Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study

Study Overview
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Brief Summary
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PNEUMOSTEM® consists of ex vivo cultured allogeneic, unrelated, human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) and it is intended for use as a cellular therapy product for prevention of Bronchopulmonary Dysplasia (BPD). This study is an open-label, single-center, dose escalation study to evaluate of safety and efficacy of PNEUMOSTEM® in premature infants at high risk for BPD.

Official Title
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A Phase I/II, Open-Label Dose Escalation Trial to Evaluate the Safety and Efficacy of Two Dose Levels of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD)

Conditions
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Bronchopulmonary Dysplasia

Intervention / Treatment
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* Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A male or female infant whose postnatal age is 3 to 14 days, inclusive (for treatment between 5 and 14 days after birth) A subject whose gestational age is between 23 and 28 weeks (23 weeks ≤ gestational age (GA) < 28 weeks) A subject whose birth weight is between 500g and 1000g, inclusive A subject who is intubated and receiving mechanical ventilation within 5-14 days after birth, with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening A subject who has had either a deterioration or no change in the setting of mechanical ventilation within the 24 hours before trial enrollment A subject whose parent/guardian can give a written informed consent Exclusion Criteria: A subject who has a congenital heart defect, except for patent ductus arteriosus (PDA), atrial septal defect (ASD) or a small, restrictive ventricular septal defect (VSD) A subject who has a serious malformation of the lung such as pulmonary hypoplasia/aplasia congenital diaphragmatic hernia, or other congenital lung anomaly A subject who has a chromosomal abnormality (e.g., Trisomy 18, Trisomy 13 or Trisomy 21) or a severe congenital malformation (e.g., hydrocephalus and encephalocele, tracheo-esophageal fistula, abdominal wall defects, and major renal anomalies) A subject who has had a severe congenital infectious disease (i.e., herpes, toxoplasmosis rubella, syphilis, HIV, etc.) A subject who has evidence of severe sepsis or septic shock due to an active infection at Screening A subject who underwent a surgical procedure within 72 hours before study drug administration or who is anticipated to have a surgical procedure within 72 hours before or following study drug administration A subject who was administered surfactant within 24 hours before study drug administration A subject who has had a bilateral grade 3 or 4 intracranial hemorrhage A subject who has active pulmonary hemorrhage or an active air leak syndrome at Screening A subject who is currently participating in any other interventional clinical trial A subject who is, in the opinion of the Principal Investigator, considered inappropriate for the trial due to any reasons other than those listed above

Ages Eligible for Study
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Minimum Age: 3 Days
Maximum Age: 14 Days

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: Prevention
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: PNEUMOSTEM®<br>Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg | Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells<br>* Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells: Dose A: 10 million cells per kg / Dose B: 20 million cells per kg<br>* Other names: PNEUMOSTEM®;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with adverse reactions for 84 days after treatment |  | 84 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age |  | Between 84 days after treatment and 20 months of corrected age | 
| Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA) |  | 36 weeks PMA | 
| Hospital Re-admission between 84 days after treatment until 20 months of corrected age |  | Between 84 days after treatment and 20 months of corrected age | 
| Bayley Scales of Infant and Toddler Development between 84 days after treatment until 20 months of corrected age |  | Between 84 days after treatment and 20 months of corrected age | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Mesenchymal Stem Cells, Human Umbilical Cord Blood, Premature Infants, Bronchopulmonary Dysplasia, Chronic Lung Disease, Cell Therapy