Non-cystic Fibrosis Bronchiectasis
Conditions
Keywords
Chronic respiratory disease, Inflammation, Chronic bacterial infection
Brief summary
This study is a proof-of-concept phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose selection study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (human normal immunoglobulin G \[IgG\] inhalation solution) compared with placebo over 52 weeks. The primary aim of the study is to characterize the overall effect of CSL787 as well as the dose response of 2 active treatment regimens of inhaled CSL787 administered to participants with NCFB toward prolonging the time to first (TTF) exacerbation.
Interventions
Sponsors
CSL Behring
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
This is a randomized, double-blind, parallel-group, placebo-controlled study.
Eligibility
Sex/Gender
ALL
Age
18 Years to 85 Years
Healthy volunteers
No
Inclusion criteria
* Adult between the ages of 18 to 85 years * Capable of spontaneous sputum production at Screening. * Primary diagnosis of NCFB confirmed by chest computed tomography (CT) scan, where bronchiectasis has been documented by a radiologist. Diagnosis in the medical records based on documented historical scans is acceptable if the chest CT, scan confirming the participant's NCFB diagnosis was performed within 12 months before enrollment. Participants for whom no chest CT scan results are available within the previous 12 months will undergo a chest CT scan during the Screening Period. * Exacerbation history within the previous 1 year defined as one of the following: * greater than or equal (≥) 2 documented exacerbations requiring oral and/or intravenous (IV) antibiotic therapy to treat a pulmonary infection. OR * 1 documented exacerbation requiring oral and/or IV antibiotic therapy to treat a pulmonary infection and a St. George's Respiratory Questionnaire (SGRQ) Symptoms score of greater than (\>) 40 at Screening. * Note: Other medications to treat NCFB such as: oral macrolides, or dipeptidyl peptidase-1 (DPP-1) inhibitors are allowed, provided ≥ 1 historical exacerbation occurred while on the medication for ≥ 3 months at a stable dose. * Postbronchodilator percent predicted forced expiratory volume in 1 second (FEV1%) \> 35% and forced expiratory volume in 1 second (FEV1) ≥ 1 L obtained in accordance with American Thoracic Society (ATS) / European Respiratory Society (ERS) standards for spirometry during Screening and at Baseline on Day 1.
Exclusion criteria
* History of severe or uncontrolled asthma, or bronchospasm in response to inhaled therapies including inhaled antibiotics. * Known or suspected hypersensitivity, or other severe reactions, to the investigational product (IP), to any excipients of the IP, or to other immunoglobulins. * Primary diagnosis of other pulmonary disorders, including chronic obstructive pulmonary disease (COPD), asthma, or diffuse panbronchiolitis (DPB), as determined by the investigator. * Use of oral, IV, or inhaled antibiotic therapy to treat a pulmonary exacerbation or any other condition within 4 weeks before Baseline measurements on Day 1.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to first (TTF) Exacerbation | Up to Week 52 | TTF exacerbation, where an exacerbation is defined as a deterioration in ≥ 3 of the following symptoms for ≥ 48 hours: cough; sputum volume and / or consistency; sputum purulence; breathlessness and / or exercise tolerance; fatigue and / or malaise; hemoptysis AND a clinician determines that antibiotic therapy is required. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of exacerbations over the Treatment and Observation Period | Up to Week 52 | — |
| Number of Participants Achieving a Minimally Clinically Important Difference (MCID) in the Quality of Life-Bronchiectasis (QoL-B) Respiratory Symptoms Scale | Up to Week 52 | The QoL-B questionnaire is a self-administered measure assessing symptoms, functioning, and health-related quality of life for patients with NCFB. This questionnaire contains 37 items on 8 different scales (Respiratory symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions, and Treatment Burden). For each scale, scores are standardized on a 0-to-100-point scale; higher scores indicate better health-related quality of life. |
| Percentage of Participants Achieving a MCID in the QoL-B Respiratory Symptoms Scale | Up to Week 52 | The QoL-B questionnaire is a self-administered measure assessing symptoms, functioning, and health-related quality of life for patients with NCFB. This questionnaire contains 37 items on 8 different scales (Respiratory symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions, and Treatment Burden). For each scale, scores are standardized on a 0-to-100-point scale; higher scores indicate better health-related quality of life. |
| Change From Baseline in QoL-B Respiratory Symptoms Scale | Baseline to Week 52 | The QoL-B questionnaire is a self-administered measure assessing symptoms, functioning, and health-related quality of life for patients with NCFB. This questionnaire contains 37 items on 8 different scales (Respiratory symptoms, Physical, Role, Emotional and Social Functioning, Vitality, Health Perceptions, and Treatment Burden). For each scale, scores are standardized on a 0-to-100-point scale; higher scores indicate better health-related quality of life. |
| Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria Isolated from Sputum | Baseline up to Week 52 | — |
| Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs) | Up to Week 56 | — |
| Percentage of Participants with TEAEs and SAEs | Up to Week 56 | — |
Countries
Australia, Japan
Contacts
CONTACTTrial Registration Coordinator
STUDY_DIRECTORStudy Director
CSL Behring
Outcome results
None listed