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

Study Overview
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Brief Summary
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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

Detailed Description
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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.

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

Conditions
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Bronchopulmonary Dysplasia

Intervention / Treatment
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* Biological: PNEUMOSTEM®
* Biological: normal saline


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 60 Months

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

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

What is the study measuring?
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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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Umbilical Cord Blood Derived Mesenchymal Stem Cells, Bronchopulmonary dysplasia, Premature infants