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Budesonide With Intratracheal Surfactants in Extremely Preterm Infants

Phase 1
Conditions
Bronchopulmonary Dysplasia
Respiratory Distress Syndrome in Premature Infant
Interventions
Registration Number
NCT04019106
Lead Sponsor
University of Manitoba
Brief Summary

This is a phase I/II trial in preterm infants aimed at identifying the optimal dose of budesonide with bovine lipid extract surfactant as vehicle for intratracheal administration.

Detailed Description

Premature infants of gestational age less than 29 weeks with respiratory distress syndrome and clinical indication for surfactant administration will be recruited for this Phase I/II open-label study.

A total of 30 subjects will be recruited from 2 neonatal intensive care units:

1. Children's Hospital-Health Sciences Centre (HSC), Winnipeg

2. St. Boniface General Hospital, Winnipeg, MB

3 groups of 10 infants each will receive single dose of intratracheal budesonide (0.0625 mg/kg, 0.125 mg/kg, and 0.25 mg/kg) with BLES surfactant (5 ml/kg). PK/PD analysis will be done using clinical parameters, serum biomarkers, tracheal aspirate biomarkers and plasma budesonide levels obtained at fixed intervals.

The duration of subject participation will involve 12-17 weeks for the clinical intervention, depending on gestational age at birth and discharge date. Participants will be followed until 40 weeks or discharge, whichever comes first.

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
30
Inclusion Criteria
  1. Male or female infant born between 23 and 28+6 weeks of GA
  2. Infant diagnosed with RDS according to clinical protocol criteria
  3. Able to adhere to surfactant administration protocol
  4. The patient is born in the study centre.
  5. Subject's parent(s)/legal guardian(s) has provided signed and dated informed consent and authorization to use protected health information, as required by national and local regulations.
  6. In the investigator's opinion, the subject's parent(s)/legal guardian(s) understand(s) and can comply with protocol requirements, instructions, and protocol-stated restrictions, and is likely to complete the study as planned.
Exclusion Criteria
  1. Older than five days at inclusion.
  2. Presence of known clinically significant congenital heart disease or other major congenital malformation
  3. Subjects with clinically significant laboratory abnormalities which are deemed by the investigator to represent a safety risk to participation in this study. Other laboratory parameters outside the reference range for the subject's age may be included if the investigator considers the abnormalities unlikely to introduce additional risk factors and will not interfere with data interpretation.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Dosing Level 2Budesonide in bovine lipid extract surfactant (BLES)0.125 mg/kg Budesonide in bovine lipid extract surfactant (BLES)
Dosing Level 1Budesonide in bovine lipid extract surfactant (BLES)0.0625 mg/kg Budesonide in bovine lipid extract surfactant (BLES)
Dosing Level 3Budesonide in bovine lipid extract surfactant (BLES)0.25 mg/kg Budesonide in bovine lipid extract surfactant (BLES)
Primary Outcome Measures
NameTimeMethod
Area under the curve from serial budesonide levelsAt 24 hour time point following dosing

Blood samples will be drawn from patients to determine the serum budesonide levels to determine the area under the curve

Secondary Outcome Measures
NameTimeMethod
Neonatal Mortalityup to 40 weeks PMA or discharge, whichever comes first

Survival of the infants

VentilationStrategytill 36 weeks PMA or discharge, whichever comes first

Duration and modality of ventilation used in the preterm infants

Respiratory Severity Scoreat baseline and till 36 weeks PMA or discharge, whichever comes first

The product of Fraction of inspired oxygen and mean airway pressure will be used to estimate the respiratory severity score

Percentage of Participants with Pulmonary Hemorrhageat baseline and 48 hours after budesonide with surfactant administration

Clinical signs of pallor, cyanosis, bradycardia, apnoea and blood gas changes. Radiographic evidences of patchy infiltrates to complete opacification of lung fields.

Percentage of Participants with Pneumothorax on Chest X-rayat baseline and 48 hours after budesonide with surfactant administration

Identified in X-ray as hyperlucent shadow outside the lungs without pulmonary vascular markings, with or without mediastinal shift

Percentage of Participants with Necrotising Enterocolitis (NEC)48 hours after budesonide with surfactant administration

presence or absence of NEC will be compared across the 3 dosing groups and within the dosing groups.

Concentration of Inflammatory Biomarkers in SerumBaseline, 24 hours, 48 hours and 1 week.

Cytokines IL-1 β, IL-6, IL-8, IL-10, CCL2 and TNF-ɑ will be analyzed in serum samples obtained from the infants following BITS administration using commercially available ELISA kits.

Bronchopulmonary Dysplasia free survivalat 36 weeks PMA or discharge, whichever comes first

NICHD criteria will be used to diagnose and grade the infants for presence of BPD.

Concentration of Inflammatory Biomarkers in Tracheal AspiratesBaseline, 24 hours, 48 hours,1 week, 4 weeks and 36 weeks Gestational Age

Tracheal aspirates will be centrifuged to isolate a large aggregate surfactant fraction that will be assayed for both phospholipid (surfactant recovery) and total protein concentration. The supernatant fraction after surfactant isolation will be assayed for total protein, and selected cytokines (IL-1 β, IL-6, IL-8, IL-10, CCL2 and TNF-ɑ)

Duration of Hospital Stayfrom day 0 (birth date) to 40 weeks
Duration of Supplemental Oxygentill 36 weeks PMA or discharge, whichever comes first
Percentage of Participants with Hypothalamic pituitary axis (HPA) suppressionat 0 and 24 hours after dosing

Cortisol levels will be measured

Percentage of Participants with Spontaneous Intestinal Perforation (SIP) on abdominal X-rayat baseline and 48 hours after budesonide with surfactant administration

Abdominal X-ray showing presence of free air. Presence or absence of SIP will be compared across the 3 dosing groups and within the dosing groups.

Level of Supplemental Oxygen Administeredat baseline and at 36 week Post menstrual age or discharge, whichever comes first

the concentration of supplemental oxygen given at discharge or 36 weeks PMA compared to baseline.

Presence of Respiratory Supportat 36 week Post menstrual age or discharge, whichever comes first

the presence or absence of any method of respiratory support at discharge or 36 weeks PMA compared to baseline.

Percentage of Participants with Intra-ventricular Hemorrhageat baseline and 48 hours after budesonide with surfactant administration

presence or absence of will be compared across the 3 dosing groups and within the dosing groups.

Percentage of Participants with Sepsisat baseline and till 36 weeks PMA or discharge, whichever comes first

As per the third international consensus definitions for sepsis and septic shock (Sepsis-3)

Percentage of Participants with Severe Retinopathy at Prematuritybaseline and 48 hours after budesonide with surfactant administration

retinopathy of ≥grade III will be recorded

Trial Locations

Locations (2)

St. Boniface General Hospital

🇨🇦

Winnipeg, Manitoba, Canada

Children's Hospital-Health Science Centre

🇨🇦

Winnipeg, Manitoba, Canada

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