BI 1305-0014
Investigational Medication Study for Bronchiectasis
Brief description of study
This study is open to adults and adolescents aged 12 to under 18 with bronchiectasis. People can participate in this study if they produce sputum and have had flare-ups (also called exacerbations).
The purpose of this study is to find out whether a medicine called BI 1291583 helps people with bronchiectasis. Participants are put into 2 groups randomly, which means by chance. One group takes BI 1291583 tablets and the other group takes placebo tablets. A placebo tablet looks like the BI 1291583 tablet but does not contain any medicine. Participants take 1 tablet once a day for up to 1 year and 6 months.
Participants are in the study for up to 1 year and 8 months. During this time, participants visit the study site up to 10 times and get about 13 phone calls from the site staff. Participants regularly complete a diary on a smartphone about their bronchiectasis symptoms and study doctors regularly check for any changes. The study doctors document when participants experience flare-ups. The number of flare-ups is compared between the participants who receive BI 1291583 and those who receive the placebo. The study doctors also regularly check participants' health and take note of any unwanted effects.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Bronchiectasis
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Age: 12 years or above
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Gender: All
Inclusion criteria:
- Male or female participants. Woman of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per International Council of Harmonisation (ICH) M3 (R2) that result in a low failure rate of less than 1 % per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the participant information.
- Signed and dated written informed consent and assent, if applicable, prior to admission to the study, in accordance with GCP and local legislation.
- Age of participants when signing the informed consent/assent ≥12 years.
-- Adolescents need to weigh at least 35 kg at Visit 1.
- Clinical history consistent with bronchiectasis (e.g. cough, chronic sputum production, recurrent respiratory infections) and investigator confirmed diagnosis of bronchiectasis by CT scan where bronchiectasis has been documented by a radiologist.
Participants whose past CT scan image records are not available will undergo a chest CT scan during Screening. Historical scans must not be older than five years.
- Adult participants should be able to produce sputum for Pseudomonas aeruginosa assessment during the screening period.
- History of documented pulmonary exacerbations (assessed and recorded by the
investigator) requiring antibiotic treatment. In the 12 months before Visit 1,
participants must have had either:
- at least 2 exacerbations, or
- at least 1 exacerbation and an St. George's Respiratory Questionnaire (SGRQ) Symptoms score of >40 at screening Visit 1 (adults only)
- at least 1 exacerbation and high symptom burden according to the investigator's judgement (adolescents only) For participants on oral or inhaled antibiotics as chronic treatment for bronchiectasis and participants on Cystic Fibrosis Transmembrane Conductance Regulator Modulator Therapy (CFTR-MT), at least one exacerbation must have occurred since initiation of antibiotics or CFTR-MT.
Exclusion criteria:
- Any new or newly diagnosed condition of primary or secondary immunodeficiency within 1 year before randomisation.
- Allergic bronchopulmonary aspergillosis being treated or requiring treatment.
- Tuberculosis or non-tuberculosis mycobacterial infection being treated or requiring treatment
- Any findings in the medical examination and/or laboratory value assessed at Screening Visit 1 or during screening period, that in the opinion of the investigator may put the participant at risk by participating in the trial.
- Any clinically relevant (at the discretion of the investigator) acute respiratory infection or ongoing pulmonary exacerbation at screening visit or during the screening unless recovered in the opinion of the investigator prior to Visit 2.
- Any relevant pulmonary, gastrointestinal, hepatic, renal, cardiovascular, metabolic, immunological, hormonal, or other disorder that, in the opinion of the investigator, may put the participant at risk by participating in the study.
- Major surgery (major according to the investigator's assessment) performed within 6 weeks prior to randomisation or scheduled during trial period.
- Any documented active or suspected malignancy or history of malignancy within 5 years prior to screening, except appropriately treated in situ non-melanoma skin cancers or in situ carcinoma of uterine cervix.
- Evidence or medical history of moderate or severe liver disease (Child-Pugh score B or C hepatic impairment).
- estimated Glomerular Filtration Rate (eGFR) according to Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) formula (adults) or Chronic Kidney Disease Under 25 (CKiD-U25) (adolescents) <30 mL/min at Visit 1.
- Previous treatment with a dipeptidyl peptidase-1 (DPP1) (Cathepsin C (CatC)) inhibitor. (Note: Participants that were randomised and only received placebo in studies with DPP1 (CatC) inhibitor are allowed.) Further exclusion criteria apply.
This study investigates bronchiectasis. The purpose of this study is to evaluate whether an investigational medication can help people with bronchiectasis.
The study evaluates the safety and effectiveness of the investigational medication for people with bronchiectasis. It focuses on understanding how the medication impacts flare-ups and overall health.
- Who can participate: This study is open to males and females aged 12 years and older with bronchiectasis. Participants must produce sputum and have a history of flare-ups. Adolescents need to weigh at least 35 kg.
- Study details: Participants will take one tablet daily for up to 1 year and 6 months. The study includes a placebo, which is an inactive substance that looks like the investigational medicine but does not contain any medicine.
- Study timelines and visits: The study will last 1 year and 8 months. The study requires 10 visits.
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