Bronchopulmonary Dysplasia
Conditions
Keywords
Mesenchymal Stem Cells, Human Umbilical Cord Blood, Premature Infants, Bronchopulmonary Dysplasia, Chronic Lung Disease, Cell Therapy
Brief summary
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.
Interventions
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells: Dose A: 10 million cells per kg / Dose B: 20 million cells per kg
Sponsors
Medipost Co Ltd.
Medipost, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Days to 14 Days
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of participants with adverse reactions for 84 days after treatment | 84 days |
Secondary
| Measure | 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 |
Countries
United States
Outcome results
None listed