Bronchopulmonary DysplasiaPhase 1/2NCT02381366
Safety and Efficacy of PNEUMOSTEM® in Premature Infants at High Risk for Bronchopulmonary Dysplasia (BPD) - a US Study
A Phase 1/2 study testing Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells in people with Bronchopulmonary Dysplasia, run by Medipost, Inc. at 1 site in United States.
Trial summary
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). Read more about this study
- Who is it for?
- Ages 3 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
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2015to May 2018
- Phase
- Phase 1/2
- 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 3 to 14 days
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Rush University Medical Center
Chicago, Illinois, 60612
About this study
Official title: 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.
- Conditions
- Bronchopulmonary Dysplasia
- Being tested
- Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells
- Keywords
- Mesenchymal Stem Cells, Human Umbilical Cord Blood, Premature Infants, Bronchopulmonary Dysplasia, Chronic Lung Disease, Cell Therapy
- 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®
Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells:
Dose A: 10 million cells per kg / Dose B: 20 million cells per kg
| Study group | Role | What they receive |
|---|---|---|
| PNEUMOSTEM® | Experimental | Dose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg 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 participants with adverse reactions for 84 days after treatment
Measured: 84 days
Secondary outcomes (4)
Number of participants with adverse reactions between 84 days after treatment and 20 months of corrected age
Measured: Between 84 days after treatment and 20 months of corrected age
Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)
Measured: 36 weeks PMA
Hospital Re-admission between 84 days after treatment until 20 months of corrected age
Measured: 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
Measured: Between 84 days after treatment and 20 months of corrected age
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Mar 2015 (estimated)
- Study startMar 2015
- Primary completionSep 2016
- Study completionMay 2018
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 1/2
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medipost, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Medipost Co Ltd.
- Responsible party
- Sponsor
- Investigators
- Steven Powell, MD, Principal investigator, Rush University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02381366
- Sponsor's protocol ID
- MD-BPD-US001
- Status last verified
- Aug 2018
- First submitted
- 15 Feb 2015
- Last updated
- 16 Aug 2018
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
Powell SB, Silvestri JM. Safety of Intratracheal Administration of Human Umbilical Cord Blood Derived Mesenchymal Stromal Cells in Extremely Low Birth Weight Preterm Infants. J Pediatr. 2019 Jul;210:209-213.e2. doi: 10.1016/j.jpeds.2019.02.029. Epub 2019 Apr 13.
PubMed 30992220
Study at a glance
From the registry record, last updated by the sponsor on 16 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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)
- ClinicalTrials.gov ID
- NCT02381366
- Acronym / sponsor ID
- MD-BPD-US001
- Conditions
- Bronchopulmonary Dysplasia
- Keywords
- Mesenchymal Stem Cells, Human Umbilical Cord Blood, Premature Infants, Bronchopulmonary Dysplasia, Chronic Lung Disease, Cell Therapy
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Medipost, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Medipost Co Ltd.
- Study start
- Mar 2015 (actual)
- Primary completion
- Sep 2016 (actual)
- Study completion
- May 2018 (actual)
- First posted
- 6 Mar 2015 (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® 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 design
- Primary purpose
- Prevention
- 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 CellsDose A: PNEUMOSTEM® 10 million cells per kg Dose B: PNEUMOSTEM® 20 million cells per kg |
Who may qualify
- Ages
- 3 to 14 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (11 listed)
- 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
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®Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells: Dose A: 10 million cells per kg / Dose B: 20 million cells per kg | Biological | PNEUMOSTEM® |
Primary outcome
Number of participants with adverse reactions for 84 days after treatment
Time frame: 84 days
4 secondary outcomes
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
Incidence of moderate/severe BPD or death at 36 weeks postmenstrual age (PMA)
Time frame: 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
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
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 16 Aug 2018.
- Derived from this study: PubMed 30992220Powell SB, Silvestri JM. Safety of Intratracheal Administration of Human Umbilical Cord Blood Derived Mesenchymal Stromal Cells in Extremely Low Birth Weight Preterm Infants. J Pediatr. 2019 Jul;210:209-213.e2. doi: 10.1016/j.jpeds.2019.02.029. Epub 2019 Apr 13.
- Individual participant dataSharing planned: Undecided
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.
Steven Powell, MD principal investigator
Rush University 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 1/2 study testing Human Umbilical Cord Blood Derived-Mesenchymal Stem Cells in people with Bronchopulmonary Dysplasia, run by Medipost, Inc. at 1 site in United States.
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 6 inclusion and 11 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 16 Aug 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 United States. 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 participants with adverse reactions for 84 days after treatment (time frame: 84 days). 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 NCT02381366, 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 16 Aug 2018.