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

NCT01828957 | Completed Phase 2 DMC

• 1 other identifier: MP-CR-009
• Study Type: interventional
• Target: 69
• Locations: 1 country
• Sites: 2

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.

Conditions

Bronchopulmonary Dysplasia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (16)

• 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

Study Arms (2)

Pneumostem®

EXPERIMENTAL

A single intratracheal administration of Pneumostem® (1.0 x 10\^7 cells/kg)

Biological: Pneumostem®

normal saline

PLACEBO COMPARATOR

A single intratracheal administration of normal saline

Other: Normal Saline

Interventions

Pneumostem® BIOLOGICAL

Also known as: Human umbilical cord blood-derived mesenchymal stem cells

Pneumostem®

Normal Saline OTHER

normal saline

Eligibility Criteria

• Age: 5 Days - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Medipost Co Ltd.: lead

Study Sites (2)

Asan Medical Center

Seoul, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Related Publications (1)

• 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.

  PMID: 33876883 DERIVED

Related Links

• Related Info

MeSH Terms

Conditions

Bronchopulmonary Dysplasia; Premature Birth

Interventions

Saline Solution

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

Intervention Hierarchy (Ancestors)

Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Study Officials

• Won-Soon Park, MD, PhD

  Department of Pediatrics, Samsung Medical Center

  PRINCIPAL INVESTIGATOR

• Ai-Rhan Kim

  Department of Neonatology, Asan Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2013
• First Posted: April 11, 2013
• Study Start: April 1, 2013
• Primary Completion: May 1, 2015
• Study Completion: August 1, 2015
• Last Updated: September 6, 2019

Record last verified: 2017-04

Locations

KR (2)