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A Safety Study of IV Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for BPD

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

Status
Terminated
Phases
Phase 1
Study type
Interventional
Source
ClinicalTrials.gov
Registry ID
NCT03857841
Enrollment
3
Registered
2019-02-28
Start date
2019-10-09
Completion date
2021-05-20
Last updated
2021-10-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Bronchopulmonary Dysplasia

Keywords

Bronchopulmonary Dysplasia, Mesenchymal Stem Cell-derived Extracellular Vesicles, Preterm Neonates

Brief 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).

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.

Interventions

BIOLOGICALUNEX-42

UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline.

BIOLOGICALPhosphate-buffered saline

Phosphate-buffered saline

Sponsors

United Therapeutics
Lead SponsorINDUSTRY

Study design

Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)

Eligibility

Sex/Gender
ALL
Age
3 Days to 14 Days
Healthy volunteers
No

Inclusion criteria

1. Infant whose postnatal age was 3 to 14 days 2. 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) 3. Endotracheally intubated and receiving mechanical ventilation at the time of Screening and randomization. 4. Not expected to be extubated within the next 24 hours after randomization. 5. The subject had a parent/guardian who gave written informed consent.

Exclusion criteria

1. Had a congenital heart defect, except for PDA, atrial septal defect or a small/moderate, restrictive ventricular septal defect. 2. Had a serious malformation of the lung, such as pulmonary hypoplasia/aplasia, congenital diaphragmatic hernia, or any other congenital lung anomaly. 3. Was being treated with inhaled nitric oxide. 4. 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). 5. Had a known severe congenital infectious disease (ie, herpes, toxoplasmosis rubella, syphilis, human immunodeficiency virus, cytomegalovirus, etc). 6. High clinical suspicion of active systemic infection, severe sepsis, or septic shock during Screening. 7. 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. 8. Had a Grade 3 or 4 intracranial hemorrhage. 9. Had active pulmonary hemorrhage. 10. The subject was currently participating in any other interventional clinical study. 11. 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.

Design outcomes

Primary

MeasureTime frameDescription
Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came firstThe 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.

Countries

United States

Participant flow

Participants by arm

ArmCount
20 Pmol Phospholipid/kg Body Weight
UNEX-42 administered at 20 pmol phospholipid/kg body weight UNEX-42: UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline.
2
60 Pmol Phospholipid/kg Body Weight
UNEX-42 administered at 60 pmol phospholipid/kg body weight UNEX-42: UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline.
0
200 Pmol Phospholipid/kg Body Weight
UNEX-42 administered at 200 pmol phospholipid/kg body weight UNEX-42: UNEX-42 is a preparation of extracellular vesicles that are secreted from human bone marrow-derived mesenchymal stem cells suspended in phosphate-buffered saline.
0
Placebo
Phosphate-buffered saline Phosphate-buffered saline: Phosphate-buffered saline
1
Total3

Withdrawals & dropouts

PeriodReasonFG000FG001FG002FG003
Overall StudyDeath1000
Overall StudyStudy Terminated by Sponsor1000

Baseline characteristics

CharacteristicTotalPlacebo20 Pmol Phospholipid/kg Body Weight60 Pmol Phospholipid/kg Body Weight200 Pmol Phospholipid/kg Body Weight
Age, Customized
Postnatal Age - 12 Days
1 participants0 participants1 participants——
Age, Customized
Postnatal Age - 5 Days
1 participants1 participants0 participants——
Age, Customized
Postnatal Age - 7 Days
1 participants0 participants1 participants——
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants0 Participants1 Participants——
Ethnicity (NIH/OMB)
Not Hispanic or Latino
2 Participants1 Participants1 Participants——
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants——
Gestation Age at Birth
23 Weeks/0 Days
1 participants1 participants0 participants——
Gestation Age at Birth
24 Weeks/1 Day
1 participants0 participants1 participants——
Gestation Age at Birth
24 Weeks/5 Days
1 participants0 participants1 participants——
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants0 Participants0 Participants——
Race (NIH/OMB)
Asian
0 Participants0 Participants0 Participants——
Race (NIH/OMB)
Black or African American
3 Participants1 Participants2 Participants——
Race (NIH/OMB)
More than one race
0 Participants0 Participants0 Participants——
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants0 Participants0 Participants——
Race (NIH/OMB)
Unknown or Not Reported
0 Participants0 Participants0 Participants——
Race (NIH/OMB)
White
0 Participants0 Participants0 Participants——
Region of Enrollment
United States
3 participants1 participants2 participants——
Sex: Female, Male
Female
3 Participants1 Participants2 Participants0 Participants0 Participants
Sex: Female, Male
Male
0 Participants0 Participants0 Participants0 Participants0 Participants

Adverse events

Event typeEG000
affected / at risk
EG001
affected / at risk
EG002
affected / at risk
EG003
affected / at risk
deaths
Total, all-cause mortality
1 / 20 / 00 / 00 / 1
other
Total, other adverse events
2 / 20 / 00 / 01 / 1
serious
Total, serious adverse events
1 / 20 / 00 / 00 / 1

Outcome results

Primary

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.

Time frame: From Day 1 to 40 Weeks Post-menstrual Age or Hospital Discharge, whichever came first

Population: All subjects randomized and received clinical study material.

ArmMeasureValue (COUNT_OF_PARTICIPANTS)
20 Pmol Phospholipid/kg Body WeightNumber of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)2 Participants
60 Pmol Phospholipid/kg Body WeightNumber of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)0 Participants
200 Pmol Phospholipid/kg Body WeightNumber of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)0 Participants
PlaceboNumber of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)1 Participants

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026