Treatment of Pediatric Bronchiolitis Obliterans by Airway Basal Stem Cells
ConditionPediatric Bronchiolitis Obliterans
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age28-18
SponsorRegend Therapeutics
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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;
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Airway Basal Stem Cells
Biological/VaccineAirway Basal Stem Cells
Treatment groups
12 Participants
are divided into 1 treatment groupGroup A: Airway Basal Stem CellsExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Change in clinical symptoms
Cough, wheezing, respiratory rate are included in clinical symptomes. Both status and frequency would be evaluated.
Time frame
12 and 24 weeks after treatment2
Changes in oxygen therapy
Oxygen saturation (SpO₂), oxygen therapy settings, and daily duration of oxygen therapy would be evaluated.
Time frame
24 weeks after treatmentSecondary outcomes
1
Changes in general condition
Rate of weight gain, time of independent activity are evaluated as general condition.
Time frame
24 weeks after treatment2
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.
Time frame
12 and 24 weeks after treatment3
Change in forced expiratory volume in one second (FEV1) from baseline
FEV1 is the volume of breath exhaled with effort in one second.
Time frame
12 and 24 weeks after treatment4
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
Time frame
12 and 24 weeks after treatmentSponsors and contacts
Click on the lead sponsor to view all of their trials.
Regend Therapeutics
Lead sponsor
Shanghai Children's Hospital
Collaborator
Source ClinicalTrials.gov