This study is to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in up to four escalating doses to preterm neonates 26 to 28 weeks gestational age who are receiving nCPAP for RDS compared to neonates receiving nCPAP alone.
This study was a multicenter, randomized, controlled, open-label, dose-escalation study, conducted to evaluate the safety and tolerability of lucinactant for inhalation in conjunction with nasal continuous positive airway pressure (nCPAP) in comparison with nCPAP alone. The study was to evaluate the safety and tolerability of lucinactant for inhalation, administered as an aerosol in 4 escalating doses. For this study, lucinactant for inhalation refers to the active investigational agent, lyophilized lucinactant, in combination with the prototype investigational delivery device. Reconstituted lyophilized lucinactant was aerosolized by the investigational device and introduced into the nCPAP circuit. Those randomized to the control arm continued to receive nCPAP alone. Dose assignments were unblinded, as the primary objective of this study was safety and tolerability. Preterm neonates with respiratory distress syndrome (RDS) between 26 and 28 completed weeks PMA who were within the first 20 hours after birth and who had successful implementation of controlled nCPAP within 90 minutes of birth were considered to be potential subjects. Before study enrollment, legal guardians were provided a written informed consent form (ICF) for each potential subject.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
48
Lucinactant for inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)
nCPAP therapy
Loma Linda University Medical Center
Loma Linda, California, United States
Number of Participants With Peri-Dosing Adverse Events - Initial Dose
Number of Participants with adverse events that were experienced during the initial study treatment
Time frame: Randomization to 24 Hours Post Randomization
Number of Participants With Air Leak
Number of participants with air leak (includes pneumothorax, pulmonary interstitial emphysema (PIE), pneumomediastinum, pneumopericardium, subcutaneous emphysema)
Time frame: 7 days
Number of Participants With Worsening of Respiratory Status Criteria
Number of participants with worsening in one of 12 respiratory status criteria through 72 hours post randomization (need for additional surfactant therapy, desaturation \< 80%, heart rate \< 100 bpm, sustained fraction of inspired oxygen (FiO2) \> 0.50, arterial carbon dioxide (PCO2) \> 65 mmHg, sustained apnea, persistent arterial pH \< 7.2, intubation for any reason, nCPAP \> 7 cmH2O, initiation of intermittent positive pressure ventilation, death, principal investigator determination of worsening status)
Time frame: Randomization to 72 Hours Post Randomization
Bronchopulmonary Dysplasia
Number of participants with bronchopulmonary dysplasia (BPD) and number of participants alive and without BPD at 36 weeks post-menstrual age (PMA)
Time frame: Randomization to 36 weeks PMA
Number of Participants With Nasal Continuous Positive Airway Pressure (nCPAP) Failure
Participants who required intubation for mechanical ventilation or surfactant administration were defined as having failed nCPAP
Time frame: Randomization to 72 Hours Post Randomization
Death
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Sharp Mary Birch Hospital for Women and Newborns
San Diego, California, United States
Christiana Care Health System
Newark, Delaware, United States
University of Miami
Miami, Florida, United States
University of Louisville
Louisville, Kentucky, United States
University of Nebraska Medical Center
Omaha, Nebraska, United States
Columbia University College of Physicians and Surgeons, New York Presbyterian Hospital - Morgan Stanley Children's Hospital
New York, New York, United States
New Hanover Regional Medical Center
Wilmington, North Carolina, United States
Providence St. Vincent Medical Center
Portland, Oregon, United States
Women and Infants Hospital
Providence, Rhode Island, United States
...and 10 more locations
Number of participants who died during the study
Time frame: Randomization to 36 weeks PMA
FiO2
Observed and change from baseline measurements for fraction of inspired oxygen (FiO2). Values represent the amount (fraction) of oxygen in the air the participant inspires; the values themselves do not have units. The normal amount of oxygen in air ("room air") is 21%, or 0.21.
Time frame: Randomization to 72 hours post randomization
Number of Participants With Complications of Prematurity
Number of participants with pre-specified common complications of prematurity.
Time frame: Randomization to 36 weeks PMA