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Trial record 2 of 3 for:    BPD - Bronchopulmonary Dysplasia | Boston Children's Hospital

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

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ClinicalTrials.gov Identifier: NCT03857841
Recruitment Status : Active, not recruiting
First Posted : February 28, 2019
Last Update Posted : March 30, 2021
Sponsor:
Information provided by (Responsible Party):
United Therapeutics

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.

Condition or disease Intervention/treatment Phase
Bronchopulmonary Dysplasia Biological: UNEX-42 Biological: Phosphate-buffered saline Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 18 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : June 20, 2019
Estimated Primary Completion Date : December 2021
Estimated Study Completion Date : December 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: 20 pmol phospholid/kg body weight
UNEX-42 administered at 20 pmol phospholid/kg body weight
Biological: 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.

Experimental: 60 pmol phospholid/kg body weight
UNEX-42 administered at 60 pmol phospholid/kg body weight
Biological: 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.

Experimental: 200 pmol phospholid/kg body weight
UNEX-42 administered at 200 pmol phospholid/kg body weight
Biological: 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.

Placebo Comparator: Placebo
Phosphate-buffered saline
Biological: Phosphate-buffered saline
Phosphate-buffered saline
Other Name: Placebo




Primary Outcome Measures :
  1. 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 will be evaluated by the number of subjects with treatment-emergent adverse events, including death, computed by dose cohort and overall during the Post-treatment Phase.


Secondary Outcome Measures :
  1. Incidence of BPD at 36 Weeks Post-menstrual Age [ Time Frame: 36 Weeks Post-menstrual Age ]
    The number of subjects with BPD at 36 Weeks Post-menstrual age will be summarized by dose cohort and placebo.

  2. Incidence of Death at 36 Weeks Post-menstrual Age [ Time Frame: 36 Weeks Post-menstrual Age ]
    The number of deaths at 36 Weeks Post-menstrual age will be summarized by dose cohort and placebo.

  3. Severity of BPD (mild, moderate, severe) at Week 36 Post-menstrual Age [ Time Frame: 36 Weeks Post-menstrual Age ]
    The number of subjects' BPD severity (mild, moderate, severe) will be summarized by dose cohort and placebo.



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Ages Eligible for Study:   up to 14 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03857841


Locations
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United States, Colorado
University of Colorado Hospital
Aurora, Colorado, United States, 80045
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
United States, Mississippi
University of Mississippi Medical Center
Jackson, Mississippi, United States, 39216
United States, Missouri
Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
Sponsors and Collaborators
United Therapeutics
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Responsible Party: United Therapeutics
ClinicalTrials.gov Identifier: NCT03857841    
Other Study ID Numbers: UNX-BP-101
First Posted: February 28, 2019    Key Record Dates
Last Update Posted: March 30, 2021
Last Verified: March 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Bronchopulmonary Dysplasia
Ventilator-Induced Lung Injury
Lung Injury
Lung Diseases
Respiratory Tract Diseases
Infant, Premature, Diseases
Infant, Newborn, Diseases