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

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Brief Title

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

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

Brief Summary

      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.
    

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 Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of participant with adverse reaction

Secondary Outcome

 Incidence of BPD at 36 Week's postmenstrual age

Condition

Bronchopulmonary Dysplasia

Intervention

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Study Arms / Comparison Groups

 PNEUMOSTEM®
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

9

Start Date

December 2010

Completion Date

December 2011

Primary Completion Date

September 2011

Eligibility 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
      

Gender

All

Ages

N/A - 14 Days

Accepts Healthy Volunteers

No

Contacts

Won-Soon Park, M.D., PhD., , 

Location Countries

Korea, Republic of

Location Countries

Korea, Republic of

Administrative Informations


NCT ID

NCT01297205

Organization ID

MP-CR-006


Responsible Party

Sponsor

Study Sponsor

Medipost Co Ltd.


Study Sponsor

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


Verification Date

April 2014