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

Phase 2RecruitingNCT07048262

CSL Behring450 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

450

Conditions

Non-cystic Fibrosis Bronchiectasis

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 85 Years

Medical Language ↔ Plain English

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.

Locations (13)

The Prince Charles Hospital

Queensland, Australia

RECRUITING

Westmead Hospital

Westmead, Australia

RECRUITING

Fukuoka University Chikushi Hopsital

Chikushino-shi, Japan

RECRUITING

Kyusho Central Hospital of the Mutual Aid Association of Public School Teachers

Fukuoka, Japan

RECRUITING

Ibaraki Prefectural Central Hospital

Ibaraki, Japan

RECRUITING

Kazunori Tobino Iizuka Hospital

Iizuka-shi, Japan

RECRUITING

National Hospital Organization Minami Kyoto Hospital

Kyoto, Japan

RECRUITING

Matsusaka Municipal Hospital

Mie, Japan

RECRUITING

National Hospital Organization Kinki Chuo Chest Medical Center

Osaka, Japan

RECRUITING

Shimonoseki City Hospital

Shimonoseki-shi, Japan

RECRUITING

...and 3 more locations

Outcomes

Primary Outcomes

Time to first (TTF) Exacerbation

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.

Time frame: Up to Month 12

Secondary Outcomes

Annualized Exacerbation Rate (AER) (Exacerbation Event Rate Per-participant Year)

Time frame: Up to Month 12

Number of Participants Achieving a Clinically Important Difference in the Quality of Life-Bronchiectasis (QoL-B) Respiratory Symptoms Scale

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.

Time frame: Up to Month 12

Percentage of Participants Achieving a Clinically Important Difference in the QoL-B Respiratory Symptoms Scale

Time frame: Up to Month 12

Change From Baseline in QoL-B Respiratory Symptoms Scale

Time frame: From Baseline to Months 6 and 12

Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria Isolated from Sputum

Time frame: From Baseline to Month 1

Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs)

Time frame: Up to Month 13

Percentage of Participants with TEAEs and SAEs

Time frame: Up to Month 13

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

Overview

Conditions

Interventions

Eligibility

Locations (13)

Outcomes

Primary Outcomes

Secondary Outcomes

Central Contacts