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CompletedNCT03385330BPD STARUpdated Apr 30, 2024Results posted

BPD Saturation TARgeting

An interventional study of LOWER oxygen saturation target group and HIGHER oxygen saturation target group in Bronchopulmonary Dysplasia, Chronic Lung Disease of Prematurity and Chronic Lung Disease, sponsored by Children's Hospital of Philadelphia. Completed at 3 sites in United States. Open to participants aged 34 Weeks to 44 Weeks. Per ClinicalTrials.gov, last updated 2024-04-30.

Sponsored by Children's Hospital of Philadelphia · Not applicable, Interventional, and Treatment

Phase
Not applicable
Study type
Interventional
Enrollment
50
Allocation
Randomized
Ages
34 Weeks to 44 Weeks
Sex
All
01

Study summary

Bronchopulmonary dysplasia (BPD), or chronic lung disease of prematurity, affects nearly half of extremely preterm infants.This study evaluates the use of supplemental oxygen to manage infants with established BPD. Participants will be randomly placed in either a higher oxygen saturation group or a lower oxygen saturation target group.

Read the detailed description

Bronchopulmonary Dysplasia is diagnosed only in babies who are born prematurely, and affects about half of extremely preterm infants. The incidence of BPD has increased over time. It is most commonly defined as oxygen dependence at 36 weeks postmenstrual age (PMA).

Infants with BPD face more than doubled odds of death after 36 weeks PMA or disability at 5 years compared to preterm infants without BPD. BPD is associated with abnormal lung function throughout childhood and significantly increases health care costs. Cognitive and respiratory outcomes are closely linked throughout the life course; thus, optimal long--term management of BPD during infancy may ultimately improve cognitive outcomes of this high--risk population.

Supplemental oxygen is a lifesaving therapy for premature infants; yet, there is limited evidence about the safety or efficacy of using supplemental oxygen to target higher versus lower oxygen saturations in infants with established BPD.

Infants between the ages of 34-44 weeks post-menstrual age with moderate or severe BPD will be randomly assigned to higher or lower oxygen saturation target ranges. The study intervention will begin in the hospital and will continue at home until 6 months corrected age. When infants are discharged with supplemental oxygen, this will be titrated according to a study algorithm in order to ensure that the target saturations are maintained throughout the study period.

02

Conditions studied

  • Bronchopulmonary Dysplasia
  • Chronic Lung Disease of Prematurity
  • Chronic Lung Disease

Keywords

  • BPD
  • Prematurity
  • Supplemental Oxygen
  • Oximetry
  • Infants
03

Who can participate

Ages eligible
34 Weeks to 44 Weeks
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Pre-term males or females infants born at \<30 0/7 weeks gestation at birth
  • Current age 34 0/7 to 43 6/7 weeks postmenstrual age
  • Diagnosis of moderate or severe Bronchopulmonary Dysplasia based on the NIH consensus definition
  • Infant has never been discharged to home from the hospital

Exclusion criteria

Exclusion Criteria:

  • Congenital anomaly or oncologic process likely to affect growth or respiratory status
  • Hemoglobinopathy or other blood disorder likely to affect oxygen saturations
  • Contraindication to nasal cannula use (for example, severe nasal septal breakdown).
  • Pulmonary hypertension requiring pharmacotherapy at the time of screening/enrollment.
  • Tracheostomy
  • Intubated during entire eligibility period
04

Study design

Phase
Not applicable
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
50 participants (actual)

Study arms

  • Active comparator
    LOWER oxygen saturation target group

    Oxygen saturation (SpO2) target range 90--94%. The study intervention will begin in the hospital and will continue at home until 6 months corrected age (CA). Overnight continuous oximetry will be transmitted wirelessly to the study team. In hospital, inspired oxygen will be adjusted as needed to maintain target SpO2. Monitoring will continue after discharge, and oxygen flow will be titrated monthly according to a standardized algorithm.

    Other: LOWER oxygen saturation target group

  • Active comparator
    HIGHER oxygen saturation target group

    Oxygen saturation target range greater than or equal to 96%.The study intervention will begin in the hospital and will continue at home until 6 months CA. Overnight continuous oximetry will be transmitted wirelessly to the study team. In hospital, inspired oxygen will be adjusted as needed to maintain target SpO2. Monitoring will continue after discharge, and oxygen flow will be titrated monthly according to a standardized algorithm.

    Other: HIGHER oxygen saturation target group

Interventions

  • OtherLOWER oxygen saturation target group

    We will randomize infants with moderate or severe BPD to lower SpO2 target ranges. The study team will then use novel technology to monitor infants starting at randomization, continuing after discharge and until 6 months corrected age, titrating supplemental oxygen to achieve these target SpO2 ranges.

  • OtherHIGHER oxygen saturation target group

    We will randomize infants with moderate or severe BPD to higher SpO2 target ranges. The study team will then use novel technology to monitor infants starting at randomization, continuing after discharge and until 6 months corrected age, titrating supplemental oxygen to achieve these target SpO2 ranges.

05

What researchers measure

Primary outcomes

  1. Intermittent Hypoxemia (IH) Events Per 8 Hours of Monitoring Time

    Incidence of intermittent hypoxia (IH, defined as SpO2 \<80% for \>=30 seconds), reported as median number of events per 8 hours of monitoring time.

    Time frame: Between discharge and 6 months corrected age

Secondary outcomes

  1. Duration of Hypoxia - Proportion of Monitored Time With Oxygen Saturation <80%

    Total exposure to hypoxia - proportion of monitored time with SpO2 \<80%

    Time frame: Between discharge and 6 months corrected age

  2. Change in Weight Z-score

    Change in weight Z-score. A Z-score of 0 represents average weight. Positive change in Z score indicates increasing weight relative to the population average weight. Negative change in Z score indicates decreasing weight relative to the population average weight.

    Time frame: Between randomization and 6 months corrected age

  3. Change in Length Z-score

    Change in length Z-score. A Z-score of 0 represents average length. Positive change in Z score indicates increasing length relative to the population average length. Negative change in Z score indicates decreasing length relative to the population average length.

    Time frame: Between randomization and 6 months corrected age

  4. Change in Head Circumference Z-score

    Change in head circumference (HC) Z-score. A Z-score of 0 represents average HC. Positive change in Z score indicates increasing HC relative to the population average HC. Negative change in Z score indicates decreasing HC relative to the population average HC.

    Time frame: Between randomization and 6 months corrected age

  5. Number of Participants With Re-hospitalization

    Any re-hospitalization

    Time frame: Between discharge and 6 months corrected age

  6. Number of Participants With Respiratory Medication Use

    Number of patients with inhaled respiratory medication use

    Time frame: At 6 months corrected age

  7. Number of Participants With Visits to Emergency Room or Urgent Care for Respiratory Reasons

    Any visits to ER or urgent care for respiratory health-related problems

    Time frame: Between discharge and 6 months corrected age

  8. Feeding

    Quality of infant oral feeding skills, parent reports that the child's feeding times are stressful or very stressful for themselves

    Time frame: At 6 months corrected age

  9. Development

    Bayley Scales of Infant Development screening test, which is a standardized assessment of cognitive, motor, and language development in infancy and early childhood. Outcome will be reported as number of children considered "at risk" in any domain.

    Time frame: At 6 months corrected age

06

Results

Posted Apr 30, 2024

Participant flow

Participant flow — Overall Study
MilestoneLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Started2822
Completed2319
Not completed53

Outcome measures

PrimaryIntermittent Hypoxemia (IH) Events Per 8 Hours of Monitoring Time

Incidence of intermittent hypoxia (IH, defined as SpO2 \<80% for \>=30 seconds), reported as median number of events per 8 hours of monitoring time.

Time frame:
Between discharge and 6 months corrected age
Reported as:
Median · IH events per 8 hours of monitoring time
Intermittent Hypoxemia (IH) Events Per 8 Hours of Monitoring Time
IH events per 8 hours of monitoring timeLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Intermittent Hypoxemia (IH) Events Per 8 Hours of Monitoring Time2.2 (1.0 to 4.2)1.2 (0.3 to 3.1)
SecondaryDuration of Hypoxia - Proportion of Monitored Time With Oxygen Saturation <80%

Total exposure to hypoxia - proportion of monitored time with SpO2 \<80%

Time frame:
Between discharge and 6 months corrected age
Reported as:
Median · proportion of monitored time
Duration of Hypoxia - Proportion of Monitored Time With Oxygen Saturation <80%
proportion of monitored timeLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Duration of Hypoxia - Proportion of Monitored Time With Oxygen Saturation <80%0.005 (0.001 to 0.016)0.004 (0.001 to 0.013)
SecondaryChange in Weight Z-score

Change in weight Z-score. A Z-score of 0 represents average weight. Positive change in Z score indicates increasing weight relative to the population average weight. Negative change in Z score indicates decreasing weight relative to the population average weight.

Time frame:
Between randomization and 6 months corrected age
Reported as:
Mean · delta Z-score
Change in Weight Z-score
delta Z-scoreLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Change in Weight Z-score0.6 ± 1.40.5 ± 1.2
SecondaryChange in Length Z-score

Change in length Z-score. A Z-score of 0 represents average length. Positive change in Z score indicates increasing length relative to the population average length. Negative change in Z score indicates decreasing length relative to the population average length.

Time frame:
Between randomization and 6 months corrected age
Reported as:
Mean · delta Z-score
Change in Length Z-score
delta Z-scoreLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Change in Length Z-score1.7 ± 1.31.2 ± 1.4
SecondaryChange in Head Circumference Z-score

Change in head circumference (HC) Z-score. A Z-score of 0 represents average HC. Positive change in Z score indicates increasing HC relative to the population average HC. Negative change in Z score indicates decreasing HC relative to the population average HC.

Time frame:
Between randomization and 6 months corrected age
Reported as:
Mean · delta Z-score
Change in Head Circumference Z-score
delta Z-scoreLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Change in Head Circumference Z-score0.5 ± 1.30.4 ± 1.7
SecondaryNumber of Participants With Re-hospitalization

Any re-hospitalization

Time frame:
Between discharge and 6 months corrected age
Reported as:
Count of participants · Participants
Number of Participants With Re-hospitalization
ParticipantsLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Number of Participants With Re-hospitalization96
SecondaryNumber of Participants With Respiratory Medication Use

Number of patients with inhaled respiratory medication use

Time frame:
At 6 months corrected age
Reported as:
Count of participants · Participants
Number of Participants With Respiratory Medication Use
ParticipantsLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Number of Participants With Respiratory Medication Use66
SecondaryNumber of Participants With Visits to Emergency Room or Urgent Care for Respiratory Reasons

Any visits to ER or urgent care for respiratory health-related problems

Time frame:
Between discharge and 6 months corrected age
Reported as:
Count of participants · Participants
Number of Participants With Visits to Emergency Room or Urgent Care for Respiratory Reasons
ParticipantsLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Number of Participants With Visits to Emergency Room or Urgent Care for Respiratory Reasons52
SecondaryFeeding

Quality of infant oral feeding skills, parent reports that the child's feeding times are stressful or very stressful for themselves

Time frame:
At 6 months corrected age
Reported as:
Count of participants · Participants
Feeding
ParticipantsLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Feeding10
SecondaryDevelopment

Bayley Scales of Infant Development screening test, which is a standardized assessment of cognitive, motor, and language development in infancy and early childhood. Outcome will be reported as number of children considered "at risk" in any domain.

Time frame:
At 6 months corrected age
Reported as:
Count of participants · Participants
Development
ParticipantsLOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target Group
Development83

Adverse events

Collected over Data were collected between enrollment and 6 months corrected age. Non-serious events are listed at a 0% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
LOWER Oxygen Saturation Target Group0/28 (0%)0/28 (0%)0/28 (0%)
HIGHER Oxygen Saturation Target Group0/22 (0%)0/22 (0%)0/22 (0%)

Baseline characteristics

Age, Continuous
Age, Continuous(post-menstrual age (PMA) in weeks)LOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target GroupTotal
Mean39.9 ± 2.340.5 ± 2.240.2 ± 2.3
Sex: Female, Male
Sex: Female, Male(Participants)LOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target GroupTotal
Female14923
Male141327
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)LOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target GroupTotal
Hispanic or Latino325
Not Hispanic or Latino252045
Unknown or Not Reported000
Race (NIH/OMB)
Race (NIH/OMB)(Participants)LOWER Oxygen Saturation Target GroupHIGHER Oxygen Saturation Target GroupTotal
American Indian or Alaska Native000
Asian112
Native Hawaiian or Other Pacific Islander000
Black or African American191534
White7512
More than one race112
Unknown or Not Reported000
07

Study locations

3 sites
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, United States
  • Pennsylvania Hospital
    Philadelphia, Pennsylvania 19107, United States
08

References and documents

Study documents

  • Protocol and statistical analysis plan · Mar 5, 2021
  • Informed consent form · Oct 6, 2021

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No

09

Registry details

Key details

Study ID
NCT03385330
Lead sponsor
Children's Hospital of Philadelphia
Collaborators
Thrasher Research Fund
Responsible party
Sara B. DeMauro, MD MSCE (Assistant Professor of Pediatrics, Children's Hospital of Philadelphia) — Principal investigator
First posted
Dec 28, 2017
Start date
Jun 1, 2018
Primary completion
Feb 1, 2023
Completion
Sep 1, 2023
Results posted
Apr 30, 2024
Last update
Apr 30, 2024

Study contacts

Sara DeMauro, MD
principal investigator · The Childrens Hospital of Pennsylvania

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
Yes
View the source record on ClinicalTrials.gov ↗

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