Treatment of Pediatric Bronchiolitis Obliterans by Airway Basal Stem Cells

Exploratory Study of Airway Basal Stem Cells on Treatment of Pediatric Bronchiolitis Obliterans

Bronchiolitis obliterans (BO) is a chronic lung disease which was initiated with injury of the bronchiolar epithelium and resulted in nonuniform luminal obliteration or narrowing. Among children, the most common form of BO is post-infectious BO with a lack of treatment guidelines or standard therapy. In this study, an open, single-armed study is performed to preliminarily evaluate the safety and efficacy of airway basal stem cells on treatment of pediatric BO.

Study Overview

• Status: Recruiting
• Conditions: Pediatric Bronchiolitis Obliterans
• Intervention / Treatment: Biological: Airway Basal Stem Cells

Detailed Description

The study is co-sponsored by Regend Therapeutics and Shanghai Children's Hospital.

• Study Type: Interventional
• Enrollment (Estimated): 12
• Phase: Early Phase 1

Contacts and Locations

• Study Contact: Name: Liling Qian, Professor and Chief Physician; Phone Number: 086-021-62474880; Email: qianliling@shchildren.com.cn

Study Locations

• China
  • Shanghai, China, 200000
    • Recruiting
    • Shanghai Children's Hospital, School of Medicine, Shanghai Jiao Tong University
    • Contact: Lei He; Phone Number: 086-021-52976581; Email: hl@shchildren.com.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Males and females, aged between 28 days and 18 years;
• Diagnosed with bronchiolitis obliterans according to the guidelines;
• Meeting at least one of the following: FEV1 < 55% of predicted value; requiring continuous assisted mechanical ventilation or oxygen therapy;
• None of acute infections within the past four weeks;
• Tolerating bronchoscopy;
• The child and/or parent(s) provide informed consent, and are able to understand and adhere to scheduled visits, treatments, laboratory tests, and other study procedures.

Exclusion Criteria:

• Subjects with bronchiolitis obliterans syndrome (BOS) who are on a current cGVHD treatment regimen at screening.
• At the time of screening, subject who is positive in each of treponema pallidum antibody (TP-Ab), human immunodeficiency virus (HIV) antibody, hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody test. Hepatitis B virus carriers with stable current condition can be enrolled. Cured hepatitis C patients with negative result in HCV ribonucleic acid (RNA) test can be enrolled as well.
• Subject who is assessed to have major lung diseases other than BO by investigators at screening or who has other severe systemic diseases within 6 months prior to screening and is considered to be unsuitable for this study by investigators.
• Presence of severe coagulation dysfunction at screening and may compromise the safety of bronchoscopy in the investigator's judgment.
• Subjects requiring long-term maintenance anticoagulant therapy or antiplatelet aggregation therapy, and for whom, in the investigator's assessment, the medication cannot be discontinued within a week prior to cell collection and infusion.
• Subjects with suicide risk or a history of psychiatric disorders at screening.
• Participation in another interventional clinical study within 3 months prior to screening.
• Poor compliance, making him or her difficult to complete the study.
• Subjects who is considered to be unsuitable for this study in in the opinion of the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Airway Basal Stem Cells	Biological: Airway Basal Stem Cells; Airway Basal Stem Cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in clinical symptoms	Cough, wheezing, respiratory rate are included in clinical symptomes. Both status and frequency would be evaluated.	12 and 24 weeks after treatment
Changes in oxygen therapy	Oxygen saturation (SpO₂), oxygen therapy settings, and daily duration of oxygen therapy would be evaluated.	24 weeks after treatment

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in general condition	Rate of weight gain, time of independent activity are evaluated as general condition.	24 weeks after treatment
Change in lung diffusing capacity for carbon monoxide (DLCO) from baseline	DLCO is a measure of the conductance of CO across the alveolar-capillary membrane and its binding with hemoglobin.	12 and 24 weeks after treatment
Change in forced expiratory volume in one second (FEV1) from baseline	FEV1 is the volume of breath exhaled with effort in one second.	12 and 24 weeks after treatment
Change in forced vital capacity (FVC) from baseline	FVC is the full amount of air that can be exhaled with effort in a complete breath	12 and 24 weeks after treatment
Change in high resolution computed tomography (HRCT) from baseline	HRCT scan is a type of CT scan that shows detailed pictures of lungs	24 weeks after treatment
Bronchiolitis Obliterans (BO) exacerbation	Frequency and severity of events will be evaluated	24 weeks after treatment

Collaborators and Investigators

• Sponsor: Regend Therapeutics
• Collaborators: Shanghai Children's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): November 11, 2025
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027

Study Registration Dates

• First Submitted: November 16, 2025
• First Submitted That Met QC Criteria: November 16, 2025
• First Posted (Estimated): November 20, 2025

Study Record Updates

• Last Update Posted (Actual): January 14, 2026
• Last Update Submitted That Met QC Criteria: January 12, 2026
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: Bronchiolitis obliterans; Airway Basal Stem Cells; Bronchiolar Epithelium; Pedatric Bronchiolitis Obliterans
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Bronchial Diseases; Lung Diseases, Obstructive; Bronchiolitis; Bronchitis; Bronchiolitis Obliterans
• Other Study ID Numbers: BO-V1.2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

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