A 4-week study to test different doses of BI 1265162 in adolescents and adults with cystic fibrosis using the Respimat® inhaler - BALANCE - CF(TM)1

A randomised, double-blind, placebo-controlled and parallel group trial to evaluate efficacy and safety of twice daily inhaled doses of BI 1265162 delivered by Respimat® inhaler as add-on therapy to standard of care over 4 weeks in patients with cystic fibrosis - BALANCE - CF(TM)1

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-000261-21-IE

Enrollment

Registered

2019-05-30

Start date

2019-07-26

Completion date

Unknown

Last updated

2020-03-11

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Cystic Fibrosis MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Product Name: BI 1265162 10 µg Pharmaceutical Form: Inhalation solution INN or Proposed INN: not yet available Other descriptive name: BI 1265162

Eligibility

Sex/Gender

All

Inclusion criteria

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

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;

Design outcomes

Primary

Measure	Time frame
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	—

Measure

Time frame

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

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Secondary

Measure	Time frame
Timepoint(s) of evaluation of this end point: After 4 weeks of treatment; 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 • Change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) total score after 4 weeks of treatment • Change from baseline in Cough and Sputum Assessment Questionnaire (CASA-Q) (4 separate sub-scores) after 4 weeks of treatment • Percentage of patients with treatment-emergent Adverse Events (AE) up to Day 36 • Ct,N (concentration of the analyte in plasma at time t following dose N) • Cpre,N (predose concentration measured for dose N) • AUC0-t,N (area under the concentration-time curve of the analyte in plasma until t hours after dose N)	—

Measure

Time frame

Timepoint(s) of evaluation of this end point: After 4 weeks of treatment; 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 • Change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ-R) total score after 4 weeks of treatment • Change from baseline in Cough and Sputum Assessment Questionnaire (CASA-Q) (4 separate sub-scores) after 4 weeks of treatment • Percentage of patients with treatment-emergent Adverse Events (AE) up to Day 36 • Ct,N (concentration of the analyte in plasma at time t following dose N) • Cpre,N (predose concentration measured for dose N) • AUC0-t,N (area under the concentration-time curve of the analyte in plasma until t hours after dose N)

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Countries

Belgium, Canada, France, Germany, Ireland, Spain, Sweden, United Kingdom, United States

Contacts

Public ContactCT Disclosure & Data Transparency

Boehringer Ingelheim Pharma GmbH & Co. KG

clintriage.rdg@boehringer-ingelheim.com18002430127

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026