Phase 2
N=149
Aerosolized Surfactant in Neonatal RDS
Respiratory Distress Syndrome, Newborn
Bottom Line
View on ClinicalTrials.gov: NCT02294630 ↗Enrolled (actual)
149
Serious AEs
0.0%
Results posted
Aug 2021
Primary outcome: Primary: Number of Participants With Adverse Events as a Measure of Safety and Feasibility — 3; 8; 4; 12 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Surfactant (Drug)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Sood, Beena G., MD, MS
- Primary completion
- Jul 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Events as a Measure of Safety and Feasibility |
3; 8; 4; 12 | — |
| PRIMARY Patient Status as Evaluated by Dose Level |
3; 3; 5; 4; 3; 8 | — |
| PRIMARY Short Term Efficacy as Assessed by Need for Intubation |
3; 3; 5; 4 | 0.399 |
| SECONDARY Blood Gas Parameters - pH |
7.35; 7.36; 7.36; 7.35 | — |
| SECONDARY Blood Gas Parameters - pCO2 |
45; 45; 43; 45 | — |
| SECONDARY Pulse Oximetry |
95.95; 96.95; 97.37; 97.47 | — |
| SECONDARY Vital Signs - Heart Rate |
134; 142; 138; 136 | — |
| SECONDARY Vital Signs - Respiratory Rate |
47; 47; 46; 47 | — |
| SECONDARY Vital Signs - Systolic Blood Pressure |
54; 51; 54; 53 | — |
| SECONDARY Number of Doses of Surfactant - Aerosolized & Intratracheal |
10; 14; 13; 14; 27; 24 | — |
| SECONDARY Pneumothorax, Pneumomediastinum or Other Air Leak |
0; 1; 0; 1 | — |
| SECONDARY Changes in Cerebral Oxygenation From Baseline as Evaluated at End of Study Intervention |
79; 80; 78; 79 | — |
| SECONDARY Changes in Surfactant Activity in Gastric Aspirates |
10.9; 9.5; 8.8; 8.0 | — |
| SECONDARY Cumulative Duration of Non-invasive and Invasive Ventilation |
4.5; 7.0; 7.9; 6.1; 1.3; 0.9 | — |
| SECONDARY Duration of Supplemental Oxygen, Intensive Care, Hospital Stay |
7.4; 9.0; 10.7; 8.9; 29.9; 26.4 | — |
| SECONDARY Age at Start of Feeds, Feeding Progression, Age at Full Enteral Feeds |
1.3; 1.6; 1.8; 1.5; 11.2; 7.8 | — |
| SECONDARY Need for Blood Transfusions |
10; 6; 8; 7 | — |
| SECONDARY Growth Parameters |
2153; 2222; 2235; 2281 | — |
| SECONDARY Morbidities Associated With Prematurity |
0; 1; 0; 1; 0; 1 | — |
| SECONDARY Survival to Hospital Discharge |
37; 38; 35; 39 | — |
| SECONDARY Survival to Discharge Without Severe Morbidity |
37; 35; 33; 35 | — |
Summary
Respiratory distress syndrome (RDS), caused by surfactant deficiency, is the leading cause of mortality and morbidity in preterm infants. Intratracheal instillation, the only approved means of surfactant delivery, requires endotracheal intubation and mechanical ventilation with their attendant risks. Interventions that decrease need for intubation and mechanical ventilation like noninvasive ventilation (NIV) including nasal continuous positive airway pressure, high flow nasal cannula or nasal intermittent mandatory ventilation are increasingly being used for initial respiratory support in preterm neonates with RDS to improve outcomes. Aerosolized surfactant delivered during NIV is an innovative and promising concept for the treatment of RDS - retaining the advantages of early surfactant with alveolar recruitment while obviating the risks of intubation and mechanical ventilation. The investigators overall hypothesis is that treatment of RDS with aerosolized surfactant in preterm infants undergoing NIV is safe and feasible and will result in short-term improvement in oxygenation and ventilation. The objective of this proposal is to perform a single-center unblinded Phase II randomized clinical trial of aerosolized surfactant for the treatment of RDS in preterm neonates undergoing NIV. Funding Source - FDA-OOPD.
Eligibility Criteria
Inclusion Criteria
- Infants admitted to the NICU at Hutzel Women's Hospital (HWH)/Children's Hospital of Michigan (CHM)
- Gestational age of 240/7-366/7 weeks
- Postnatal age ≤ 24 hours
- Clinical diagnosis of RDS based on (i) presence of at least two of the four classic symptoms (need of supplemental oxygen, tachypnea, intercostal retractions or grunting), and (ii) exclusion of other causes of respiratory failure and (iii) Clinician intent to administer surfactant if infant requires intubation
- Respiratory support with NIV (CPAP or NIPPV or HFNC) with FiO2 ≥25% or PEEP ≥ 4 cmH20 or HFNC rate ≥ 2 LPM for ≤8 hours
- Written informed consent from parent/guardian
Exclusion Criteria
- Previous receipt of surfactant
- Infants with respiratory distress who are unstable and require immediate intubation
- Active air leak syndrome (e.g. pneumothorax, pneumomediastinum)
- Lethal congenital malformations; death anticipated within first 3 days of life; decision to withhold support
- Serious abdominal, cardiac, airway or respiratory malformations including tracheal esophageal fistula, intestinal atresia, omphalocele, gastroschisis, pulmonary hypoplasia, or diaphragmatic hernia
- Neuromuscular disorder resulting in respiratory compromise
Data sourced from ClinicalTrials.gov (NCT02294630). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.