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

Primary Purpose

Bronchopulmonary Dysplasia

Status

Completed

Phase

Phase 1

Locations

Korea, Republic of

Study Type

Interventional

Intervention

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

About this trial

This is an interventional treatment trial for Bronchopulmonary Dysplasia focused on measuring Human Umbilical Cord Blood Derived Mesenchymal Stem Cells, Bronchopulmonary dysplasia, Premature infants

Eligibility Criteria

5 Days - 14 Days (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Samsung Medical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PNEUMOSTEM®

Arm Description

Outcomes

Primary Outcome Measures

Number of participant with adverse reaction

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

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

Full Information

NCT ID

NCT01297205

First Posted

February 11, 2011

Last Updated

April 3, 2014

Sponsor

Medipost Co Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT01297205

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2014

Overall Recruitment Status

Completed

Study Start Date

December 2010 (undefined)

Primary Completion Date

September 2011 (Actual)

Study Completion Date

December 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Medipost Co Ltd.

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Bronchopulmonary Dysplasia

Keywords

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

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

9 (Actual)

8. Arms, Groups, and Interventions

Arm Title

PNEUMOSTEM®

Arm Type

Experimental

Intervention Type

Biological

Intervention Name(s)

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Other Intervention Name(s)

PNEUMOSTEM®

Intervention Description

Dose A - 10 million cells per kg Dose B - 20 million cells per kg Single intratracheal administration

Primary Outcome Measure Information:

Title

Number of participant with adverse reaction

Description

Time Frame

12 weeks from the day of treatment

Secondary Outcome Measure Information:

Title

Incidence of BPD at 36 Week's postmenstrual age

Description

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

Time Frame

36 week's postmenstrual age

10. Eligibility

Sex

All

Minimum Age & Unit of Time

5 Days

Maximum Age & Unit of Time

14 Days

Accepts Healthy Volunteers

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

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

Organizational Affiliation

Samsung Medical Center

Official's Role

Principal Investigator

Facility Information:

Facility Name

Samsung Medical Center

City

Seoul

Country

Korea, Republic of

12. IPD Sharing Statement

Citations:

PubMed Identifier

24508444

Citation

Chang YS, Ahn SY, Yoo HS, Sung SI, Choi SJ, Oh WI, Park WS. Mesenchymal stem cells for bronchopulmonary dysplasia: phase 1 dose-escalation clinical trial. J Pediatr. 2014 May;164(5):966-972.e6. doi: 10.1016/j.jpeds.2013.12.011. Epub 2014 Feb 6.

Results Reference

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

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