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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Observational

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

Brief Summary: 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 Summary:

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.


Sponsor: Samsung Medical Center

Current Primary Outcome: Number of subjects with Adverse Drug Reaction [ Time Frame: at corrected age of 21 months (±3 months) ]

Blood test, chest x-ray, physical exam


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • 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) ]

    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 ]
    Body weight, Head circumference, Height : growth percentile


Original Secondary Outcome: Better 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) ]

Exploratory efficacy will be evaluated based on the comparative analysis of the Bayely test results of the 9 subjects who were treated with Pneumostem® during the early part of the Phase I study and a group of 45 patients who were born prematurely and suffered from the same respiratory conditions but not treated with Pneumostem®.


Information By: Samsung Medical Center

Dates:
Date Received: June 27, 2012
Date Started: September 2011
Date Completion: September 2017
Last Updated: October 12, 2016
Last Verified: October 2016