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

Bronchopulmonary Dysplasia Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02381366
• Other study ID #: MD-BPD-US001
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: March 2015
• Est. completion date: May 2018

Study information

• Verified date: August 2018
• Source: Medipost America Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: May 2018
• Est. primary completion date: September 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 14 Days

Eligibility

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

Related Conditions & MeSH terms

• Bronchopulmonary Dysplasia
• Hyperplasia
• Premature Birth

Intervention

Biological:
• Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells: Dose A: 10 million cells per kg / Dose B: 20 million cells per kg

Locations

Country	Name	City	State
United States	Rush University Medical Center	Chicago	Illinois

Sponsors (2)

Lead Sponsor	Collaborator
Medipost America Inc.	Medipost Co Ltd.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with adverse reactions for 84 days after treatment		84 days
Secondary	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
Secondary	Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)		36 weeks PMA
Secondary	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
Secondary	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