Clinical trial NCT02381366: Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study
Official title: 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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Bronchopulmonary Dysplasia
Keywords: Mesenchymal Stem Cells
Human Umbilical Cord Blood
Premature Infants
Bronchopulmonary Dysplasia
Chronic Lung Disease
Cell Therapy
Lead sponsor: Medipost America Inc.
Sponsor class: INDUSTRY
Collaborators: name: Medipost Co Ltd.
First posted: 2015-03-06
Enrollment: 12
Sex: ALL
Minimum age (years): 3
Maximum age (years): 14
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with adverse reactions for 84 days after treatment
timeFrame: 84 days
Secondary outcomes:
measure: Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age
timeFrame: Between 84 days after treatment and 20 months of corrected age
measure: Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)
timeFrame: 36 weeks PMA
measure: Hospital Re-admission between 84 days after treatment until 20 months of corrected age
timeFrame: Between 84 days after treatment and 20 months of corrected age
measure: Bayley Scales of Infant and Toddler Development between 84 days after treatment until 20 months of corrected age
timeFrame: Between 84 days after treatment and 20 months of corrected age
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
Study officials:
affiliation: Rush University Medical Center
name: Steven Powell, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Powell SB, Silvestri JM. Safety of Intratracheal Administration of Human Umbilical Cord Blood Derived Mesenchymal Stromal Cells in Extremely Low Birth Weight Preterm Infants. J Pediatr. 2019 Jul;210:209-213.e2. doi: 10.1016/j.jpeds.2019.02.029. Epub 2019 Apr 13.