Clinical trial NCT01828957: Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature Infants
Official title: Randomized, Double-blind, Multi-center, Phase II Clinical Trial to Evaluate the Efficacy and Safety of Pneumostem® Versus a Control Group for Treatment of Bronchopulmonary Dysplasia in Premature Infants
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Bronchopulmonary Dysplasia
Keywords: Human Umbilical Cord Blood Derived Mesenchymal Stem Cells
Bronchopulmonary dysplasia
Premature infants
Lead sponsor: Medipost Co Ltd.
Sponsor class: INDUSTRY
First posted: 2013-04-11
Enrollment: 69
Sex: ALL
Minimum age (years): 5
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of this study is to evaluate the efficacy and safety of a single intratracheal administration of Pneumostem® for treatment of Bronchopulmonary Dysplasia (BPD) in high-risk premature infants by comparing Pneumostem-treated group with a control group.


Eligibility criteria: Inclusion Criteria:

* Age: 5 - 14 days since birth
* Fetal gestational age: ≥23 weeks and \<29 weeks
* Birth weight: ≥500g and ≤1250g
* Premature infant of equal to or less than 2 weeks of age who is receiving a ventilator therapy at a rate of \> 12 breath/min and \> 25% oxygen
* Patient whose ventilator setting has not been changed and who has shown aggravation of the illness within the 24 hours prior to the study enrollment
* Patient with a written consent form signed by a legal representative or a parent upon explanation of the clinical trial

Exclusion Criteria:

* Patient with concurrent cyanotic or acyanotic congenital heart diseases, except for patent ductus arteriosus
* Patient with a concurrent severe lung malformation (i.e. Pulmonary hypoplasia, congenital diaphragmatic hernia, congenital cystic lung disease)
* Patient with a concurrent severe lung malformation with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
* Patient with a concurrent severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
* Patient withCRP \> 30 mg/dL; Severe sepsis or shock
* Patient who is scheduled for or expected to undergo a surgical procedure 72 hours prior to/following the administration of the study drug
* Patient who has been administered with a surfactant within the 24 hours prior to the administration of the study drug
* Patient with severe intracranial hemorrhage ≥ grade 3 or 4
* Patient with active pulmonary hemorrhage or active air leak syndrome at the time of screening
* Patient with a history of participating in other clinical studies
* Patient who is allergic to Gentamicin
* Patient who is considered inappropriate to participate in the study by the investigator

Primary outcomes:
measure: Incidence of BPD (moderate to severe) or mortality at 36 weeks PMA
timeFrame: 36 weeks PMA
Secondary outcomes:
measure: Intubation duration
timeFrame: 36 weeks PMA
measure: Incidence of BPD
timeFrame: 28-days since birth
measure: Survival rate
timeFrame: 28-days since birth, 36 weeks PMA, and termination of the trial
measure: Duration of ventilator dependence
timeFrame: Week 24
measure: Duration of CPAP treatment
timeFrame: Week 24
measure: Postnatal steroid use (%) for the purpose of ventilator weaning
timeFrame: Week 24
measure: Cumulative duration of oxygen use
timeFrame: Week 24
measure: Incidence of Retinopathy of Prematurity (ROP) of Grade III or more
timeFrame: Week 24
measure: Retinopathy of Prematurity (ROP) that require treatment with avastin or laser
timeFrame: Week 24
measure: Growth velocity (Z-score)
timeFrame: Week 24
measure: Length of stay prior to the first discharge from the hospital
timeFrame: duration of the hospital stay, an expected average of approximately 3 months since birth
measure: Incidence of adverse events
timeFrame: Week 24
measure: Clinically significant laboratory findings
timeFrame: Week 24
measure: Incidence of pneumothorax that require intubation
timeFrame: Week 24
measure: Incidence of moderate to severe pulmonary hemorrhage
timeFrame: Week 24
measure: Incidence of intraventricular hemorrhage of grade 3 or more
timeFrame: Week 24
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: Department of Pediatrics, Samsung Medical Center
name: Won-Soon Park, MD, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Department of Neonatology, Asan Medical Center
name: Ai-Rhan 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.