Down SyndromeTRIONCT01808508
Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
A study testing Continuous positive airway pressure in people with Down Syndrome and Obstructive Sleep Apnea Syndrome, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 8 to 20 years
- Conditions the study is for
- Down Syndrome, Obstructive Sleep Apnea Syndrome
- What is being tested?
- Continuous positive airway pressure
- Type of treatment
- Medical device
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2010to Sep 2014
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Aged 8-20 years
- Down syndrome (based on the characteristic phenotype)
- Families provide informed consent and child provides assent
Who cannot join
- Subjects and families who do not speak English well enough to undergo psychometric testing.
- Subjects living in institutions where there is no primary caregiver to participate in the neurocognitive/behavioral battery.
- Major chronic lung disease such as chronic aspiration.
- Previous or current CPAP therapy.
- Participation in a weight loss program.
- Craniofacial or neuromuscular conditions other than those associated with DS.
- Untreated hypothyroidism. Participants with normal thyroid function tests will be included in the study.
Trial locations and contacts
1 site in 1 country.
United States1 site
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
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.
Read the 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.
- Conditions
- Down Syndrome, Obstructive Sleep Apnea Syndrome
- Being tested
- Continuous positive airway pressure, Sham or placebo continuous positive airway pressure
- Keywords
- Down syndrome, Obstructive sleep apnea syndrome, Continuous positive airway pressure, sham or placebo CPAP
- Medical subject headings
- Down Syndrome, Sleep Apnea, Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Continuous positive airway pressureDevice
Also called CPAP
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.
Sham or placebo continuous positive airway pressureDevice
Also called Sham or placebo CPAP
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.
| Study group | Role | What they receive |
|---|---|---|
| Group 1- Continuous positive airway pressure (CPAP) | Active comparator | Group 1 will receive therapeutic CPAP for 4 months. Receives: Device: Continuous positive airway pressure |
| Group 2-Sham Continuous positive airway pressure (CPAP) | Placebo comparator | Group 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months. Receives: Device: Sham or placebo continuous positive airway pressure |
| Group 3- No Intervention | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Epworth Sleepiness Scale From Baseline to End of Study
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).
Measured: 4 Months
Secondary outcomes (3)
Change in Child Behavior Checklist (CBCL) Total Score From Baseline to 4 Months
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).
Measured: 4 Months
Change in the Left Ventricular (LV) Mass Index Score From Baseline to 4 Months
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.
Measured: 4 Months
Change in the Distance Walked on a 6 Minute Walk Test From Baseline to 4 Months
As secondary outcome of the second aim, we will use the distance walked during the 6 minute walk test.
Measured: 4 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Mar 2013 (estimated)
- Study startSep 2010
- Primary completionMar 2014
- Study completionSep 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Carole Marcus, MD, Principal investigator, Children's Hospital of Philadelphia
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01808508
- Sponsor's protocol ID
- 11-007964
- Acronym
- TRIO
- Status last verified
- Dec 2015
- First submitted
- 7 Mar 2013
- Last updated
- 8 Feb 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Obstructive Sleep Apnea and Neurocognitive and Cardiovascular Function in Children With Down Syndrome
- ClinicalTrials.gov ID
- NCT01808508
- Acronym / sponsor ID
- TRIO / 11-007964
- Conditions
- Down Syndrome, Obstructive Sleep Apnea Syndrome
- Keywords
- Down syndrome, Obstructive sleep apnea syndrome, Continuous positive airway pressure, sham or placebo CPAP
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Mar 2014 (actual)
- Study completion
- Sep 2014 (actual)
- First posted
- 11 Mar 2013 (estimated)
- Results
- Posted 8 Feb 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Group 1- Continuous positive airway pressure (CPAP) | Active comparator | Device: Continuous positive airway pressureGroup 1 will receive therapeutic CPAP for 4 months. |
| Group 2-Sham Continuous positive airway pressure (CPAP) | Placebo comparator | Device: Sham or placebo continuous positive airway pressureGroup 2 are individuals with OSAS who will receive sham or placebo continuous positive airway pressure (CPAP) for 4 months. |
| Group 3- No Intervention | 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. |
Who may qualify
- Ages
- 8 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Aged 8-20 years
- Down syndrome (based on the characteristic phenotype)
- Families provide informed consent and child provides assent
Key exclusion criteria (7 listed)
- Subjects and families who do not speak English well enough to undergo psychometric testing.
- Subjects living in institutions where there is no primary caregiver to participate in the neurocognitive/behavioral battery.
- Major chronic lung disease such as chronic aspiration.
- Previous or current CPAP therapy.
- Participation in a weight loss program.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Continuous positive airway pressureAlso: CPAPContinuous positive airway pressure is a machine used with sleep which compresses air delivered via a nasal mask and thus helps stent the airway open. | Device | Group 1- Continuous positive airway pressure (CPAP) |
| Sham or placebo continuous positive airway pressureAlso: Sham or placebo CPAPSham 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. | Device | Group 2-Sham Continuous positive airway pressure (CPAP) |
Primary outcome
Change in Epworth Sleepiness Scale From Baseline to End of Study
Time frame: 4 Months
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).
3 secondary outcomes
Change in Child Behavior Checklist (CBCL) Total Score From Baseline to 4 Months
Time frame: 4 Months
Change in the Left Ventricular (LV) Mass Index Score From Baseline to 4 Months
Time frame: 4 Months
Change in the Distance Walked on a 6 Minute Walk Test From Baseline to 4 Months
Time frame: 4 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carole Marcus, MD principal investigator
Children's Hospital of Philadelphia
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Continuous positive airway pressure in people with Down Syndrome and Obstructive Sleep Apnea Syndrome, run by Children's Hospital of Philadelphia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Feb 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Feb 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Epworth Sleepiness Scale From Baseline to End of Study (time frame: 4 Months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2016.