Positive Airway Pressure (PAP) for Children With Down Syndrome (DS) and Obstructive Sleep Apnea Syndrome (OSAS)

Positive Airway Pressure for the Treatment of the Obstructive Sleep Apnea Syndrome in Children With Down Syndrome (Stage 2)

Determine the efficacy of family-informed intervention (INT) vs standard clinical care over a period of twelve months in children with obstructive sleep apnea and Down Syndrome.

Study Overview

• Status: Completed
• Conditions: Obstructive Sleep Apnea; Down Syndrome
• Intervention / Treatment: Behavioral: Family-informed intervention; Behavioral: Standard Clinical Care

Detailed Description

Evaluate the effect of PAP adherence on quality of life, neurobehavioral, and healthcare utilization in children with DS and OSAS. It is being hypothesized that, irrespective of study arm, increased PAP adherence will be associated with better quality of life, neurobehavioral, and healthcare utilization outcomes.

Evaluate the effect of PAP adherence in children with DS and OSAS on patient-centered outcomes and family-relevant outcomes identified during the R61 phase of this research. It is being hypothesized that irrespective of study arm, increased PAP adherence will be associated with better patient-centered outcomes, and family-relevant outcomes identified during the first year of this research.

Determine the efficacy of INT vs. CON in promoting PAP adherence. It is being hypothesized that that children receiving INT will show significantly increased objectively-measured PAP adherence at 6 months compared with those receiving CON (Aim 4A). As a secondary aim (4B), it will be evaluated whether the improved adherence is maintained over 12 months. It is being hypothesized that participants initially randomized to the INT arm will have better adherence at 12 months compared to those in the CON arm.

Use mixed methods during the randomized controlled trial to identify family perceptions, such as empowerment and self-efficacy, regarding PAP use in youth with OSAS and DS. It is being hypothesized that INT-PAP will be associated with more positive perceptions compared to CON.

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • University of Miami
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • The Children's Hospital of Philadelphia

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Clinical referral for PAP initiation to treat OSAS
• Ages 6-18 years
• Children are able to cooperate with testing
• Have not used PAP for the past 3 years prior to enrollment

Exclusion Criteria:

• Major illnesses, such as leukemia or severe cyanotic congenital heart disease listed for cardiac transplant.
• Family planning to move out of the city within the next year
• Children in foster care
• Child with previously treated with PAP
• Caregivers who do not speak English well enough to complete behavioral and performance measures.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Family-informed intervention (INT); Multiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team.	Behavioral: Family-informed intervention; Multiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team
Active Comparator: Standard Clinical Care; Support which is given as part of the standard clinical care for patients who are currently prescribed PAP.	Behavioral: Standard Clinical Care; Support which is given as part of the standard clinical care for patients who are currently prescribed PAP.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Nights Adherent to PAP Therapy from Initiation to 6 Months	PAP adherence will be assessed as the percentage of nights in which the participant is adherent to PAP therapy from initiation (T0) through 6 months (T6). This is calculated as the number of nights meeting adherence criteria divided by the total number of nights during the participant's study participation period, multiplied by 100.	6 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Minutes of PAP Use on Nights Used	PAP adherence will be measured as the number of minutes of PAP use per night among nights with any recorded PAP use, calculated per participant from PAP initiation through the end of the 6-month intervention period.	6 months
Change in PedsQL Total Score (Quality of Life)	Quality of life will be measured using the Pediatric Quality of Life Inventory (PedsQL) total score. The PedsQL is a 5-point scale ranging from 0 to 4(0 = Never, 4 = Always), with higher scores indicating better quality of life.	Baseline, 6 months, and 12 months
Change in Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) Parent Report Score	Executive functioning will be assessed using the Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2), a parent-reported questionnaire that evaluates behaviors associated with executive functioning. Scores are reported as standardized T-scores, with higher scores indicating greater executive dysfunction	Baseline, 6 months, and 12 months
Percentage of Nights Adherent to PAP Therapy from Initiation to 12 Months	PAP adherence will be assessed as the percentage of nights in which the participant is adherent to PAP therapy from initiation (T0) through 12 months (T12). This is calculated as the number of nights meeting adherence criteria divided by the total number of days during the participant's study participation period, multiplied by 100.	12 months

Collaborators and Investigators

• Sponsor: University of Miami
• Collaborators: University of Pennsylvania; National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health (NIH); Children's Hospital Medical Center, Cincinnati
• Investigators: Principal Investigator: Ignacio E. Tapia, M.D., M.S., University of Miami

Publications and helpful links

General Publications

• Xanthopoulos MS, Byars K, Meinzen-Derr J, Ebensen A, Xiao R, Heubi C, Gurbani NS, Ishman SL, Bradford R, Hicks S, Redline S, Tapia IE. Standard medical care versus enhanced interdisciplinary care for implementation of positive airway pressure in youth with Down syndrome: a randomised controlled trial protocol. BMJ Open. 2025 Nov 23;15(11):e110990. doi: 10.1136/bmjopen-2025-110990.

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2021
• Primary Completion (Actual): September 30, 2025
• Study Completion (Actual): September 30, 2025

Study Registration Dates

• First Submitted: October 10, 2019
• First Submitted That Met QC Criteria: October 17, 2019
• First Posted (Actual): October 21, 2019

Study Record Updates

• Last Update Posted (Actual): May 29, 2026
• Last Update Submitted That Met QC Criteria: May 26, 2026
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Keywords: Obstructive Sleep Apnea; Down Syndrome; Positive Airway Pressure; PAP
• Additional Relevant MeSH Terms: Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, Inborn; Respiratory Tract Diseases; Neurobehavioral Manifestations; Respiration Disorders; Sleep Wake Disorders; Congenital Abnormalities; Abnormalities, Multiple; Apnea; Sleep Disorders, Intrinsic; Dyssomnias; Intellectual Disability; Sleep Apnea Syndromes; Chromosome Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Sleep Apnea, Obstructive; Down Syndrome
• Other Study ID Numbers: 20230780; R33HL151253 (U.S. NIH Grant/Contract); R61HL151253 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No