Bronchopulmonary DysplasiaNCT02023788
Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study
An observational study of Bronchopulmonary Dysplasia and Respiratory Tract Infections, run by Medipost Co Ltd. at 1 site in South Korea.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 45 to 63 months
- Conditions the study is for
- Bronchopulmonary Dysplasia, Respiratory Tract Infections, Premature Birth of Newborn
- What is being tested?
- PNEUMOSTEM
- How many people
- 8 participants
- Where
- 1 siteSouth Korea
- Study dates
- Apr 2014to Oct 2016
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 45 to 63 months
- All sexes
- No healthy volunteers
- Child
Study population: 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
Who may be able to join
- 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
Who cannot join
- -Infants whose parent or legal guardian does not consent to participate in this follow-up study
Trial locations and contacts
1 site in 1 country.
About this 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.
Read the detailed description
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®.
- Being tested
- PNEUMOSTEM
- Keywords
- human umbilical cord blood-derived mesenchymal stem cells, bronchopulmonary dysplasia, respiratory tract infection, Premature infants
- Medical subject headings
- Bronchopulmonary Dysplasia, Respiratory Tract Infections, Premature Birth
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PNEUMOSTEMBiological
Also called human umbilical cord blood-derived mesenchymal stem cells
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.
| Study group | Role | What they receive |
|---|---|---|
| Pneumostem® | Low Dose Group (3 subjects): 1.0 x 10^7 cells/kg, High Dose Group (6 subjects): 2.0 x 10^7 cells/kg
Intervention: Biological: Pneumostem® Receives: Biological: PNEUMOSTEM |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of subjects with adverse drug reactions
adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam
Measured: 60 months (corrected age)
Secondary outcomes (4)
Respiratory outcomes
- 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)
Measured: 60 months (corrected age)
Survival
Measured: 60 months (corrected age)
Z-score
- weight
- height
- head circumference
- percentile
Measured: 60 months (corrected age)
Potential neurological development test outcomes
- K-ASQ (Korean Ages and Stages Questionnaires),
- Bayley test (BSID III)
Measured: 60 months (corrected age)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Dec 2013 (estimated)
- Study startApr 2014
- Primary completionOct 2016
- Study completionOct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bronchopulmonary DysplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medipost Co Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Won-Soon Park, MD, PhD, Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02023788
- Sponsor's protocol ID
- MP-CR-006-F/U-5y
- Status last verified
- Apr 2017
- First submitted
- 17 Dec 2013
- Last updated
- 4 Sep 2018
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Sep 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-term Safety and Efficacy Follow-up Study of PNEUMOSTEM® in Patients Who Completed PNEUMOSTEM® Phase-I Study
- ClinicalTrials.gov ID
- NCT02023788
- Acronym / sponsor ID
- MP-CR-006-F/U-5y
- Keywords
- human umbilical cord blood-derived mesenchymal stem cells, bronchopulmonary dysplasia, respiratory tract infection, Premature infants
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Medipost Co Ltd.Industry (pharma, biotech, devices)
- Study start
- Apr 2014
- Primary completion
- Oct 2016 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 30 Dec 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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®.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pneumostem® | – | Biological: PNEUMOSTEMLow Dose Group (3 subjects): 1.0 x 10^7 cells/kg, High Dose Group (6 subjects): 2.0 x 10^7 cells/kg Intervention: Biological: Pneumostem® |
Who may qualify
- Ages
- 45 to 63 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- 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
Key inclusion criteria (2 listed)
- 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
Key exclusion criteria (1 listed)
- -Infants whose parent or legal guardian does not consent to participate in this follow-up study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PNEUMOSTEMAlso: human umbilical cord blood-derived mesenchymal stem cellsA 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. | Biological | Pneumostem® |
Primary outcome
Number of subjects with adverse drug reactions
Time frame: 60 months (corrected age)
adverse drug reactions, clinically significant laboratory findings, vital signs, physical exam
4 secondary outcomes
Respiratory outcomes
Time frame: 60 months (corrected age)
Survival
Time frame: 60 months (corrected age)
Z-score
Time frame: 60 months (corrected age)
Potential neurological development test outcomes
Time frame: 60 months (corrected age)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Sep 2018.
- Phase-I: 2 Year Follow-Up Safety and Efficacy Study of Pneumostem® in Premature Infants With Bronchopulmonary DysplasiaLink provided by the sponsor.
- Phase-I:Safety and Efficacy Evaluation of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary DysplasiaLink provided by the sponsor.
- Phase-II: Efficacy and Safety Evaluation of Pneumostem® Versus a Control Group for Treatment of BPD in Premature InfantsLink provided by the sponsor.
Related trials for Bronchopulmonary Dysplasia
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Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia
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Controlled Remote Monitoring and Optimization of Oxygen Therapy in Preterm Infants
BPD - Bronchopulmonary Dysplasia, Pulmonary Hypertension
Not yet recruitingPhase 1/2
Little Lungs Study
Invasive Mechanical Ventilation, Severe Respiratory Distress Syndrome
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Strap To Reduce Apnea in Preterm Infants: A Randomized Crossover Trial
Apnea of Prematurity, Intermittent Hypoxemia
Recruiting
Ventilator Pressure and Optimization of Compliance and Hemodynamics
Bronchopulmonary Dysplasia, Pulmonary Hypertension Due to Lung Diseases and Hypoxia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Won-Soon Park, MD, PhD principal investigator
Samsung Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Bronchopulmonary Dysplasia and Respiratory Tract Infections, run by Medipost Co Ltd. at 1 site in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 4 Sep 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of subjects with adverse drug reactions (time frame: 60 months (corrected age)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Sep 2018.