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

CSL Behring

Status and phase: Enrolling; Phase 2

Conditions: Non-cystic Fibrosis Bronchiectasis

Treatments: Drug: Placebo; Biological: CSL787; Device: Nebulizer

Study type: Interventional

Funder types: Industry

Identifiers

NCT07048262

2024-518821-13-00 (Other Identifier)

CSL787_2001

Details and patient eligibility

About

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.

Enrollment: 450 estimated patients

Sex: All

Ages: 18 to 85 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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450 participants in 3 patient groups, including a placebo group

CSL787 High Dose

Experimental group

Description:

Participants in this arm will receive a high dose of CSL787.

Treatment:

Device: Nebulizer

Biological: CSL787

CSL787 Low Dose

Experimental group

Description:

Participants in this arm will receive a low dose of CSL787.

Treatment:

Device: Nebulizer

Biological: CSL787

Placebo

Placebo Comparator group

Description:

Participants in this arm will receive placebo.

Treatment:

Device: Nebulizer

Drug: Placebo