A Safety Study of IV Stem Cell-derived Extracellular Vesicles (UNEX-42) in Preterm Neonates at High Risk for BPD
Condition: Bronchopulmonary Dysplasia
MeSH terms: Bronchopulmonary Dysplasia
Intervention: UNEX-42; Phosphate-buffered saline
Intervention description: 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 saline
Phase: Phase 1
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 3.0
Sex: All
Minimum age: 3 Days
Maximum age: 14 Days
Healthy volunteers: No
Keywords: Bronchopulmonary Dysplasia; Mesenchymal Stem Cell-derived Extracellular Vesicles; Preterm Neonates

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).
    

Eligibility criteria:

        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.