Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature Infants

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Bronchopulmonary Dysplasia

Intervention / Treatment
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* Biological: Pneumostem®
* Other: Normal Saline


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 5 Days
Maximum Age: 14 Days

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pneumostem®<br>A single intratracheal administration of Pneumostem® (1.0 x 10^7 cells/kg) | Biological: Pneumostem®<br> <br> * Other names: Human umbilical cord blood-derived mesenchymal stem cells;|
| Placebo Comparator: normal saline<br>A single intratracheal administration of normal saline | Other: Normal Saline<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of BPD (moderate to severe) or mortality at 36 weeks PMA | Incidence of BPD (moderate to severe) or mortality rate at 36 weeks PMA | 36 weeks PMA | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Intubation duration |  | 36 weeks PMA | 
| Incidence of BPD |  | 28-days since birth | 
| Survival rate |  | 28-days since birth, 36 weeks PMA, and termination of the trial | 
| Duration of ventilator dependence |  | Week 24 | 
| Duration of CPAP treatment |  | Week 24 | 
| Postnatal steroid use (%) for the purpose of ventilator weaning |  | Week 24 | 
| Cumulative duration of oxygen use |  | Week 24 | 
| Incidence of Retinopathy of Prematurity (ROP) of Grade III or more |  | Week 24 | 
| Retinopathy of Prematurity (ROP) that require treatment with avastin or laser |  | Week 24 | 
| Growth velocity (Z-score) |  | Week 24 | 
| Length of stay prior to the first discharge from the hospital |  | duration of the hospital stay, an expected average of approximately 3 months since birth | 
| Incidence of adverse events |  | Week 24 | 
| Clinically significant laboratory findings |  | Week 24 | 
| Incidence of pneumothorax that require intubation |  | Week 24 | 
| Incidence of moderate to severe pulmonary hemorrhage |  | Week 24 | 
| Incidence of intraventricular hemorrhage of grade 3 or more |  | Week 24 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Umbilical Cord Blood Derived Mesenchymal Stem Cells, Bronchopulmonary dysplasia, Premature infants