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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-000261-21 | EudraCT Number |
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Not due to safety reasons
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The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic fibrosis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Placebo | Placebo Comparator | 2 puffs ofmatching placebowere inhaledorally via theRespimat®inhaler twice dailyfor a treatmentperiod of 4 weeksin patients withcystic fibrosis. |
|
| BI 1265162 50 μg b.i.d. | Experimental | 2 puffs of 25micrograms (μg)BI 1265162(Total: 50μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:100μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis. |
|
| BI 1265162 100 μg b.i.d. | Experimental | 2 puffs of 50micrograms (μg)BI 1265162(Total: 100μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:200μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis. |
|
| BI 1265162 200 μg b.i.d. | Experimental | 2 puffs of 100micrograms (μg)BI 1265162(Total: 200μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:400μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis. |
|
| BI 1265162 20 μg b.i.d. | Experimental | 2 puffs of 10micrograms (μg)BI 1265162(Total: 20μg)were inhaledorally via theRespimat®inhaler twice daily(b.i.d., daily dose:40μg) for atreatment periodof 4 weeks inpatients withcystic fibrosis. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BI 1265162 | Drug | Inhalation solution |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Percent Predicted Trough Forced Expiratory Volume in 1 Second (FEV1) After 4 Weeks of Treatment | Trough FEV1 was measured within 30 minutes prior to dosing of study medication. | At 30 minutes prior to dosing in Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Lung Clearance Index (LCI) Assessed by N2 Multiple Breath Washout (N2MBW) Procedure After 4 Weeks of Treatment | Change from baseline in Lung Clearance Index (LCI) assessed by N2 Multiple Breath Washout (N2MBW) procedure after 4 weeks of treatment was reported. LCI was calculated as the ratio of cumulative expired volume (CEV) to functional residual capacity (FRC), which was LCI = CEV (milliliter/kilogram) / FRC (milliliter/kilogram) and hence, LCI was "Unitless". The change from baseline after 4 weeks of treatment in LCI was then calculated as the LCI value measured after 4 weeks of treatment at Day 29 minus the LCI value measured at baseline on Day 1. |
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Inclusion Criteria:
Male or female patients, 12 years of age or older at screening;
Documented diagnosis of cystic fibrosis including:
Patients able to perform acceptable spirometric manoeuvres according to American Thoracic Society (ATS) standards;
FEV1 ≥ 40% and ≤ 90% of predicted values at screening and predose at Visit 2;
Women of childbearing potential (WOCBP) must be willing and able to use highly effective methods of birth control per ICH M3 (R2) that result in a failure rate of less than1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the patient (or patient's legal guardian) information;
Signed and dated written informed consent and assent in accordance with ICH Harmonized Guideline for Good Clinical Practice (GCP) and local legislation prior to admission in the trial.
Exclusion Criteria:
Evidence of acute upper or lower respiratory tract infection within 4 weeks prior to randomization based on investigator's judgement;
Pulmonary exacerbation requiring use of i.v./oral/inhaled antibiotics or oral corticosteroids within 4 weeks prior to randomisation;
Patients with history of Acute Tubular Necrosis (ATN);
Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated basal cell carcinoma of the skin or in situ carcinoma of uterine cervix;
Patients unable to inhale trial drug in an appropriate manner from the Respimat® inhaler based on investigator's judgement;
Patients who have started a new chronic medication for CF within 4 weeks of randomisation;
Patients who have previously received a lung transplant or patients who are currently on a waiting list to receive a lung transplant;
Patients with a significant history of allergy/hypersensitivity (including medication allergy) which is deemed relevant to the trial as judged by the investigator or with a known hypersensitivity to trial drug or its components. "Significance" in this context refers to any increased risk of hypersensitivity reaction to trial medication;
Any clinically significant laboratory abnormalities at screening as judged by the investigator, or any of the following:
Clinically significant disease or medical condition other than CF or CF-related conditions that, in the opinion of the investigator, would compromise the safety of the patient or the data quality. This includes significant haematological, hepatic, renal, cardiovascular and neurologic disease. Patients with diabetes may participate if their disease is under good control prior to screening;
Patients not expected to comply with the protocol requirements or not expected to complete the trial as scheduled;
Previous randomisation in this trial;
Currently enrolled in another investigational device or drug trial, or less than 30 days or six half-lives (whichever is greater) since ending another investigational device or drug trial(s), or receiving other investigational treatment(s);
Chronic alcohol or drug abuse or any condition that, in the investigator's opinion, makes them an unreliable trial patient or unlikely to complete the trial;
Women who are pregnant, nursing, or who plan to become pregnant while in the trial.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nemours Children's Hospital | Orlando | Florida | 32827 | United States | ||
| Riley Hospital for Children at Indiana University Health |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34385272 | Derived | Goss CH, Fajac I, Jain R, Seibold W, Gupta A, Hsu MC, Sutharsan S, Davies JC, Mall MA. Efficacy and safety of inhaled ENaC inhibitor BI 1265162 in patients with cystic fibrosis: BALANCE-CF 1, a randomised, phase II study. Eur Respir J. 2022 Feb 17;59(2):2100746. doi: 10.1183/13993003.00746-2021. Print 2022 Feb. | |
| 33313307 | Derived |
| Label | URL |
|---|---|
| Related Info | View source |
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After the study is completed and the primary manuscript is accepted for publishing, researchers can use this following link https:// trials.boehringer-ingelheim.com/trial_results/ clinical_submission_documents.html to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Also, Researchers can use the following link http://trials.boehringeringelheim. com/ to find information in order to 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. The data shared are the raw clinical study data sets.
After all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication.
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 both the independent review panel and the sponsor, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a 'Data Sharing Agreement'.
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 trial aimed to assess the efficacy, safety, and pharmacokinetics of different dose regimens of BI 1265162 taken twice daily by the Respimat® inhaler versus placebo in adult and adolescent patients with cystic fibrosis for a 4-week treatment period. Study was terminated without recruiting any adolescent patients.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | 2 puffs of matching placebo were inhaled orally via the Respimat® inhaler twice daily for a treatment period of 4 weeks in patients with cystic fibrosis. |
| FG001 | BI 1265162 20μg b.i.d. |
| 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 18, 2019 | Mar 25, 2021 |
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|
| Placebo | Drug | Inhalation solution |
|
| At pre-dose in Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
| Change From Baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) Total Score After 4 Weeks of Treatment | The adult/adolescent format of the CFQ-R consists of 50 questions (qts) dividing into 12 domains: Physical functioning(8 qts), role limitations(4 qts), vitality(4 qts), emotional functioning(5 qts), social functioning(6 qts), body image(3 qts), eating disturbance(3 qts), treatment burden(3 qts), health perceptions(3 qts), weight(1 qts), respiratory symptoms(7 qts), and digestive system(3 qts). The score of some qts is first reversed if reversed coded, so that the score for each of the 50 qts ranges from 1 to 4 points (less symptoms). Then, a domain score for a domain with N qts is calculated as (sum of the scores of the N qts - N)/(N ✕ 4 - N) ✕ 100. Each domain score ranges from 0 to 100 (better health). The CFQ-R total score is summing up the domain scores and ranges from 0 to 1200 (better quality of life). The change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) total score after 4 weeks of treatment was reported. | At Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
| Change From Baseline in Cough and Sputum Assessment Questionnaire (CASA-Q) (4 Separate Sub-scores) After 4 Weeks of Treatment | The 20-item Sputum Assessment Questionnaire (CASA-Q) consisted of 4 domains: Cough Symptoms Domain (3 items), Cough Impact Domain (8 items), Sputum Symptoms Domain (3 items), and Sputum Impact Domain (6 items). Score of each item has been reversed such that better responses have higher score, which ranges from 1 (worse) to 5 (better health). For each domain, the domain score was calculated by summing up the scores of the respective items and scaling to a value ranging from 0 to 100, with higher score associated with fewer symptoms/less impact due to cough or sputum. The 4 domain scores (sub-scores) were reported. | At Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
| Percentage of Patients With Treatment-emergent Adverse Events (AE) up to Day 36 | Percentage of patients with any treatment-emergent Adverse Events (AE) up to day 36 was reported. | From Day 1 (baseline) until end of 4 weeks of treatment period (Day 29) plus 7 days of follow-up, up to 36 days. |
| Concentration of BI 1265162 in Plasma at 0.083 Hour at Steady State Following Dose 15 (C0.083,ss,15) | Concentration of BI 1265162 in plasma at 0.083 hour at steady state following dose 15 (C0.083,ss,15) was reported. | At 5 minutes (around 0.083 hours) post dosing at steady state on Day 8 for dose 15 (morning dose on Day 8). |
| Concentration of BI 1265162 in Plasma at 0.083 Hour at Steady State Following Dose 57 (C0.083,ss,57) | Concentration of BI 1265162 in plasma at 0.083 hour at steady state following dose 57 (C0.083,ss,57) was reported. | At 5 minutes (around 0.083 hours) post dosing at steady state on Day 29 for dose 57 (morning dose on Day 29). |
| Pre-dose Concentration Measured of BI 1265162 in Plasma at Steady State After Dose 15 (Cpre,ss, 15) | Pre-dose concentration measured of BI 1265162 in plasma at steady state after dose 15 (Cpre,ss, 15) was reported. | At pre-dose (taken within 60 minutes prior to dosing) at steady state on Day 8 for dose 15 (morning dose on Day 8). |
| Pre-dose Concentration Measured of BI 1265162 in Plasma at Steady State After Dose 57 (Cpre,ss, 57) | Pre-dose concentration measured of BI 1265162 in plasma at steady state after dose 57 (Cpre,ss, 57) was reported. | At pre-dose (taken within 60 minutes prior to dosing) at steady state on Day 29 for dose 57 (morning dose on Day 29). |
| Area Under the Concentration-time Curve of BI 1265162 in Plasma From 0 to 4 Hours at Steady State After Dose 15 (AUC0-4,ss,15) | Area under the concentration-time curve of BI 1265162 in plasma from 0 to 4 hours at steady state after dose 15 (AUC0-4,ss,15) was reported. | At pre-dose (taken within 60 minutes prior to dosing) and 5 minutes (min), 30 min, 1 hour, and 4 hours post dosing at steady state on Day 8 for dose 15 (morning dose on Day 8). |
| Indianapolis |
| Indiana |
| 46202 |
| United States |
| The University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27517 | United States |
| Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15224 | United States |
| Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| University of Texas Southwestern Medical Center | Dallas | Texas | 75390 | United States |
| Virginia Commonwealth University Health Systems | Richmond | Virginia | 23219 | United States |
| University of Washington | Seattle | Washington | 98195 | United States |
| Brussels - UNIV UZ Brussel | Brussels | 1090 | Belgium |
| UZ Leuven | Leuven | 3000 | Belgium |
| St. Paul's Hospital | Vancouver | British Columbia | V1Y 1S1 | Canada |
| Centre Hospitalier de l'Universite de Montreal (CHUM) | Montreal | Quebec | H2X 3E4 | Canada |
| HOP Arnaud de Villeneuve | Montpellier | 34295 | France |
| HOP Robert Debré | Paris | 75019 | France |
| HOP Cochin | Paris | 75679 | France |
| HOP Lyon Sud | Pierre-Bénite | 69495 | France |
| HOP Perharidy | Roscoff | 29684 | France |
| Charité - Universitätsmedizin Berlin | Berlin | 13353 | Germany |
| Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH | Essen | 45239 | Germany |
| Universitätsklinikum Gießen und Marburg GmbH | Giessen | 35385 | Germany |
| Medizinische Hochschule Hannover | Hanover | 30625 | Germany |
| Universitätsklinikum Tübingen | Tübingen | 72076 | Germany |
| Hospital Vall d'Hebron | Barcelona | 08035 | Spain |
| Sahlgrenska US, Göteborg | Gothenburg | 413 45 | Sweden |
| Stockholm CF-Center , B59, Huddinge Universitetssjukhus | Stockholm | 141 86 | Sweden |
| Royal Brompton Hospital | London | SW3 6NP | United Kingdom |
| Goss CH, Jain R, Seibold W, Picard AC, Hsu MC, Gupta A, Fajac I. An innovative phase II trial to establish proof of efficacy and optimal dose of a new inhaled epithelial sodium channel inhibitor BI 1265162 in adults and adolescents with cystic fibrosis: BALANCE-CFTM 1. ERJ Open Res. 2020 Dec 7;6(4):00395-2020. doi: 10.1183/23120541.00395-2020. eCollection 2020 Oct. |
2 puffs of 10 micrograms (μg) BI 1265162 (Total: 20μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 40μg) for a treatment period of 4 weeks in patients with cystic fibrosis.
| FG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| FG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| FG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| COMPLETED |
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| NOT COMPLETED |
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Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | 2 puffs of matching placebo were inhaled orally via the Respimat® inhaler twice daily for a treatment period of 4 weeks in patients with cystic fibrosis. |
| BG001 | BI 1265162 20μg b.i.d. | 2 puffs of 10 micrograms (μg) BI 1265162 (Total: 20μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 40μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| BG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| BG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| BG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| BG005 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| 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 |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
| |||||||||||
| Trough forced expiratory volume in one second (FEV1) percent predicted | Baseline trough FEV1 percent (%) predicted was defined as the last measurement taken on day 1 before first study drug administration and was measured within 30 minutes prior to dosing. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. One participant in the BI 1265162 200 microgram group did not have valid baseline trough FEV1 percent predicted value measured. | Mean | Standard Deviation | Percentage of predicted trough FEV1 |
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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 | Change From Baseline in Percent Predicted Trough Forced Expiratory Volume in 1 Second (FEV1) After 4 Weeks of Treatment | Trough FEV1 was measured within 30 minutes prior to dosing of study medication. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. Only participants with non-missing outcome measured were included in the analysis. | Posted | Mean | Standard Deviation | Percentage of predicted trough FEV1 | At 30 minutes prior to dosing in Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
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| Secondary | Change From Baseline in Lung Clearance Index (LCI) Assessed by N2 Multiple Breath Washout (N2MBW) Procedure After 4 Weeks of Treatment | Change from baseline in Lung Clearance Index (LCI) assessed by N2 Multiple Breath Washout (N2MBW) procedure after 4 weeks of treatment was reported. LCI was calculated as the ratio of cumulative expired volume (CEV) to functional residual capacity (FRC), which was LCI = CEV (milliliter/kilogram) / FRC (milliliter/kilogram) and hence, LCI was "Unitless". The change from baseline after 4 weeks of treatment in LCI was then calculated as the LCI value measured after 4 weeks of treatment at Day 29 minus the LCI value measured at baseline on Day 1. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. Only participants with non-missing outcome measured were included in the analysis. | Posted | Mean | Standard Deviation | Unitless | At pre-dose in Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
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| Secondary | Change From Baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) Total Score After 4 Weeks of Treatment | The adult/adolescent format of the CFQ-R consists of 50 questions (qts) dividing into 12 domains: Physical functioning(8 qts), role limitations(4 qts), vitality(4 qts), emotional functioning(5 qts), social functioning(6 qts), body image(3 qts), eating disturbance(3 qts), treatment burden(3 qts), health perceptions(3 qts), weight(1 qts), respiratory symptoms(7 qts), and digestive system(3 qts). The score of some qts is first reversed if reversed coded, so that the score for each of the 50 qts ranges from 1 to 4 points (less symptoms). Then, a domain score for a domain with N qts is calculated as (sum of the scores of the N qts - N)/(N ✕ 4 - N) ✕ 100. Each domain score ranges from 0 to 100 (better health). The CFQ-R total score is summing up the domain scores and ranges from 0 to 1200 (better quality of life). The change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) total score after 4 weeks of treatment was reported. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. Only participants with non-missing outcome measured were included in the analysis. | Posted | Mean | Standard Deviation | Score on a scale | At Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
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| Secondary | Change From Baseline in Cough and Sputum Assessment Questionnaire (CASA-Q) (4 Separate Sub-scores) After 4 Weeks of Treatment | The 20-item Sputum Assessment Questionnaire (CASA-Q) consisted of 4 domains: Cough Symptoms Domain (3 items), Cough Impact Domain (8 items), Sputum Symptoms Domain (3 items), and Sputum Impact Domain (6 items). Score of each item has been reversed such that better responses have higher score, which ranges from 1 (worse) to 5 (better health). For each domain, the domain score was calculated by summing up the scores of the respective items and scaling to a value ranging from 0 to 100, with higher score associated with fewer symptoms/less impact due to cough or sputum. The 4 domain scores (sub-scores) were reported. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. Only participants with non-missing outcome measured were included in the analysis. | Posted | Mean | Standard Deviation | Score on a scale | At Day 1 (baseline) and Day 29 (end of 4-week treatment period). |
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| Secondary | Percentage of Patients With Treatment-emergent Adverse Events (AE) up to Day 36 | Percentage of patients with any treatment-emergent Adverse Events (AE) up to day 36 was reported. | Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received. | Posted | Number | Percentage of participants | From Day 1 (baseline) until end of 4 weeks of treatment period (Day 29) plus 7 days of follow-up, up to 36 days. |
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| Secondary | Concentration of BI 1265162 in Plasma at 0.083 Hour at Steady State Following Dose 15 (C0.083,ss,15) | Concentration of BI 1265162 in plasma at 0.083 hour at steady state following dose 15 (C0.083,ss,15) was reported. | Pharmacokinetic set (PKS): The PKS included all patients in the treated set who provided at least one pharmacokinetic parameter. Only participants with non-missing outcome measured were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | picomole/liter (pmol/L) | At 5 minutes (around 0.083 hours) post dosing at steady state on Day 8 for dose 15 (morning dose on Day 8). |
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| Secondary | Concentration of BI 1265162 in Plasma at 0.083 Hour at Steady State Following Dose 57 (C0.083,ss,57) | Concentration of BI 1265162 in plasma at 0.083 hour at steady state following dose 57 (C0.083,ss,57) was reported. | Pharmacokinetic set (PKS): The PKS included all patients in the treated set who provided at least one pharmacokinetic parameter. Only participants with non-missing outcome measured were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | picomole/liter (pmol/L) | At 5 minutes (around 0.083 hours) post dosing at steady state on Day 29 for dose 57 (morning dose on Day 29). |
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| Secondary | Pre-dose Concentration Measured of BI 1265162 in Plasma at Steady State After Dose 15 (Cpre,ss, 15) | Pre-dose concentration measured of BI 1265162 in plasma at steady state after dose 15 (Cpre,ss, 15) was reported. | Pharmacokinetic set (PKS): The PKS included all patients in the treated set who provided at least one pharmacokinetic parameter. Only participants with non-missing outcome measured were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | picomole/liter (pmol/L) | At pre-dose (taken within 60 minutes prior to dosing) at steady state on Day 8 for dose 15 (morning dose on Day 8). |
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| Secondary | Pre-dose Concentration Measured of BI 1265162 in Plasma at Steady State After Dose 57 (Cpre,ss, 57) | Pre-dose concentration measured of BI 1265162 in plasma at steady state after dose 57 (Cpre,ss, 57) was reported. | Pharmacokinetic set (PKS): The PKS included all patients in the treated set who provided at least one pharmacokinetic parameter. Only participants with non-missing outcome measured were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | picomole/liter (pmol/L) | At pre-dose (taken within 60 minutes prior to dosing) at steady state on Day 29 for dose 57 (morning dose on Day 29). |
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| Secondary | Area Under the Concentration-time Curve of BI 1265162 in Plasma From 0 to 4 Hours at Steady State After Dose 15 (AUC0-4,ss,15) | Area under the concentration-time curve of BI 1265162 in plasma from 0 to 4 hours at steady state after dose 15 (AUC0-4,ss,15) was reported. | Pharmacokinetic set (PKS): The PKS included all patients in the treated set who provided at least one pharmacokinetic parameter. Only participants with non-missing outcome measured were included in the analysis. | Posted | Geometric Mean | Geometric Coefficient of Variation | hours * picomole/liter (h*pmol/L) | At pre-dose (taken within 60 minutes prior to dosing) and 5 minutes (min), 30 min, 1 hour, and 4 hours post dosing at steady state on Day 8 for dose 15 (morning dose on Day 8). |
|
From Day 1 (baseline) until end of 4 weeks of treatment period (Day 29) plus 7 days of follow-up, up to 36 days.
Treated set (TS): The TS included all patients who were randomized and treated with at least one dose of study drug. The treatment assignment was determined based on the first treatment the subjects received.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | 2 puffs of matching placebo were inhaled orally via the Respimat® inhaler twice daily for a treatment period of 4 weeks in patients with cystic fibrosis. | 1 | 18 | 1 | 18 | 11 | 18 |
| EG001 | BI 1265162 20μg b.i.d. | 2 puffs of 10 micrograms (μg) BI 1265162 (Total: 20μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 40μg) for a treatment period of 4 weeks in patients with cystic fibrosis. | 0 | 6 | 0 | 6 | 0 | 6 |
| EG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. | 0 | 5 | 0 | 5 | 2 | 5 |
| EG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. | 0 | 5 | 0 | 5 | 2 | 5 |
| EG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. | 0 | 18 | 1 | 18 | 15 | 18 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Hypoglycaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Lacrimation increased | Eye disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Vessel puncture site haematoma | General disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Acute sinusitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Infective pulmonary exacerbation of cystic fibrosis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Pneumonia bacterial | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Rhinitis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Vulvovaginal candidiasis | Infections and infestations | MedDRA 23.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Aspartate aminotransferase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Pulmonary function test decreased | Investigations | MedDRA 23.0 | Systematic Assessment |
| |
| Hyperkalaemia | Metabolism and nutrition disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchial hyperreactivity | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Bronchial obstruction | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Nasal polyps | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Sputum increased | Respiratory, thoracic and mediastinal disorders | MedDRA 23.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 23.0 | Systematic Assessment |
|
This trial was prematurely discontinued with only adult patients being recruited.
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 | May 18, 2020 | Mar 25, 2021 | SAP_001.pdf |
Not provided
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| 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 |
Not provided
Not provided
|
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| Other |
| OG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
|
| OG001 | BI 1265162 20μg b.i.d. | 2 puffs of 10 micrograms (μg) BI 1265162 (Total: 20μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 40μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG002 | BI 1265162 50μg b.i.d. | 2 puffs of 25 micrograms (μg) BI 1265162 (Total: 50μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 100μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG003 | BI 1265162 100μg b.i.d. | 2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG004 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG003 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG003 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG003 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
| OG003 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|
2 puffs of 50 micrograms (μg) BI 1265162 (Total: 100μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 200μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
| OG003 | BI 1265162 200μg b.i.d. | 2 puffs of 100 micrograms (μg) BI 1265162 (Total: 200μg) were inhaled orally via the Respimat® inhaler twice daily (b.i.d., daily dose: 400μg) for a treatment period of 4 weeks in patients with cystic fibrosis. |
|
|