Brief Title
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)
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.
Study Phase
Phase 1/Phase 2
Study Type
Interventional
Primary Outcome
Number of participants with adverse reactions for 84 days after treatment
Secondary Outcome
Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age
Condition
Bronchopulmonary Dysplasia
Intervention
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Study Arms / Comparison Groups
PNEUMOSTEM®
Description: Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
12
Start Date
March 2015
Completion Date
May 2018
Primary Completion Date
September 2016
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
Gender
All
Ages
N/A - 14 Days
Accepts Healthy Volunteers
No
Contacts
Steven Powell, MD, ,
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT02381366
Organization ID
MD-BPD-US001
Responsible Party
Sponsor
Study Sponsor
Medipost America Inc.
Collaborators
Medipost Co Ltd.
Study Sponsor
Steven Powell, MD, Principal Investigator, Rush University Medical Center
Verification Date
August 2018