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

NCT01808508 | Completed Results DMC

• 1 other identifier: 11-007964
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief 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. 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.

Conditions

Down Syndrome; Obstructive Sleep Apnea Syndrome

Keywords

Down syndrome; Obstructive sleep apnea syndrome; Continuous positive airway pressure; sham or placebo CPAP

Outcome Measures

Primary Outcomes (1)

• 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).

  4 Months

Secondary Outcomes (3)

• Change in Child Behavior Checklist (CBCL) Total Score From Baseline to 4 Months

  4 Months

• Change in the Left Ventricular (LV) Mass Index Score From Baseline to 4 Months

  4 Months

• Change in the Distance Walked on a 6 Minute Walk Test From Baseline to 4 Months

  4 Months

Study Arms (3)

Group 1- Continuous positive airway pressure (CPAP)

ACTIVE COMPARATOR

Group 1 will receive therapeutic CPAP for 4 months.

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.

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.

Interventions

Continuous positive airway pressure DEVICE

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.

Also known as: CPAP

Group 1- Continuous positive airway pressure (CPAP)

Sham or placebo continuous positive airway pressure DEVICE

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.

Also known as: Sham or placebo CPAP

Group 2-Sham Continuous positive airway pressure (CPAP)

Eligibility Criteria

• Age: 8 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Conditions

Down Syndrome; Sleep Apnea, Obstructive

Interventions

Continuous Positive Airway Pressure

Condition Hierarchy (Ancestors)

Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Sleep Apnea Syndromes; Apnea; Respiration Disorders; Respiratory Tract Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders

Intervention Hierarchy (Ancestors)

Positive-Pressure Respiration; Respiration, Artificial; Airway Management; Therapeutics; Respiratory Therapy

Limitations and Caveats

This preliminary study was limited by the small sample size, and the few children with Down Syndrome but normal breathing during sleep.

Results Point of Contact

• Title: Carole L. Marcus, MBBCh
• Organization: Children's Hospital of Philadelphia

marcus@email.chop.edu

267-426-5842

Study Officials

• Carole Marcus, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2013
• First Posted: March 11, 2013
• Study Start: September 1, 2010
• Primary Completion: March 1, 2014
• Study Completion: September 1, 2014
• Last Updated: February 8, 2016
• Results First Posted: February 8, 2016

Record last verified: 2015-12

Locations

US (1)