Dose Range Finding, Efficacy, and Safety Study of Nebulized CSL787 in Adults With Non-cystic Fibrosis Bronchiectasis (NCFB)

Not Applicable

Not yet recruiting

• Conditions: Non-cystic Fibrosis Bronchiectasis
• Interventions: Biological: CSL787; Drug: Placebo; Device: Nebulizer

• Registration Number: NCT07048262
• Lead Sponsor: CSL Behring

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 (nebulized immunoglobulin G \[IgG\]) compared with placebo over 6 to 12 months to prolong the time to first (TTF) exacerbation as a primary endpoint.

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 over a period of greater than or equal to (\>=) 6 months but not exceeding 12 months toward prolonging the TTF exacerbation.

Detailed Description

Not available

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-06-24
• Study First Submitted QC: 2025-06-24
• Last Update Submitted: 2025-06-24
• Study First Posted: 2025-07-02
• Last Update Posted: 2025-07-02
• Study Start: 2025-07-27
• Primary Completion: 2028-02-28
• Study Completion: 2028-03-28

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 450

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) total score > 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 6 weeks before Baseline.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
CSL787 High Dose	CSL787	Participants in this arm will receive a high dose of CSL787.
CSL787 High Dose	Nebulizer	Participants in this arm will receive a high dose of CSL787.
CSL787 Low Dose	CSL787	Participants in this arm will receive a low dose of CSL787.
CSL787 Low Dose	Nebulizer	Participants in this arm will receive a low dose of CSL787.
Placebo	Placebo	Participants in this arm will receive placebo.

Primary Outcome Measures

Name	Time	Method
TTF Exacerbation 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 Outcome Measures

Name	Time	Method
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.
Annualized Exacerbation Rate (AER) (Exacerbation Event Per-participant Year of follow up)	Up to Month 12
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.
Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria Isolated from Sputum	From Baseline to Month 1
Percentage of Participants with TEAEs and SAEs	Up to Month 13
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.
Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs)	Up to Month 13