Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study

Study Overview
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Brief Summary
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This is a 5-year long-term follow-up study of open label, single-center, phase I clinical trial to evaluate the safety and efficacy of PNEUMOSTEM® in premature infants with bronchopulmonary dysplasia.

Detailed Description
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Bronchopulmonary dysplasia (BPD) is the most common cause of death for prematurely born babies with low birth weights. In addition, many children who recover from this 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. The purpose of the study is to evaluate 3-5 year long term safety and efficacy in patients who completed the earlier part of the phase I clinical trial of PNEUMOSTEM®.

Official Title
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Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study

Conditions
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Bronchopulmonary Dysplasia, Respiratory Tract Infections, Premature Birth of Newborn

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 were enrolled in the 2-year follow-up study (NCT01632475) of phase 1 clinical trial for the safety and efficacy evaluations of PNEUMOSTEM® treatment in premature infants with bronchopulmonary dysplasia Infants with a written consent form signed by a parent or legal guardian Exclusion Criteria: -Infants whose parent or legal guardian does not consent to participate in this follow-up study

Ages Eligible for Study
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Minimum Age: 45 Months
Maximum Age: 63 Months

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 Intervention: Biological: Pneumostem® | Biological: PNEUMOSTEM<br>* A single intratracheal administration Low Dose Group (3 patients): 1.0 x 10^7 cells/kg High Dose Group (6 patients): 2.0 x 10^7 cells/kg * The subjects were administered with Pneumostem in the earlier part of the Phase I study. No drug/biologics will be administered to any subject 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 reactions | adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam | 60 months (corrected age) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Respiratory outcomes | hospital readmission rates and length of stay whether medical interventions such as oxygen, steroid, or bronchodilator therapy was done and duration of the therapy Frequency of Emergency Room visit (total number of visits/ number of visits due to respiratory illnesses) | 60 months (corrected age) | 
| Survival |  | 60 months (corrected age) | 
| Z-score | weight height head circumference percentile | 60 months (corrected age) | 
| Potential neurological development test outcomes | K-ASQ (Korean Ages and Stages Questionnaires), Bayley test (BSID III) | 60 months (corrected age) | 

 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, respiratory tract infection, Premature infants