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

• ClinicalTrials.gov Identifier: NCT01828957
• Recruitment Status: Completed
• First Posted: April 11, 2013
• Last Update Posted: September 6, 2019
• Sponsor: Medipost Co Ltd.
• Information provided by (Responsible Party): Medipost Co Ltd.

Study Description

Brief Summary:

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.

Condition or disease	Intervention/treatment	Phase
Bronchopulmonary Dysplasia	Biological: Pneumostem®; Other: Normal Saline	Phase 2

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 69 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Study Start Date: April 2013
• Actual Primary Completion Date: May 2015
• Actual Study Completion Date: August 2015

Arms and Interventions

Arm	Intervention/treatment
Experimental: Pneumostem®; A single intratracheal administration of Pneumostem® (1.0 x 10^7 cells/kg)	Biological: Pneumostem®; Other Name: Human umbilical cord blood-derived mesenchymal stem cells
Placebo Comparator: normal saline; A single intratracheal administration of normal saline	Other: Normal Saline

Outcome Measures

Primary Outcome Measures :

1. Incidence of BPD (moderate to severe) or mortality at 36 weeks PMA [ Time Frame: 36 weeks PMA ]
   Incidence of BPD (moderate to severe) or mortality rate at 36 weeks PMA

Secondary Outcome Measures :

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

Eligibility Criteria

• Ages Eligible for Study: 5 Days to 14 Days (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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

Contacts and Locations

Locations

Korea, Republic of

Asan Medical Center

Seoul, Korea, Republic of

Samsung Medical Center

Seoul, Korea, Republic of

Sponsors and Collaborators

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

More Information

Additional Information:

Related Info 

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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.

• Responsible Party: Medipost Co Ltd.
• ClinicalTrials.gov Identifier: NCT01828957
• Other Study ID Numbers: MP-CR-009
• First Posted: April 11, 2013
• Last Update Posted: September 6, 2019
• Last Verified: April 2017

Keywords provided by Medipost Co Ltd.:

Human Umbilical Cord Blood Derived Mesenchymal Stem Cells; Bronchopulmonary dysplasia; Premature infants

Additional relevant MeSH terms:

Bronchopulmonary Dysplasia; Premature Birth; Obstetric Labor, Premature; Obstetric Labor Complications; Pregnancy Complications; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases