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

August 14, 2018 updated by: Medipost America Inc.

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)

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 Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

12

Phase

  • Phase 2
  • 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

  • United States
    • Illinois
      • Chicago, Illinois, United States, 60612
        • Rush University Medical Center

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

3 days to 2 weeks (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: PNEUMOSTEM®
Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg
Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells

Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells:

Dose A: 10 million cells per kg / Dose B: 20 million cells per kg

Other Names:
  • PNEUMOSTEM®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of participants with adverse reactions for 84 days after treatment
Time Frame: 84 days
84 days

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age
Time Frame: Between 84 days after treatment and 20 months of corrected age
Between 84 days after treatment and 20 months of corrected age
Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)
Time Frame: 36 weeks PMA
36 weeks PMA
Hospital Re-admission between 84 days after treatment until 20 months of corrected age
Time Frame: Between 84 days after treatment and 20 months of corrected age
Between 84 days after treatment and 20 months of corrected age
Bayley Scales of Infant and Toddler Development between 84 days after treatment until 20 months of corrected age
Time Frame: Between 84 days after treatment and 20 months of corrected age
Between 84 days after treatment and 20 months of corrected age

Collaborators and Investigators

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

Sponsor

Medipost America Inc.

Collaborators

Medipost Co Ltd.

Investigators

  • Principal Investigator: Steven Powell, MD, Rush University 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

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 (Actual)

March 1, 2015

Primary Completion (Actual)

September 1, 2016

Study Completion (Actual)

May 1, 2018

Study Registration Dates

First Submitted

February 15, 2015

First Submitted That Met QC Criteria

March 2, 2015

First Posted (Estimate)

March 6, 2015

Study Record Updates

Last Update Posted (Actual)

August 16, 2018

Last Update Submitted That Met QC Criteria

August 14, 2018

Last Verified

August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MD-BPD-US001

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

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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