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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

CSL Behring LLC

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Respiratory Tract Diseases [C08]

Decision date (initial)

2025-10-01

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

External identifiers

EU CT number

2024-518821-13-00

ClinicalTrials.gov

NCT07048262

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy

To evaluate the effect of nebulized CSL787 (human normal immunoglobulin for nebulization) on the TTF exacerbation.

Conditions and MedDRA coding

Non-cystic Fibrosis Bronchiectasis

Study design 1 period

Regulatory references

Scientific advice from competent authorities

European Medicines Agency, Pharmaceuticals And Medical Devices Agency, Food And Drug Administration

Plan to share IPD

Yes

IPD plan description

CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at [email protected].

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Adult between the ages of 18 to 85 years
2. 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
3. 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: maintenance treatment with 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
4. 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 3

1. History of bronchospasm in response to inhaled therapies including inhaled antibiotics
2. Primary diagnosis of other pulmonary disorders, including chronic obstructive pulmonary disease (COPD) asthma or, diffuse panbronchiolitis (DPB), as determined by the investigator.
3. Pulmonary exacerbation requiring antibiotic therapy within the 6 weeks before Baseline.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. TTF (Time to first) Exacerbation

Secondary endpoints 7

1. Annualized Exacerbation Rate (AER) (Exacerbation Event Rate Per Participant-year of Follow-up)
2. Number of Participants Achieving a Clinically Important Difference in the Quality of Life–Bronchiectasis (QoL-B) Respiratory Symptoms Scale
3. Percentage of Participants Achieving a Clinically Important Difference in the QoL-B Respiratory Symptoms Scale
4. Change From Baseline in QoL-B Respiratory Symptoms Scale
5. Change From Baseline in Total Colony-forming Unit (CFUs) for Pathogenic Bacteria isolated from Sputum
6. Number of Participants with Treatment-emergent Adverse events (TEAEs) and Serious Adverse Events (SAEs)
7. Percentage of Participants with TEAEs and SAEs

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

CSL Behring LLC

Sponsor organisation

CSL Behring LLC

Address

1020 1st Avenue

City

King Of Prussia

Postcode

19406-1310

Country

United States

Scientific contact point

Organisation

CSL Behring LLC

Contact name

Study Director

Public contact point

Organisation

CSL Behring LLC

Contact name

Trial Registration Coordinator

Third parties 15

Locations

10 EU/EEA countries · 75 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 191 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

7 submissions — initial application plus substantial / non-substantial modifications