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

NCT ID: NCT02381366

Last Updated: 2018-08-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-31
• Study Completion Date: 2018-05-31

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.

Conditions

Bronchopulmonary Dysplasia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

PNEUMOSTEM®

Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg

Group Type: EXPERIMENTAL

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Intervention Type: BIOLOGICAL

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells:

Dose A: 10 million cells per kg / Dose B: 20 million cells per kg

Interventions

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

Intervention Type: BIOLOGICAL

Other Intervention Names

PNEUMOSTEM®

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

• Minimum Eligible Age: 3 Days
• Maximum Eligible Age: 14 Days
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Medipost Co Ltd.

INDUSTRY

Sponsor Role: collaborator

Medipost America Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Steven Powell, MD

Role: PRINCIPAL_INVESTIGATOR

Rush University Medical Center

Locations

Rush University Medical Center

Chicago, Illinois, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 30992220 (View on PubMed)

Other Identifiers

MD-BPD-US001

Identifier Type: -

Identifier Source: org_study_id