A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhaler

Phase 1

Conditions: Cystic Fibrosis
MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

Registration Number: EUCTR2019-000261-21-ES

Lead Sponsor: Boehringer Ingelheim, S.A.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 98

Inclusion Criteria

1. Male or female patients / 12 years of age or older at screening;
2. Documented diagnosis of cystic fibrosis including:
• positive sweat chloride = 60 mEq/L, by pilocarpine iontophoresis or
• a genotype with 2 identifiable mutations consistent with cystic fibrosis accompanied by one or more clinical features with cystic fibrosis phenotype;
3. FEV1 = 40% and = 90% of predicted values at screening and at predose at Visit 2 (randomisation).
Are the trial subjects under 18? yes
Number of subjects for this age range: 21
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 77
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Evidence of acute upper or lower respiratory tract infection within 4 weeks prior to randomisation based on investigator’s judgement;
2. Pulmonary exacerbation requiring use of i.v./oral/inhaled antibiotics or oral corticosteroids within 4 weeks prior to randomisation;
3. Women of childbearing potential

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The primary objective of this trial is to assess the efficacy, safety and pharmacokinetics of twice daily inhaled doses of 20 µg, 50 µg, 100µg and 200 µg of BI 1265162 delivered by Respimat® inhaler versus placebo in adolescents and adult patients with cystic fibrosis.;Secondary Objective: Not applicable;Primary end point(s): The primary endpoint to assess efficacy of BI 1265162 is the change from baseline in percent predicted trough Forced Expiratory Volume in 1 Second (FEV1) after 4 weeks of treatment.;Timepoint(s) of evaluation of this end point: After 4 weeks of treatment

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Change from baseline in Lung Clearance Index (LCI) assessed by N2 Multiple Breath Washout (N2MBW) procedure after 4 weeks of treatment<br>• Change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) total score after 4 weeks of treatment<br>• Change from baseline in Cough and Sputum Assessment Questionnaire (CASA-Q) (4 separate sub-scores) after 4 weeks of treatment<br>• Percentage of patients with treatment-emergent Adverse Events (AE) up to Day 36<br>• Cmax,N (maximum measured concentration of the analyte in plasma following dose N) up to Day 36<br>• Cpre,N (predose concentration measured for dose N) up to Day 29<br>• AUC0-t,N (area under the concentration-time curve of the analyte in plasma until t hours after dose N) up to Day 36;Timepoint(s) of evaluation of this end point: After 4 weeks of treatment

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