A Study to Assess the Efficacy, Safety, and Tolerability of Brensocatib in Participants With Non-Cystic Fibrosis Bronchiectasis

JRCT ID: jRCT2031210048

Registered date:22/04/2021

Basic Information

• Recruitment status: Not Recruiting
• Health condition(s) or Problem(s) studied: Non-Cystic Fibrosis Bronchiectasis
• Date of first enrollment: 01/12/2020
• Target sample size: 1660
• Countries of recruitment: Argentina,Japan,Australia,Japan,Austria,Japan,Belgium,Japan,Brazil,Japan,Turkey,Japan,Canada,Japan,Chile,Japan,Colombia,Japan,Denmark,Japan,France,Japan,Germany,Japan,Greece,Japan,UK,Japan,Ireland,Japan,Israel,Japan,Italy,Japan,Korea, Republic of,Japan,Latvia,Japan,Malaysia,Japan,Mexico,Japan,Netherlands,Japan,New Zealand,Japan,Peru,Japan,USA,Japan,Portugal,Japan,Thailand,Japan,Slovakia,Japan,Spain,Japan,Taiwan,Japan
• Study type: Interventional

Intervention(s)

- Experimental: Brensocatib 10 mg
Participants will receive brensocatib 10 mg once daily, for 52 weeks.

- Experimental: Brensocatib 25 mg
Participants will receive brensocatib 25 mg once daily, for 52 weeks.

- Placebo Comparator: Placebo
Participants will receive a brensocatib-matching placebo, tablets orally, once daily, for 52 weeks.

Outcome(s)

• Primary Outcome: Annualized Rate of Pulmonary Exacerbations (PEs) over the 52-weeks treatment period

Secondary Outcome

- Time to First Pulmonary Exacerbation (PE) over the 52-week treatment period
- Percentage of Participants who are Pulmonary Exacerbation (PE) Free over the 52-week treatment period
- Change from Baseline in Postbronchodilator Forced Expiratory Volume in 1 second (FEV1) at Week 52
- Annualized Rate of Severe Pulmonary Exacerbations (PEs) over the 52-week treatment period
- Change from Baseline to Week 52 in Quality of Life Questionnaire- Bronchiectasis (QOL-B) Respiratory Symptoms Domain Score in Adult Participants
- Number of Participants who Experience at Least one treatment-emergent adverse events (TEAEs) over 56 weeks
- Change from Baseline in clinical laboratory parameters, vital signs, and ECG
- Plasma Concentration of Brensocatib at Select Time Points up to Week 52

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: <= 85age old
• Gender: Both

Include criteria

- Provide their signed study informed consent to participate.
a. Adolescent participants must have signed study assent form to participate, and the adolescent parent or legal guardian must have provided signed informed consent for the adolescent to participate.
- Clinical history consistent with non-cystic fibrosis bronchiectasis (NCFBE) (cough, chronic sputum production and/or recurrent respiratory infections) that is confirmed by chest computerized tomography (CT) scan.
- At least 2 PEs defined by need for antibiotic prescription by a physician for the signs and symptoms of respiratory infections in the past 12 months before the Screening Visit.
a. Adolescent participants are required to have at least 1 pulmonary exacerbation in the prior 12 months.
- Women must be post-menopausal (defined as no menses for 12 months without an alternative medical cause), surgically sterile, or using highly effective double barrier contraception (ie, methods that in combination achieve <1% unintended pregnancy rates per year) from Day 1 to at least 90 days after the last dose.
- Male participants with female partners of childbearing potential must be using effective contraception from Day 1 to at least 90 days after the last dose.
- Male participants with pregnant or non-pregnant women of child-bearing potential partners must use condoms to avoid potential exposure to the embryo/fetus.

Exclude criteria

- A primary diagnosis of chronic obstructive pulmonary disease (COPD) or asthma as judged by the Investigator.
- Bronchiectasis due to cystic fibrosis.
- Current smokers as defined per Centers for Disease Control (CDC).
- Known or suspected immunodeficiency disorder, including history of invasive opportunistic infections.
- Known history of human immunodeficiency virus (HIV) infection.
- Currently being treated for nontuberculous mycobacteria (NTM) lung infection, allergic bronchopulmonary aspergillosis, or tuberculosis (TB).
- Active and current symptomatic infection by COVID-19.
- Inability to follow the procedures of the study (eg, due to language problems or psychological disorders).
- Receiving medications or therapy that are prohibited as concomitant medications.
- Previously participated in a clinical trial for brensocatib.
- Received any live attenuated vaccine within 4 weeks prior to the first administration of brensocatib.
- Suffering an exacerbation 4 weeks before Screening or during the Screening period.
- Adult participants only: Have compliance issues with completion of electronic diary entries during the Screening Period and in the opinion of the Investigator, compliance is unlikely to improve during the study.
- Participated in any other interventional clinical studies within 3 months before Screening Visit.
- History of alcohol or drug abuse within 6 months prior to the Screening Visit.
- Is the Investigator or any Sub-Investigator, research assistant, pharmacist, study coordinator, other staff or relative thereof directly involved in the conduct of the study.
- Known history of hypersensitivity to brensocatib or any of its excipients.

Related Information

• Primary Sponsor: Fernandez C
• Secondary ID(s): NCT04594369,2020-003688-25