Trial Outcomes & Findings for Steroids for Pediatric Apnea Research in Kids (NCT NCT02180672)
NCT ID: NCT02180672
Last Updated: 2021-11-10
Results Overview
Efficacy measure to assess acute response to nasal steroids. Obstructive apnea hypopnea index (events per hour). Per inclusion/exclusion criteria the expected range for this study at baseline is between 2 and 30 events per hour. Please note that lower and upper ranges can be broader at the 3- and 12-month points. The obstructive apnea hypopnea index (OAHI) is the sum of obstructive apneas and hypopneas, and mixed apneas divided by the total hours of sleep. Hence, the unit used is events per hour. It ranges from 0 events per hour(meaning no obstructive apneas at all) until 400 events per hour approximately. Higher values indicate more severe obstructive sleep apnea or worse outcome.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
211 participants
Primary outcome timeframe
3 months
Results posted on
2021-11-10
Participant Flow
Screening: polysomnography, ophthalmologic exam, DXA scan. 75 children did not meet PSG inclusion criteria, 1 child had a coloboma, and 1 child had abnormal DXA scan.
Participant milestones
| Measure |
Nasal Steroids
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Overall Study
STARTED
|
91
|
43
|
|
Overall Study
COMPLETED
|
86
|
36
|
|
Overall Study
NOT COMPLETED
|
5
|
7
|
Reasons for withdrawal
| Measure |
Nasal Steroids
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Overall Study
Lost to Follow-up
|
5
|
7
|
Baseline Characteristics
The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
Baseline characteristics by cohort
| Measure |
Nasal Steroids
n=91 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=43 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
Total
n=134 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
8.24 years
STANDARD_DEVIATION 2.17 • n=91 Participants
|
8.10 years
STANDARD_DEVIATION 2.03 • n=43 Participants
|
8.17 years
STANDARD_DEVIATION 2.10 • n=134 Participants
|
|
Sex: Female, Male
Female
|
45 Participants
n=91 Participants
|
20 Participants
n=43 Participants
|
65 Participants
n=134 Participants
|
|
Sex: Female, Male
Male
|
46 Participants
n=91 Participants
|
23 Participants
n=43 Participants
|
69 Participants
n=134 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=91 Participants
|
0 Participants
n=43 Participants
|
0 Participants
n=134 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=91 Participants
|
0 Participants
n=43 Participants
|
0 Participants
n=134 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=91 Participants
|
0 Participants
n=43 Participants
|
0 Participants
n=134 Participants
|
|
Race (NIH/OMB)
Black or African American
|
65 Participants
n=91 Participants
|
25 Participants
n=43 Participants
|
90 Participants
n=134 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=91 Participants
|
5 Participants
n=43 Participants
|
15 Participants
n=134 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=91 Participants
|
0 Participants
n=43 Participants
|
0 Participants
n=134 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
16 Participants
n=91 Participants
|
13 Participants
n=43 Participants
|
29 Participants
n=134 Participants
|
|
Region of Enrollment
United States
|
91 participants
n=91 Participants
|
43 participants
n=43 Participants
|
134 participants
n=134 Participants
|
|
OAHI
|
7.85 events per hour
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
6.51 events per hour
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
7.52 events per hour
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Nasal Obstrction Symptoms Evaluation (NOSE)
|
5.59 units on a scale
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
4.47 units on a scale
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
5.03 units on a scale
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
The Epworth Sleepiness Scale
|
9.09 units on a scale
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
7.58 units on a scale
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
8.34 units on a scale
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Pediatric Quality of Life (PedsQL)
|
73.91 units on a scale
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
78.44 units on a scale
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
76.175 units on a scale
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Behavior Rating Inventory of Executive Function (BRIEF)
|
50.86 T-Score
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
47.11 T-Score
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
48.985 T-Score
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Conners Abbreviated Symptom Questionnaire
|
8.19 T-Score
n=86 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
5.97 T-Score
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
7.08 T-Score
n=122 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Conners Continuous Performance Test (CPT)
|
54.8 T-Score
n=82 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
56.1 T-Score
n=36 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
55.45 T-Score
n=118 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
|
Purdue Peg Board
|
-1.06 z-Score
n=85 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
-1.39 z-Score
n=35 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
-1.225 z-Score
n=120 Participants • The total number of analyzed is different than the total number of participants is due to participants withdrawing themselves from the study or being lost to follow up.
|
PRIMARY outcome
Timeframe: 3 monthsEfficacy measure to assess acute response to nasal steroids. Obstructive apnea hypopnea index (events per hour). Per inclusion/exclusion criteria the expected range for this study at baseline is between 2 and 30 events per hour. Please note that lower and upper ranges can be broader at the 3- and 12-month points. The obstructive apnea hypopnea index (OAHI) is the sum of obstructive apneas and hypopneas, and mixed apneas divided by the total hours of sleep. Hence, the unit used is events per hour. It ranges from 0 events per hour(meaning no obstructive apneas at all) until 400 events per hour approximately. Higher values indicate more severe obstructive sleep apnea or worse outcome.
Outcome measures
| Measure |
Nasal Steroids
n=86 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=36 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Obstructive Apnea Hypopnea Index
|
7.69 events per hour
Interval 5.39 to 9.99
|
6.33 events per hour
Interval 4.09 to 8.58
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: The obstructive apnea hypopnea index (OAHI) is the sum of obstructive apneas and hypopneas, and mixed apneas divided by the total hours of sleep. Hence, the unit used is events per hour. It ranges from 0 events per hour(meaning no obstructive apneas at all) until 400 events per hour approximately. Higher values indicate more severe obstructive sleep apnea or worse outcome.
Efficacy measure to assess duration of response to nasal steroids. Obstructive apnea hypopnea index (events per hour). Per inclusion/exclusion criteria the expected range for this study at baseline is between 2 and 30 events per hour. Please note that lower and upper ranges can be broader at the 3- and 12-month points.
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
OAHI
|
6.11 events per hour
Interval 3.97 to 8.24
|
6.27 events per hour
Interval 3.98 to 8.55
|
SECONDARY outcome
Timeframe: 12 monthsNasal Obstruction Symptom Evaluation (NOSE) scale, a validated scale of nasal obstructive symptoms. The scales ranges from 0-100 with higher values indicating worse nasal obstruction
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Nasal Obstruction Symptom Evaluation (NOSE)
|
3.66 score on a scale
Interval 2.54 to 4.77
|
5.31 score on a scale
Interval 3.43 to 7.2
|
SECONDARY outcome
Timeframe: 12 monthsEpworth Sleepiness scale is a measure of sleepiness that ranges from 0-24, with higher values indicating sleepiness
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
The Epworth Sleepiness Scale
|
7.68 score on a scale
Interval 6.24 to 9.12
|
8.56 score on a scale
Interval 6.68 to 10.45
|
SECONDARY outcome
Timeframe: 12 monthPediatric Quality of Life Inventory (PedsQL), a well-validated, generic measure of global quality of life, in which scores range from 0 to 100, with higher scores indicating better quality of life
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Pediatric Quality of Life Inventory (PedsQL)
|
78.69 score on a scale
Interval 72.93 to 84.45
|
73.67 score on a scale
Interval 66.66 to 80.68
|
SECONDARY outcome
Timeframe: 12 monthsBehavior Rating Inventory of Executive Function \[BRIEF\] Global Executive Composite T score, comprising summary measures of behavioral regulation and metacognition \[with mean scores of 50 and standard deviation of 10, with higher scores indicating worse functioning\]
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Behavior Rating Inventory of Executive Function (BRIEF)
|
47.42 T-Score
Interval 43.93 to 50.91
|
49.5 T-Score
Interval 44.01 to 54.99
|
SECONDARY outcome
Timeframe: 12 monthsA parent-rated measure of symptoms of attention problems, yielding T scores with a mean of 50 and a standard deviation of 10 (higher scores indicate worse functioning).
Outcome measures
| Measure |
Nasal Steroids
n=36 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Conners Abbreviated Symptom Questionnaire
|
5.62 T-Score
Interval 3.73 to 7.52
|
6.50 T-Score
Interval 3.84 to 9.16
|
SECONDARY outcome
Timeframe: 12 monthsThe Purdue Peg Board is a widely used test of fine motor coordination, yields z-scores with a mean of 0 and a standard deviation of 1, with higher scores indicating better performance.
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=31 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Purdue Peg Board
|
-1.12 z-Score
Interval -1.7 to -0.54
|
-1.31 z-Score
Interval -1.79 to -0.84
|
SECONDARY outcome
Timeframe: 12 monthsThe CPT is a performance measure of sustained attention that yields T scores with a mean of 50 and a standard deviation of 10 (higher scores indicate worse functioning)
Outcome measures
| Measure |
Nasal Steroids
n=36 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=29 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Conners Continuous Performance Test (CPT)
|
52.67 T-Score
Interval 49.07 to 56.27
|
52.92 T-Score
Interval 48.74 to 57.11
|
SECONDARY outcome
Timeframe: 12-MonthsThe Child Behavior Checklist (CBCL), is a widely used and validated caregiver-completed survey of behavior competencies that yields standardized, age-adjusted scores on internalizing, externalizing and attentional behavior difficulties81, 82. All scores are T scores with a mean of 50 and standard deviation of 10 (higher scores indicate worse functioning).
Outcome measures
| Measure |
Nasal Steroids
n=38 Participants
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=32 Participants
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Child Behavior Checklist
Internalizing
|
49.92 T-Score
Interval 47.01 to 52.83
|
51.94 T-Score
Interval 47.46 to 56.42
|
|
Child Behavior Checklist
Externalizing
|
51.89 T-Score
Interval 48.45 to 55.34
|
52 T-Score
Interval 47.34 to 56.66
|
|
Child Behavior Checklist
Total Behavior Problems
|
51.45 T-Score
Interval 48.47 to 54.43
|
51.19 T-Score
Interval 45.99 to 56.39
|
|
Child Behavior Checklist
Attention
|
55.13 T-Score
Interval 53.47 to 56.79
|
57.38 T-Score
Interval 54.15 to 60.6
|
Adverse Events
Nasal Steroids
Serious events: 1 serious events
Other events: 66 other events
Deaths: 66 deaths
Placebo
Serious events: 0 serious events
Other events: 16 other events
Deaths: 16 deaths
Serious adverse events
| Measure |
Nasal Steroids
n=86 participants at risk
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=36 participants at risk
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Endocrine disorders
Ketotic Hypoglycemia
|
1.2%
1/86 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
0.00%
0/36 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
Other adverse events
| Measure |
Nasal Steroids
n=86 participants at risk
Participants randomly assigned to this study arm will receive a 3-month course of nasal steroids (nasal fluticasone).
Nasal Fluticasone: One spray per nostril, per day.
|
Placebo
n=36 participants at risk
Participants randomly assigned to this study arm will receive a 3-month course of placebo nasal spray (saline).
Placebo: One spray per nostril, per day.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Lower Respiratory Tract (Including Asthma)
|
7.0%
6/86 • Number of events 6 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
25.0%
9/36 • Number of events 9 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Tract (including cough)
|
43.0%
37/86 • Number of events 37 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
19.4%
7/36 • Number of events 7 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
|
Gastrointestinal disorders
Gastrointestinal Tract
|
11.6%
10/86 • Number of events 10 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
5.6%
2/36 • Number of events 2 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
|
Infections and infestations
Other Infections
|
9.3%
8/86 • Number of events 8 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
8.3%
3/36 • Number of events 3 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
|
Respiratory, thoracic and mediastinal disorders
Sleep Apnea Exacerbation
|
4.7%
4/86 • Number of events 4 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
0.00%
0/36 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
|
General disorders
Other Illness
|
36.0%
31/86 • Number of events 31 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
13.9%
5/36 • Number of events 5 • Adverse events were collected monthly at telephone calls at months 2, 4, 5, 7, 8, 10 and 11, and during in-person visits at months 1, 3, 6, 9 and 12.
|
Additional Information
Ignacio Tapia, MD, Principal Investigator, Associate Professor
The Children's Hospital of Philadelphia
Phone: (267) 426-5842
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place