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, ,
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
February 2021