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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
12

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Number of participants with adverse reactions for 84 days after treatment
Time Frame: 84 days
84 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Medipost America Inc.

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Medipost Co Ltd.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
September 1, 2016

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
May 1, 2018

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 15, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 2, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 6, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 16, 2018

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 14, 2018

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2018

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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