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration
Measured: at baseline, at week 24
Phase I: Occurrence of any dose limiting toxicities (DLTs) up to Week 24 after drug administration
Measured: up to 24 weeks
Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration
Measured: at baseline, at week 24
Phase II: Occurrence of any serious adverse events (SAEs) up to Week 24 after drug administration
Measured: up to 24 weeks
Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration
Measured: up to 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jul 2024
- Study start4 Nov 2024
- Primary completion3 Feb 2026
- Study completion3 Feb 2026
Why it stopped: Sponsor decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Boehringer IngelheimThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Model description
- 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.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boehringer Ingelheim
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06515002
- Sponsor's protocol ID
- 1504-0001
- Other IDs
- 2023-503281-23-00, U1111-1291-0800, 1504.1, 2023
- Status last verified
- Apr 2026
- First submitted
- 18 Jul 2024
- Last updated
- 15 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Seamless Phase I/II Trial With an Initial Open-label Dose Escalation Part and a Subsequent Randomised, Double-blind, Placebo-controlled Expansion Part to Evaluate the Safety, Tolerability, and Efficacy of a Single Dose of BI 3720931, an Inhaled Lentiviral Vector Gene Therapy, in Adult People With Cystic Fibrosis Who Are Ineligible for CFTR Modulators (Lenticlair 1)
- ClinicalTrials.gov ID
- NCT06515002
- Acronym / sponsor ID
- 1504-0001
- Conditions
- Cystic Fibrosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Boehringer IngelheimIndustry (pharma, biotech, devices)
- Study start
- 4 Nov 2024 (actual)
- Primary completion
- 3 Feb 2026 (actual)
- Study completion
- 3 Feb 2026 (actual)
- First posted
- 23 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 6
- Model description
- 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.
| Arm | Type | Receives |
|---|---|---|
| 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Cystic fibrosis (CF)-pulmonary phenotype and a confirmed diagnosis of CF:
- 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 (2 listed)
- 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 requiring chronic use of systemic corticosteroids or immunosuppressants to treat another condition Further exclusion criteria apply.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BI 3720931BI 3720931 | Genetic | 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 |
| PlaceboPlacebo | Drug | Phase II: Placebo group |
| InhalerInhaler for application of BI 3720931 and placebo | Device | 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 |
Primary outcome
Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration
Time frame: 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
Time frame: at baseline, at week 8
6 secondary outcomes
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
Time frame: up to 12 weeks
Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration
Time frame: at baseline, at week 24
Phase I: Occurrence of any dose limiting toxicities (DLTs) up to Week 24 after drug administration
Time frame: up to 24 weeks
Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration
Time frame: at baseline, at week 24
Phase II: Occurrence of any serious adverse events (SAEs) up to Week 24 after drug administration
Time frame: up to 24 weeks
Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration
Time frame: up to 24 weeks
Results and safety
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2026.
- Related InfoLink provided by the sponsor.
- Individual participant dataSharing planned: No. Clinical studies sponsored by Boehringer Ingelheim, phases I to IV, interventional and non-interventional, are in scope for sharing of the raw clinical study data and clinical study documents. Exceptions might apply, e.g. studies in products where Boehringer Ingelheim is not the license holder; studies regarding pharmaceutical formulations and associated analytical methods, and studies pertinent to pharmacokinetics using human biomaterials; studies conducted in a single center or targeting rare diseases (in case of low number of patients and therefore limitations with anonymization). For more details refer to: https://www.clinicalstudies.boehringer-ingelheim.com/msw/datasharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing BI 3720931 and Inhaler in people with Cystic Fibrosis, run by Boehringer Ingelheim at 5 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 15 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France, Italy, Netherlands, Spain, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration (time frame: up to 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2026.