Non-cystic Fibrosis Bronchiectasis
Conditions
Keywords
Chronic respiratory disease, Inflammation, Chronic bacterial infection
Brief summary
This study is a phase 2b, multicenter, randomized, double-blind, placebo-controlled, parallel-group, dose range finding study designed to explore the efficacy, safety, and tolerability of 2 active treatment regimens of CSL787 (immunoglobulin G \[IgG\] inhalation solution) compared with placebo over a period of 6 to 12 months independent of the occurrence of pulmonary exacerbations. 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 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 * 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 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 either 1 of the following: * \>= 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 \> 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 percentage of the predicted normal forced expiratory volume in 1 second of expiration \[FEV1% predicted\] \> 35% and forced expiratory volume in 1 second (FEV1) \>= 1 liter (L) obtained in accordance with American Thoracic Society (ATS) / European Respiratory Society (ERS) standards for spirometry during Screening and at Baseline.
Exclusion criteria
* History of 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 immunoglobulin. * Primary diagnosis of other pulmonary disorders, including chronic obstructive pulmonary disease (COPD) asthma or, diffuse panbronchiolitis (DPB), as determined by the investigator. * Pulmonary exacerbation requiring antibiotic therapy within the 4 weeks before Baseline.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to first (TTF) Exacerbation | Up to Month 12 | 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 Participants Achieving a Clinically Important Difference in the Quality of Life-Bronchiectasis (QoL-B) Respiratory Symptoms Scale | Up to Month 12 | 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 Clinically Important Difference in the QoL-B Respiratory Symptoms Scale | Up to Month 12 | 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 | From Baseline to Months 6 and 12 | 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. |
| Annualized Exacerbation Rate (AER) (Exacerbation Event Rate Per-participant Year) | Up to Month 12 | — |
| Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs) | Up to Month 13 | — |
| Percentage of Participants with TEAEs and SAEs | Up to Month 13 | — |
| Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria Isolated from Sputum | From Baseline to Month 1 | — |
Countries
Australia, Japan
Contacts
Primary ContactTrial Registration Coordinator
Outcome results
None listed