Follow-Up Study of Safety and Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia
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.
|Study Design:||Observational Model: Case-Only
Time Perspective: Prospective
|Official Title:||Long Term Follow-Up Study of the Safety and Exploratory Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia|
- Number of subjects with Adverse Drug Reaction [ Time Frame: at corrected age of 21 months (±3 months) ] [ Designated as safety issue: Yes ]Blood test, chest x-ray, physical exam
- 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® [ Time Frame: at corrected age of 10 months (±2 months) and 21 months (±3 months) ] [ Designated as safety issue: No ]
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.
- Growth [ Time Frame: Corrected gestational age of 4-6months, 8-12months, 18-24months ] [ Designated as safety issue: No ]Body weight, Head circumference, Height : growth percentile
|Study Start Date:||September 2011|
|Estimated Study Completion Date:||September 2017|
|Estimated Primary Completion Date:||September 2017 (Final data collection date for primary outcome measure)|
Low Dose Group (3 subjects): 1.0 x 10^7 cells/kg, High Dose Group (6 subjects): 2.0 x 10^7 cells/kg
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.
Other Name: Human umbilical cord blood-derived mesenchymal stem cells
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01632475
|Korea, Republic of|
|Samsung Medical Center|
|Seoul, Korea, Republic of|
|Samsung Medical Center|
|Seoul, Korea, Republic of, 135-710|
|Principal Investigator:||Won-Soon Park, MD, PhD||Samsung Medical Center|