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.
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®.
Study Type
OBSERVATIONAL
Enrollment
8
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.
Samsung Medical Center
Seoul, South Korea
Number of subjects with adverse drug reactions
adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam
Time frame: 60 months (corrected age)
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)
Time frame: 60 months (corrected age)
Survival
Time frame: 60 months (corrected age)
Z-score
* weight * height * head circumference * percentile
Time frame: 60 months (corrected age)
Potential neurological development test outcomes
* K-ASQ (Korean Ages and Stages Questionnaires), * Bayley test (BSID III)
Time frame: 60 months (corrected age)
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