Follow-up Safety and Efficacy Evaluation on Subjects Who Completed PNEUMOSTEM® Phase-II 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 (NCT01828957) 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: corrected age of 6, 12,18, 24, 36, 48, and 60 months.

Official Title
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Follow-up Safety and Efficacy Evaluation on Subjects Who Completed the Initial Stage of PNEUMOSTEM® Phase-II 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 completed the safety and efficacy evaluations in Pneumostem Phase II clinical trial 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: 7 Months
Maximum Age: 7 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® (1.0 x 10^7 cells/kg) | Biological: Pneumostem®<br> <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> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Respiratory outcome: readmission rate and duration of the hospital stay due to respiratory infection |  | 6, 12, 18, 24, 36, 48, and 60 months, corrected age | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Whether the subject is receiving medical treatments and if so, duration of the treatment (use of oxygen, steroid, or brochodilator) |  | 6, 12, 18, 24, 36, 48, and 60 months, corrected age | 
| Number of admissions to Emergency Room | Total number of admissions to Emergency Room and number of admissions to Emergency Room due to resiratory problems | 6, 12, 18, 24, 36, 48, and 60 months, corrected age | 
| Survival |  | 6, 12, 18, 24, 36, 48, and 60 months, corrected age | 
| Growth measured by Z-score |  | 6, 12, 18, 24, 36, 48, and 60 months, corrected age | 
| Neurological developmental status on K-ASQ, Bayley Scale, Gross Motor Function Classification System (GMFCS) for Cerebral Palsy |  | 24 months, corrected age | 
| Deafness or Blindness |  | 24 months, corrected age | 
| Number of adverse events |  | 6, 12, 24,36, 48, and 60 months, corrected age | 
| Significant changes in vital signs |  | 6, 12, 24, 36, 48, and 60 months, corrected age | 
| Significant changes in physical exam |  | 6, 12, 24, 36, 48, and 60 months, corrected age | 

 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