Ignite Creation Date:
2025-12-25 @ 3:44 AM
Ignite Modification Date:
2026-01-09 @ 11:51 AM
Study NCT ID:
NCT06515002
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-11-24
First Post:
2024-07-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D009330', 'term': 'Nebulizers and Vaporizers'}], 'ancestors': [{'id': 'D004864', 'term': 'Equipment and Supplies'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR'], 'maskingDescription': 'Phase I: open-label Phase II: double-blind'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': '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).\n\nPhase 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.\n\nAn 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.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 36}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2024-11-04', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2028-07-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-19', 'studyFirstSubmitDate': '2024-07-18', 'studyFirstSubmitQcDate': '2024-07-18', 'lastUpdatePostDateStruct': {'date': '2025-11-24', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2024-07-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-03-14', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Phase I: Occurrence of any drug-related, treatment-emergent adverse events (AE) up to Week 24 after drug administration', 'timeFrame': 'up to 24 weeks'}, {'measure': 'Phase II: Absolute change from baseline in forced expiratory volume in 1 second, percent of predicted value (FEV1pp) at Week 8 after drug administration', 'timeFrame': 'at baseline, at week 8'}], 'secondaryOutcomes': [{'measure': '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', 'timeFrame': 'up to 12 weeks'}, {'measure': 'Phase I: Absolute change from baseline in FEV1pp at Week 24 after drug administration', 'timeFrame': 'at baseline, at week 24'}, {'measure': 'Phase I: Occurrence of any dose limiting toxicities (DLTs) up to Week 24 after drug administration', 'timeFrame': 'up to 24 weeks'}, {'measure': 'Phase II: Absolute change from baseline in FEV1pp at Week 24 after drug administration', 'timeFrame': 'at baseline, at week 24'}, {'measure': 'Phase II: Occurrence of any serious adverse events (SAEs) up to Week 24 after drug administration', 'timeFrame': 'up to 24 weeks'}, {'measure': 'Phase II: Occurrence of any drug-related, treatment-emergent AEs up to Week 24 after drug administration', 'timeFrame': 'up to 24 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Cystic Fibrosis']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://www.mystudywindow.com', 'label': 'Related Info'}]}, 'descriptionModule': {'briefSummary': "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.\n\nThis 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.\n\nDoctors 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)."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key inclusion criteria:\n\n* Cystic fibrosis (CF)-pulmonary phenotype and a confirmed diagnosis of CF:\n\n * Positive sweat chloride ≥60 mmol/L by pilocarpine iontophoresis OR\n * 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\n* 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\n* Trial participants able to perform acceptable spirometric maneuvers according to American Thoracic Society/European Respiratory Society 2019 standards\n* 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\n* 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.\n\nKey exclusion criteria:\n\n* 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\n* Trial participants requiring chronic use of systemic corticosteroids or immunosuppressants to treat another condition Further exclusion criteria apply.'}, 'identificationModule': {'nctId': 'NCT06515002', 'briefTitle': 'A Study to Test How Well BI 3720931 is Tolerated and Whether it Improves Lung Function in People With Cystic Fibrosis (Lenticlair™ 1)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Boehringer Ingelheim'}, 'officialTitle': '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)', 'orgStudyIdInfo': {'id': '1504-0001'}, 'secondaryIdInfos': [{'id': '2023-503281-23-00', 'type': 'REGISTRY', 'domain': 'CTIS'}, {'id': 'U1111-1291-0800', 'type': 'REGISTRY', 'domain': 'WHO International Clinical Trials Registry Platform (ICTRP)'}, {'id': '1504.1', 'type': 'OTHER', 'domain': 'Sponsor ID'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase I: Dose group 1 (low dose)', 'interventionNames': ['Genetic: BI 3720931', 'Device: Inhaler']}, {'type': 'EXPERIMENTAL', 'label': 'Phase I: Dose group 2 (medium dose)', 'interventionNames': ['Genetic: BI 3720931', 'Device: Inhaler']}, {'type': 'EXPERIMENTAL', 'label': 'Phase I: Dose group 3 (high dose)', 'interventionNames': ['Genetic: BI 3720931', 'Device: Inhaler']}, {'type': 'EXPERIMENTAL', 'label': 'Phase II: Dose group 1', 'interventionNames': ['Genetic: BI 3720931', 'Device: Inhaler']}, {'type': 'EXPERIMENTAL', 'label': 'Phase II: Dose group 2', 'interventionNames': ['Genetic: BI 3720931', 'Device: Inhaler']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Phase II: Placebo group', 'interventionNames': ['Drug: Placebo', 'Device: Inhaler']}], 'interventions': [{'name': 'BI 3720931', 'type': 'GENETIC', 'description': 'BI 3720931', 'armGroupLabels': ['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']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Placebo', 'armGroupLabels': ['Phase II: Placebo group']}, {'name': 'Inhaler', 'type': 'DEVICE', 'description': 'Inhaler for application of BI 3720931 and placebo', 'armGroupLabels': ['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']}]}, 'contactsLocationsModule': {'locations': [{'zip': '34295', 'city': 'Montpellier', 'country': 'France', 'facility': 'HOP Gui de Chauliac', 'geoPoint': {'lat': 43.61093, 'lon': 3.87635}}, {'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'HOP Necker', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '16147', 'city': 'Genova', 'country': 'Italy', 'facility': 'Istituto G. Gaslini', 'geoPoint': {'lat': 45.21604, 'lon': 11.87211}}, {'zip': '00165', 'city': 'Roma', 'country': 'Italy', 'facility': 'Osp. Pediatrico Bambin Gesù', 'geoPoint': {'lat': 44.99364, 'lon': 11.10642}}, {'zip': '3584 CX', 'city': 'Utrecht', 'country': 'Netherlands', 'facility': 'Universitair Medisch Centrum Utrecht', 'geoPoint': {'lat': 52.09083, 'lon': 5.12222}}, {'zip': '08035', 'city': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Universitari Vall D Hebron', 'geoPoint': {'lat': 41.38879, 'lon': 2.15899}}, {'zip': 'CF64 2XX', 'city': 'Cardiff', 'country': 'United Kingdom', 'facility': 'University Hospital Llandough', 'geoPoint': {'lat': 51.48, 'lon': -3.18}}, {'zip': 'G51 4TF', 'city': 'Glasgow', 'country': 'United Kingdom', 'facility': 'Queen Elizabeth University Hospital', 'geoPoint': {'lat': 55.86515, 'lon': -4.25763}}, {'zip': 'SW3 6JY', 'city': 'London', 'country': 'United Kingdom', 'facility': 'Royal Brompton Hospital', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}, {'zip': 'M23 9LT', 'city': 'Manchester', 'country': 'United Kingdom', 'facility': 'Wythenshawe Hospital', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}]}, 'ipdSharingStatementModule': {'url': 'https://www.mystudywindow.com/msw/datasharing', 'infoTypes': ['STUDY_PROTOCOL', 'SAP', 'CSR'], 'timeFrame': 'One year after the approval has been granted by major Regulatory Authorities and after the primary manuscript has been accepted for publication, or after termination of the development program.', 'ipdSharing': 'YES', 'description': 'Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement".\n\nFurthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website.', 'accessCriteria': "For study documents - upon signing of a 'Document Sharing Agreement'. For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement."}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Boehringer Ingelheim', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}