Pediatric Bronchiolitis Obliterans
Conditions
Keywords
Airway Basal Stem Cells, Bronchiolitis obliterans, Bronchiolar Epithelium, Pedatric Bronchiolitis Obliterans
Brief summary
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.
Detailed description
The study is co-sponsored by Regend Therapeutics and Shanghai Children's Hospital.
Interventions
BIOLOGICALAirway Basal Stem Cells
Airway Basal Stem Cells
Sponsors
Shanghai Children's Hospital
Regend Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
28 Days to 18 Years
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in clinical symptoms | 12 and 24 weeks after treatment | Cough, wheezing, respiratory rate are included in clinical symptomes. Both status and frequency would be evaluated. |
| Changes in oxygen therapy | 24 weeks after treatment | Oxygen saturation (SpO₂), oxygen therapy settings, and daily duration of oxygen therapy would be evaluated. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in forced expiratory volume in one second (FEV1) from baseline | 12 and 24 weeks after treatment | FEV1 is the volume of breath exhaled with effort in one second. |
| Change in forced vital capacity (FVC) from baseline | 12 and 24 weeks after treatment | FVC is the full amount of air that can be exhaled with effort in a complete breath |
| Changes in general condition | 24 weeks after treatment | Rate of weight gain, time of independent activity are evaluated as general condition. |
| Bronchiolitis Obliterans (BO) exacerbation | 24 weeks after treatment | Frequency and severity of events will be evaluated |
| Change in high resolution computed tomography (HRCT) from baseline | 24 weeks after treatment | HRCT scan is a type of CT scan that shows detailed pictures of lungs |
| Change in lung diffusing capacity for carbon monoxide (DLCO) from baseline | 12 and 24 weeks after treatment | DLCO is a measure of the conductance of CO across the alveolar-capillary membrane and its binding with hemoglobin. |
Countries
China
Contacts
Primary ContactLiling Qian, Professor and Chief Physician
Outcome results
None listed