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Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome (TRIO)

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ClinicalTrials.gov Identifier: NCT01808508
Recruitment Status : Completed
First Posted : March 11, 2013
Results First Posted : February 8, 2016
Last Update Posted : February 8, 2016
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Philadelphia

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Down Syndrome
Obstructive Sleep Apnea Syndrome
Interventions Device: Continuous positive airway pressure
Device: Sham or placebo continuous positive airway pressure
Enrollment 27
Recruitment Details Children were recruited from The Children's Hospital of Philadelphia Trisomy 21 Program and community support groups.
Pre-assignment Details 27 subjects were consented, 4 subjects were ineligible before randomization. Subjects with an obstructive apnea hypopnea index (AHI) of at least 5/hr were randomized to CPAP or sham CPAP (Group 1 or 2) for 4 months in a double-blind fashion. Subjects with normal baseline polysomnograms (AHI <1.5/hr) were followed without CPAP (Group 3).
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
Period Title: Overall Study
Started 11 9 3
Completed 10 9 3
Not Completed 1 0 0
Reason Not Completed
Lost to Follow-up             1             0             0
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3 - No Intervention Total
Hide Arm/Group Description

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group Total of all reporting groups
Overall Number of Baseline Participants 11 9 3 23
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Median (Inter-Quartile Range)
Unit of measure:  Years
Number Analyzed 11 participants 9 participants 3 participants 23 participants
10.0
(9.0 to 11.5)
12.0
(9.0 to 15.0)
12
(10.0 to 13.5)
10
(9.0 to 14.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants 9 participants 3 participants 23 participants
Female
2
  18.2%
5
  55.6%
2
  66.7%
9
  39.1%
Male
9
  81.8%
4
  44.4%
1
  33.3%
14
  60.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 11 participants 9 participants 3 participants 23 participants
11 9 3 23
1.Primary Outcome
Title Change in Epworth Sleepiness Scale From Baseline to End of Study
Hide Description The primary aim of the study is to assess the relationship between obstructive sleep apnea syndrome (OSAS) and the neurocognitive and behavioral outcomes of individuals with Down syndrome. Sleepiness was assessed using the pediatric version of the Epworth Sleepiness Scale (ESS). The ESS is a self-administered questionnaire with 8 questions. It provides a measure of a person’s general level of daytime sleepiness, or average sleep propensity in daily life. The ESS asks people to rate, on a 4-point scale (0, low to 3, high) their usual chances of dozing off or falling asleep in 8 different situations or activities that most people engage in as part of their daily lives. The total ESS score provides an estimate of a general characteristic of each person's average level of sleepiness in daily life (0= no chance of dozing/no daytime sleepiness to 24=high chance of dozing/lots of daytime sleepiness).
Time Frame 4 Months
Hide Outcome Measure Data
Hide Analysis Population Description
1 OSAS subject randomized to CPAP did not return for follow-up
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description:

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
Overall Number of Participants Analyzed 10 9 3
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
-1
(-4 to 0)
-0.5
(-3 to 0.75)
1
(0.5 to 3.5)
2.Secondary Outcome
Title Change in Child Behavior Checklist (CBCL) Total Score From Baseline to 4 Months
Hide Description The change in behavioral domain was measured by the Child Check Behavior List. The CBCL is a widely used method of identifying problem behavior in children. Problems are identified by a respondent who knows the child well, usually a parent or other care giver. There are 2 versions based on the child's age (CBCL/1½-5 for use children 18 months-5 years; the CBCL/6-18 for children aged 6-18 years). The checklists consists of a number of statements about the child's behavior and responses are recorded on a scale: 0 = Not True, 1 = Somewhat or Sometimes True, 2 = Very True or Often True. The preschool checklist contains 100 questions and, school-age checklist contains 120 questions. 8 sub-scores (1 for each of 8 syndromes: anxious/depressed, withdrawn depressed, somatic complaints, social problems, thought problems, attention problems, rule-breaking behavior and aggressive behavior) are calculated, each ranging from 0 (normal) to 16 (clinical behavior).
Time Frame 4 Months
Hide Outcome Measure Data
Hide Analysis Population Description
1 OSAS subject randomized to CPAP did not return for follow-up
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description:

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
Overall Number of Participants Analyzed 10 9 3
Median (Inter-Quartile Range)
Unit of Measure: units on a scale
-2
(-4.5 to 0)
2
(0.75 to 5.25)
2
(1 to 4.5)
3.Secondary Outcome
Title Change in the Left Ventricular (LV) Mass Index Score From Baseline to 4 Months
Hide Description The change in left ventricular mass index score, measured on echocardiography, was used to assess the relationship between obstructive sleep apnea syndrome and cardiovascular function of individuals with Down syndrome. Left ventricular (LV) mass was calculated from M-mode measurements of the LV end-diastolic dimension, the thickness of the interventricular septum and the thickness of the LV posterior wall, and presented as a z-score.
Time Frame 4 Months
Hide Outcome Measure Data
Hide Analysis Population Description
1 OSAS subject randomized to CPAP did not return for follow-up
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description:

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
Overall Number of Participants Analyzed 10 9 3
Median (Inter-Quartile Range)
Unit of Measure: z-score
-0.06
(-0.58 to 1.57)
-1.18
(-1.97 to 0.00)
0.36
(0.12 to 2.71)
4.Secondary Outcome
Title Change in the Distance Walked on a 6 Minute Walk Test From Baseline to 4 Months
Hide Description As secondary outcome of the second aim, we will use the distance walked during the 6 minute walk test.
Time Frame 4 Months
Hide Outcome Measure Data
Hide Analysis Population Description
The majority of subjects were unable to follow instructions properly and therefore none could successfully complete the test. Thus the results were not considered to be valid.
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description:

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
Overall Number of Participants Analyzed 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame 4 Months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Hide Arm/Group Description

Group 1 will receive therapeutic CPAP for 4 months.

Continuous positive airway pressure: Continuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open.

Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.

Sham or placebo continuous positive airway pressure: Sham or placebo CPAP is a machine used instead of therapeutic CPAP.

This machine is similar to a therapeutic CPAP machine but has built in leaks and does not deliver pressure. It is not effective in treating OSAS.

Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.
All-Cause Mortality
Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/11 (0.00%)      0/9 (0.00%)      0/3 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Group 1- Continuous Positive Airway Pressure (CPAP) Group 2-Sham Continuous Positive Airway Pressure (CPAP) Group 3- No Intervention
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   1/11 (9.09%)      2/9 (22.22%)      1/3 (33.33%)    
Gastrointestinal disorders       
Abdominal Pain   0/11 (0.00%)  0 1/9 (11.11%)  1 0/3 (0.00%)  0
Infections and infestations       
Lyme Disease   0/11 (0.00%)  0 1/9 (11.11%)  1 0/3 (0.00%)  0
Skin and subcutaneous tissue disorders       
Pressure Sore  [1]  1/11 (9.09%)  1 0/9 (0.00%)  0 1/3 (33.33%)  1
Indicates events were collected by systematic assessment
[1]
On forehead from probe used during sleep study
This preliminary study was limited by the small sample size, and the few children with Down Syndrome but normal breathing during sleep.
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Name/Title: Carole L. Marcus, MBBCh
Organization: Children's Hospital of Philadelphia
Phone: 267-426-5842
Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT01808508     History of Changes
Other Study ID Numbers: 11-007964
First Submitted: March 7, 2013
First Posted: March 11, 2013
Results First Submitted: December 4, 2015
Results First Posted: February 8, 2016
Last Update Posted: February 8, 2016