Severe Bronchopulmonary Dysplasia
Conditions
Brief summary
This study is to evaluate the efficacy and safety of PNEUMOSTEM® for the Prevention and Treatment of Severe Bronchopulmonary Dysplasia (Severe BPD) in Premature Infants. Half of subjects will receive PNEUMOSTEM, while the other half will receive a placebo.
Detailed description
Bronchopulmonary dysplasia (BPD) is a chronic lung disease in which premature infants and it results in significant morbidity and mortality. PNEUMOSTEM is intended to prevent and treat BPD by modulating inflammation and repairing damaged lung tissue in premature infants through paracrine effects.
Interventions
BIOLOGICALPNEUMOSTEM
human umbilical cord blood-derived mesenchymal stem cells
OTHERPlacebo
normal saline
Sponsors
Medipost Co Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
No minimum to 13 Days
Healthy volunteers
No
Inclusion criteria
at screening and randomization 1. 23 weeks to \< 25 weeks of gestational age 2. 500g to 1,250g body weight at birth 3. premature infant within postnatal 13 days of age 4. use ventilator with ventilation rate \>12 breaths/min or oxygen supply \> 25%, or use high frequency ventilator (HFV) at IP administration 1. premature infant within postnatal 5 to 14 days of age 2. No improvement in ventilator setting 24 hours prior to administration of IP
Exclusion criteria
1. subject with cyanotic congenital heart disease or non-cyanotic congenital heart disease that can cause heart failure 2. subject with pulmonary hypoplasia, congenital diaphragmatic hernia, or serious lung malformation such as congenital cystic lung disease 3. subject with chromosome disorder with serious malformation (i.e. Edward syndrome, patau syndrome, Down syndrome, etc.), severe congenital malformation (i.e. hydrocephalus, encephalocele, etc.), or severe congenital infection (i.e., herpes, toxoplasmosis, rubella, syphilis, AIDS, etc.) 4. subject with serious sepsis as active infection or shock due to sepsis 5. subject with grade 3 or 4 of bilateral intraventricular hemorrhage 6. at screening, subject with active pulmonary hemorrhage or active air leak syndrome 7. subject who underwent/will undergo surgery within 72 hours before/after investigational product (IP) administration 8. subject who is expected to be treated with surfactant within 24 hours prior to IP administration 9. subject who is expected to be allergic to gentamicin (Birth mother's allergy for gentamicin will be confirmed). 10. subject who have previously participated in other clinical trials 11. subject who is considered ineligible by investigator due to other medical reasons
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of subjects who have severe BPD or are dead | 36 weeks postmenstrual age (PMA) | Percentage of subjects who have severe BPD or are dead |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of subjects by severity of BPD | prenatal 28 days/36 weeks PMA | Percentage of subjects by severity of BPD |
| Percentage of subjects in death due to lung disease | prenatal 28 days/36 weeks PMA and study end timepoint | Percentage of subjects in death due to lung disease |
| intubation duration | up to 24 weeks | intubation duration |
| ventilation duration | up to 24 weeks | ventilation duration |
| continuous positive airway pressure (CPAP) treatment duration | up to 24 weeks | continuous positive airway pressure (CPAP) treatment duration |
| treatment duration with supplemental oxygen | up to 24 weeks | treatment duration with supplemental oxygen |
| Percentage of subjects who have moderate/severe BPD or are dead | 36 weeks PMA | Percentage of subjects who have moderate/severe BPD or are dead |
| Retinopathy of prematurity (ROP) with stage III or higher | up to 24 weeks | number of subjects with ROP with stage III or higher |
| number of subjects with retinopathy of prematurity that needs bevacizumab or laser therapy | up to 24 weeks | number of subjects with retinopathy of prematurity that needs bevacizumab or laser therapy |
| z-score | up to 24 weeks (visit 10) | percentile for body weight, height, and head circumference |
| days in hospitalization | up to 24 weeks | days in hospitalization |
| changes in tracheal suction fluid examination | from screening to 7 days after IP administration (visit 5) | changes in tracheal suction fluid examination |
| % of subjects treated with steroid for weaning ventilator | up to 24 weeks | % of subjects treated with steroid for weaning ventilator |
Countries
South Korea
Outcome results
None listed