Trial Outcomes & Findings for Feasibility Study of Aerosolized Surfaxin in the Prevention of Respiratory Distress Syndrome (RDS) in Premature Infants (NCT NCT00807235)
NCT ID: NCT00807235
Last Updated: 2012-06-13
Results Overview
TERMINATED
PHASE2
17 participants
24 hours
2012-06-13
Participant Flow
Neonates were enrolled between January and August, 2005. This was an open-label study with 2 treatment groups (Regimen 1 and Regimen 2). Eligible neonates were sequentially enrolled and stratified by GA into 2 strata for each regimen. For each regimen, enrollment in Stratum 1 (30 to 32 GA) was completed before enrollment in Stratum 2 (28 to 29 GA).
Participant milestones
| Measure |
Lucinactant - 3 Hour Interval
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Overall Study
STARTED
|
11
|
6
|
|
Overall Study
COMPLETED
|
10
|
6
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
| Measure |
Lucinactant - 3 Hour Interval
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
Baseline Characteristics
Feasibility Study of Aerosolized Surfaxin in the Prevention of Respiratory Distress Syndrome (RDS) in Premature Infants
Baseline characteristics by cohort
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
Total
n=17 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
11 Participants
n=93 Participants
|
6 Participants
n=4 Participants
|
17 Participants
n=27 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=93 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=27 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=93 Participants
|
2 Participants
n=4 Participants
|
9 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=93 Participants
|
4 Participants
n=4 Participants
|
8 Participants
n=27 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=93 Participants
|
6 participants
n=4 Participants
|
17 participants
n=27 Participants
|
|
Gestational Age
|
29.8 weeks
STANDARD_DEVIATION 1.40 • n=93 Participants
|
30.7 weeks
STANDARD_DEVIATION 0.99 • n=4 Participants
|
30.1 weeks
STANDARD_DEVIATION 1.31 • n=27 Participants
|
PRIMARY outcome
Timeframe: 24 hoursPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Respiratory Distress Syndrome
|
4 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 0.5, 1, 2, 4, 6, 12, 18, 24, 36, 48, 60, 72 hoursPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
AUC for FiO₂calculated using the trapezoidal rule. Missing data imputed using last observation carried forward
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Area Under the Curve (AUC) for Fraction of Inspired Oxygen (FiO₂)
|
0.36 percent O₂*hours
Standard Deviation 0.15
|
0.25 percent O₂*hours
Standard Deviation 0.08
|
SECONDARY outcome
Timeframe: 72 hoursPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
a/A ratio is a relative way to judge the lungs ability to transport O₂. It compares the partial pressure of O₂in the alveoli (A) to the partial pressure of O₂in the artery (a). It is calculated by dividing the partial pressure of O₂in the artery, abbreviated PaO2, by the partial pressure of O₂in the alveoli using the alveolar gas equation, abbreviated PAO2. A value of 0.80 or above is normal, a value of 0.60 or below may be incompatible with spontaneous breathing, and a value below 0.22 indicates severe lung disease.
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Arterial Alveolar (a/A) O₂Ratio
|
0.46 mm Hg over mm Hg (ratio of pressures)
Standard Deviation 0.25
|
0.53 mm Hg over mm Hg (ratio of pressures)
Standard Deviation 0.29
|
SECONDARY outcome
Timeframe: Through 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat). Time in days calculated for neonates who met failure criteria (3 for Regimen 1, 2 for Regimen 2)
Failure criteria defined as rescue with bolus surfactant and mechanical ventilation
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Time to Meet Failure Criteria
|
0.49 days
Standard Deviation 0.38
|
0.30 days
Standard Deviation 0.35
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Bronchopulmonary Dysplasia (BPD)
|
1 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants Alive and Without BPD
|
10 participants
|
5 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Intraventricular Hemorrhage (IVH)/Periventricular Leukomalacia (PVL)
|
1 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Patent Ductus Arteriosus (PDA)
|
3 participants
|
3 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Necrotizing Enterocolitis (NEC)
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Pulmonary Hemorrhage
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Acquired Sepsis
|
2 participants
|
1 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Incidence of Mortality
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: 28 daysPopulation: This was a phase 2, pilot, estimation study. Hence, sample size calculations were not performed. All enrolled infants analyzed (intent-to-treat).
Outcome measures
| Measure |
Lucinactant - 3 Hour Interval
n=11 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 Participants
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Number of Participants With Air Leak
|
0 participants
|
0 participants
|
Adverse Events
Lucinactant - 3 Hour Interval
Lucinactant - 1 Hour Interval
Serious adverse events
| Measure |
Lucinactant - 3 Hour Interval
n=11 participants at risk
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 participants at risk
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Infections and infestations
Sepsis neonatal
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Investigations
Oxygen saturation decreased
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal apnoeic attack
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal respiratory distress syndrome
|
27.3%
3/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal respiratory failure
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Vascular disorders
Pulmonary hypertension NOS
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
Other adverse events
| Measure |
Lucinactant - 3 Hour Interval
n=11 participants at risk
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 3 hours
|
Lucinactant - 1 Hour Interval
n=6 participants at risk
Aerosolized Lucinactant via nasal continuous positive airway pressure (nCPAP) w/ Retreatment after 1 hour
|
|---|---|---|
|
Blood and lymphatic system disorders
Anaemia neonatal
|
18.2%
2/11 • 28 days
|
50.0%
3/6 • 28 days
|
|
Cardiac disorders
Bradycardia NOS
|
9.1%
1/11 • 28 days
|
50.0%
3/6 • 28 days
|
|
Congenital, familial and genetic disorders
Patent ductus arteriosus
|
36.4%
4/11 • 28 days
|
50.0%
3/6 • 28 days
|
|
Gastrointestinal disorders
Abdominal distension
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Gastrointestinal disorders
Gastro-oesophageal reflux disease
|
0.00%
0/11 • 28 days
|
33.3%
2/6 • 28 days
|
|
General disorders
Oedema NOS
|
9.1%
1/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Hepatobiliary disorders
Jaundice neonatal
|
27.3%
3/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Infections and infestations
Abscess NOS
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Infections and infestations
Pneumonia NOS
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Infections and infestations
Sepsis neonatal
|
18.2%
2/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Investigations
Oxygen saturation decreased
|
54.5%
6/11 • 28 days
|
50.0%
3/6 • 28 days
|
|
Metabolism and nutrition disorders
Acidosis NOS
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Metabolism and nutrition disorders
Hyperglycaemia NOS
|
9.1%
1/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Metabolism and nutrition disorders
Hypermagnesaemia
|
27.3%
3/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Metabolism and nutrition disorders
Hypocalcaemia
|
36.4%
4/11 • 28 days
|
33.3%
2/6 • 28 days
|
|
Metabolism and nutrition disorders
Hypoglycaemia neonatal
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Metabolism and nutrition disorders
Hyponatraemia
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Metabolism and nutrition disorders
Metabolic acidosis NOS
|
27.3%
3/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Pregnancy, puerperium and perinatal conditions
Feeding problem in newborn
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Psychiatric disorders
Agitation neonatal
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Bronchopulmonary dysplasia
|
27.3%
3/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Hypercapnia
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Nasal passage irritation
|
18.2%
2/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal apnoeic attach
|
90.9%
10/11 • 28 days
|
100.0%
6/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal respiratory distress syndrome
|
54.5%
6/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Neonatal respiratory failure
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary haemorrhage
|
0.00%
0/11 • 28 days
|
16.7%
1/6 • 28 days
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Skin and subcutaneous tissue disorders
Skin irritation
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Vascular disorders
Intraventricular haemorrhage neonatal
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Vascular disorders
Neonatal hypotension
|
9.1%
1/11 • 28 days
|
0.00%
0/6 • 28 days
|
|
Vascular disorders
Pulmonary hypertension NOS
|
9.1%
1/11 • 28 days
|
16.7%
1/6 • 28 days
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: GT60