Trial Outcomes & Findings for Inhaled Nitric Oxide and Neuroprotection in Premature Infants (NCT NCT00515281)

NCT ID: NCT00515281

Last Updated: 2024-10-17

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2/PHASE3
• Target enrollment: 273 participants
• Primary outcome timeframe: Two years
• Results posted on: 2024-10-17

Participant Flow

Participant milestones

Measure	INO Control (Short iNO) INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Overall Study STARTED	138	135
Overall Study COMPLETED	138	135
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Inhaled Nitric Oxide and Neuroprotection in Premature Infants

Baseline characteristics by cohort

Measure	INO Control (Short iNO) n=138 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.	Total n=273 Participants Total of all reporting groups
Age, Continuous	27.2 Weeks STANDARD_DEVIATION 2.2 • n=5 Participants	26.8 Weeks STANDARD_DEVIATION 2.1 • n=7 Participants	27.0 Weeks STANDARD_DEVIATION 2.2 • n=5 Participants
Sex: Female, Male Female	63 Participants n=5 Participants	63 Participants n=7 Participants	126 Participants n=5 Participants
Sex: Female, Male Male	75 Participants n=5 Participants	72 Participants n=7 Participants	147 Participants n=5 Participants
Race/Ethnicity, Customized Black	99 Participants n=5 Participants	91 Participants n=7 Participants	190 Participants n=5 Participants
Race/Ethnicity, Customized Non-black	39 Participants n=5 Participants	44 Participants n=7 Participants	83 Participants n=5 Participants
Region of Enrollment United States	138 participants n=5 Participants	135 participants n=7 Participants	273 participants n=5 Participants

PRIMARY outcome

Timeframe: Two years

Population: Data were not collected (due to funding cuts).

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: 36 weeks of age corrected (BPD) or before discharge (death)

Need for respiratory support at 36 weeks post-menstrual age, either as positive pressure or supplemental oxygen to maintain oxygen saturations >90%, or death before discharge

Outcome measures

Measure	INO Control (Short iNO) n=138 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Bronchopulmonary Dysplasia (BPD) or Death	65 Participants	83 Participants

SECONDARY outcome

Timeframe: through discharge (up to 400 days)

Death before discharge

Outcome measures

Measure	INO Control (Short iNO) n=138 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Death	19 Participants	13 Participants

SECONDARY outcome

Timeframe: 36 weeks of age corrected

Need for respiratory support at 36 weeks post-menstrual age (PMA), either as positive pressure or supplemental oxygen to maintain oxygen saturations >90%

Outcome measures

Measure	INO Control (Short iNO) n=138 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Bronchopulmonary Dysplasia	51 Participants	71 Participants

SECONDARY outcome

Timeframe: 40 weeks PMA

Outcome measures

Measure	INO Control (Short iNO) n=138 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Supplemental Oxygen Use	30 Participants	28 Participants

SECONDARY outcome

Timeframe: 36 weeks of age corrected (BPD) or before discharge (death)

Need for respiratory support at 36 weeks post-menstrual age, either as positive pressure or supplemental oxygen to maintain oxygen saturations >90%, or death before discharge among infants < 750g

Outcome measures

Measure	INO Control (Short iNO) n=41 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=41 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
BPD or Death in Infants Weighing < 750g	36 Participants	37 Participants

SECONDARY outcome

Timeframe: 36 weeks of age corrected (BPD) or before discharge (death)

Need for respiratory support at 36 weeks post-menstrual age, either as positive pressure or supplemental oxygen to maintain oxygen saturations >90%, or death before discharge among infants 750-999g

Outcome measures

Measure	INO Control (Short iNO) n=43 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=44 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
BPD or Death in Infants Weighing 750-999g	20 Participants	36 Participants

SECONDARY outcome

Timeframe: 36 weeks of age corrected (BPD) or before discharge (death)

Need for respiratory support at 36 weeks post-menstrual age, either as positive pressure or supplemental oxygen to maintain oxygen saturations >90%, or death before discharge among Black infants

Outcome measures

Measure	INO Control (Short iNO) n=99 Participants INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=91 Participants The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
BPD or Death in Black Infants	50 Participants	53 Participants

Adverse Events

INO Control (Short iNO)

Serious events: 53 serious events

Other events: 0 other events

Deaths: 19 deaths

INO Treatment (Long iNO)

Serious events: 61 serious events

Other events: 0 other events

Deaths: 13 deaths

Serious adverse events

Measure	INO Control (Short iNO) n=138 participants at risk INO will be given to infants in the control group for the first 7 days of the study gas, then O2 or room air, as clinically appropriate, on the 8th day, until 33 weeks corrected age inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm.	INO Treatment (Long iNO) n=135 participants at risk The treatment group will receive iNO, combined with O2 or room air, until 33 weeks corrected age. inhaled nitric oxide: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of subject's lungs. The subject will remain on inhaled nitric oxide until he/she reaches 33 weeks of gestation if assigned to the treatment arm, or until the 8th day of the study if he/she is assigned to the control arm. oxygen: The amount of gas will be carefully controlled and adjusted to the level that best improves the function of the subject's lungs. Subjects in the control arm of the study will receive oxygen beginning on the 8th day of the study until he/she reaches 33 weeks of gestation.
Vascular disorders Severe IVH/PVL	9.4% 13/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	11.9% 16/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.
Respiratory, thoracic and mediastinal disorders Pulmonary hemorrhage	1.4% 2/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	3.7% 5/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.
Respiratory, thoracic and mediastinal disorders Pneumorthorax	9.4% 13/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	7.4% 10/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.
Respiratory, thoracic and mediastinal disorders Pulmonary interstitial emphysema (PIE)	13.8% 19/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	19.3% 26/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.
Cardiac disorders Symptomatic patent ductus arteriosis (PDA)	32.6% 45/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	30.4% 41/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.
Gastrointestinal disorders Necrotizing enterocolitis (NEC) of Bell's stage 2 severity or greater	10.1% 14/138 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.	14.1% 19/135 • through discharge (up to 400 days) Standard perinatal morbidities were tracked and reported and all are considered serious adverse events (SAEs). Non-serious adverse events were not defined or tracked.

Other adverse events

Adverse event data not reported

Additional Information

Michael D. Schreiber, MD

University of Chicago

Phone: 773-702-6205

Email: mschreiber@bsd.uchicago.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place