Trial Outcomes & Findings for Comparing Auto-adjustable Positive Airway Pressure to Continuous Positive Airway Pressure in Children With Sleep Apnea (NCT NCT01846988)

NCT ID: NCT01846988

Last Updated: 2021-04-23

Results Overview

The aim of this study is to establish APAP as an effective treatment modality for OSAS in the pediatric population. We hypothesize that APAP can effectively approximate the pressure determined by PSG CPAP titration as measured by the average 90th pressure percentile and average CPAP pressure delivered to the patient. The in-lab CPAP titration PSG following initial diagnosis of OSA is the current standard of therapy. APAP would potentially bridge the gap between the time of diagnosis and the follow up PSG for CPAP titration, allowing for more prompt intervention.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

15 participants

Primary outcome timeframe

First visit after APAP run-in at 6-8 weeks

Results posted on

2021-04-23

Participant Flow

15 subjects started study procedures. 5 subjects withdrew after completing the APAP pressurized comparison phase and only 10 subjects were randomized to crossover treatment. All randomized subjects completed the study. Data analysis includes the 5 withdrawals completing APAP pressurized comparison plus the 10 subjects completing study procedures.

Participant milestones

Participant milestones
Measure
APAP Pressurized Comparison
An in-hospital titration study will average 90th pressure percentile and average CPAP pressure derived from the device will be compared to the CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 1
Randomized to APAP for 4 weeks followed by crossover to CPAP for 4 weeks after APAP settings for 4-8 weeks.
Group 2
Randomized to CPAP for 4 weeks followed by crossover to APAP for 4 weeks after APAP settings for 4-8 weeks.
APAP Pressurize Comparison (4-8 Weeks)
STARTED
15
0
0
APAP Pressurize Comparison (4-8 Weeks)
COMPLETED
15
0
0
APAP Pressurize Comparison (4-8 Weeks)
NOT COMPLETED
0
0
0
Intervention #1 - 4 Weeks
STARTED
0
5
5
Intervention #1 - 4 Weeks
COMPLETED
0
5
5
Intervention #1 - 4 Weeks
NOT COMPLETED
0
0
0
Intervention #2 - 4 Weeks
STARTED
0
5
5
Intervention #2 - 4 Weeks
COMPLETED
0
5
5
Intervention #2 - 4 Weeks
NOT COMPLETED
0
0
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
APAP Pressurized Comparison
n=15 Participants
Comparison usage to determine APAP average 90% pressure (APAP P90) for initial treatment period and to assess adherence for randomization periods for 6-8 weeks or 4-6 weeks. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 1 Crossover Treatment
Randomized to APAP for 4 weeks followed by crossover to CPAP for 4 weeks after APAP settings for 4-8 weeks.
Group 2 Crossover Treatment
Randomized to CPAP for 4 weeks followed by crossover to APAP for 4 weeks after APAP settings for 4-8 weeks.
Total
n=15 Participants
Total of all reporting groups
Age, Categorical
<=18 years
15 Participants
n=15 Participants
0 Participants
0 Participants
15 Participants
n=15 Participants
Age, Categorical
Between 18 and 65 years
0 Participants
n=15 Participants
0 Participants
0 Participants
0 Participants
n=15 Participants
Age, Categorical
>=65 years
0 Participants
n=15 Participants
0 Participants
0 Participants
0 Participants
n=15 Participants
Age, Continuous
14.3 years
STANDARD_DEVIATION 3.1 • n=15 Participants
14.3 years
STANDARD_DEVIATION 3.1 • n=15 Participants
Sex: Female, Male
Female
7 Participants
n=15 Participants
7 Participants
n=15 Participants
Sex: Female, Male
Male
8 Participants
n=15 Participants
8 Participants
n=15 Participants
Race and Ethnicity Not Collected
0 Participants
Race and Ethnicity were not collected from any participant.
Region of Enrollment
United States
15 participants
n=15 Participants
15 participants
n=15 Participants

PRIMARY outcome

Timeframe: First visit after APAP run-in at 6-8 weeks

Population: The APAP pressure comparison group included 15 participants. After this initial period, 5 participants withdrew from the study. The remaining 10 participants were randomized to Group 1 or 2.

The aim of this study is to establish APAP as an effective treatment modality for OSAS in the pediatric population. We hypothesize that APAP can effectively approximate the pressure determined by PSG CPAP titration as measured by the average 90th pressure percentile and average CPAP pressure delivered to the patient. The in-lab CPAP titration PSG following initial diagnosis of OSA is the current standard of therapy. APAP would potentially bridge the gap between the time of diagnosis and the follow up PSG for CPAP titration, allowing for more prompt intervention.

Outcome measures

Outcome measures
Measure
APAP Pressure Comparison
n=15 Participants
APAP pressure comparison period was 6-8 weeks. APAP average 90th pressure percentile derived from machine download was compared to CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 2
Combined CPAP data regardless of order of randomization
Group 2
Michigan pediatric sleep questionnaire score administered after CPAP randomization period regardless of the order of randomization.
Difference Between Optimal Pressure (cm H2O) Derived From APAP run-in Period Versus Result of CPAP Titration Study
APAP 90 percent pressure
7.3 cmH2O
Standard Deviation 2.6
Difference Between Optimal Pressure (cm H2O) Derived From APAP run-in Period Versus Result of CPAP Titration Study
CPAP titration PSG pressure
9.4 cmH2O
Standard Deviation 2.6

PRIMARY outcome

Timeframe: end of study visit at 12-16 weeks

Population: For data analysis we combined APAP data together and CPAP data together regardless of the order of randomization, yielding 10 subjects for APAP adherence and 10 subjects for CPAP adherence. In the data analysis below the column labeled Group 1 contains all APAP data and Group 2 contains all CPAP data.

Randomized crossover period comparison of adherence between APAP and CPAP calculated from therapy usage hours.

Outcome measures

Outcome measures
Measure
APAP Pressure Comparison
n=10 Participants
APAP pressure comparison period was 6-8 weeks. APAP average 90th pressure percentile derived from machine download was compared to CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 2
n=10 Participants
Combined CPAP data regardless of order of randomization
Group 2
Michigan pediatric sleep questionnaire score administered after CPAP randomization period regardless of the order of randomization.
Adherence Comparison of APAP vs.CPAP (% Days Device Used and % Days Device Used >4 Hours)
% days device used
62.5 percentage of days
Standard Deviation 27.2
61.4 percentage of days
Standard Deviation 25.6
Adherence Comparison of APAP vs.CPAP (% Days Device Used and % Days Device Used >4 Hours)
% days device used >4h
42.7 percentage of days
Standard Deviation 29.0
44.3 percentage of days
Standard Deviation 29.4

SECONDARY outcome

Timeframe: 12-16 weeks

Comparison of Michigan pediatric sleep questionnaire score between baseline (at time of enrollment in the study) and each group (APAP, CPAP) after randomization period for the 10 subjects who completed the study. The scale range is from 0 to 1, Normal \<0.33, at risk is 0.33-0.55 and \>0.55 abnormal.

Outcome measures

Outcome measures
Measure
APAP Pressure Comparison
n=10 Participants
APAP pressure comparison period was 6-8 weeks. APAP average 90th pressure percentile derived from machine download was compared to CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 2
n=10 Participants
Combined CPAP data regardless of order of randomization
Group 2
n=10 Participants
Michigan pediatric sleep questionnaire score administered after CPAP randomization period regardless of the order of randomization.
Michigan Pediatric Sleep Questionnaire
0.65 score on a scale
Standard Deviation 0.2
0.44 score on a scale
Standard Deviation 0.2
0.39 score on a scale
Standard Deviation 0.2

SECONDARY outcome

Timeframe: 12-16 weeks

Comparison of Epworth Sleepiness scale questionnaire score between baseline (at time of enrollment in the study) and each group (APAP, CPAP) after randomization period for the 10 subjects who completed the study. The scale range is from 0 to 24, with a value of 10 or greater suggesting significance for sleep disordered breathing.

Outcome measures

Outcome measures
Measure
APAP Pressure Comparison
n=10 Participants
APAP pressure comparison period was 6-8 weeks. APAP average 90th pressure percentile derived from machine download was compared to CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 2
n=10 Participants
Combined CPAP data regardless of order of randomization
Group 2
n=10 Participants
Michigan pediatric sleep questionnaire score administered after CPAP randomization period regardless of the order of randomization.
Epworth Sleepiness Scale Questionnaire
10.3 score on a scale
Standard Deviation 6.2
7.6 score on a scale
Standard Deviation 5.0
7.2 score on a scale
Standard Deviation 4.3

SECONDARY outcome

Timeframe: 12-16 weeks

Comparison of Pediatric Quality of Life Inventory questionnaire score between baseline (at time of enrollment in the study) and each group (APAP, CPAP) after randomization period for the 10 subjects who completed the study. The scale scores range between 0-100. Higher scores indicate better health related quality of life. Healthy: 81.34-100, at risk: 81.33-65.4, unhealthy \<65.4

Outcome measures

Outcome measures
Measure
APAP Pressure Comparison
n=10 Participants
APAP pressure comparison period was 6-8 weeks. APAP average 90th pressure percentile derived from machine download was compared to CPAP pressure determined by CPAP titration PSG. Subjects will then be randomized to Group 1 first, or Group 2 first, then crossover to the other group.
Group 2
n=10 Participants
Combined CPAP data regardless of order of randomization
Group 2
n=10 Participants
Michigan pediatric sleep questionnaire score administered after CPAP randomization period regardless of the order of randomization.
Comparison of Pediatric Quality of Life Inventory Questionnaire Score Between Baseline and Each Randomization Period
61.7 score on a scale
Standard Deviation 17.9
65.9 score on a scale
Standard Deviation 20.2
68.5 score on a scale
Standard Deviation 18.3

Adverse Events

APAP Pressurized Comparison

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Group 1 Crossover Treatment

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Group 2 Crossover Treatment

Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Dr. Neepa Gurbani, Assistant Professor

Cincinnati Children's Hospital Medical Center

Phone: (513) 736-1814

Results disclosure agreements

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