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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT01808508
First received: March 7, 2013
Last updated: January 8, 2016
Last verified: December 2015
  Purpose

Many individuals with Down syndrome (DS) have breathing problems during sleep. This is called obstructive sleep apnea syndrome (OSAS). OSAS is very common in individuals with Down syndrome because of the shape of their face and tongue and because of their low muscle tone. OSAS can cause a lot of health problems including behavioral and learning problems as well as heart problems.

The purpose of this research study is to look at the effects of treating OSAS in individuals with Down syndrome with a machine called Continuous Positive Airway Pressure (CPAP). The investigators want to see if treatment of OSAS improves learning, behavior and heart problems.


Condition Intervention
Down Syndrome
Obstructive Sleep Apnea Syndrome
Device: Continuous positive airway pressure
Device: Sham or placebo continuous positive airway pressure

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome

Resource links provided by NLM:


Further study details as provided by Children's Hospital of Philadelphia:

Primary Outcome Measures:
  • Change in Epworth Sleepiness Scale From Baseline to End of Study [ Time Frame: 4 Months ] [ Designated as safety issue: No ]
    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).


Secondary Outcome Measures:
  • Change in Child Behavior Checklist (CBCL) Total Score From Baseline to 4 Months [ Time Frame: 4 Months ] [ Designated as safety issue: No ]
    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).

  • Change in the Left Ventricular (LV) Mass Index Score From Baseline to 4 Months [ Time Frame: 4 Months ] [ Designated as safety issue: No ]
    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.

  • Change in the Distance Walked on a 6 Minute Walk Test From Baseline to 4 Months [ Time Frame: 4 Months ] [ Designated as safety issue: No ]
    As secondary outcome of the second aim, we will use the distance walked during the 6 minute walk test.


Enrollment: 27
Study Start Date: September 2010
Study Completion Date: September 2014
Primary Completion Date: March 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Group 1- Continuous positive airway pressure (CPAP)
Group 1 will receive therapeutic CPAP for 4 months.
Device: 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.
Other Name: CPAP
Placebo Comparator: Group 2-Sham Continuous positive airway pressure (CPAP)
Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months.
Device: 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.
Other Name: Sham or placebo CPAP
No Intervention: Group 3- No Intervention
Group 3 are individuals with normal breathing during sleep who will not receive any intervention and will be used as a control group.

Detailed Description:

All individuals participating in the study will be evaluated for obstructive sleep apnea syndrome (OSAS) with a baseline sleep study. Based on the results of the sleep study,individuals with OSAS will be randomly assigned into two groups. One group will receive continuous positive airway pressure (CPAP) and the other intervention group will receive sham CPAP(placebo) for four months. CPAP is a machine with a mask that fits over the nose, is worn during sleep and helps keep the airway open. Sham CPAP (placebo) is a machine that looks and sounds like a CPAP machine but does not give pressure so it does not treat OSAS. Individuals with normal breathing during the sleep study will not receive a machine, and will serve as controls.

Individuals will also have baseline tests including blood tests, an echocardiogram (pictures of the heart), a walking test and learning tests. After 4 months, the baseline tests will be repeated in all participants. At the end of the study, all individuals with OSAS will get treatment.

  Eligibility

Ages Eligible for Study:   8 Years to 20 Years   (Child, Adult)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Aged 8-20 years
  2. Down syndrome (based on the characteristic phenotype)
  3. Families provide informed consent and child provides assent

Exclusion Criteria:

  1. Subjects and families who do not speak English well enough to undergo psychometric testing.
  2. Subjects living in institutions where there is no primary caregiver to participate in the neurocognitive/behavioral battery.
  3. Major chronic lung disease such as chronic aspiration.
  4. Previous or current CPAP therapy.
  5. Participation in a weight loss program.
  6. Craniofacial or neuromuscular conditions other than those associated with DS.
  7. Untreated hypothyroidism. Participants with normal thyroid function tests will be included in the study.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01808508

Locations
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Sponsors and Collaborators
Children's Hospital of Philadelphia
Investigators
Principal Investigator: Carole Marcus, MD Children's Hospital of Philadelphia
  More Information

Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT01808508     History of Changes
Other Study ID Numbers: 11-007964 
Study First Received: March 7, 2013
Results First Received: December 4, 2015
Last Updated: January 8, 2016
Health Authority: United States: Institutional Review Board
United States: Food and Drug Administration

Keywords provided by Children's Hospital of Philadelphia:
Down syndrome
Obstructive sleep apnea syndrome
Continuous positive airway pressure
sham or placebo CPAP

Additional relevant MeSH terms:
Down Syndrome
Syndrome
Apnea
Sleep Apnea Syndromes
Sleep Apnea, Obstructive
Disease
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms, Respiratory
Signs and Symptoms
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Nervous System Diseases
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn

ClinicalTrials.gov processed this record on July 28, 2016