Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Premature Neonates

Study Overview
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Brief Summary
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The primary objective of this study is to evaluate the safety and tolerability of aerosolized surfactant, specifically lucinactant for inhalation, administered in escalating inhaled doses to preterm neonates 29 to 34 weeks gestational age who are receiving nasal continuous positive airway pressure (nCPAP) for respiratory distress syndrome (RDS), compared to neonates receiving nCPAP alone.

Official Title
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A Multicenter, Randomized, Open-Label, Controlled Trial to Assess the Safety and Tolerability of Lucinactant for Inhalation in Preterm Neonates 29 to 34 Weeks PMA: Dose Escalation and Study Extension

Conditions
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Respiratory Distress Syndrome

Intervention / Treatment
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* Drug: Lucinactant for Inhalation
* Device: nCPAP alone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Informed consent from a legally authorized representative Gestational age 29 to 34 completed weeks (34 weeks 6 days) post menstrual age (PMA) Successful implementation of controlled nCPAP within 90 minutes after birth Spontaneous breathing Chest radiograph consistent with RDS Need for moderate levels of supplemental oxygen and nCPAP to maintain oxygen saturation of 88% to 95% for at least 30 minutes within the first 21 hours after birth Exclusion Criteria: Heart rate that cannot be stabilized above 100 beats/minute within 5 minutes of birth Recurrent episodes of apnea occurring after the initial newborn resuscitation period (ie, 10 minutes after birth) requiring intermittent positive pressure breaths using inflating pressures above the set CPAP pressure administered manually or mechanically through any patient interface A 5 minute Apgar score < 5 Major congenital malformation(s) and cranial/facial abnormalities that preclude nCPAP, diagnosed antenatally or immediately after birth Other diseases or conditions potentially interfering with cardiopulmonary function (eg, hydrops fetalis or congenital infection such as TORCH) Known or suspected chromosomal abnormality or syndrome Prolong rupture of membranes (PROM) > 2 weeks Evidence of hemodynamic instability requiring vasopressors or steroids for hemodynamic support and/or presumed clinical sepsis Need for endotracheal intubation and mechanical ventilation Has been administered: another investigational agent or exposure to an investigational medical device, any other surfactant agent, steroid treatment after birth

Ages Eligible for Study
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Minimum Age: 29 Weeks
Maximum Age: 34 Weeks

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Sequential Assignment
Interventional Model Description: Sequential escalating dosing groups, each group contained an active and control arm of equal size.
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Aerosolized lucinactant (25 mg/kg)<br>25 mg total phospholipids (TPL)/kg: Lucinactant for inhalation with nCPAP | Drug: Lucinactant for Inhalation<br>* Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)<br>* Other names: Aerosurf;|
| Experimental: Aerosolized lucinactant (50 mg/kg)<br>50 mg TPL/kg: Lucinactant for inhalation with nCPAP | Drug: Lucinactant for Inhalation<br>* Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)<br>* Other names: Aerosurf;|
| Experimental: Aerosolized lucinactant (75 mg/kg)<br>75 mg TPL/kg: Lucinactant for inhalation with nCPAP | Drug: Lucinactant for Inhalation<br>* Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)<br>* Other names: Aerosurf;|
| Experimental: Aerosolized lucinactant (100 mg/kg)<br>100 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met. | Drug: Lucinactant for Inhalation<br>* Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)<br>* Other names: Aerosurf;|
| Experimental: Aerosolized lucinactant (150 mg/kg)<br>150 mg TPL/kg: Lucinactant for inhalation with nCPAP; repeat dosing possible if criteria met. | Drug: Lucinactant for Inhalation<br>* Lucinactant for Inhalation refers to the active investigational agent, lucinactant, in combination with the investigational delivery device (drug-device combination product)<br>* Other names: Aerosurf;|
| Active Comparator: nCPAP alone<br>nCPAP therapy alone | Device: nCPAP alone<br>* nCPAP therapy<br>* Other names: nasal continuous positive airway pressure;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Peri-Dosing Events | Pre-specified adverse events that occured during treatment administration; does not include nCPAP alone | Within 48 Hours after Initiation of Study Treatment | 
| All Cause Mortality |  | Within 36 Weeks PMA | 
| Oxygen Saturation Levels | Oxygen saturation as determined by pulse oximetry | Within 3 Hours of Randomization | 
| Serum Electrolytes |  | 24 Hours Post Randomization | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PCO2 | Arterial carbon dioxide | Within 3 Hours of Randomization |