Clinical trial NCT01632475: Follow-Up Study of Safety and Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia
Official title: Long Term Follow-Up Study of the Safety and Exploratory Efficacy of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Bronchopulmonary Dysplasia
Keywords: Human umbilical cord blood-derived mesenchymal stem cells
Premature infants
Bronchopulmonary dysplasia
Lung injury
Lung diseases
Hyperplasia
Ventilator-induced lung injury
Respiratory lung diseases
Pathologic Processes
Infant, Premature, Diseases
Infant, Newborn, Diseases
Lead sponsor: Samsung Medical Center
Sponsor class: OTHER
Collaborators: name: Medipost Co Ltd.
First posted: 2012-07-03
Enrollment: 9
Sex: ALL
Minimum age (years): 4
Maximum age (years): 2
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Infants who enrolled in the study of phase 1 clinical trial to evaluate the safety and efficacy of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia. (NCT01297205)

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 description: 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.

Eligibility criteria: Inclusion Criteria:

* all Infants who enrolled in the Phase 1 PNEUMOSTEM® clinical trial (NCT01297205)

Exclusion Criteria:

* Infants whose parent or legal guardian did not want to participate in the study

Primary outcomes:
measure: Number of subjects with Adverse Drug Reaction
timeFrame: at corrected age of 21 months (±3 months)
Secondary outcomes:
measure: 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®
timeFrame: at corrected age of 10 months (±2 months) and 21 months (±3 months)
measure: Growth
timeFrame: Corrected gestational age of 4-6months, 8-12months, 18-24months
Other outcomes:
Locations:
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
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:
citation: Ahn SY, Chang YS, Kim JH, Sung SI, Park WS. Two-Year Follow-Up Outcomes of Premature Infants Enrolled in the Phase I Trial of Mesenchymal Stem Cells Transplantation for Bronchopulmonary Dysplasia. J Pediatr. 2017 Jun;185:49-54.e2. doi: 10.1016/j.jpeds.2017.02.061. Epub 2017 Mar 21.