Clinical trial NCT03392467: PNEUMOSTEM for the Prevention and Treatment of Severe BPD in Premature Infants
Official title: A Multi-center, Randomized, Double-blind, Parallel, Placebo-controlled Phase II Clinical Trial to Evaluate the Efficacy and Safety of PNEUMOSTEM for the Prevention and Treatment of Severe Bronchopulmonary Dysplasia in Premature Infants
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Severe Bronchopulmonary Dysplasia
Lead sponsor: Medipost Co Ltd.
Sponsor class: INDUSTRY
First posted: 2018-01-08
Enrollment: 60
Sex: ALL
Maximum age (years): 13
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: PREVENTION

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.

Eligibility criteria: 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

Primary outcomes:
measure: Percentage of subjects who have severe BPD or are dead
timeFrame: 36 weeks postmenstrual age (PMA)
Secondary outcomes:
measure: Percentage of subjects who have moderate/severe BPD or are dead
timeFrame: 36 weeks PMA
measure: Percentage of subjects by severity of BPD
timeFrame: prenatal 28 days/36 weeks PMA
measure: Percentage of subjects in death due to lung disease
timeFrame: prenatal 28 days/36 weeks PMA and study end timepoint
measure: intubation duration
timeFrame: up to 24 weeks
measure: ventilation duration
timeFrame: up to 24 weeks
measure: continuous positive airway pressure (CPAP) treatment duration
timeFrame: up to 24 weeks
measure: treatment duration with supplemental oxygen
timeFrame: up to 24 weeks
measure: % of subjects treated with steroid for weaning ventilator
timeFrame: up to 24 weeks
measure: Retinopathy of prematurity (ROP) with stage III or higher
timeFrame: up to 24 weeks
measure: number of subjects with retinopathy of prematurity that needs bevacizumab or laser therapy
timeFrame: up to 24 weeks
measure: z-score
timeFrame: up to 24 weeks (visit 10)
measure: days in hospitalization
timeFrame: up to 24 weeks
measure: changes in tracheal suction fluid examination
timeFrame: from screening to 7 days after IP administration (visit 5)
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Asan medical Center
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
affiliation: Samsung Medical Center
name: Wonsoon Park
role: PRINCIPAL_INVESTIGATOR
affiliation: Asan Medical Center
name: Airhan Kim
role: PRINCIPAL_INVESTIGATOR
References:
citation: Ahn SY, Chang YS, Lee MH, Sung SI, Lee BS, Kim KS, Kim AR, Park WS. Stem cells for bronchopulmonary dysplasia in preterm infants: A randomized controlled phase II trial. Stem Cells Transl Med. 2021 Aug;10(8):1129-1137. doi: 10.1002/sctm.20-0330. Epub 2021 Apr 20.