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

NCT03857841 | Terminated Phase 1 Results DMC FDA Drug

• 1 other identifier: UNX-BP-101
• Study Type: interventional
• Target: 3
• Locations: 1 country
• Sites: 6

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).

Conditions

Bronchopulmonary Dysplasia

Keywords

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

Outcome Measures

Primary Outcomes (1)

• 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.

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

Study Arms (4)

20 pmol phospholipid/kg body weight

EXPERIMENTAL

UNEX-42 administered at 20 pmol phospholipid/kg body weight

Biological: UNEX-42

60 pmol phospholipid/kg body weight

EXPERIMENTAL

UNEX-42 administered at 60 pmol phospholipid/kg body weight

Biological: UNEX-42

200 pmol phospholipid/kg body weight

EXPERIMENTAL

UNEX-42 administered at 200 pmol phospholipid/kg body weight

Biological: UNEX-42

Placebo

PLACEBO COMPARATOR

Phosphate-buffered saline

Biological: Phosphate-buffered saline

Interventions

UNEX-42 BIOLOGICAL

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

20 pmol phospholipid/kg body weight; 200 pmol phospholipid/kg body weight; 60 pmol phospholipid/kg body weight

Phosphate-buffered saline BIOLOGICAL

Phosphate-buffered saline

Also known as: Placebo

Placebo

Eligibility Criteria

• Age: 3 Days - 14 Days
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• United Therapeutics: lead

Study Sites (6)

University of Colorado Hospital

Aurora, Colorado, 80045, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02215, United States

Location

University of Mississippi Medical Center

Jackson, Mississippi, 39216, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

MeSH Terms

Conditions

Bronchopulmonary Dysplasia

Condition Hierarchy (Ancestors)

Ventilator-Induced Lung Injury; Lung Injury; Lung Diseases; Respiratory Tract Diseases; Infant, Premature, Diseases; Infant, Newborn, Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Global Medical Information
• Organization: United Therapeutics Corp.

clinicaltrials@unither.com

919-485-8350

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 20, 2019
• First Posted: February 28, 2019
• Study Start: October 9, 2019
• Primary Completion: May 20, 2021
• Study Completion: May 20, 2021
• Last Updated: October 12, 2021
• Results First Posted: October 12, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)