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

This study is currently recruiting participants. (see Contacts and Locations)
Verified January 2014 by Children's Hospital of Philadelphia
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier:
NCT01808508
First received: March 7, 2013
Last updated: January 14, 2014
Last verified: January 2014
  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:
  • Sleepiness [ Time Frame: at baseline ] [ 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. The primary outcome for this aim is measuring sleepiness with the pediatric version of the Epworth Sleepiness Scale.


Secondary Outcome Measures:
  • Behavioral domain [ Time Frame: at baseline ] [ Designated as safety issue: No ]
    The behavioral domain is the secondary outcome of the primary aim of the study as measured by the Child Check Behavior List or the Adult Check Behavior List based on the individual's age.

  • The distance walked on a 6 minute walk test [ Time Frame: at baseline ] [ Designated as safety issue: No ]
    As secondary outcome of the second aim, we will use the distance walked during the 6 minute walk test.

  • Left ventricular mass index [ Time Frame: at baseline ] [ Designated as safety issue: No ]

    The secondary aim of the study is to assess the relationship between the obstructive sleep apnea syndrome and cardiovascular function of individuals with Down syndrome. As secondary outcome we will use the left ventricular mass index as measured on echocardiography.We hypothesize that subjects with OSAS who are randomized to therapeutic CPAP will have greater improvements in cardiac structure/function than will subjects randomized to sham CPAP or DS control subjects without OSAS.

    Furthermore, we hypothesize that these improvements will correlate with the degree of CPAP use. We hypothesize that subjects with OSAS who are randomized to therapeutic CPAP will have greater improvements in cardiac structure/function than will subjects randomized to sham CPAP or DS control subjects without OSAS.



Other Outcome Measures:
  • Changes in sleepiness as measured by the pediatric version of the Epworth Sleepiness Scale [ Time Frame: after 4 months ] [ Designated as safety issue: No ]

    The third aim of the study is to determine the effect of 4 months of treatment of OSAS on the neurocognitive and cardiovascular function of individuals with DS. Subjects with DS and OSAS will be randomized to 4 months of treatment with therapeutic CPAP or sham CPAP, and objective CPAP adherence will be monitored. DS controls with normal breathing during sleep will be followed for 4 months without treatment.

    We hypothesize the individuals with OSAS and DS who receive CPAP will have greater improvement in their neurocognitive and behavioral testing versus individuals with OSAS on sham CPAP.

    Furthermore, we hypothesize that these improvements will correlate with the degree of CPAP use.



Estimated Enrollment: 87
Study Start Date: September 2010
Estimated Study Completion Date: June 2015
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Group 1- CPAP

Subjects will be divided into 3 study groups, based on the initial polysomnogram: subjects with OSAS randomized to therapeutic CPAP (Group 1) or sham CPAP (Group 2), and those without OSAS (Group 3). The OSAS group will be randomized to receive either therapeutic CPAP or sham CPAP (placebo).

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 CPAP
Group 2 are individuals with OSAS who will receive sham or placebo 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
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
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

Contacts
Contact: Sofia KONSTANTINOPOULOU, MD 267-426-5842 konstantinopoulous@email.chop.edu
Contact: Carole Marcus 267-426-5842 marcus@email.chop.edu

Locations
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Sofia Konstantinopoulou, MD    267-426-5842    konstantinopoulous@email.chop.edu   
Contact: Carole Marcus, MD    267-426-5842    marcus@email.chop.edu   
Principal Investigator: Carole Marcus, MD         
Sub-Investigator: Sofia Konstantinopoulou, MD         
Sponsors and Collaborators
Children's Hospital of Philadelphia
Investigators
Principal Investigator: Carole Marcus, MD Children's Hospital of Philadelphia
  More Information

No publications provided

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
Last Updated: January 14, 2014
Health Authority: 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
Apnea
Sleep Apnea Syndromes
Sleep Apnea, Obstructive
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms, Respiratory
Signs and Symptoms
Mental Retardation
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Disorders

ClinicalTrials.gov processed this record on July 29, 2014