Clinical trial NCT05865886: A Study to Test How Well BI 1291583 is Tolerated by People With Cystic Fibrosis Bronchiectasis (Clairafly™)
Official title: A Randomized, 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™)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Bronchiectasis
Lead sponsor: Boehringer Ingelheim
Sponsor class: INDUSTRY
First posted: 2023-05-19
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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.


Eligibility criteria: Inclusion Criteria:

1. Age of patients when signing the informed consent ≥18 years
2. 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)
3. 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
4. History of pulmonary exacerbations requiring antibiotic treatment. In the 12 months before Visit 1, patients must have had either:

   1. at least 2 exacerbations, or
   2. at least 1 exacerbation and an St. George's Respiratory Questionnaire (SGRQ) Symptoms score of \>40 at screening visit 1 For patients on stable oral or inhaled antibiotics as chronic treatment for BE, at least one exacerbation must have occurred while on stable antibiotics.
5. Patients must be able to provide spontaneous or induced sputum samples. Further inclusion criteria apply.

Exclusion Criteria:

1. Moderate or severe liver disease (defined by Child-Pugh score B or C hepatic impairment) or Aspartate aminotransferase (AST) and/or Alanine aminotransferase (ALT) \> 3.0x Upper limit of normal (ULN) at Visit 1
2. Estimated glomerular filtration rate (eGFR) according to Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula \< 30 mL/min at Visit 1
3. Absolute blood neutrophil count \< 1,000/mm\^3 (equivalent to \< 1000 cells/μL or \< 10\^9 cells/L) at Visit 1
4. Any findings in the medical examination (including blood pressure (BP), pulse rate (PR), or electrocardiogram (ECG)) and/or laboratory value assessed at Visit 1 or during screening period that in the opinion of the investigator may put the patient at risk by participating in the trial
5. Positive serological tests for hepatitis B, hepatitis C (also confirmed with Hepatitis C virus ribonucleic acid (HCV RNA)), or human immunodeficiency virus (HIV) infection, or known infection status.

Further exclusion criteria apply.

Primary outcomes:
measure: Occurrence of Treatment emergent Adverse Events (TEAEs) up to 16 weeks from first drug administration
timeFrame: up to 16 weeks
Secondary outcomes:
measure: Relative change from baseline in neutrophil elastase (NE) activity in sputum at week 8 after first drug administration
timeFrame: at week 8
measure: Area under the concentration-time curve of the analyte in plasma over a uniform dosing interval (AUCτ) for the first dose
timeFrame: up to 8 hours
measure: Maximum measured concentration of the analyte in plasma (Cmax) for the first dose
timeFrame: up to 8 hours
measure: Area under the concentration-time curve of the analyte in plasma at steady state over a uniform dosing interval (AUCτ,ss)
timeFrame: up to 12 weeks
measure: Maximum measured concentration of the analyte in plasma (Cmax) at steady state (Cmax,ss)
timeFrame: up to 12 weeks
Other outcomes:
Locations:
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: New York
country: United States
facility: Northwell Health Physician Partners
state: New York
city: Leuven
country: Belgium
facility: UZ Leuven
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Essen
country: Germany
facility: Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Jena
country: Germany
facility: Universitätsklinikum Jena
city: Firenze
country: Italy
facility: Azienda Ospedaliera Meyer
city: Verona
country: Italy
facility: A.O. Univ. Integrata di Verona
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC, location VUMC
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
city: Barcelona
country: Spain
facility: Hospital Vall d'Hebron-Barcelona-47683
Study officials:
References: