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| ID | Type | Description | Link |
|---|---|---|---|
| 2022-502835-21-00 | Registry Identifier | CTIS | |
| U1111-1291-2567 | Registry Identifier | WHO Registry |
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This study is open to adults aged 18 years and older with cystic fibrosis bronchiectasis.
The purpose of this study is to find out whether a medicine called BI 1291583 is tolerated by people with cystic fibrosis bronchiectasis. Participants are put randomly into 2 groups. One group takes BI 1291583 tablets and the other group takes placebo tablets. Placebo tablets look like BI 1291583 tablets but do not contain any medicine. Participants in both groups take 1 tablet once a day for 12 weeks. Participants have twice the chance of being placed in the BI 1291583 group than in the placebo group.
Participants are in the study for about 6 months. During this time, they visit the study site 7 times. At the visits, the doctors check the health of the participants and note any health problems that could have been caused by BI 1291583.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 5 mg BI 1291583 | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BI 1291583 | Drug | Once daily oral administration of 5 milligram (mg) tablets of BI 1291583 for 12 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Occurrence of Any Treatment Emergent Adverse Events | Occurrence of any treatment emergent adverse events is expressed as percentages of participants with treatment emergent adverse events. Percentages are rounded to one decimal place. | From first dose of trial drug administration until last dose of trial drug administration plus 28 days of residual effect period, up to 16 weeks. |
| Measure | Description | Time Frame |
|---|---|---|
| Relative Change From Baseline in NE Activity, in Sputum, at Week 8 After First Drug Administration | Relative change from baseline in neutrophil elastase (NE) activity, in sputum, at Week 8 after first drug administration is reported. Relative change from baseline at Week 8 in neutrophil elastase was calculated as below: Relative fluorescence unit (RFU) at Week 8-Relative fluorescence unit at baseline)*100%/Relative fluorescence unit at baseline. |
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Inclusion Criteria:
Age of patients when signing the informed consent ≥18 years
Historical clinical diagnosis of Cystic fibrosis (CF) (symptoms of CF and sweat chloride ≥ 60 mmol/L and/or 2 CF-causing Cystic fibrosis transmembrane conductance regulator (CFTR) mutations)
Investigator-confirmed diagnosis of Bronchiectasis (BE) by Computed tomography (CT) scan and clinical history consistent with BE (e.g., cough, chronic sputum production, recurrent respiratory infections). Subjects whose past chest CT records are not available will undergo a chest CT scan during Screening. Historical scans must not be older than 5 years
History of pulmonary exacerbations requiring antibiotic treatment. In the 12 months before Visit 1, patients must have had either:
Patients must be able to provide spontaneous or induced sputum samples. Further inclusion criteria apply.
Exclusion Criteria:
Further exclusion criteria apply.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Jewish Health | Denver | Colorado | 80206 | United States | ||
| Central Florida Pulmonary Group |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39175217 | Derived | Badorrek P, Diefenbach C, Kogler H, Eleftheraki A, Seitz F, Hohlfeld JM. Novel cathepsin C inhibitor, BI 1291583, intended for treatment of bronchiectasis: Phase I characterization in healthy volunteers. Clin Transl Sci. 2024 Aug;17(8):e13891. doi: 10.1111/cts.13891. |
| Label | URL |
|---|---|
| Related Info | View source |
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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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All subjects were screened for eligibility prior to participation in the trial. Subjects attended a specialist site which ensured that they (the subjects) strictly met all inclusion and none of the exclusion criteria. Subjects were not to be allocated to a treatment group if any of the entry criteria were violated.
This was a randomised, double-blind, placebo-controlled, parallel group trial evaluating safety, tolerability, pharmacodynamics and pharmacokinetics of BI 1291583 one tablet once daily over 12 weeks versus placebo in adult patients with cystic fibrosis bronchiectasis (Clairafly™).
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants with cystic fibrosis bronchiectasis administered orally once daily tablets of placebo matching BI 1291583 for 12 weeks. |
| FG001 | 5 mg BI 1291583 | Participants with cystic fibrosis bronchiectasis administered orally once daily 5 milligram (mg) tablets of BI 1291583 for 12 weeks. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Nov 21, 2023 | Oct 2, 2025 |
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| Placebo to BI 1291583 | Drug | Once daily oral administration of tablets of placebo matching BI 1291583 for 12 weeks. |
|
| Before the first drug administration (baseline: mean value of screening and Week 0 prior to the first treatment intake) and Week 8 after first study drug administration. |
| Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h After the First Dose (AUC0-6) | Area under the curve of BI 1291583 in plasma over a uniform dosing interval tau=6h after the first dose (AUC0-6) is reported. | Before drug administration and 1 hour (h), 3.5h, 6h after administration of the first dose of BI 1291583 at Day 1. |
| Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h at Steady State (AUC0-6,ss) | Area under the curve of BI 1291583 in plasma over a uniform dosing interval tau=6h at steady state (AUC0-6,ss) is reported. | Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85. |
| Maximum Measured Concentration of BI 1291583 in Plasma After the First Dose | Maximum measured concentration of BI 1291583 in plasma after the first dose is reported. | Before drug administration and 1 hour (h), 3.5h, 6h, and 8h after administration of the first dose of BI 1291583 at Day 1. |
| Maximum Measured Concentration of BI 1291583 in Plasma at Steady State | Maximum measured concentration of BI 1291583 in plasma at steady state is reported. | Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85. |
| Altamonte Springs |
| Florida |
| 32701 |
| United States |
| Chest Medicine Associates | South Portland | Maine | 04106 | United States |
| Northwell Health Physician Partners | New York | New York | 10028 | United States |
| Columbia University Medical Center-New York Presbyterian Hospital | New York | New York | 10032 | United States |
| Oregon Health and Sciences University | Portland | Oregon | 97239 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Brussels - UNIV UZ Brussel | Brussels | 1090 | Belgium |
| HOP Arnaud de Villeneuve | Montpellier | 34295 | France |
| Hôpital Cochin | Paris | 75679 | France |
| Hôpital Charles Nicolle | Rouen | 76031 | France |
| Charité - Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH | Essen | 45239 | Germany |
| Medizinische Hochschule Hannover | Hanover | 30625 | Germany |
| Klinikum der Universität München AÖR | München | 80336 | Germany |
| Azienda Ospedaliera Meyer | Florence | 50139 | Italy |
| Istituto G. Gaslini | Genova | 16147 | Italy |
| IRCCS Fondazione Ospedale Maggiore | Milan | 20122 | Italy |
| AOU San Luigi Gonzaga | Orbassano (TO) | 10043 | Italy |
| Azienda Ospedaliera Universitaria Integrata Verona | Verona | 37126 | Italy |
| Amsterdam UMC, location VUMC | Amsterdam | 1081 HV | Netherlands |
| Universitair Medisch Centrum Utrecht | Utrecht | 3584 CX | Netherlands |
| Hospital Universitari Vall d'Hebron | Barcelona | 08035 | Spain |
| Hospital Universitario Ramon Y Cajal | Madrid | 28049 | Spain |
| COMPLETED | Completed planned treatment |
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| NOT COMPLETED |
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|
Treated Set (TS): included all randomised patients who were documented to have taken at least one dose of investigational treatment.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants with cystic fibrosis bronchiectasis administered orally once daily tablets of placebo matching BI 1291583 for 12 weeks. |
| BG001 | 5 mg BI 1291583 | Participants with cystic fibrosis bronchiectasis administered orally once daily 5 milligram (mg) tablets of BI 1291583 for 12 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Occurrence of Any Treatment Emergent Adverse Events | Occurrence of any treatment emergent adverse events is expressed as percentages of participants with treatment emergent adverse events. Percentages are rounded to one decimal place. | Treated Set (TS): included all randomised patients who were documented to have taken at least one dose of investigational treatment. | Posted | Number | Percentage of participants | From first dose of trial drug administration until last dose of trial drug administration plus 28 days of residual effect period, up to 16 weeks. |
|
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| |||||||||||||||||||||||||||||
| Secondary | Relative Change From Baseline in NE Activity, in Sputum, at Week 8 After First Drug Administration | Relative change from baseline in neutrophil elastase (NE) activity, in sputum, at Week 8 after first drug administration is reported. Relative change from baseline at Week 8 in neutrophil elastase was calculated as below: Relative fluorescence unit (RFU) at Week 8-Relative fluorescence unit at baseline)*100%/Relative fluorescence unit at baseline. | Treated Set (TS): included all randomised patients who were documented to have taken at least one dose of investigational treatment. Only participants with a value at baseline and at Week 8 are reported. | Posted | Mean | Standard Deviation | percent change in RFU | Before the first drug administration (baseline: mean value of screening and Week 0 prior to the first treatment intake) and Week 8 after first study drug administration. |
| ||||||||||||||||||||||||||||||
| Secondary | Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h After the First Dose (AUC0-6) | Area under the curve of BI 1291583 in plasma over a uniform dosing interval tau=6h after the first dose (AUC0-6) is reported. | Pharmacokinetic set (PKS): This patient set included all patients from the treated set (TS) who provided at least one post-dose plasma BI 1291583 concentration that was not excluded due to a protocol deviation relevant to the evaluation of pharmacokinetic (PK), or due to PK non-evaluability. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours *nanomole/Liter (h*nmol/L) | Before drug administration and 1 hour (h), 3.5h, 6h after administration of the first dose of BI 1291583 at Day 1. |
|
| |||||||||||||||||||||||||||||
| Secondary | Area Under the Curve of BI 1291583 in Plasma Over a Uniform Dosing Interval Tau=6h at Steady State (AUC0-6,ss) | Area under the curve of BI 1291583 in plasma over a uniform dosing interval tau=6h at steady state (AUC0-6,ss) is reported. | Pharmacokinetic set (PKS): This patient set included all patients from the treated set (TS) who provided at least one post-dose plasma BI 1291583 concentration that was not excluded due to a protocol deviation relevant to the evaluation of pharmacokinetic (PK), or due to PK non-evaluability. Only participants with evaluable results for this PK parameter are reported. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours *nanomole/Liter (h*nmol/L) | Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85. |
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| |||||||||||||||||||||||||||||
| Secondary | Maximum Measured Concentration of BI 1291583 in Plasma After the First Dose | Maximum measured concentration of BI 1291583 in plasma after the first dose is reported. | Pharmacokinetic set (PKS): This patient set included all patients from the treated set (TS) who provided at least one post-dose plasma BI 1291583 concentration that was not excluded due to a protocol deviation relevant to the evaluation of pharmacokinetic (PK), or due to PK non-evaluability. | Posted | Geometric Mean | Geometric Coefficient of Variation | nmol/L | Before drug administration and 1 hour (h), 3.5h, 6h, and 8h after administration of the first dose of BI 1291583 at Day 1. |
|
| |||||||||||||||||||||||||||||
| Secondary | Maximum Measured Concentration of BI 1291583 in Plasma at Steady State | Maximum measured concentration of BI 1291583 in plasma at steady state is reported. | Pharmacokinetic set (PKS): This patient set included all patients from the treated set (TS) who provided at least one post-dose plasma BI 1291583 concentration that was not excluded due to a protocol deviation relevant to the evaluation of pharmacokinetic (PK), or due to PK non-evaluability. Only participants with evaluable results for this PK parameter are reported. | Posted | Geometric Mean | Geometric Coefficient of Variation | nmol/L | Before drug administration at Day 85 and 1 hour (h), 3.5h, 6h after administration of BI 1291583 at Day 85. |
|
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All-Cause Mortality, Serious Adverse Events and other adverse events: From first dose of trial drug administration until last dose of trial drug administration plus 28 days of residual effect period, up to 16 weeks.
Treated Set (TS): included all randomised patients who were documented to have taken at least one dose of investigational treatment.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants with cystic fibrosis bronchiectasis administered orally once daily tablets of placebo matching BI 1291583 for 12 weeks. | 0 | 7 | 1 | 7 | 6 | 7 |
| EG001 | 5 mg BI 1291583 | Participants with cystic fibrosis bronchiectasis administered orally once daily 5 milligram (mg) tablets of BI 1291583 for 12 weeks. | 0 | 15 | 5 | 15 | 13 | 15 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hepatic cirrhosis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Infective exacerbation of bronchiectasis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Splenomegaly | Blood and lymphatic system disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Tachycardia | Cardiac disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Ear discomfort | Ear and labyrinth disorders | MedDRA 27.0 | Systematic Assessment |
| |
| Conjunctivitis allergic | Eye disorders | MedDRA 27.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Enteritis | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Gingival recession | Gastrointestinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Hepatic fibrosis | Hepatobiliary disorders | MedDRA 27.0 | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Pneumonia viral | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Sputum purulent | Infections and infestations | MedDRA 27.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 27.0 | Systematic Assessment |
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| Blood creatine phosphokinase increased | Investigations | MedDRA 27.0 | Systematic Assessment |
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| Forced expiratory volume decreased | Investigations | MedDRA 27.0 | Systematic Assessment |
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| Prothrombin time prolonged | Investigations | MedDRA 27.0 | Systematic Assessment |
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| Hypoalbuminaemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
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| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 27.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Hypotonia | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Sleep deficit | Nervous system disorders | MedDRA 27.0 | Systematic Assessment |
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| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 27.0 | Systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Blister | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 27.0 | Systematic Assessment |
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Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Boehringer Ingelheim, Call Center | Boehringer Ingelheim | 1-800-243-0127 | clintriage.rdg@boehringer-ingelheim.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Oct 17, 2024 | Oct 2, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D001987 | Bronchiectasis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D001982 | Bronchial Diseases |
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| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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