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

NCT02381366 | Completed Phase 1 DMC

• 1 other identifier: MD-BPD-US001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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

Keywords

Mesenchymal Stem Cells; Human Umbilical Cord Blood; Premature Infants; Bronchopulmonary Dysplasia; Chronic Lung Disease; Cell Therapy

Outcome Measures

Primary Outcomes (1)

• Number of participants with adverse reactions for 84 days after treatment

  84 days

Secondary Outcomes (4)

• 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

• Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)

  36 weeks PMA

• 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

• 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

Study Arms (1)

PNEUMOSTEM®

EXPERIMENTAL

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

Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Interventions

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells BIOLOGICAL

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

Also known as: PNEUMOSTEM®

PNEUMOSTEM®

Eligibility Criteria

• Age: 3 Days - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Medipost, Inc.: lead
• Medipost Co Ltd.: collaborator

Study Sites (1)

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Related Publications (1)

• 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.

  PMID: 30992220 DERIVED

MeSH Terms

Conditions

Bronchopulmonary Dysplasia; Premature Birth

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Obstetric Labor, Premature; Obstetric Labor Complications; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases

Study Officials

• Steven Powell, MD

  Rush University Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: PREVENTION
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 15, 2015
• First Posted: March 6, 2015
• Study Start: March 1, 2015
• Primary Completion: September 1, 2016
• Study Completion: May 1, 2018
• Last Updated: August 16, 2018

Record last verified: 2018-08

Locations

US (1)