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

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Brief Title

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

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

Brief Summary

      A multicenter, placebo-controlled, randomized, dose escalation, safety, and tolerability
      study of UNEX-42 in infants born at <27 weeks of GA at high risk for BPD.
    

Detailed Description

      Subjects will be assessed during Screening and Baseline (prior to randomization) to determine
      eligibility for the study. After randomization, subjects will be monitored in the hospital
      through 40 Weeks PMA or the time of hospital discharge (whichever comes first).

      The following efficacy and safety assessments will occur 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, and Respiratory Severity Score.

      Safety Assessments: physical examination, vital signs, adverse events, predefined
      complications of prematurity, clinical laboratory parameters, and chest x-ray.

      Enrollment between cohorts will pause for data review by a Data Monitoring Committee to
      evaluate the data available after each of the first 2 cohorts have been enrolled. Dose
      administration for Cohort 1 will occur such that there is 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. This procedure will be followed for
      each cohort.

      Subjects that complete the Post-treatment Phase (including those that are discharged from
      hospital prior to 40 Weeks PMA) will continue into the Long-term Outcome Phase and will be
      assessed through 1 year of corrected age.
    

Study Phase

Phase 1

Study Type

Interventional


Primary Outcome

Number of Subjects With Treatment-emergent Adverse Events During the Post-treatment Phase (Safety and Tolerability)

Secondary Outcome

 Incidence of BPD at 36 Weeks Post-menstrual Age

Condition

Bronchopulmonary Dysplasia

Intervention

UNEX-42

Study Arms / Comparison Groups

 20 pmol phospholid/kg body weight
Description:  UNEX-42 administered at 20 pmol phospholid/kg body weight

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

18

Start Date

June 20, 2019

Completion Date

December 2021

Primary Completion Date

December 2021

Eligibility Criteria

        Inclusion Criteria:

          1. Infant whose postnatal age is 3 to 14 days

          2. Subjects meet 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 (FiO2 ≥35% 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 has a parent/guardian who gives written informed consent.

        Exclusion Criteria:

          1. Has a congenital heart defect, except for PDA, atrial septal defect or a
             small/moderate, restrictive ventricular septal defect.

          2. Has a serious malformation of the lung, such as pulmonary hypoplasia/aplasia,
             congenital diaphragmatic hernia, or any other congenital lung anomaly.

          3. Being treated with inhaled nitric oxide.

          4. Has 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. Has 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 is anticipated to have a surgical procedure
             (requiring admission to an operating room) within 72 hours before or following
             randomization.

          8. Has had a Grade 3 or 4 intracranial hemorrhage.

          9. Has active pulmonary hemorrhage.

         10. The subject is currently participating in any other interventional clinical study.

         11. The subject is, in the opinion of the Investigator, so ill that death is inevitable,
             or is considered inappropriate for the study for any reason(s) other than those listed
             above.
      

Gender

All

Ages

N/A - 14 Days

Accepts Healthy Volunteers

No

Contacts

Rob Grover, 919-425-5506, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03857841

Organization ID

UNX-BP-101


Responsible Party

Sponsor

Study Sponsor

United Therapeutics


Study Sponsor

Rob Grover, Study Director, United Therapeutics


Verification Date

March 2020