Bronchopulmonary DysplasiaPhase 1NCT01297205
Safety and Efficacy Evaluation of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia
A Phase 1 study testing Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells in people with Bronchopulmonary Dysplasia, run by Medipost Co Ltd. at 1 site in South Korea.
Trial summary
PNEUMOSTEM® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with bronchopulmonary dysplasia. This study is to assess the safety and the efficacy of this study drug. Read more about this study
- Who is it for?
- Ages 5 to 14 days
- Conditions the study is for
- Bronchopulmonary Dysplasia
- What is being tested?
- Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteSouth Korea
- Study dates
- Dec 2010to Dec 2011
- Phase
- Phase 1
- 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 5 to 14 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Birth weight range: 500g~1250g
- Fetal gestational age: 23 weeks to 29 weeks
- Premature infants who cannot do spontaneous breathing, which ventilation rate is less than 12 breaths per min of ventilation rate and 25% of oxygen demand
- Premature infants who does not improve the breathing or worse within 24 hours prior to enrollment of this study
- Written consent form signed by a legal representative or a parent
Who cannot join
- Cyanotic or acyanotic congenital heart diseases except patent ductus arteriosus
- Severe lung malformation (i.e. Pulmonary hypoplasia, congenital diaphragmatic hernia, congenital cystic lung disease)
- Severe lung malformation with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
- Severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
- CRP > 30 mg/dL; Severe sepsis or shock
- Premature infants who is going to or expected to have surgery 72 hours before/after this study drug administration
Show all 12Show fewer
- Surfactant administration within 24 hours prior to this study drug administration
- Severe intracranial hemorrhage ≥ grade 3 or 4
- Premature infants who have active pulmonary hemorrhage or active air leak syndrome at the time point of screening
- History of other clinical studies as a participant
- Premature infants who are allergic to Gentamicin
- Premature infants who is considered inappropriate by the investigators
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Open Label, Single-Center, Phase 1 Clinical Study to Evaluate the Safety and the Efficacy of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia
PNEUMOSTEM® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with bronchopulmonary dysplasia. This study is to assess the safety and the efficacy of this study drug.
Read the detailed description
Bronchopulmonary dysplasia (BPD) is most common cause of death of children who were born prematurely, with low birth weights. In addition, many children who were recovered from this disease are suffering from many side effects such as prolonged hospitalization, pulmonary hypertension, and failure to thrive.
The purpose of BPD treatment is to make a baby be able to do spontaneous breathing and to spontaneous breathing a baby needs much energy and because of this a baby may have difficulty to feed. For this reason, medication with steroid, diuretic and respiratory drugs are currently used. However, there is no effective cure so far.
It has been reported that bone marrow derived mesenchymal stem cells (BM-MSC) can differentiate to pulmonary epithelial and pulmonary endothelial cells. Some animal studies showed that BM-MSC differentiated to 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® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with BPD. The main purpose of this study is to evaluate the safety and the tolerability of this study drug and to establish the maximum toxicity dose. The latent efficacy will also be assessed.
- Conditions
- Bronchopulmonary Dysplasia
- Being tested
- Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
- Keywords
- Human Umbilical Cord Blood Derived Mesenchymal Stem Cells, Bronchopulmonary dysplasia, Premature infants
- Medical subject headings
- Bronchopulmonary Dysplasia, Premature Birth
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Human Umbilical Cord Blood Derived-Mesenchymal Stem CellsBiological
Also called PNEUMOSTEM®
Dose A - 10 million cells per kg Dose B - 20 million cells per kg Single intratracheal administration
| Study group | Role | What they receive |
|---|---|---|
| PNEUMOSTEM® | Experimental | Not described Receives: Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participant with adverse reaction
Number of paitents with normal rage of vital signs and laboratory examination Chest x-ray result, Duration of ventilator dependence, Duration of CPAP treatment, Duration of intubation, Occurrence of pneumothorax, Occurrence of intraventricular hemorrhage, Postnatal steroid use (%), Dose of surfactant (%) Cumulative duration of oxygen use
Measured: 12 weeks from the day of treatment
Secondary outcomes (1)
Incidence of BPD at 36 Week's postmenstrual age
Incidence of BPD at 36 Week's postmenstrual age (PMA)and 28 week's PMA Survival rate at 28 days after birth and 36 week's PMA
Measured: 36 week's postmenstrual age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Feb 2011 (estimated)
- Study startDec 2010
- Primary completionSep 2011
- Study completionDec 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medipost Co Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Won-Soon Park, M.D., PhD., Principal investigator, Samsung Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01297205
- Sponsor's protocol ID
- MP-CR-006
- Status last verified
- Apr 2014
- First submitted
- 11 Feb 2011
- Last updated
- 7 Apr 2014
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Chang YS, Ahn SY, Yoo HS, Sung SI, Choi SJ, Oh WI, Park WS. Mesenchymal stem cells for bronchopulmonary dysplasia: phase 1 dose-escalation clinical trial. J Pediatr. 2014 May;164(5):966-972.e6. doi: 10.1016/j.jpeds.2013.12.011. Epub 2014 Feb 6.
PubMed 24508444
Study at a glance
From the registry record, last updated by the sponsor on 7 Apr 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open Label, Single-Center, Phase 1 Clinical Study to Evaluate the Safety and the Efficacy of PNEUMOSTEM® Treatment in Premature Infants With Bronchopulmonary Dysplasia
- ClinicalTrials.gov ID
- NCT01297205
- Acronym / sponsor ID
- MP-CR-006
- Conditions
- Bronchopulmonary Dysplasia
- Keywords
- Human Umbilical Cord Blood Derived Mesenchymal Stem Cells, Bronchopulmonary dysplasia, Premature infants
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Medipost Co Ltd.Industry (pharma, biotech, devices)
- Study start
- Dec 2010
- Primary completion
- Sep 2011 (actual)
- Study completion
- Dec 2011 (actual)
- First posted
- 16 Feb 2011 (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.
PNEUMOSTEM® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with bronchopulmonary dysplasia. This study is to assess the safety and the efficacy of this study drug.
Detailed description
Bronchopulmonary dysplasia (BPD) is most common cause of death of children who were born prematurely, with low birth weights. In addition, many children who were recovered from this disease are suffering from many side effects such as prolonged hospitalization, pulmonary hypertension, and failure to thrive.
The purpose of BPD treatment is to make a baby be able to do spontaneous breathing and to spontaneous breathing a baby needs much energy and because of this a baby may have difficulty to feed. For this reason, medication with steroid, diuretic and respiratory drugs are currently used. However, there is no effective cure so far.
It has been reported that bone marrow derived mesenchymal stem cells (BM-MSC) can differentiate to pulmonary epithelial and pulmonary endothelial cells. Some animal studies showed that BM-MSC differentiated to 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® is human umbilical cord blood derived mesenchymal stem cells and it is intended to treat premature infants with BPD. The main purpose of this study is to evaluate the safety and the tolerability of this study drug and to establish the maximum toxicity dose. The latent efficacy will also be assessed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PNEUMOSTEM® | Experimental | Biological: Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells |
Who may qualify
- Ages
- 5 to 14 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Birth weight range: 500g~1250g
- Fetal gestational age: 23 weeks to 29 weeks
- Premature infants who cannot do spontaneous breathing, which ventilation rate is less than 12 breaths per min of ventilation rate and 25% of oxygen demand
- Premature infants who does not improve the breathing or worse within 24 hours prior to enrollment of this study
- Written consent form signed by a legal representative or a parent
Key exclusion criteria (12 listed)
- Cyanotic or acyanotic congenital heart diseases except patent ductus arteriosus
- Severe lung malformation (i.e. Pulmonary hypoplasia, congenital diaphragmatic hernia, congenital cystic lung disease)
- Severe lung malformation with chromosome anomalies (i.e. Edward syndrome, Patau syndrome, Down syndrome, etc) or severe congenital malformation (Hydrocephalus, Encephalocele, etc)
- Severe congenital infection (i.e. Herpes, Toxoplasmosis, Rubella, Syphilis, AIDS, etc)
- CRP > 30 mg/dL; Severe sepsis or shock
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 |
|---|---|---|
| Human Umbilical Cord Blood Derived-Mesenchymal Stem CellsAlso: PNEUMOSTEM®Dose A - 10 million cells per kg Dose B - 20 million cells per kg Single intratracheal administration | Biological | PNEUMOSTEM® |
Primary outcome
Number of participant with adverse reaction
Time frame: 12 weeks from the day of treatment
Number of paitents with normal rage of vital signs and laboratory examination Chest x-ray result, Duration of ventilator dependence, Duration of CPAP treatment, Duration of intubation, Occurrence of pneumothorax, Occurrence of intraventricular hemorrhage, Postnatal steroid use (%), Dose of surfactant (%) Cumulative duration of oxygen use
1 secondary outcome
Incidence of BPD at 36 Week's postmenstrual age
Time frame: 36 week's postmenstrual 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 7 Apr 2014.
- Derived from this study: PubMed 24508444Chang YS, Ahn SY, Yoo HS, Sung SI, Choi SJ, Oh WI, Park WS. Mesenchymal stem cells for bronchopulmonary dysplasia: phase 1 dose-escalation clinical trial. J Pediatr. 2014 May;164(5):966-972.e6. doi: 10.1016/j.jpeds.2013.12.011. Epub 2014 Feb 6.
Related trials for Bronchopulmonary Dysplasia
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Controlled Remote Monitoring and Optimization of Oxygen Therapy in Preterm Infants
BPD - Bronchopulmonary Dysplasia, Pulmonary Hypertension
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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
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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, M.D., 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?
A Phase 1 study testing Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells in people with Bronchopulmonary Dysplasia, 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 5 inclusion and 12 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 7 Apr 2014. 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.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 participant with adverse reaction (time frame: 12 weeks from the day of treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01297205, which also lists 1 linked publication.
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 7 Apr 2014.