Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study

Completed

• Conditions: Bronchopulmonary Dysplasia; Respiratory Tract Infections; Premature Birth of Newborn
• Interventions: Biological: PNEUMOSTEM

• Registration Number: NCT02023788
• Lead Sponsor: Medipost Co Ltd.

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®.

Study Timeline

• Study First Submitted: 2013-12-17
• Study First Submitted QC: 2013-12-23
• Study First Posted: 2013-12-30
• Study Start: 2014-04
• Primary Completion: 2016-10
• Study Completion: 2016-10
• Status Verified: 2017-04
• Last Update Submitted: 2018-08-30
• Last Update Posted: 2018-09-04

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 8

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Pneumostem®	PNEUMOSTEM	Low Dose Group (3 subjects): 1.0 x 10\^7 cells/kg, High Dose Group (6 subjects): 2.0 x 10\^7 cells/kg Intervention: Biological: Pneumostem®

Primary Outcome Measures

Name	Time	Method
Number of subjects with adverse drug reactions	60 months (corrected age)	adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam

Secondary Outcome Measures

Name	Time	Method
Respiratory outcomes	60 months (corrected age)	* 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)
Survival	60 months (corrected age)
Z-score	60 months (corrected age)	* weight; * height; * head circumference; * percentile
Potential neurological development test outcomes	60 months (corrected age)	* K-ASQ (Korean Ages and Stages Questionnaires),; * Bayley test (BSID III)

Trial Locations

Locations (1)

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of