Treatment of Pediatric Bronchiolitis Obliterans by Airway Basal Stem Cells

Regend Therapeutics

Status and phase

Enrolling

Early Phase 1

Conditions

Pediatric Bronchiolitis Obliterans

Treatments

Biological: Airway Basal Stem Cells

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT07239895

BO-V1.2

Details and patient eligibility

About

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.

Full description

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

Enrollment

12 estimated patients

Sex

All

Ages

28 days to 18 years old

Volunteers

No Healthy Volunteers

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.