Safety and Efficacy Evaluation of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Medipost Co Ltd.
ClinicalTrials.gov Identifier:
NCT01297205
First received: February 11, 2011
Last updated: April 3, 2014
Last verified: April 2014
  Purpose

PNEUMOSTEM® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with bronchopulmonary dysplasia. This study is to assess the safety and the efficacy of this study drug.


Condition Intervention Phase
Bronchopulmonary Dysplasia
Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open Label, Single-Center, Phase 1 Clinical Study to Evaluate the Safety and the Efficacy of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia

Resource links provided by NLM:


Further study details as provided by Medipost Co Ltd.:

Primary Outcome Measures:
  • Number of participant with adverse reaction [ Time Frame: 12 weeks from the day of treatment ] [ Designated as safety issue: Yes ]
    Number of paitents with normal rage of vital signs and laboratory examination Chest x-ray result, Duration of ventilator dependence, Duration of CPAP treatment, Duration of intubation, Occurrence of pneumothorax, Occurrence of intraventricular hemorrhage, Postnatal steroid use (%), Dose of surfactant (%) Cumulative duration of oxygen use


Secondary Outcome Measures:
  • Incidence of BPD at 36 Week's postmenstrual age [ Time Frame: 36 week's postmenstrual age ] [ Designated as safety issue: No ]
    Incidence of BPD at 36 Week's postmenstrual age (PMA)and 28 week's PMA Survival rate at 28 days after birth and 36 week's PMA


Enrollment: 9
Study Start Date: December 2010
Study Completion Date: December 2011
Primary Completion Date: September 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: PNEUMOSTEM® Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
Dose A - 10 million cells per kg Dose B - 20 million cells per kg Single intratracheal administration
Other Name: PNEUMOSTEM®

Detailed Description:

Bronchopulmonary dysplasia (BPD) is most common cause of death of children who were born prematurely, with low birth weights. In addition, many children who were recovered from this disease are suffering from many side effects such as prolonged hospitalization, pulmonary hypertension, and failure to thrive.

The purpose of BPD treatment is to make a baby be able to do spontaneous breathing and to spontaneous breathing a baby needs much energy and because of this a baby may have difficulty to feed. For this reason, medication with steroid, diuretic and respiratory drugs are currently used. However, there is no effective cure so far.

It has been reported that bone marrow derived mesenchymal stem cells (BM-MSC) can differentiate to pulmonary epithelial and pulmonary endothelial cells. Some animal studies showed that BM-MSC differentiated to bronchial cells and type 2 pneumocytes in rats with pneumonia and improve the fibrosis that occur after administration of bleomycin. Based on the findings, it is considered that mesenchymal stem cell therapy can help regenerate the damaged lung as well as BPD that cause lung inflammation, fibrosis, deficiency of type 2 pneumocytes, and so on.

PNEUMOSTEM® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with BPD. The main purpose of this study is to evaluate the safety and the tolerability of this study drug and to establish the maximum toxicity dose. The latent efficacy will also be assessed.

  Eligibility

Ages Eligible for Study:   up to 14 Days
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Birth weight range: 500g~1250g
  • Fetal gestational age: 23 weeks to 29 weeks
  • Premature infants who cannot do spontaneous breathing, which ventilation rate is less than 12 breaths per min of ventilation rate and 25% of oxygen demand
  • Premature infants who does not improve the breathing or worse within 24 hours prior to enrollment of this study
  • Written consent form signed by a legal representative or a parent

Exclusion Criteria:

  • Cyanotic or acyanotic congenital heart diseases except patent ductus arteriosus
  • Severe lung malformation (i.e. Pulmonary hypoplasia, congenital diaphragmatic hernia, congenital cystic lung disease)
  • Severe lung malformation with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
  • Severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
  • CRP > 30 mg/dL; Severe sepsis or shock
  • Premature infants who is going to or expected to have surgery 72 hours before/after this study drug administration
  • Surfactant administration within 24 hours prior to this study drug administration
  • Severe intracranial hemorrhage ≥ grade 3 or 4
  • Premature infants who have active pulmonary hemorrhage or active air leak syndrome at the time point of screening
  • History of other clinical studies as a participant
  • Premature infants who are allergic to Gentamicin
  • Premature infants who is considered inappropriate by the investigators
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01297205

Locations
Korea, Republic of
Samsung Medical Center
Seoul, Korea, Republic of
Sponsors and Collaborators
Medipost Co Ltd.
Investigators
Principal Investigator: Won-Soon Park, M.D., PhD. Samsung Medical Center
  More Information

Additional Information:
No publications provided

Responsible Party: Medipost Co Ltd.
ClinicalTrials.gov Identifier: NCT01297205     History of Changes
Other Study ID Numbers: MP-CR-006
Study First Received: February 11, 2011
Last Updated: April 3, 2014
Health Authority: Korea: Food and Drug Administration

Keywords provided by Medipost Co Ltd.:
Human Umbilical Cord Blood Derived Mesenchymal Stem Cells
Bronchopulmonary dysplasia
Premature infants

Additional relevant MeSH terms:
Bronchopulmonary Dysplasia
Ventilator-Induced Lung Injury
Lung Injury
Lung Diseases
Respiratory Tract Diseases
Infant, Premature, Diseases
Infant, Newborn, Diseases

ClinicalTrials.gov processed this record on April 17, 2014