Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study
Phase 1
Completed
- Conditions
- Bronchopulmonary Dysplasia
- Interventions
- Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
- Registration Number
- NCT02381366
- Lead Sponsor
- Medipost America Inc.
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 12
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description PNEUMOSTEM® Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg
- Primary Outcome Measures
Name Time Method Number of participants with adverse reactions for 84 days after treatment 84 days
- Secondary Outcome Measures
Name Time Method 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 Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA) 36 weeks PMA 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 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
Trial Locations
- Locations (1)
Rush University Medical Center
🇺🇸Chicago, Illinois, United States