Bronchopulmonary DysplasiaPhase 1NCT03857841
A Safety Study of IV Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for BPD
A Phase 1 study testing UNEX-42 and Phosphate-buffered saline in people with Bronchopulmonary Dysplasia, run by United Therapeutics at 6 sites in United States.
Trial summary
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at <27 weeks of gestational age (GA) at high risk for 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?
- UNEX-42 and Phosphate-buffered saline
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 6 sitesUnited States
- Study dates
- 9 Oct 2019to 20 May 2021
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Infant whose postnatal age was 3 to 14 days
- Subjects met the following oxygen and birth weight criteria based on gestational age: 23 weeks to 24 weeks 6 days (any birth weight, any oxygen requirement) or 25 weeks to 26 weeks 6 days (fraction of inspired oxygen [FiO2] ≥35% [sustained for >2 hours] at any point during postnatal Days 1 to 14 AND birth weight ≤750 g)
- Endotracheally intubated and receiving mechanical ventilation at the time of Screening and randomization.
- Not expected to be extubated within the next 24 hours after randomization.
- The subject had a parent/guardian who gave written informed consent.
Who cannot join
- Had a congenital heart defect, except for PDA, atrial septal defect or a small/moderate, restrictive ventricular septal defect.
- Had a serious malformation of the lung, such as pulmonary hypoplasia/aplasia, congenital diaphragmatic hernia, or any other congenital lung anomaly.
- Was being treated with inhaled nitric oxide.
- Had a known chromosomal abnormality (eg, Trisomy 18, Trisomy 13, or Trisomy 21) or a severe congenital malformation (eg, hydrocephalus and encephalocele, trachea-esophageal fistula, abdominal wall defects, and major renal anomalies).
- Had a known severe congenital infectious disease (ie, herpes, toxoplasmosis rubella, syphilis, human immunodeficiency virus, cytomegalovirus, etc).
- High clinical suspicion of active systemic infection, severe sepsis, or septic shock during Screening.
Show all 11Show fewer
- Underwent a surgical procedure (requiring admission to an operating room) within 72 hours before randomization or who was anticipated to have a surgical procedure (requiring admission to an operating room) within 72 hours before or following randomization.
- Had a Grade 3 or 4 intracranial hemorrhage.
- Had active pulmonary hemorrhage.
- The subject was currently participating in any other interventional clinical study.
- The subject was, in the opinion of the Investigator, so ill that death was inevitable, or was considered inappropriate for the study for any reason(s) other than those listed above.
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
University of Colorado Hospital
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Brigham and Women's Hospital
Boston, Massachusetts, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Children's Mercy Hospital
Kansas City, Missouri, 64108
About this study
Official title: A Safety Study of Intravenous Infusion of Bone Marrow Mesenchymal Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for Bronchopulmonary Dysplasia
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at <27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).
Read the detailed description
The study was discontinued by the Sponsor on 24 February 2021 due to a business decision, not related to reasons of safety or efficacy. Only data listings were created; no summary or inferential analyses were performed.
Subjects were assessed during Screening and Baseline (prior to randomization) for eligibility in the study. Subjects then received a single IV dose of UNEX-42 at 20 pmol phospholipid/kg body weight, or placebo. After randomization, subjects were monitored in the hospital through 40 Weeks postmentrual age (PMA) or the time of hospital discharge (whichever came first).
The following efficacy and safety assessments occurred during the course of the study:
Efficacy Assessments: incidence and severity of BPD, duration of hospitalization, duration of mechanical ventilation, duration of supplemental oxygen therapy, duration of postnatal steroids, tracheal aspirate inflammatory biomarkers, and Respiratory Severity Score.
Safety Assessments: physical examination, vital signs, adverse events, predefined complications of prematurity, clinical laboratory parameters, and chest x-ray.
Dose administration for Cohort 1 occurred so that there was an observational period of 3 days between dosing the first, second, and third subject to assure the opportunity for safety assessments in at least 1 subject on active treatment. In addition, enrollment between cohorts was to be paused for data review by a Data Monitoring Committee to evaluate the data available after each of the first 2 cohorts were enrolled.
Subjects that completed the Post-treatment Phase (including those that were discharged from the hospital prior to 40 Weeks PMA) continued into the Long-term Outcome Phase and were assessed through 1 year of corrected age.
- Conditions
- Bronchopulmonary Dysplasia
- Being tested
- UNEX-42, Phosphate-buffered saline
- Keywords
- Bronchopulmonary Dysplasia, Mesenchymal Stem Cell-derived Extracellular Vesicles, Preterm Neonates
- Medical subject headings
- Bronchopulmonary Dysplasia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UNEX-42Biological
UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline.
Phosphate-buffered salineBiological
Also called Placebo
Phosphate-buffered saline
| Study group | Role | What they receive |
|---|---|---|
| 20 pmol phospholipid/kg body weight | Experimental | UNEX-42 administered at 20 pmol phospholipid/kg body weight Receives: Biological: UNEX-42 |
| 60 pmol phospholipid/kg body weight | Experimental | UNEX-42 administered at 60 pmol phospholipid/kg body weight Receives: Biological: UNEX-42 |
| 200 pmol phospholipid/kg body weight | Experimental | UNEX-42 administered at 200 pmol phospholipid/kg body weight Receives: Biological: UNEX-42 |
| Placebo | Placebo comparator | Phosphate-buffered saline Receives: Biological: Phosphate-buffered saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)
The safety and tolerability of UNEX-42 in subjects with BPD was evaluated by the number of subjects with treatment-emergent adverse events, including death, computed by dose cohort and overall during the Post-treatment Phase.
Measured: From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Feb 2019
- Study start9 Oct 2019
- Primary completion20 May 2021
- Study completion20 May 2021
Why it stopped: The study was discontinued due to a business decision; no safety concerns were noted.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- United Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03857841
- Sponsor's protocol ID
- UNX-BP-101
- Status last verified
- Sep 2021
- First submitted
- 20 Feb 2019
- Last updated
- 12 Oct 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Safety Study of Intravenous Infusion of Bone Marrow Mesenchymal Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for Bronchopulmonary Dysplasia
- ClinicalTrials.gov ID
- NCT03857841
- Acronym / sponsor ID
- UNX-BP-101
- Conditions
- Bronchopulmonary Dysplasia
- Keywords
- Bronchopulmonary Dysplasia, Mesenchymal Stem Cell-derived Extracellular Vesicles, Preterm Neonates
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was discontinued due to a business decision; no safety concerns were noted.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- United TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 9 Oct 2019 (actual)
- Primary completion
- 20 May 2021 (actual)
- Study completion
- 20 May 2021 (actual)
- First posted
- 28 Feb 2019 (actual)
- Results
- Posted 12 Oct 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability study of UNEX-42 in infants born at <27 weeks of gestational age (GA) at high risk for bronchopulmonary dysplasia (BPD).
Detailed description
The study was discontinued by the Sponsor on 24 February 2021 due to a business decision, not related to reasons of safety or efficacy. Only data listings were created; no summary or inferential analyses were performed.
Subjects were assessed during Screening and Baseline (prior to randomization) for eligibility in the study. Subjects then received a single IV dose of UNEX-42 at 20 pmol phospholipid/kg body weight, or placebo. After randomization, subjects were monitored in the hospital through 40 Weeks postmentrual age (PMA) or the time of hospital discharge (whichever came first).
The following efficacy and safety assessments occurred during the course of the study:
Efficacy Assessments: incidence and severity of BPD, duration of hospitalization, duration of mechanical ventilation, duration of supplemental oxygen therapy, duration of postnatal steroids, tracheal aspirate inflammatory biomarkers, and Respiratory Severity Score.
Safety Assessments: physical examination, vital signs, adverse events, predefined complications of prematurity, clinical laboratory parameters, and chest x-ray.
Dose administration for Cohort 1 occurred so that there was an observational period of 3 days between dosing the first, second, and third subject to assure the opportunity for safety assessments in at least 1 subject on active treatment. In addition, enrollment between cohorts was to be paused for data review by a Data Monitoring Committee to evaluate the data available after each of the first 2 cohorts were enrolled.
Subjects that completed the Post-treatment Phase (including those that were discharged from the hospital prior to 40 Weeks PMA) continued into the Long-term Outcome Phase and were assessed through 1 year of corrected age.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 20 pmol phospholipid/kg body weight | Experimental | Biological: UNEX-42UNEX-42 administered at 20 pmol phospholipid/kg body weight |
| 60 pmol phospholipid/kg body weight | Experimental | Biological: UNEX-42UNEX-42 administered at 60 pmol phospholipid/kg body weight |
| 200 pmol phospholipid/kg body weight | Experimental | Biological: UNEX-42UNEX-42 administered at 200 pmol phospholipid/kg body weight |
| Placebo | Placebo comparator | Biological: Phosphate-buffered salinePhosphate-buffered saline |
Who may qualify
- Ages
- 3 to 14 days
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- Infant whose postnatal age was 3 to 14 days
- Subjects met the following oxygen and birth weight criteria based on gestational age: 23 weeks to 24 weeks 6 days (any birth weight, any oxygen requirement) or 25 weeks to 26 weeks 6 days (fraction of inspired oxygen [FiO2] ≥35% [sustained for >2 hours] at any point during postnatal Days 1 to 14 AND birth weight ≤750 g)
- Endotracheally intubated and receiving mechanical ventilation at the time of Screening and randomization.
- Not expected to be extubated within the next 24 hours after randomization.
- The subject had a parent/guardian who gave written informed consent.
Key exclusion criteria (11 listed)
- Had a congenital heart defect, except for PDA, atrial septal defect or a small/moderate, restrictive ventricular septal defect.
- Had a serious malformation of the lung, such as pulmonary hypoplasia/aplasia, congenital diaphragmatic hernia, or any other congenital lung anomaly.
- Was being treated with inhaled nitric oxide.
- Had a known chromosomal abnormality (eg, Trisomy 18, Trisomy 13, or Trisomy 21) or a severe congenital malformation (eg, hydrocephalus and encephalocele, trachea-esophageal fistula, abdominal wall defects, and major renal anomalies).
- Had a known severe congenital infectious disease (ie, herpes, toxoplasmosis rubella, syphilis, human immunodeficiency virus, cytomegalovirus, etc).
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 |
|---|---|---|
| UNEX-42UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline. | Biological | 20 pmol phospholipid/kg body weight, 200 pmol phospholipid/kg body weight, 60 pmol phospholipid/kg body weight |
| Phosphate-buffered salineAlso: PlaceboPhosphate-buffered saline | Biological | Placebo |
Primary outcome
Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)
Time frame: From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first
The safety and tolerability of UNEX-42 in subjects with BPD was evaluated by the number of subjects with treatment-emergent adverse events, including death, computed by dose cohort and overall during the Post-treatment Phase.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Oct 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Bronchopulmonary Dysplasia
Recruiting
Optimization of Saturation Targets And Resuscitation Trial (OptiSTART)
Premature Infants, Bronchopulmonary Dysplasia
Recruiting
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
Not yet recruiting
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.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing UNEX-42 and Phosphate-buffered saline in people with Bronchopulmonary Dysplasia, run by United Therapeutics at 6 sites 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 5 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: stopped early, as of the sponsor's last update on 12 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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. Some participants in this study may receive a placebo. 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?
Yes. Results are posted in the registry (first posted 12 Oct 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability) (time frame: From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first). 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 12 Oct 2021.