Trial Outcomes & Findings for Continuous Positive Airway Pressure to Improve Milder Obstructive Sleep Apnea (NCT NCT00089752)

NCT ID: NCT00089752

Last Updated: 2017-07-24

Results Overview

The primary endpoint is change after 8 weeks of treatment from baseline value (controlling for baseline value) in the 30-item Functional Outcomes of Sleep Questionnaire (FOSQ) that will be used to test the primary study hypothesis that patients with milder OSA (RDI 5-30) on active treatment will demonstrate greater mean change for the Total score from baseline to 8 weeks treatment. The FOSQ is designed to assess the impact of excessive sleepiness on functional status. The instrument has established content validity, test-retest reliability (r= 0.91), and internal consistency (alpha = 0.96). The scale ranges from 5 - 20 with normal functional status being a value greater than 17.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 281 participants
• Primary outcome timeframe: 8 weeks
• Results posted on: 2017-07-24

Participant Flow

Participants were recruited from the sleep disorders clinic from each of the 5 participating centers. Potential participants were approached by study staff at each site once their medical record indicated that they may be candidates for the study. Recruitment commenced 1-11-05 and ended 4-18-2008.

Participant milestones

Measure	Active CPAP Continuous positive pressure device that delivers therapeutic positive airway pressure to maintain airway patency worn continuously for each night of treatment.	Sham CPAP A device that looks and sounds like an active continuous positive pressure device (CPAP) that delivers ineffective pressure, i.e.,less than 1 cm H20 pressure compared to greater than 5 cm H20 for active treatment. Like active CPAP treatment it is worn every night.
Overall Study STARTED	141	140
Overall Study COMPLETED	121	118
Overall Study NOT COMPLETED	20	22

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Continuous Positive Airway Pressure to Improve Milder Obstructive Sleep Apnea

Baseline characteristics by cohort

Measure	Active CPAP n=121 Participants CPAP device	Sham CPAP n=118 Participants Sham CPAP device	Total n=239 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	116 Participants n=5 Participants	112 Participants n=7 Participants	228 Participants n=5 Participants
Age, Categorical >=65 years	5 Participants n=5 Participants	6 Participants n=7 Participants	11 Participants n=5 Participants
Sex: Female, Male Female	55 Participants n=5 Participants	44 Participants n=7 Participants	99 Participants n=5 Participants
Sex: Female, Male Male	66 Participants n=5 Participants	74 Participants n=7 Participants	140 Participants n=5 Participants
Region of Enrollment United States	68 participants n=5 Participants	68 participants n=7 Participants	136 participants n=5 Participants
Region of Enrollment Canada	53 participants n=5 Participants	50 participants n=7 Participants	103 participants n=5 Participants

PRIMARY outcome

Timeframe: 8 weeks

Population: Consented and randomized participants who completed the FOSQ with mild or moderate obstructive sleep apnea.

The primary endpoint is change after 8 weeks of treatment from baseline value (controlling for baseline value) in the 30-item Functional Outcomes of Sleep Questionnaire (FOSQ) that will be used to test the primary study hypothesis that patients with milder OSA (RDI 5-30) on active treatment will demonstrate greater mean change for the Total score from baseline to 8 weeks treatment. The FOSQ is designed to assess the impact of excessive sleepiness on functional status. The instrument has established content validity, test-retest reliability (r= 0.91), and internal consistency (alpha = 0.96). The scale ranges from 5 - 20 with normal functional status being a value greater than 17.

Outcome measures

Measure	Shame CPAP n=118 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=121 Participants Active Continuous Positive Airway Pressure device
Change in the Score of the Functional Outcomes of Sleep Questionnaire at Baseline and Week 8 Treatment	-0.14 scores on a scale Standard Deviation 2.61	0.98 scores on a scale Standard Deviation 2.89

SECONDARY outcome

Timeframe: Measured at Baseline and Week 8 of treatment in ITT sample

Population: Participants randomized and who had post-treatment data in ITT analysis

Change in the score from baseline to 8 weeks treatment, controlling for baseline in the self-rated 8 item measure of daytime sleepiness with a range from 0 - 24. Lower values indicting less daytime sleepiness

Outcome measures

Measure	Shame CPAP n=102 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=105 Participants Active Continuous Positive Airway Pressure device
Change in the Score From Baseline to 8 Weeks Treatment Epworth Sleepiness Scale	-0.54 scores on a scale Standard Deviation 3.49	-2.58 scores on a scale Standard Deviation 4.28

SECONDARY outcome

Timeframe: Measured at Baseline and Week 8 treatment in the ITT sample

Population: Population who had data post-randomization following 8 wks. intervention - ITT analysis

Change in mean arterial pressure (MAP) value from baseline to 8 weeks treatment, controlling for baseline, measured by 48 hours ambulatory blood pressure device - Space Laboratories

Outcome measures

Measure	Shame CPAP n=72 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=80 Participants Active Continuous Positive Airway Pressure device
Change in Mean Arterial Daytime Pressure at Baseline and Week 8 Treatment	92 mmHg Standard Deviation 7.9	90.3 mmHg Standard Deviation 7.6

SECONDARY outcome

Timeframe: Measured at Baseline and Week 8 treatment in the ITT sample

Population: Population who had data post-randomization following 8 wks. intervention - ITT analysis

Change in the score from baseline to 8 weeks treatment, controlling for baseline, in the POMS is a reliable and valid measure of mood states that consists of 65 adjectives on which subjects' rate themselves as they feel "today" using a five-point scale. There are six mood or affective states on this test derived through factor analysis: Tension-Anxiety (score range 0-36), Depression-Dejection (score range 0 - 60), Anger-Hostility (score range 0-48), Vigor-Activity (score range 0-32), Fatigue-Inertia (score range 0-28), and Confusion-Bewilderment (score range 0-28). There is also a summary Total Mood Disturbance (TMD) score that gives a Total estimate of affective state score range 0-200). Higher scores indicate greater disability.

Outcome measures

Measure	Shame CPAP n=103 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=107 Participants Active Continuous Positive Airway Pressure device
Change in the Score From Baseline to 8 Weeks Treatment Measured by the Profile of Mood States	-0.5 scores on a scale Standard Deviation 20.1	-10.2 scores on a scale Standard Deviation 25.5

SECONDARY outcome

Timeframe: Baseline and 8 weeks of treatment in the ITT sample

Population: The Intent-to-Treat Sample includes all randomized patients receiving at least a 20 minute interval of Active during the post randomization treatment period and who had no clinically significant major violations of inclusion or exclusion criteria.

Change in the score from baseline to 8 weeks treatment, controlling for baseline, in the PVT is an objective assessment of sleepiness and measures decrements in neurobehavioral performance due to sleepiness, i.e., ability to sustain attention and respond in a timely manner to salient signals.(7) The PVT yields five highly informative metrics on the capacity for sustained attention and vigilance performance: frequency of lapses, duration of lapse domain, optimum response time, vigilance decrement function, false response frequency. We applied this conceptually valid, relatively short duration, reliable task with known psychometric properties and minimal practice/learning curves to document attentional lapses (response times > 500 msec) in performance.

Outcome measures

Measure	Shame CPAP n=96 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=102 Participants Active Continuous Positive Airway Pressure device
Change in the Number of Lapses From Baseline to 8 Weeks Treatment on the Psychomotor Vigilance Task (PVT) - Total Lapses in 20 Minute Test	2.99 number on a scale Standard Deviation 21.54	-2.91 number on a scale Standard Deviation 26.12

SECONDARY outcome

Timeframe: Baseline and Week 8 of treatment in ITT sample.

Population: The Intent-to-Treat Sample includes all randomized patients exposed to CPAP or Sham treatment during the post randomization treatment period.

Change in the score from baseline to 8 weeks treatment, controlling for baseline, in the SF-36 is a 36-item questionnaire that assesses eight health concepts: physical functioning, bodily pain, role limitations due to physical problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate greater disability with a range of scores from 0-100.

Outcome measures

Measure	Shame CPAP n=85 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=84 Participants Active Continuous Positive Airway Pressure device
Change in the Score From Baseline to 8 Weeks Treatment on the SF36 - Physical	-0.19 scores on a scale Standard Deviation 6.99	3.92 scores on a scale Standard Deviation 7.46

SECONDARY outcome

Timeframe: Baseline and after 8 weeks of treatment in ITT sample

Population: The Intent-to-Treat Sample includes all randomized patients exposed to CPAP or Sham treatment during the post randomization treatment period.

Change in the score from baseline to 8 weeks treatment, controlling for baseline, in the SF-36 is a 36-item questionnaire that assesses eight health concepts: physical functioning, bodily pain, role limitations due to physical problems, role limitations due to personal or emotional problems, emotional well-being, social functioning, energy/fatigue, and general health perceptions. Higher scores indicate greater disability with a range of scores from 0-100.

Outcome measures

Measure	Shame CPAP n=85 Participants Ineffective (placebo) Continuous Positive Airway Pressure device	CPAP n=84 Participants Active Continuous Positive Airway Pressure device
Change in the Score From Baseline to 8 Weeks Treatment SF-36 Mental Component	2.00 scores on a scale Standard Deviation 8.10	3.38 scores on a scale Standard Deviation 9.83

Adverse Events

Active CPAP

Serious events: 6 serious events

Other events: 93 other events

Deaths: 0 deaths

Sham CPAP

Serious events: 11 serious events

Other events: 94 other events

Deaths: 0 deaths

Serious adverse events

Measure	Active CPAP n=121 participants at risk Continuous positive pressure device that delivers therapeutic (prescribed \> 5 CM H2O) positive airway pressure worn continuously for each night of treatment for 8 wks.	Sham CPAP n=118 participants at risk A device worn continuously every night that looks and sounds like an active continuous positive pressure device (CPAP) that delivers ineffective pressure, i.e.,less than 1 cm H20 pressure for 8 wks.
Gastrointestinal disorders Gastrointestinal	0.83% 1/121 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	2.5% 3/118 • Number of events 4 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Cardiac disorders Cardiac	0.83% 1/121 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	1.7% 2/118 • Number of events 2 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Musculoskeletal and connective tissue disorders Musculoskeletal	1.7% 2/121 • Number of events 2 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.00% 0/118 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Nervous system disorders Pain	0.83% 1/121 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.00% 0/118 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
General disorders Constitutional Symptoms	0.83% 1/121 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.00% 0/118 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Renal and urinary disorders Renal	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.85% 1/118 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Musculoskeletal and connective tissue disorders Dermatology/Skin	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.85% 1/118 • Number of events 2 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Respiratory, thoracic and mediastinal disorders Pulmonary	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.85% 1/118 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Blood and lymphatic system disorders Hemorrhage/Bleeding	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.85% 1/118 • Number of events 2 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Blood and lymphatic system disorders Blood/Bone Marrow	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	0.85% 1/118 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Blood and lymphatic system disorders Infection	0.00% 0/121 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	1.7% 2/118 • Number of events 2 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.

Other adverse events

Measure	Active CPAP n=121 participants at risk Continuous positive pressure device that delivers therapeutic (prescribed \> 5 CM H2O) positive airway pressure worn continuously for each night of treatment for 8 wks.	Sham CPAP n=118 participants at risk A device worn continuously every night that looks and sounds like an active continuous positive pressure device (CPAP) that delivers ineffective pressure, i.e.,less than 1 cm H20 pressure for 8 wks.
Immune system disorders Rhinitis Allergic nos/ Allergy Other-Possibly related to study	22.3% 27/121 • Number of events 28 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	29.7% 35/118 • Number of events 35 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Skin and subcutaneous tissue disorders Dermatology/Skin - Possibly related to study	16.5% 20/121 • Number of events 20 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	18.6% 22/118 • Number of events 22 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
General disorders Constitutional Symptoms	3.3% 4/121 • Number of events 25 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	7.6% 9/118 • Number of events 9 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Gastrointestinal disorders Gastrointestinal	12.4% 15/121 • Number of events 23 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	17.8% 21/118 • Number of events 36 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Infections and infestations Infection - Not related to study	11.6% 14/121 • Number of events 24 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	21.2% 25/118 • Number of events 47 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Musculoskeletal and connective tissue disorders Musculoskeletal/Soft Tissue- Not related to study	5.8% 7/121 • Number of events 8 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	8.5% 10/118 • Number of events 15 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Nervous system disorders Neurology - Not related to study	5.8% 7/121 • Number of events 9 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	8.5% 10/118 • Number of events 12 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
General disorders Pain- possibly related to study	38.8% 47/121 • Number of events 102 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	43.2% 51/118 • Number of events 116 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Respiratory, thoracic and mediastinal disorders Pulmonary/Upper Respiratory - Possibly related to study	9.1% 11/121 • Number of events 14 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	12.7% 15/118 • Number of events 22 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.
Infections and infestations Syndromes - Not related to study	0.83% 1/121 • Number of events 1 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.	6.8% 8/118 • Number of events 12 • 5 years From beginning of recruitment until study termination (5 years) . All non-serious adverse events reported and unspecified relative to whether it was associated with the intervention.

Additional Information

Terri E. Weaver, PhD, RN, FAAN

University of Pennsylvania School of Nursing

Phone: 215-898-2992

Email: tew@nursing.upenn.edu

Results disclosure agreements

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