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

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.

Study Overview

• Status: Completed
• Conditions: Respiratory Tract Infections; Premature Birth of Newborn; Bronchopulmonary Dysplasia
• Intervention / Treatment: Biological: PNEUMOSTEM

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 Type: Observational
• Enrollment (Actual): 8

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 5 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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

Description

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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort

Intervention / Treatment

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®

Biological: PNEUMOSTEM; A single intratracheal administration Low Dose Group (3 patients): 1.0 x 10^7 cells/kg High Dose Group (6 patients): 2.0 x 10^7 cells/kg * The subjects were administered with Pneumostem in the earlier part of the Phase I study. No drug/biologics will be administered to any subject during this part of the study.; Other Names: human umbilical cord blood-derived mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of subjects with adverse drug reactions	adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam	60 months (corrected age)

Secondary Outcome Measures

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

Collaborators and Investigators

• Sponsor: Medipost Co Ltd.

Publications and helpful links

Helpful Links

• Phase-I: 2 Year Follow-Up Safety and Efficacy Study of Pneumostem® in Premature Infants With Bronchopulmonary Dysplasia
• Phase-I:Safety and Efficacy Evaluation of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia
• Phase-II: Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature Infants

Study record dates

Study Major Dates

• Study Start: April 1, 2014
• Primary Completion (ACTUAL): October 1, 2016
• Study Completion (ACTUAL): October 1, 2016

Study Registration Dates

• First Submitted: December 17, 2013
• First Submitted That Met QC Criteria: December 23, 2013
• First Posted (ESTIMATE): December 30, 2013

Study Record Updates

• Last Update Posted (ACTUAL): September 4, 2018
• Last Update Submitted That Met QC Criteria: August 30, 2018
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Keywords: Premature infants; bronchopulmonary dysplasia; respiratory tract infection; human umbilical cord blood-derived mesenchymal stem cells
• Additional Relevant MeSH Terms: Infections; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Lung Injury; Infant, Premature, Diseases; Ventilator-Induced Lung Injury; Premature Birth; Respiratory Tract Infections; Bronchopulmonary Dysplasia
• Other Study ID Numbers: MP-CR-006-F/U-5y