Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) – a US Study

Brief Title

Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study

Official Title

A Phase I/II, Open-Label Dose Escalation Trial to Evaluate the Safety and Efficacy of Two Dose Levels of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD)

Brief Summary

      PNEUMOSTEM® consists of ex vivo cultured allogeneic, unrelated, human umbilical cord
      blood-derived mesenchymal stem cells (hUCB-MSCs) and it is intended for use as a cellular
      therapy product for prevention of Bronchopulmonary Dysplasia (BPD). This study is an
      open-label, single-center, dose escalation study to evaluate of safety and efficacy of
      PNEUMOSTEM® in premature infants at high risk for BPD.
    

Study Phase

Phase 1/Phase 2

Study Type

Interventional

Primary Outcome

Number of participants with adverse reactions for 84 days after treatment

Secondary Outcome

 Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age

Condition

Bronchopulmonary Dysplasia

Intervention

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Study Arms / Comparison Groups

 PNEUMOSTEM®

Description:  Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg

Publications

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

Recruitment Status

Biological

Estimated Enrollment

12

Start Date

March 2015

Completion Date

May 2018

Primary Completion Date

September 2016

Eligibility Criteria

        Inclusion Criteria:

          -  A male or female infant whose postnatal age is 3 to 14 days, inclusive (for treatment
             between 5 and 14 days after birth)

          -  A subject whose gestational age is between 23 and 28 weeks (23 weeks ≤ gestational age
             (GA) < 28 weeks)

          -  A subject whose birth weight is between 500g and 1000g, inclusive

          -  A subject who is intubated and receiving mechanical ventilation within 5-14 days after
             birth, with a fraction of inspired oxygen (FiO2) of 0.25 or greater at Screening

          -  A subject who has had either a deterioration or no change in the setting of mechanical
             ventilation within the 24 hours before trial enrollment

          -  A subject whose parent/guardian can give a written informed consent

        Exclusion Criteria:

          -  A subject who has a congenital heart defect, except for patent ductus arteriosus
             (PDA), atrial septal defect (ASD) or a small, restrictive ventricular septal defect
             (VSD)

          -  A subject who has a serious malformation of the lung such as pulmonary
             hypoplasia/aplasia congenital diaphragmatic hernia, or other congenital lung anomaly

          -  A subject who has a chromosomal abnormality (e.g., Trisomy 18, Trisomy 13 or Trisomy
             21) or a severe congenital malformation (e.g., hydrocephalus and encephalocele,
             tracheo-esophageal fistula, abdominal wall defects, and major renal anomalies)

          -  A subject who has had a severe congenital infectious disease (i.e., herpes,
             toxoplasmosis rubella, syphilis, HIV, etc.)

          -  A subject who has evidence of severe sepsis or septic shock due to an active infection
             at Screening

          -  A subject who underwent a surgical procedure within 72 hours before study drug
             administration or who is anticipated to have a surgical procedure within 72 hours
             before or following study drug administration

          -  A subject who was administered surfactant within 24 hours before study drug
             administration

          -  A subject who has had a bilateral grade 3 or 4 intracranial hemorrhage

          -  A subject who has active pulmonary hemorrhage or an active air leak syndrome at
             Screening

          -  A subject who is currently participating in any other interventional clinical trial

          -  A subject who is, in the opinion of the Principal Investigator, considered
             inappropriate for the trial due to any reasons other than those listed above
      

Gender

All

Ages

3 Days - 14 Days

Accepts Healthy Volunteers

No

Contacts

Steven Powell, MD, , 

Location Countries

United States

Location Countries

United States

NCT ID

NCT02381366

Organization ID

MD-BPD-US001

Responsible Party

Sponsor

Study Sponsor

Medipost America Inc.

Collaborators

 Medipost Co Ltd.

Study Sponsor

Steven Powell, MD, Principal Investigator, Rush University Medical Center

Verification Date

August 2018