PNEUMOSTEM for the Prevention and Treatment of Severe BPD in Premature Infants

A Multi-center, Randomized, Double-blind, Parallel, Placebo-controlled Phase II Clinical Trial to Evaluate the Efficacy and Safety of PNEUMOSTEM for the Prevention and Treatment of Severe Bronchopulmonary Dysplasia in Premature Infants

This study is to evaluate the efficacy and safety of PNEUMOSTEM® for the Prevention and Treatment of Severe Bronchopulmonary Dysplasia (Severe BPD) in Premature Infants. Half of subjects will receive PNEUMOSTEM, while the other half will receive a placebo.

Study Overview

• Status: Completed
• Conditions: Severe Bronchopulmonary Dysplasia
• Intervention / Treatment: Other: Placebo; Biological: PNEUMOSTEM

Detailed Description

Bronchopulmonary dysplasia (BPD) is a chronic lung disease in which premature infants and it results in significant morbidity and mortality. PNEUMOSTEM is intended to prevent and treat BPD by modulating inflammation and repairing damaged lung tissue in premature infants through paracrine effects.

• Study Type: Interventional
• Enrollment (Actual): 60
• 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: No older than 1 week (Child)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

at screening and randomization

1. 23 weeks to < 25 weeks of gestational age
2. 500g to 1,250g body weight at birth
3. premature infant within postnatal 13 days of age
4. use ventilator with ventilation rate >12 breaths/min or oxygen supply > 25%, or use high frequency ventilator (HFV)

at IP administration

1. premature infant within postnatal 5 to 14 days of age
2. No improvement in ventilator setting 24 hours prior to administration of IP

Exclusion Criteria:

1. subject with cyanotic congenital heart disease or non-cyanotic congenital heart disease that can cause heart failure
2. subject with pulmonary hypoplasia, congenital diaphragmatic hernia, or serious lung malformation such as congenital cystic lung disease
3. subject with chromosome disorder with serious malformation (i.e. Edward syndrome, patau syndrome, Down syndrome, etc.), severe congenital malformation (i.e. hydrocephalus, encephalocele, etc.), or severe congenital infection (i.e., herpes, toxoplasmosis, rubella, syphilis, AIDS, etc.)
4. subject with serious sepsis as active infection or shock due to sepsis
5. subject with grade 3 or 4 of bilateral intraventricular hemorrhage
6. at screening, subject with active pulmonary hemorrhage or active air leak syndrome
7. subject who underwent/will undergo surgery within 72 hours before/after investigational product (IP) administration
8. subject who is expected to be treated with surfactant within 24 hours prior to IP administration
9. subject who is expected to be allergic to gentamicin (Birth mother's allergy for gentamicin will be confirmed).
10. subject who have previously participated in other clinical trials
11. subject who is considered ineligible by investigator due to other medical reasons

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo; normal saline	Other: Placebo; normal saline
Experimental: PNEUMOSTEM; human umbilical cord blood derived mesenchymal stem cell (hUCB-MSC)	Biological: PNEUMOSTEM; human umbilical cord blood-derived mesenchymal stem cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of subjects who have severe BPD or are dead	Percentage of subjects who have severe BPD or are dead	36 weeks postmenstrual age (PMA)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of subjects who have moderate/severe BPD or are dead	Percentage of subjects who have moderate/severe BPD or are dead	36 weeks PMA
Percentage of subjects by severity of BPD	Percentage of subjects by severity of BPD	prenatal 28 days/36 weeks PMA
Percentage of subjects in death due to lung disease	Percentage of subjects in death due to lung disease	prenatal 28 days/36 weeks PMA and study end timepoint
intubation duration	intubation duration	up to 24 weeks
ventilation duration	ventilation duration	up to 24 weeks
continuous positive airway pressure (CPAP) treatment duration	continuous positive airway pressure (CPAP) treatment duration	up to 24 weeks
treatment duration with supplemental oxygen	treatment duration with supplemental oxygen	up to 24 weeks
% of subjects treated with steroid for weaning ventilator	% of subjects treated with steroid for weaning ventilator	up to 24 weeks
Retinopathy of prematurity (ROP) with stage III or higher	number of subjects with ROP with stage III or higher	up to 24 weeks
number of subjects with retinopathy of prematurity that needs bevacizumab or laser therapy	number of subjects with retinopathy of prematurity that needs bevacizumab or laser therapy	up to 24 weeks
z-score	percentile for body weight, height, and head circumference	up to 24 weeks (visit 10)
days in hospitalization	days in hospitalization	up to 24 weeks
changes in tracheal suction fluid examination	changes in tracheal suction fluid examination	from screening to 7 days after IP administration (visit 5)

Collaborators and Investigators

• Sponsor: Medipost Co Ltd.
• Investigators: Principal Investigator: Wonsoon Park, Samsung Medical Center; Principal Investigator: Airhan Kim, 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.

Study record dates

Study Major Dates

• Study Start (Actual): August 13, 2018
• Primary Completion (Actual): January 9, 2024
• Study Completion (Actual): October 18, 2024

Study Registration Dates

• First Submitted: December 20, 2017
• First Submitted That Met QC Criteria: January 2, 2018
• First Posted (Actual): January 8, 2018

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 22, 2025
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Respiratory Tract Diseases; Lung Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Lung Injury; Ventilator-Induced Lung Injury; Bronchopulmonary Dysplasia
• Other Study ID Numbers: MP-CR-012

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No