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

April 3, 2014 updated by: Medipost Co Ltd.

Open Label, Single-Center, Phase 1 Clinical Study to Evaluate the Safety and the Efficacy of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

9

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

5 days to 2 weeks (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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 Names:
  • PNEUMOSTEM®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participant with adverse reaction
Time Frame: 12 weeks from the day of treatment
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
12 weeks from the day of treatment

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of BPD at 36 Week's postmenstrual age
Time Frame: 36 week's postmenstrual age
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
36 week's postmenstrual age

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medipost Co Ltd.

Investigators

  • Principal Investigator: Won-Soon Park, M.D., PhD., Samsung Medical Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2010

Primary Completion (Actual)

September 1, 2011

Study Completion (Actual)

December 1, 2011

Study Registration Dates

First Submitted

February 11, 2011

First Submitted That Met QC Criteria

February 15, 2011

First Posted (Estimate)

February 16, 2011

Study Record Updates

Last Update Posted (Estimate)

April 7, 2014

Last Update Submitted That Met QC Criteria

April 3, 2014

Last Verified

April 1, 2014

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MP-CR-006

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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