Follow-up Study of Safety and Efficacy in Subjects Who Completed PNEUMOSTEM® Phase II (MP-CR-012) Clinical Trial

This is a follow-up study to investigate the long-term safety and efficacy of PNEUMOSTEM® versus placebo, for the treatment of BPD in premature infants. Subjects who participated in and completed the initial stage of the Phase II trial (NCT03392467) will be followed-up until 60 months of corrected age

Study Overview

• Status: Active, not recruiting
• Conditions: Bronchopulmonary Dysplasia; BPD
• Intervention / Treatment: Biological: PNEUMOSTEM®; Biological: normal saline

Detailed Description

Subjects who completed the initial stage of the Phase II clinical trial will be followed-up at 7 additional visits: 6, 12,18, 24 months corrected age, 36, 48, and 60 months after birth.

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2

Contacts and Locations

Study Locations

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

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 5 years (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Subject who enrolled in phase 2 clinical trial of PNEUMOSTEM® to evaluate efficacy and safety, and were administered with investigational product
• Subject with a written consent form signed by a legal representative or a parent upon explanation of the clinical trial

Exclusion Criteria:

• Subject whose parent or legal representative does not agree to participate in the study
• Subject who is considered inappropriate to participate in the study by the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PNEUMOSTEM®; A single intratracheal administration of Pneumostem® (10.0 x 10^6 cells/kg)	Biological: PNEUMOSTEM®; A single intratracheal administration of PNEUMOSTEM® (1.0 x 10^7 cells/kg); Other Names: Human umbilical cord blood-derived mesenchymal stem cells
Placebo Comparator: normal saline; A single intratracheal administration of normal saline	Biological: normal saline; A single intratracheal administration of normal saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	6 months corrected age
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	12 months corrected age
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection is recorded and compared with control group	18 months corrected age
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	24 months corrected age
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	36 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	39 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	42 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	45 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	48 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	51 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	54 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	57 months after birth
Respiratory outcome: Number of hospitalizations	Total number of hospitalizations and the number of hospital stay due to respiratory infection	60 months after birth

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mortality	check whether patient is alive or dead at evaluation time point	6, 12, 18, 24 months corrected age, 36, 48, and 60 months after birth
Growth measured by Z-score	Growth measured by Z-score	6, 12, 18, 24 months corrected age, 36, 48, and 60 months after birth
Neurological developmental status: Korean Developmental Screening Test for infants and children(K-DST)	Neurological developmental status on Korean Developmental Screening Test for infants and children(K-DST)	6, 12, 18, 24 months corrected age,36, 48, and 60 months after birth
Deafness or Blindness	Deafness: audiometry /Blindness: examination of extraocular muscle, fundus photography, optical biometry	24 months corrected age
Bayley Scales of Infant and Toddler Development (Third Edition)	Scale: cognitive, motor, language, social-emotional, adaptive behavior	18~24months corrected age, 36~42months after birth
Gross Motor Function Classification System for Cerebral Palsy	Gross Motor Function Classification System for Cerebral Palsy	24 months corrected age
Number of adverse events	Number of adverse events	6, 12, 18, 24 months corrected age,36, 48, and 60 months after birth
Number of admissions to Emergency Room (ER)	Total number of admissions to Emergency Room (ER) and number of admission to Emergency Room (ER) due to respiratory infection at each evaluation time point	6, 12, 18, 24months corrected age, 36, 39, 42, 45, 48, 51, 54, 57 and 60 months after birth
Medical treatment records	whether or not, the patient is receiving medical treatment(use of oxygen, steroid, or bronchodilator, diuretics, Sildenafil) and if receiving treatment, the duration of the treatment at each evaluation time point	6, 12, 18, 24months corrected age, 36, 39, 42, 45, 48, 51, 54, 57 and 60 months after birth

Collaborators and Investigators

• Sponsor: Medipost Co Ltd.
• Investigators: Principal Investigator: Wonsoon Park, MD, PhD, Department of Pediatrics, Samsung Medical Center; Principal Investigator: Ai-Rhan Kim, MD, PhD, Department of Neonatology, Asan Medical Center

Study record dates

Study Major Dates

• Study Start (Actual): July 5, 2019
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): June 30, 2027

Study Registration Dates

• First Submitted: June 19, 2019
• First Submitted That Met QC Criteria: June 28, 2019
• First Posted (Actual): July 1, 2019

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 4, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: Premature infants; Bronchopulmonary dysplasia; Human Umbilical Cord Blood Derived Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Lung Injury; Ventilator-Induced Lung Injury; Bronchopulmonary Dysplasia
• Other Study ID Numbers: MP-CR-012-F/U

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No