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

NCT07048262 | Recruiting Phase 2 DMC FDA Drug FDA Device

• 2 other identifiers: CSL787_20012024-518821-13-00
• Study Type: interventional
• Target: 450
• Locations: 2 countries
• Sites: 13

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.

Conditions

Non-cystic Fibrosis Bronchiectasis

Keywords

Chronic respiratory disease; Inflammation; Chronic bacterial infection

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to Month 12

Secondary Outcomes (7)

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

  Up to Month 12

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

  Up to Month 12

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

  Up to Month 12

• Change From Baseline in QoL-B Respiratory Symptoms Scale

  From Baseline to Months 6 and 12

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

  From Baseline to Month 1

Study Arms (3)

CSL787 High Dose

EXPERIMENTAL

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

Biological: CSL787; Device: Nebulizer

CSL787 Low Dose

EXPERIMENTAL

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

Biological: CSL787; Device: Nebulizer

Placebo

PLACEBO COMPARATOR

Participants in this arm will receive placebo.

Drug: Placebo; Device: Nebulizer

Interventions

CSL787 BIOLOGICAL

CSL787 high or low doses once daily (QD) will be administered via inhalation over a period of 6 to 12 months.

CSL787 High Dose; CSL787 Low Dose

Placebo DRUG

Participants will receive a matching volume of placebo QD over a period of 6 to 12 months.

Placebo

Nebulizer DEVICE

The nebulizer is a CE-marked device.

CSL787 High Dose; CSL787 Low Dose; Placebo

Eligibility Criteria

• Age: 18 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CSL Behring: lead

Study Sites (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

Japan Anti-Tuberculosis Association, Fukujuji Hospital

Tokyo, Japan

RECRUITING

Keio University Hospital

Tokyo, Japan

RECRUITING

National Hospital Organization Mie Chuo Medical Center

Tsu, Japan

RECRUITING

MeSH Terms

Conditions

Inflammation

Interventions

Nebulizers and Vaporizers

Condition Hierarchy (Ancestors)

Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Equipment and Supplies

Study Officials

• Study Director

  CSL Behring

  STUDY DIRECTOR

Central Study Contacts

Trial Registration Coordinator

CONTACT

+1 610-878-4697; clinicaltrials@cslbehring.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a randomized, double-blind, parallel-group, placebo-controlled study.

Study Record Dates

• First Submitted: June 24, 2025
• First Posted: July 2, 2025
• Study Start: September 3, 2025
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): March 28, 2028
• Last Updated: November 10, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (i.e. FDA, EMA) is complete and the primary publication is available.
• Access Criteria: Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.

Locations

JP (11); AU (2)