Aerosolized Infasurf Treatment Protocol: Aerosolized Infasurf for Spontaneously Breathing Patients With Early RDS (AERO-03)

Aero-03

Surfactant replacement therapy can be life-saving for newborn infants born with symptomatic lung surfactant deficiency causing Respiratory Distress Syndrome (RDS). Currently, such therapy requires instillation of a liquid suspension into the trachea through an endotracheal tube. Endotracheal intubations have undesirable adverse effects on fragile premature infants. Instilling surfactant as a liquid suspension into the lung is associated with adverse events due to interruption of breathing in patients who already have respiratory insufficiency. This treatment protocol is expanded access that offers the opportunity for patients to receive aerosolized Infasurf prior to FDA acting on ONY Biotech's application for approval for commercial marketing of aerosolized Infasurf.

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Inclusion Criteria: Two cohorts with different entry criteria are included. In cohort 1 are patients with a diagnosis of early RDS who are not intubated and have not received a liquid surfactant instillation. In cohort 2 are patients who received liquid surfactant in the first hour of life, have been extubated and whose RDS is worsening. Cohort 1 Inclusion Criteria 1. NICU patient, \<12 hours of age. 2. Clinical diagnosis of RDS, with or without chest X-ray data. 3. Inspired oxygen ≤40% to maintain adequate oxygen saturation. 4. Not intubated 5. Requiring: (a) nasal continuous positive airway pressure (nCPAP), or (b) high flow nasal cannula (≥2L/kg/min) (HFNC), or (c) non-invasive ventilation. Cohort 2 Inclusion Criteria 1. NICU patient, \<24 hours of age who received liquid surfactant at ≤ 1 hour of age and was extubated. 2. Clinical diagnosis of RDS, with or without chest X-ray data. 3. Inspired oxygen ≤40% to maintain adequate oxygen saturation. 4. Not intubated 5. Requiring: (a) nasal continuous positive airway pressure (nCPAP), or (b) high flow nasal cannula (≥2L/kg/min) (HFNC), or (c) non-invasive ventilation. Exclusion Criteria: * 1) Requires \>40% inspired oxygen to maintain acceptable oxygenation 2) Requires endotracheal intubation. 3) A co-existing medical condition that makes aerosol administration contraindicated in the judgment of the attending physician.

{'studyType': 'EXPANDED_ACCESS', 'expandedAccessTypes': {'treatment': True}}

2023-11-07