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NCT Number: NCT02023788

Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study

This is a 5-year long-term follow-up study of open label, single-center, phase I clinical trial to evaluate the safety and efficacy of PNEUMOSTEM® in premature infants with bronchopulmonary dysplasia.

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Key information

Conditions

Bronchopulmonary Dysplasia Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases and Pregnancy Complications Infant, Newborn, Diseases Infant, Premature, Diseases Infections Lung Diseases Lung Injury Obstetric Labor Complications Obstetric Labor, Premature Pregnancy Complications Premature Birth Premature Birth of Newborn Respiratory Tract Diseases Respiratory Tract Infections Urogenital Diseases Ventilator-Induced Lung Injury

Age range

45 month–63 month

Sex eligibility

All sexes

Study type

Observational

Primary location

Samsung Medical Center

Seoul, South Korea

About this study

Bronchopulmonary dysplasia (BPD) is the most common cause of death for prematurely born babies with low birth weights. In addition, many children who recover from this disease suffer from various complications such as prolonged hospitalization, pulmonary hypertension, and failure to thrive.

It has been reported that bone marrow-derived mesenchymal stem cells (BM-MSC) can differentiate into pulmonary epithelial and pulmonary endothelial cells. Some animal studies showed that BM-MSCs differentiate into 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® consists of human umbilical cord blood-derived mesenchymal stem cells and is intended to treat BPD in premature infants. The purpose of the study is to evaluate 3-5 year long term safety and efficacy in patients who completed the earlier part of the phase I clinical trial of PNEUMOSTEM®.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • All infants who were enrolled in the 2-year follow-up study (NCT01632475) of phase 1 clinical trial for the safety and efficacy evaluations of PNEUMOSTEM® treatment in premature infants with bronchopulmonary dysplasia
  • Infants with a written consent form signed by a parent or legal guardian

Exclusion criteria

-Infants whose parent or legal guardian does not consent to participate in this follow-up study

Treatment and study plan

PNEUMOSTEM

Biological

A single intratracheal administration

Low Dose Group (3 patients): 1.0 x 10^7 cells/kg High Dose Group (6 patients): 2.0 x 10^7 cells/kg

  • The subjects were administered with Pneumostem in the earlier part of the Phase I study. No drug/biologics will be administered to any subject during this part of the study.

Other names: human umbilical cord blood-derived mesenchymal stem cells

Primary outcomes

  1. Number of subjects with adverse drug reactions

    Time frame: 60 months (corrected age)

    adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam

Secondary outcomes

  1. Respiratory outcomes

    Time frame: 60 months (corrected age)

    • hospital readmission rates and length of stay
    • whether medical interventions such as oxygen, steroid, or bronchodilator therapy was done and duration of the therapy
    • Frequency of Emergency Room visit (total number of visits/ number of visits due to respiratory illnesses)

  2. Survival

    Time frame: 60 months (corrected age)

  3. Z-score

    Time frame: 60 months (corrected age)

    • weight
    • height
    • head circumference
    • percentile

  4. Potential neurological development test outcomes

    Time frame: 60 months (corrected age)

    • K-ASQ (Korean Ages and Stages Questionnaires),
    • Bayley test (BSID III)

Sponsors and collaborators

Lead sponsor

Medipost Co Ltd.

Industry

Registry information

Important dates

Study start
2014
Primary completion
2016
Study completion
2016
First posted
Dec 30, 2013
Registry last updated
Sep 4, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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