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

Bronchopulmonary Dysplasia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02023788
• Other study ID #: MP-CR-006-F/U-5y
• Status: Completed
• Start date: April 2014
• Est. completion date: October 2016

Study information

• Verified date: April 2017
• Source: Medipost Co Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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®.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 8
• Est. completion date: October 2016
• Est. primary completion date: October 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: 45 Months to 63 Months

Eligibility

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

Related Conditions & MeSH terms

• Bronchopulmonary Dysplasia
• Premature Birth
• Premature Birth of Newborn
• Respiratory Tract Infections

Intervention

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.

Locations

Country	Name	City	State
Korea, Republic of	Samsung Medical Center	Seoul

Sponsors (1)

Lead Sponsor	Collaborator
Medipost Co Ltd.

Country where clinical trial is conducted

Korea, Republic of, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of subjects with adverse drug reactions	adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam	60 months (corrected age)
Secondary	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)
Secondary	Survival		60 months (corrected age)
Secondary	Z-score	weight; height; head circumference; percentile	60 months (corrected age)
Secondary	Potential neurological development test outcomes	K-ASQ (Korean Ages and Stages Questionnaires),; Bayley test (BSID III)	60 months (corrected age)

External 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