Clinical trial NCT02023788: Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study
Official title: Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Bronchopulmonary Dysplasia
Respiratory Tract Infections
Premature Birth of Newborn
Keywords: human umbilical cord blood-derived mesenchymal stem cells
bronchopulmonary dysplasia
respiratory tract infection
Premature infants
Lead sponsor: Medipost Co Ltd.
Sponsor class: INDUSTRY
First posted: 2013-12-30
Enrollment: 8
Sex: ALL
Minimum age (years): 45
Maximum age (years): 63
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: 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

Brief summary: 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: 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®.

Eligibility criteria: 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

Primary outcomes:
measure: Number of subjects with adverse drug reactions
timeFrame: 60 months (corrected age)
Secondary outcomes:
measure: Respiratory outcomes
timeFrame: 60 months (corrected age)
measure: Survival
timeFrame: 60 months (corrected age)
measure: Z-score
timeFrame: 60 months (corrected age)
measure: Potential neurological development test outcomes
timeFrame: 60 months (corrected age)
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
Study officials:
affiliation: Samsung Medical Center
name: Won-Soon Park, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: