Trial Outcomes & Findings for Inhaled Nitric Oxide for Pulmonary Hypertension and Bronchopulmonary Dysplasia (NCT NCT03576885)
NCT ID: NCT03576885
Last Updated: 2025-01-15
Results Overview
Incidence of death
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
32 participants
Primary outcome timeframe
36 weeks post menstrual age
Results posted on
2025-01-15
Participant Flow
Participant milestones
| Measure |
Treatment Group - Active
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Control Group
Enrolled infants with no evidence of pulmonary hypertension will serve as the control group for incidence of death or bronchopulmonary hypertension
|
|---|---|---|---|
|
Overall Study
STARTED
|
16
|
16
|
0
|
|
Overall Study
COMPLETED
|
16
|
16
|
0
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Inhaled Nitric Oxide for Pulmonary Hypertension and Bronchopulmonary Dysplasia
Baseline characteristics by cohort
| Measure |
Treatment Group - Active
n=16 Participants
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 Participants
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Control Group
Enrolled infants with no evidence of pulmonary hypertension will serve as the control group for incidence of death or bronchopulmonary hypertension
|
Total
n=32 Participants
Total of all reporting groups
|
|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
32 Participants
n=4 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Age, Continuous
|
3 days
STANDARD_DEVIATION 1 • n=5 Participants
|
3 days
STANDARD_DEVIATION 1 • n=7 Participants
|
—
|
3 days
STANDARD_DEVIATION 1 • n=4 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
5 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
10 Participants
n=4 Participants
|
|
Sex: Female, Male
Male
|
11 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
22 Participants
n=4 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
1 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Black or African American
|
7 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
14 Participants
n=4 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
15 Participants
n=4 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
2 Participants
n=4 Participants
|
|
Region of Enrollment
United States
|
16 Participants
n=5 Participants
|
16 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
32 Participants
n=4 Participants
|
PRIMARY outcome
Timeframe: 36 weeks post menstrual ageIncidence of death
Outcome measures
| Measure |
Treatment Group - Active
n=16 Participants
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 Participants
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
|---|---|---|
|
Death
|
1 Participants
|
1 Participants
|
PRIMARY outcome
Timeframe: 36 weeks post menstrual ageIncidence of bronchopulmonary dysplasia as determined by need for positive pressure ventilation or supplemental oxygen after room air challenge test
Outcome measures
| Measure |
Treatment Group - Active
n=16 Participants
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 Participants
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
|---|---|---|
|
Bronchopulmonary (BPD) Dysplasia (Yes/no)
|
10 Participants
|
9 Participants
|
SECONDARY outcome
Timeframe: Duration of treatment or up to Day 14Pulmonary arterial pressure will be evaluated every 24-48 hours after enrollment.
Outcome measures
| Measure |
Treatment Group - Active
n=16 Participants
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 Participants
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
|---|---|---|
|
Pulmonary Hypertension
|
11 Participants
|
15 Participants
|
Adverse Events
Treatment Group - Active
Serious events: 2 serious events
Other events: 14 other events
Deaths: 1 deaths
Treatment Group - Placebo
Serious events: 5 serious events
Other events: 14 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment Group - Active
n=16 participants at risk
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 participants at risk
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
|---|---|---|
|
Nervous system disorders
IVH
|
12.5%
2/16 • Number of events 2 • Until 36 weeks post-menstrual age
|
12.5%
2/16 • Number of events 2 • Until 36 weeks post-menstrual age
|
|
Gastrointestinal disorders
NEC
|
0.00%
0/16 • Until 36 weeks post-menstrual age
|
18.8%
3/16 • Number of events 3 • Until 36 weeks post-menstrual age
|
Other adverse events
| Measure |
Treatment Group - Active
n=16 participants at risk
inhaled nitric oxide treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
inhaled nitric oxide: inhaled nitric oxide will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
Treatment Group - Placebo
n=16 participants at risk
Placebo treatment will start at 20 ppm and continue for 2 weeks or until resolution of pulmonary hypertension, whichever comes first.
Placebo: placebo will be administered starting at 20 ppm. Treatment will continue for 14 days or until resolution of pulmonary hypertension
|
|---|---|---|
|
Blood and lymphatic system disorders
MetHb% >2
|
18.8%
3/16 • Number of events 3 • Until 36 weeks post-menstrual age
|
31.2%
5/16 • Number of events 5 • Until 36 weeks post-menstrual age
|
|
Infections and infestations
Culture positive sepsis
|
31.2%
5/16 • Number of events 5 • Until 36 weeks post-menstrual age
|
56.2%
9/16 • Number of events 9 • Until 36 weeks post-menstrual age
|
|
Cardiac disorders
PDA requiring treatment
|
18.8%
3/16 • Number of events 3 • Until 36 weeks post-menstrual age
|
25.0%
4/16 • Number of events 4 • Until 36 weeks post-menstrual age
|
|
Cardiac disorders
Left Ventricular Dysfunction
|
6.2%
1/16 • Number of events 1 • Until 36 weeks post-menstrual age
|
6.2%
1/16 • Number of events 1 • Until 36 weeks post-menstrual age
|
|
Hepatobiliary disorders
Hyperbilirubinemia
|
43.8%
7/16 • Number of events 7 • Until 36 weeks post-menstrual age
|
43.8%
7/16 • Number of events 7 • Until 36 weeks post-menstrual age
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.2%
1/16 • Number of events 1 • Until 36 weeks post-menstrual age
|
18.8%
3/16 • Number of events 3 • Until 36 weeks post-menstrual age
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place