A 4-week Study to Test Different Doses of BI 1265162 in Adolescents and Adults With Cystic Fibrosis Using the Respimat® Inhaler - BALANCE - CF™1
Condition: Cystic Fibrosis
MeSH terms: Cystic Fibrosis; Fibrosis
Intervention: BI 1265162; Placebo
Intervention description: Inhalation solution; Inhalation solution
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Double (Participant, Investigator)
Enrollment: 52.0
Sex: All
Minimum age: 12 Years
Healthy volunteers: No

Summary:

      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.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Male or female patients, 12 years of age or older at screening;

          -  Documented diagnosis of cystic fibrosis including:

               -  positive sweat chloride ≥ 60 mEq/L, by pilocarpine iontophoresis OR

               -  genotype with 2 identifiable mutations consistent with cystic fibrosis
                  accompanied by one or more clinical features with cystic fibrosis phenotype;

          -  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:

               -  Potassium > upper limit of normal (ULN) in non-haemolysed blood

               -  Abnormal renal function defined as estimated Glomerular Filtration Rate (eGFR) <
                  60ml/min/1.73m²

               -  Abnormal liver function, defined by serum level of either alanine transaminase
                  (ALT), aspartate transaminase (AST) or total bilirubine ≥ 3 x upper limit of
                  normal (ULN)

          -  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.