A Translational Study for Phenotyping and Endotyping Chinese Patients With NCFBE

A Multi-center Longitudinal Observation Translational Study to Evaluate Phenotypes, Endotypes and Biomarkers in Chinese Patients With NCFBE

Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic respiratory disease characterized by a clinical syndrome of chronic productive cough and recurrent respiratory infections in the presence of abnormal and permanent dilation of the bronchi. Recent epidemiological studies have clearly shown that the prevalence and incidence of NCFBE are quickly rising both in high- and low-income countries. With the increase of prevalence, bronchiectasis brings huge medical and economic burden to the society.

In this study, the investigator will perform biomarker assessments and multi-omics analysis on NCFBE patients and healthy participants in China to validate the link of disease pathways to pathophysiological features and uncover the molecular endotypes behind clinical phenotypesof Chinese patients with NCFBE.

Study Overview

• Status: Recruiting
• Conditions: Non-cystic Fibrosis Bronchiectasis
• Intervention / Treatment: Other: Participant Follow-up

Detailed Description

Background/Rationale:

Non-cystic fibrosis bronchiectasis (NCFBE) is a chronic respiratory disease characterized by a clinical syndrome of chronic productive cough and recurrent respiratory infections in the presence of abnormal and permanent dilation of the bronchi. Recent epidemiological studies have clearly shown that the prevalence and incidence of NCFBE are quickly rising both in high- and low-income countries. With the increase of prevalence, bronchiectasis brings huge medical and economic burden to the society.

Recently published Chinese Bronchiectasis Registry study (BE-China) data showed that Chinese bronchiectasis patients exhibit unique clinical characteristics when compared to western countries. The proportion of post-infective causes and tuberculosis in China was twice that of the European Multicenter Bronchiectasis Audit and Research Collaboration (EMBARC) cohort. Moreover, Chinese patients had a lower FEV1% of predicted value, and a higher proportion of obstruction compared to the EMBARC cohort. Pseudomonas aeruginosa (PsA) was the most common pathogen in both cohorts.

Chinese patients exhibited a higher frequency of hospitalization compared to the EMBARC cohort (57.2% vs 26.4%); however, unlike the frequency of hospitalization, Chinese patients had fewer exacerbations in the year before enrollment compared to the EMBARC cohort, with most having only one exacerbation. The proportion of patients with three or more exacerbations was much higher in the EMBARC cohort than in China (12.3% vs 38.8%). Except for Aspergillus fumigatus, the positive culture rates of other pathogens were much higher in the EMBARC cohort than in China.

Significant difference in aetiology and clinical phenotypes of NCFBE has been demonstrated by many registry studies conducted in different geographical regions including EMBARC and BE-China. However, the biological processes and mechanisms driving the disease development, so-called "endotypes", have not been fully investigated within the patient populations in these studies. It is remaining unknown that if these differences reported in clinical phenotypes were truly caused by or linked to different endotypes in NCFBE.

In this study, the investigator will perform biomarker assessments and multi-omics analysis on NCFBE patients and healthy participants in China to validate the link of disease pathways to pathophysiological features and uncover the molecular endotypes behind clinical phenotypesof Chinese patients with NCFBE.

• Study Type: Observational
• Enrollment (Estimated): 320

Contacts and Locations

• Study Contact: Name: AstraZeneca Clinical Study Information Center; Phone Number: 1-877-240-9479; Email: information.center@astrazeneca.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China
      • Recruiting
      • Beijing Hospital
    • Beijing, Beijing Municipality, China
      • Active, not recruiting
      • Beijing Chaoyang Hospital Affiliated to Capital Medical University
    • Beijing, Beijing Municipality, China
      • Active, not recruiting
      • Peking University People's Hospital
  • Chongqing Municipality
    • Chongqing, Chongqing Municipality, China
      • Recruiting
      • The Second Affiliated Hospital of Chongqing Medical University
  • Fujian
    • Quanzhou, Fujian, China
      • Recruiting
      • The Second Affiliated Hospital of Fujian Medical University
  • Gansu
    • Lanzhou, Gansu, China
      • Active, not recruiting
      • Gansu Provincial Hospital
  • Guangdong
    • Shenzhen, Guangdong, China
      • Active, not recruiting
      • ShenZhen People's Hospital
    • Zhanjiang, Guangdong, China
      • Recruiting
      • Affiliated Hospital of Guangdong Medical University"
  • Guangxi
    • Nanning, Guangxi, China
      • Recruiting
      • The First Affiliated Hospital of Guangxi Medical University
  • Guizhou
    • Guiyang, Guizhou, China
      • Recruiting
      • Guizhou Provincial People's Hospital
  • Henan
    • Zhengzhou, Henan, China
      • Recruiting
      • Henan Provincial People's Hospital
    • Zhengzhou, Henan, China
      • Recruiting
      • Zhengzhou People's Hospital
  • Hubei
    • Shiya, Hubei, China
      • Recruiting
      • Shiyan Taihe Hospital
  • Hunan
    • Changsha, Hunan, China
      • Active, not recruiting
      • Xiangya Hospital of Central South University
  • Jiangsu
    • Xuzhou, Jiangsu, China
      • Active, not recruiting
      • The Affiliated Hospital of Xuzhou Medical University
  • Jiangxi
    • Nanchang, Jiangxi, China
      • Recruiting
      • The First Affiliated Hospital of Nanchang University
  • Jilin
    • Changchun, Jilin, China
      • Active, not recruiting
      • The Second Hospital of Jilin University
  • Shandong
    • Weifang, Shandong, China
      • Recruiting
      • Weifang People's Hospital
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Active, not recruiting
      • Ruijin Hospital, Shanghai Jiao Tong University School of Medicine
    • Shanghai, Shanghai Municipality, China
      • Recruiting
      • Shanghai Tongji Hospital
    • Shanghai, Shanghai Municipality, China
      • Recruiting
      • Huadong Hospital Affiliated to Fudan University
    • Shanghai, Shanghai Municipality, China
      • Recruiting
      • Shanghai Fifth People's Hospital
    • Shanghai, Shanghai Municipality, China
      • Active, not recruiting
      • Shanghai General Hospital
    • Shanghai, Shanghai Municipality, China
      • Active, not recruiting
      • Shanghai Pulmonary Hospital
    • Shanghai, Shanghai Municipality, China
      • Active, not recruiting
      • Zhongshan Hospital Affiliated to FuDan University"
  • Sichuan
    • Chengdu, Sichuan, China
      • Recruiting
      • West China Hospital of Sichuan University
    • Chengdu, Sichuan, China
      • Recruiting
      • Chengdu Fifth People's Hospital
    • Chengdu, Sichuan, China
      • Recruiting
      • Sichuan Academy of Medical Sciences · Sichuan Provincial People's Hospital
  • Yunnan
    • Anning, Yunnan, China
      • Active, not recruiting
      • Anning First People's Hospital
    • Kunming, Yunnan, China
      • Active, not recruiting
      • The First Affiliated Hospital of Kunming Medical University
  • Zhejiang
    • Hangzhou, Zhejiang, China
      • Active, not recruiting
      • The Second Affiliated Hospital of Zhejiang University School of Medicine
    • Jiaxing, Zhejiang, China
      • Active, not recruiting
      • Jiaxing Second Hospital
    • Ningbo, Zhejiang, China
      • Recruiting
      • Ningbo Fenghua District People's Hospital
    • Ningbo, Zhejiang, China
      • Active, not recruiting
      • The First Affiliated Hospital of Ningbo University
    • Wenzhou, Zhejiang, China
      • Recruiting
      • The First Affiliated Hospital of Wenzhou Medical University
  • Zhejing
    • Huzhou, Zhejing, China
      • Active, not recruiting
      • Huzhou Central Hospital
    • Jiaxing, Zhejing, China
      • Active, not recruiting
      • Jiaxing First Hospital
  • Zhengzhou
    • Xinxiang, Zhengzhou, China
      • Recruiting
      • The First Affiliated Hospital of Xinxiang Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: Yes

• Sampling Method: Non-Probability Sample

Study Population

Healthy controls will include at least 20 healthy participants aged 30 years or older, with no clinically significant lung disease or chronic lung disease.

A total of up to 300 NCFBE patients (males and females aged 18 years or older) will be recruited and enrolled into the study. Patient numbers are mainly based on feasible situation.

Approximately 30% of NCFBE patients in the study will be required to have had at least 2 exacerbations in the past 12 months.

A subset of 60 NCFBE patients and 20 healthy participants is planned for an optional bronchoscopy assessment.

Description

Inclusion Criteria:

Healthy control cohort:

· Age ≥30 years

Bronchiectasis cohort:

• Capable of giving signed informed consent.
• Participant must be ≥18 years of age, at the time of signing the ICF.
• Able to perform acceptable lung function testing according to ATS/ERS 2019 acceptability criteria.
• Able and willing to comply with the requirements of the protocol including ability to read, write, be fluent in the translated language of all participants facing questionnaires used at center, and use electronic devices (e.g. FENO and spirometry).
• Documented physician-diagnosed bronchiectasis: with a clinical history consistent with bronchiectasis (chronic cough and daily sputum production etc.) and having performed chest HRCT indicating bronchiectasis.
• Remaining clinically stable upon recruitment. Patients with exacerbations are allowed to be enrolled into the study at least 4 weeks after the end of exacerbations. If a patient experiencing exacerbation and directly enters the exacerbation visit, the patient is required to come to the study site for baseline visit (after confirming that all inclusion/exclusion criteria are met) within 4 weeks /+ 3 days after the end of exacerbation

Exclusion Criteria:

Healthy control cohort:

• Any respiratory diagnosis (asthma, COPD, bronchiectasis, pulmonary fibrosis or any other chronic respiratory condition requiring regular treatment).
• Inflammatory conditions including rheumatoid arthritis, inflammatory bowel disease, any other connective tissue disease.
• Active malignancy excluding non-melanoma skin cancer.
• Antibiotic treatment for an acute respiratory tract infection in the previous 4 weeks or current sinusitis.
• Any contraindication to study procedures including bronchoscopy.
• Current smoking or smoking in the preceding 3 months.
• Treatment with anti-coagulants.

Bronchiectasis cohort:

• Traction bronchiectasis associated with interstitial lung disease or other pulmonary disorders (e.g., pulmonary fibrosis and cystic fibrosis).
• Primary diagnosis of another pulmonary condition, including COPD, asthma. Patients with a secondary diagnosis of these pulmonary diseases will be allowed to participate as long as bronchiectasis is considered by the investigator to be the primary diagnosis.
• Any disorder, including, but not limited to, cardiovascular, gastrointestinal, hepatic, renal, neurological, musculoskeletal, infectious, endocrine, metabolic, hematological, psychiatric, or major physical impairment that is not stable in the opinion of the investigator and could:
• Affect the safety of the participant throughout the study
• Influence the findings of the study or the interpretation
• Impede the participant's ability to complete the entire duration of study
• The participant has a history of alcohol or drug abuse within the past year, which, in the opinion of the responsible physician, contra-indicates their participation.
• Active malignancy excluding non-melanoma skin cancer.
• Participants is female who is pregnant or lactating or up to 6 weeks post-partum or 6 weeks cessation of breastfeeding.
• The participant has an altered mental status at the time of informed consent.
• History or current evidence of an upper or lower respiratory infection or symptoms(including common cold) within 2 weeks of baseline assessments
• Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study center).
• Terminal disease and/or organ failure or participants otherwise considered not appropriate for the study participation.

Study Plan

How is the study designed?

Number of groups / cohorts

2

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Healthy control cohort; Healthy controls will include at least 20 healthy participants aged 30 years or older, with no clinically significant lung disease or chronic lung disease.	Other: Participant Follow-up; This is a longitudinal multi-center, observational, translational study which includes patients with a physician diagnosis of NCFBE by chest HRCT and healthy controls (at baseline only). This study will consist of a baseline visit, a 6-month (site visit or telephone visit) and a 12-month visit as well as planned unscheduled visits for exacerbation events and one optional visit for bronchoscopy.Healthy participants will be only enrolled in the baseline visit and bronchoscopy visit.
Bronchiectasis cohort; A total of up to 300 NCFBE patients (males and females aged 18 years or older) will be recruited and enrolled into the study. Patient numbers are mainly based on feasible situation. Approximately 30% of NCFBE patients in the study will be required to have had at least 2 exacerbations in the past 12 months.	Other: Participant Follow-up; This is a longitudinal multi-center, observational, translational study which includes patients with a physician diagnosis of NCFBE by chest HRCT and healthy controls (at baseline only). This study will consist of a baseline visit, a 6-month (site visit or telephone visit) and a 12-month visit as well as planned unscheduled visits for exacerbation events and one optional visit for bronchoscopy.Healthy participants will be only enrolled in the baseline visit and bronchoscopy visit.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
FEV1/FVC%	Evaluation of lung function	12 month
FEF25-75 L/s	Evaluation of small airway function	12 month
FENO ppb	Evaluation of airway inflammation: FENO.	12 month
HRCT	Evaluation of lung structure profile and change through radiological parameters.	12 month
Gene expression read counts by RNAseq	Functional and transcriptional characterization of airway immune cells, bronchial epithelial cells andsmooth muscle cells (optional).	12 month
on-set age(years-old)	Risk factor assessment: on-set age(years-old)	12 month
Sex(M/F)	Risk factor assessment: Sex(M/F)	12 month
body mass index(kg/m^2)	Risk factor assessment: body mass index(kg/m^2)	12 month
comorbidities	Risk factor assessment: comorbidities	12 month
medical history	Risk factor assessment: medical history, especially TB history	12 month
smoking status(never/current/former)	Risk factor assessment: smoking status(never/current/former)	12 month
smoking pack years(pack/year)	Risk factor assessment: smoking pack years(pack/year)	12 month
Sputum microbiology (CFU)	Bronchiectasis aetiology evaluation	12 month
Historical Exacerbation	Risk factor assessment: exacerbation number in the previous year	12 month
BSI score	Evaluation of ronchiectasis disease severity: BSI score(0-4 mild,5-8 moderate, ≥9 severe)	12 month
QoL-B-RSS	Evaluation of quality of life: Patient reported outcome: QoL-B-RSS (0-100, higher score stands for lower symptom burden and higher quality of life)	12 month
BHQ	Evaluation of quality of life: Patient reported outcome: BHQ(10-70,higher score stands for higher symptom burden and pooer quality of life )	12 month
BEST	eDiary: BEST(MCID 4 points may standfor an exacerbation.)	12 month
Treatment pattern	Evaluation of treatment pattern: inhaled antibiotic, macrolide, and mucoactive drugs,etc.	12 month
Exacerbation assessment	Exacerbation assessment: number of exacerbations per patient per year	12 month
cell percentage (%)	Measurement of immune cell (including but not limited to neutrophils and eosinophils) percentages in blood	12 month
Molecular deliverables	MUC5AC/5B in sputum	12 month
Cell counts (10^9/L)	Measurement of immune cell (including but not limited to neutrophils and eosinophils) counts in blood	12 month
Exacerbation assessment about hospitalization	Exacerbation assessment: number of exacerbations lead to hospitalization per patient per year	12 month

Collaborators and Investigators

• Sponsor: AstraZeneca
• Investigators: Principal Investigator: Jinfu Xu, Doctor, Shanghai Tongji Hospital, Tongji University School of Medicine

Study record dates

Study Major Dates

• Study Start (Actual): October 27, 2025
• Primary Completion (Estimated): July 29, 2027
• Study Completion (Estimated): July 29, 2027

Study Registration Dates

• First Submitted: September 29, 2025
• First Submitted That Met QC Criteria: November 17, 2025
• First Posted (Actual): November 24, 2025

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 20, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: NCFBE
• Other Study ID Numbers: D9186R00001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.

Yes, indicates that AZ are accepting requests for IPD, but this does not mean all requests will be shared.

• IPD Sharing Time Frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA PhRMA Data Sharing Principles. For details of our timelines, please rerefer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
• IPD Sharing Access Criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No