Trial Outcomes & Findings for Extending CPAP Therapy in Stable Preterm Infants to Increase Lung Growth and Function (NCT NCT04295564)

Last Updated: 2025-08-17

Results Overview

Establish that 2 additional weeks of CPAP in the NICU for stable preterm infants changes alveolar volume at approximately 6 months of age compared to infants who have CPAP discontinued, usual care. Measurements of alveolar volume were obtained at the same time as measurements of lung diffusion using an induced respiratory pause technique at an elevated lung volume of 30 cmH2O. During the passive expiration following the 4 second induced respiratory pause for gas exchange, carbon monoxide (CO) and helium (He) concentrations are used to calculate alveolar volume and lung diffusion. Results were expressed as averages of 2-3 measurements within 10%, adjusting for hemoglobin.

Recruitment status

COMPLETED

Study phase

Target enrollment

100 participants

Primary outcome timeframe

4 - 8 months of age

Results posted on

2025-08-17

Participant Flow

Participant milestones

Participant milestones
Measure	Extended Continuous Positive Airway Pressure (eCPAP) Participants will remain on continuous positive airway pressure (CPAP) for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	Discontinue Continuous Positive Airway Pressure (dCPAP) Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Overall Study STARTED	54	46
Overall Study COMPLETED	53	46
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Reasons for withdrawal
Measure	Extended Continuous Positive Airway Pressure (eCPAP) Participants will remain on continuous positive airway pressure (CPAP) for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	Discontinue Continuous Positive Airway Pressure (dCPAP) Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Overall Study Withdrawn by PI after discharge due to diagnosis of a rare chromosomal mutation.	1	0

Baseline Characteristics

Extending CPAP Therapy in Stable Preterm Infants to Increase Lung Growth and Function

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	eCPAP n=54 Participants Participants randomized to stay on CPAP for 2 additional weeks once CPAP stability criteria were met.	dCPAP n=46 Participants Participants randomized to discontinue CPAP once CPAP stability criteria were met.	Total n=100 Participants Total of all reporting groups
Age, Continuous	32.4 weeks of postmenstrual age STANDARD_DEVIATION 0.9 • n=5 Participants	32.4 weeks of postmenstrual age STANDARD_DEVIATION 0.8 • n=7 Participants	32.4 weeks of postmenstrual age STANDARD_DEVIATION 0.9 • n=5 Participants
Sex: Female, Male Female	19 Participants n=5 Participants	24 Participants n=7 Participants	43 Participants n=5 Participants
Sex: Female, Male Male	35 Participants n=5 Participants	22 Participants n=7 Participants	57 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	19 Participants n=5 Participants	9 Participants n=7 Participants	28 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	34 Participants n=5 Participants	37 Participants n=7 Participants	71 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants	2 Participants n=7 Participants	5 Participants n=5 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
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) White	42 Participants n=5 Participants	33 Participants n=7 Participants	75 Participants n=5 Participants
Race (NIH/OMB) More than one race	6 Participants n=5 Participants	7 Participants n=7 Participants	13 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	54 participants n=5 Participants	46 participants n=7 Participants	100 participants n=5 Participants

PRIMARY outcome

Timeframe: 4 - 8 months of age

Population: Decrease in the number of participants analyzed versus those initially randomized due to: Seven infants in the eCPAP arm did not complete VA testing due to the following reasons: 1 diagnosed with ARID1B mutation diagnosed after discharge and discontinued from study by the principal investigator; 1 with seizure disorder so could not be sedated; 3 mothers (one with a set of twins) refused sedation for the outpatient test; 1 infant with a technically unacceptable test.

Outcome measures

Outcome measures
Measure	eCPAP n=47 Participants Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=46 Participants Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Alveolar Volume	500.2 mL Standard Error 24.9	418.1 mL Standard Error 23.4

SECONDARY outcome

Timeframe: 4 - 8 months of age

Establish that 2 extra weeks of CPAP in stable preterm infants increases lung diffusion at approximately 6 months of age versus infants who have CPAP discontinued, usual care. Measurements of lung diffusion were obtained at the same time as measurements of alveolar volume using an induced respiratory pause technique at an elevated lung volume of 30 cmH2O. During the passive expiration following the 4 second induced respiratory pause for gas exchange, carbon monoxide (CO) and helium (He) concentrations are used to calculate alveolar volume and lung diffusion. Results were expressed as averages of 2-3 measurements within 10%, adjusting for hemoglobin.

Outcome measures

Outcome measures
Measure	eCPAP n=47 Participants Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=46 Participants Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Lung Diffusion	3.4 mL/min/mmHg Standard Error 0.2	2.8 mL/min/mmHg Standard Error 0.1

SECONDARY outcome

Timeframe: 4 - 8 months of age

Population: Decrease in the number of participants analyzed versus those initially randomized due to: Infants in the eCPAP arm did not complete VA testing due to the following reasons: 1 diagnosed with ARID1B mutation diagnosed after discharge and discontinued from study by the principal investigator; 1 with seizure disorder so could not be sedated; 3 mothers (one with a set of twins) refused sedation for the outpatient test; and remainder with technically unacceptable test.

Establish that 2 extra weeks of CPAP in stable preterm infants increases forced expiratory flows at approximately 6 months of age versus infants who have CPAP discontinued, usual care. Forced expiratory flows were measured using the raised volume rapid thoracic compression technique following specific American Thoracic Society and European Respiratory Society criteria for acceptance.

Outcome measures

Outcome measures
Measure	eCPAP n=38 Participants Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=38 Participants Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Forced Expiratory Flows at 50% of the Expired Volume (FEF50)	500.6 mL/sec Standard Error 18.2	437.9 mL/sec Standard Error 17.9

OTHER_PRE_SPECIFIED outcome

Timeframe: through 12 months of age (+/- 2 months)

Evaluate whether 2 extra weeks of CPAP in stable preterm infants results in lower respiratory morbidity through 12 months of age compared to infants who had CPAP discontinued, usual care. A standardized respiratory questionnaire was administered monthly through 12 months of age to ascertain this information on wheeze which was defined a priori.

Outcome measures

Outcome measures
Measure	eCPAP n=53 Participants Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=46 Participants Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Number of Participants With the Occurrence of Wheeze	23 Participants	26 Participants

OTHER_PRE_SPECIFIED outcome

Timeframe: through 12 months of age (+/- 2 months)

Evaluate whether 2 extra weeks of CPAP in stable preterm infants results in improved neurodevelopmental outcomes (Bayley III Gross Motor Scores) through 12 months of age compared to infants who had CPAP discontinued, usual care. These exams were performed by a licensed psychologist in the neurodevelopmental clinic.

Outcome measures

Outcome data not reported

Adverse Events

eCPAP

Serious events: 1 serious events

Other events: 37 other events

Deaths: 0 deaths

dCPAP

Serious events: 1 serious events

Other events: 29 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	eCPAP n=54 participants at risk Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=46 participants at risk Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Respiratory, thoracic and mediastinal disorders Vocal cord paralysis	0.00% 0/54 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	2.2% 1/46 • Number of events 1 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Infections and infestations streptococcus group B sepsis	1.9% 1/54 • Number of events 1 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	0.00% 0/46 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.

Other adverse events

Other adverse events
Measure	eCPAP n=54 participants at risk Participants will remain on CPAP for 2 additional weeks once CPAP stability criteria is met. Additional 2 weeks of CPAP: Subjects will be randomized to an additional 2 weeks of CPAP vs. discontinuing CPAP per usual care.	dCPAP n=46 participants at risk Participants will discontinue CPAP as per usual care once CPAP stability criteria is met.
Eye disorders Retinopathy of prematurity	16.7% 9/54 • Number of events 9 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	10.9% 5/46 • Number of events 5 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Respiratory, thoracic and mediastinal disorders Neonatal respiratory insufficiency	16.7% 9/54 • Number of events 16 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	13.0% 6/46 • Number of events 11 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Respiratory, thoracic and mediastinal disorders Apnea neonatal	16.7% 9/54 • Number of events 11 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	28.3% 13/46 • Number of events 18 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Metabolism and nutrition disorders Feeding disorder NOS	3.7% 2/54 • Number of events 2 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	6.5% 3/46 • Number of events 3 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Respiratory, thoracic and mediastinal disorders Bronchopulmonary dysplasia	7.4% 4/54 • Number of events 4 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	6.5% 3/46 • Number of events 3 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Skin and subcutaneous tissue disorders Nasal mucosal erythema	5.6% 3/54 • Number of events 3 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	4.3% 2/46 • Number of events 2 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Skin and subcutaneous tissue disorders Hemangioma of skin	0.00% 0/54 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	6.5% 3/46 • Number of events 3 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.
Eye disorders Anemia of prematurity	25.9% 14/54 • Number of events 14 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.	19.6% 9/46 • Number of events 9 • From consent through 12 months of age. Definition: Any untoward or unfavorable medical occurrence in a participant, including any abnormal sign (for example, abnormal physical exam or laboratory finding), symptom, or disease, temporally associated with the participant's participation in the research, whether or not considered related to the participant's participation in the research.

Additional Information

Dr. Cindy McEvoy

OHSU

Phone: 503-494-0223

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place