Trial Outcomes & Findings for Medications for Obstructive Sleep Apnea In Children With Down Syndrome (NCT NCT04115878)
NCT ID: NCT04115878
Last Updated: 2025-08-19
Results Overview
change in number of obstructive apneas and hypopneas per hour on polysomnography from baseline
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
four weeks
Results posted on
2025-08-19
Participant Flow
Enrollment was only 15 randomized participants rather than planned 24 due to recruitment delays related to the COVID-19 pandemic. 22 participants were enrolled but 7 did not qualify for randomization. 4 did not meet the requirement for apnea-hypopnea index \>5 on their baseline polysomnogram, 1 had hypoxemia unrelated to OSA on their baseline polysomnogram, 1 could not tolerate polysomnogram and 1 participant became adherent to CPAP between consent and baseline polysomnogram.
Participant milestones
| Measure |
High Dose Ato-oxy Then Low Dose Ato-oxy
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy Then High Dose Ato-oxy
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
First Intervention
STARTED
|
8
|
7
|
|
First Intervention
COMPLETED
|
7
|
6
|
|
First Intervention
NOT COMPLETED
|
1
|
1
|
|
Second Intervention
STARTED
|
7
|
6
|
|
Second Intervention
COMPLETED
|
7
|
5
|
|
Second Intervention
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Medications for Obstructive Sleep Apnea In Children With Down Syndrome
Baseline characteristics by cohort
| Measure |
All Study Participants
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 4.1 • n=93 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
5 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
6 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
11 participants
n=93 Participants
|
|
BMI percentile
|
79 percentile
STANDARD_DEVIATION 23 • n=93 Participants
|
|
Congenital heart disease
|
8 Participants
n=93 Participants
|
PRIMARY outcome
Timeframe: four weekschange in number of obstructive apneas and hypopneas per hour on polysomnography from baseline
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Obstructive Apnea-hypopnea Index (oAHI)
|
-3.79 events per hour
Standard Deviation 2.74
|
-3.51 events per hour
Standard Deviation 3.06
|
SECONDARY outcome
Timeframe: four weeksChange in OSA-18 (a measure of health-related quality of life) from baseline. The OSA-18 score ranges from 18 to 136, with lower scores indicating better health-related quality of life.
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Obstructive Sleep Apnea-18 Score (OSA-18)
|
-6.00 score on a scale
Standard Deviation 21.2
|
-6.82 score on a scale
Standard Deviation 11.91
|
SECONDARY outcome
Timeframe: four weekschange in number of arousals per hour on polysomnography from baseline
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Arousal Index
|
0.68 arousals per hour
Standard Deviation 8.24
|
2.01 arousals per hour
Standard Deviation 5.15
|
OTHER_PRE_SPECIFIED outcome
Timeframe: four weeksChange in PedsQL total score (a measure of health-related quality of life) from baseline. The PedsQL score ranges from 0-100, with higher scores indicating better health-related quality of life.
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Pediatric Quality of Life Inventory (PedsQL) Total Score
|
1.97 score on a scale
Standard Deviation 15.77
|
3.36 score on a scale
Standard Deviation 15.27
|
OTHER_PRE_SPECIFIED outcome
Timeframe: four weeksPopulation: Form not completed by one participant for high dose.
Change in Caregiver Global Impression of Change from baseline. This will be assessed on a seven point scale answering the question: Since the start of this therapy, my child's overall status is: 1. Very much improved; 2. Much improved; 3. Minimally Improved; 4. No change; 5. Minimally worse; 6. Much worse or 7. Very much worse.
Outcome measures
| Measure |
High Dose Ato-oxy
n=10 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Caregiver Global Impression of Change
|
3.70 score on a scale
Standard Deviation 0.95
|
3.45 score on a scale
Standard Deviation 1.03
|
OTHER_PRE_SPECIFIED outcome
Timeframe: four weeksChange in N1 sleep percentage on polysomnography from baseline
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
N1 Sleep Percentage
|
0.45 percentage of N1 sleep
Standard Deviation 3.49
|
0.40 percentage of N1 sleep
Standard Deviation 3.17
|
OTHER_PRE_SPECIFIED outcome
Timeframe: four weeksChange in REM sleep percentage on polysomnography from baseline
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
REM Sleep Percentage
|
-2.16 percentage of REM sleep
Standard Deviation 10.16
|
-1.41 percentage of REM sleep
Standard Deviation 8.89
|
OTHER_PRE_SPECIFIED outcome
Timeframe: four weeksChange in N3 sleep percentage on polysomnography from baseline
Outcome measures
| Measure |
High Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=11 Participants
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
N3 Sleep Percentage
|
-0.73 percentage of N3 sleep
Standard Deviation 14.58
|
-4.29 percentage of N3 sleep
Standard Deviation 13.14
|
Adverse Events
High Dose Ato-oxy
Serious events: 1 serious events
Other events: 8 other events
Deaths: 1 deaths
Low Dose Ato-oxy
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
High Dose Ato-oxy
n=14 participants at risk
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=14 participants at risk
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure and death
|
7.1%
1/14 • Number of events 1 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
0.00%
0/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
Other adverse events
| Measure |
High Dose Ato-oxy
n=14 participants at risk
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
Low Dose Ato-oxy
n=14 participants at risk
Atomoxetine and oxybutynin (ato-oxy): Low dose ato-oxy will include 0.5 mg/kg/day of atomoxetine (max 40 mg) in combination with 5 mg oxybutynin High dose ato-oxy will include 1.2 mg/kg/day atomoxetine (max 80 mg) and 5 mg oxybutynin
|
|---|---|---|
|
Psychiatric disorders
Fatigue
|
28.6%
4/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
14.3%
2/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Psychiatric disorders
Mood changes
|
28.6%
4/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
21.4%
3/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Gastrointestinal disorders
Diarrhea
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Nervous system disorders
Headaches
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
0.00%
0/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Renal and urinary disorders
Decreased urinary frequency
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
0.00%
0/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
|
Gastrointestinal disorders
Dry mouth
|
0.00%
0/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
7.1%
1/14 • Adverse event data was collected throughout time on therapy (1 month each for high and low dose ato-oxy)
Adverse events were assessed via weekly phone calls while on medications
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place