Airway basal stem cell treatment for children with bronchiolitis obliterans

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

This trial will try airway basal stem cell treatment to see if it is safe and helps children (28 days to 18 years) with bronchiolitis obliterans who have severe lung function loss or need ongoing oxygen or ventilator support.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 12 (estimated)
• Ages: 28 Days to 18 Years
• Sex: All
• Sponsor: Regend Therapeutics (Industry-sponsored)
• Locations: 1 site (Shanghai)
• Trial ID: NCT07239895 on ClinicalTrials.gov

What this trial studies

This is an open-label, single-arm Early Phase 1 study testing airway basal stem cell therapy in pediatric bronchiolitis obliterans. The trial is co-sponsored by Regend Therapeutics and Shanghai Children's Hospital and enrolls children able to tolerate bronchoscopy. Participants receive airway basal stem cell intervention and are followed for safety and preliminary signals of benefit including lung function (e.g., FEV1) and dependence on oxygen or mechanical ventilation. The goal is to gather early safety data and initial efficacy evidence to inform future larger studies.

Who should consider this trial

Good fit: Children aged 28 days to 18 years with a guideline diagnosis of bronchiolitis obliterans who have FEV1 <55% predicted or require continuous assisted mechanical ventilation or oxygen and who can tolerate bronchoscopy are the intended candidates.

Not a fit: Children with active infections, certain positive infectious serologies at screening, those on current cGVHD therapy for BOS, or those who cannot tolerate bronchoscopy or have only mild disease are unlikely to benefit from this early-phase intervention.

Why it matters

Potential benefit: If successful, the treatment could reduce airway scarring, improve lung function, and lessen the need for long-term oxygen or ventilator support in affected children.

How similar studies have performed: This airway basal stem cell approach in pediatric BO is largely novel; related stem-cell therapies for other airway diseases have shown limited early-phase signals but no established benefit yet.

Eligibility criteria

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.

Where this trial is running

Shanghai

• Shanghai Children's Hospital, School of Medicine, Shanghai Jiao Tong University — Shanghai, China (Recruiting)

Study contacts

• Study coordinator: Liling Qian, Professor and Chief Physician

Conditions Pediatric Bronchiolitis Obliterans; Airway Basal Stem Cells; Bronchiolitis obliterans; Bronchiolar Epithelium; Pedatric Bronchiolitis Obliterans