Follow-Up Study of Safety and Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia

Study Overview
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Brief Summary
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This is a long term follow-up study of the open label, single-center, phase I clinical trial to evaluate the safety of Pneumostem® in premature infants with BPD.

Detailed Description
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Bronchopulmonary dysplasia (BPD) is the most common cause of death for premature newborns with low birth weights. In addition, many children who recover from the disease suffer from various complications such as prolonged hospitalization, pulmonary hypertension, and failure to thrive. It has been reported that bone marrow-derived mesenchymal stem cells (BM-MSC) can differentiate into pulmonary epithelial and pulmonary endothelial cells. Some animal studies showed that BM-MSCs differentiate into bronchial cells and type 2 pneumocytes in rats with pneumonia and improve the fibrosis that occur after administration of bleomycin. Based on the findings, it is considered that mesenchymal stem cell therapy can help regenerate the damaged lung as well as BPD that cause lung inflammation, fibrosis, deficiency of type 2 pneumocytes, and so on. PNEUMOSTEM® consists of human umbilical cord blood-derived mesenchymal stem cells and is intended to treat BPD in premature infants. This is a long term follow-up study of the earlier part of the phase I clinical trial.

Official Title
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Long Term Follow-Up Study of the Safety and Exploratory Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia

Conditions
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Bronchopulmonary Dysplasia

Intervention / Treatment
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* Biological: Pneumostem®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: all Infants who enrolled in the Phase 1 PNEUMOSTEM® clinical trial (NCT01297205) Exclusion Criteria: Infants whose parent or legal guardian did not want to participate in the study

Ages Eligible for Study
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Minimum Age: 4 Months
Maximum Age: 2 Years

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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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Pneumostem®<br>Low Dose Group (3 subjects): 1.0 x 10^7 cells/kg, High Dose Group (6 subjects): 2.0 x 10^7 cells/kg | Biological: Pneumostem®<br>* A single intratracheal administration Low Dose Group (3 patients): 1.0 x 10^7 cells/kg, High Dose Group (6 patients): 2 x 10^7 cells/kg * The subjects were administered with Pneumostem® in the earlier part of the phase I study. No drugs/biologics are administered during this part of the study.<br>* Other names: Human umbilical cord blood-derived mesenchymal stem cells;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of subjects with Adverse Drug Reaction | Blood test, chest x-ray, physical exam | at corrected age of 21 months (±3 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Neurological development test outcome from the subjects who were treated with Pneumostem®, compared with the patients who suffered from the same conditions but not treated with Pneumostem® | Bayely test results of the 9 subjects who were treated with Pneumostem® during the early part of the Phase I study. The results of Brain MRI study performed at corrected age of 18-24 months. | at corrected age of 10 months (±2 months) and 21 months (±3 months) | 
| Growth | Body weight, Head circumference, Height : growth percentile | Corrected gestational age of 4-6months, 8-12months, 18-24months | 

 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, Premature infants, Bronchopulmonary dysplasia, Lung injury, Lung diseases, Hyperplasia, Ventilator-induced lung injury, Respiratory lung diseases, Pathologic Processes, Infant, Premature, Diseases, Infant, Newborn, Diseases