Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature Infants

Randomized, Double-blind, Multi-center, Phase II Clinical Trial to Evaluate the Efficacy and Safety of Pneumostem® Versus a Control Group for Treatment of Bronchopulmonary Dysplasia in Premature Infants

The objective of this study is to evaluate the efficacy and safety of a single intratracheal administration of Pneumostem® for treatment of Bronchopulmonary Dysplasia (BPD) in high-risk premature infants by comparing Pneumostem-treated group with a control group.

Study Overview

• Status: Completed
• Conditions: Bronchopulmonary Dysplasia
• Intervention / Treatment: Other: Normal Saline; Biological: Pneumostem®

• Study Type: Interventional
• Enrollment (Actual): 69
• Phase: Phase 2

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of
    • Asan Medical Center
  • Seoul, Korea, Republic of
    • Samsung Medical Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 5 days to 2 weeks (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Age: 5 - 14 days since birth
• Fetal gestational age: ≥23 weeks and <29 weeks
• Birth weight: ≥500g and ≤1250g
• Premature infant of equal to or less than 2 weeks of age who is receiving a ventilator therapy at a rate of > 12 breath/min and > 25% oxygen
• Patient whose ventilator setting has not been changed and who has shown aggravation of the illness within the 24 hours prior to the study enrollment
• Patient with a written consent form signed by a legal representative or a parent upon explanation of the clinical trial

Exclusion Criteria:

• Patient with concurrent cyanotic or acyanotic congenital heart diseases, except for patent ductus arteriosus
• Patient with a concurrent severe lung malformation (i.e. Pulmonary hypoplasia, congenital diaphragmatic hernia, congenital cystic lung disease)
• Patient with a concurrent severe lung malformation with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
• Patient with a concurrent severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
• Patient withCRP > 30 mg/dL; Severe sepsis or shock
• Patient who is scheduled for or expected to undergo a surgical procedure 72 hours prior to/following the administration of the study drug
• Patient who has been administered with a surfactant within the 24 hours prior to the administration of the study drug
• Patient with severe intracranial hemorrhage ≥ grade 3 or 4
• Patient with active pulmonary hemorrhage or active air leak syndrome at the time of screening
• Patient with a history of participating in other clinical studies
• Patient who is allergic to Gentamicin
• Patient who is considered inappropriate to participate in the study by the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: PARALLEL
• Masking: QUADRUPLE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Pneumostem®; A single intratracheal administration of Pneumostem® (1.0 x 10^7 cells/kg)	Biological: Pneumostem®; Other Names: Human umbilical cord blood-derived mesenchymal stem cells
PLACEBO_COMPARATOR: normal saline; A single intratracheal administration of normal saline	Other: Normal Saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence of BPD (moderate to severe) or mortality at 36 weeks PMA	Incidence of BPD (moderate to severe) or mortality rate at 36 weeks PMA	36 weeks PMA

Secondary Outcome Measures

Outcome Measure	Time Frame
Intubation duration	36 weeks PMA
Incidence of BPD	28-days since birth
Survival rate	28-days since birth, 36 weeks PMA, and termination of the trial
Duration of ventilator dependence	Week 24
Duration of CPAP treatment	Week 24
Postnatal steroid use (%) for the purpose of ventilator weaning	Week 24
Cumulative duration of oxygen use	Week 24
Incidence of Retinopathy of Prematurity (ROP) of Grade III or more	Week 24
Retinopathy of Prematurity (ROP) that require treatment with avastin or laser	Week 24
Growth velocity (Z-score)	Week 24
Length of stay prior to the first discharge from the hospital	duration of the hospital stay, an expected average of approximately 3 months since birth
Incidence of adverse events	Week 24
Clinically significant laboratory findings	Week 24
Incidence of pneumothorax that require intubation	Week 24
Incidence of moderate to severe pulmonary hemorrhage	Week 24
Incidence of intraventricular hemorrhage of grade 3 or more	Week 24

Collaborators and Investigators

• Sponsor: Medipost Co Ltd.
• Investigators: Principal Investigator: Won-Soon Park, MD, PhD, Department of Pediatrics, Samsung Medical Center; Principal Investigator: Ai-Rhan Kim, Department of Neonatology, Asan Medical Center

Publications and helpful links

General Publications

• Ahn SY, Chang YS, Lee MH, Sung SI, Lee BS, Kim KS, Kim AR, Park WS. Stem cells for bronchopulmonary dysplasia in preterm infants: A randomized controlled phase II trial. Stem Cells Transl Med. 2021 Aug;10(8):1129-1137. doi: 10.1002/sctm.20-0330. Epub 2021 Apr 20.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start: April 1, 2013
• Primary Completion (ACTUAL): May 1, 2015
• Study Completion (ACTUAL): August 1, 2015

Study Registration Dates

• First Submitted: April 2, 2013
• First Submitted That Met QC Criteria: April 8, 2013
• First Posted (ESTIMATE): April 11, 2013

Study Record Updates

• Last Update Posted (ACTUAL): September 6, 2019
• Last Update Submitted That Met QC Criteria: September 4, 2019
• Last Verified: April 1, 2017

More Information

Terms related to this study

• Keywords: Premature infants; Bronchopulmonary dysplasia; Human Umbilical Cord Blood Derived Mesenchymal Stem Cells
• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Pregnancy Complications; Obstetric Labor Complications; Obstetric Labor, Premature; Lung Injury; Infant, Premature, Diseases; Ventilator-Induced Lung Injury; Premature Birth; Bronchopulmonary Dysplasia
• Other Study ID Numbers: MP-CR-009