PAP for Children With DS and 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: Recruiting
• 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 (Estimated): 86
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Florida
    • Miami, Florida, United States, 33136
      • Recruiting
      • University of Miami
      • Contact: Ignacio E Tapia, MD; Phone Number: 305-243-5393; Email: itapia@miami.edu
      • Contact: Winnie Chauvel, BS; Phone Number: 305-2435393; Email: w.chauvel@med.miami.edu
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Recruiting
      • Cincinnati Children's Hospital Medical Center
      • Contact: Stacey Ishman, MD; Phone Number: 513-636-4944; Email: stacey.ishman@cchmc.org
      • Contact: Suzanna Hicks; Phone Number: (513) 636-4944; Email: Suzanna.Hicks@cchmc.org
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Recruiting
      • The Children's Hospital of Philadelphia
      • Contact: Ruth Bradford; Phone Number: (267) 426-5747; Email: bradford@chop.edu
      • Contact: Melissa Xanthopoulos, PhD; Phone Number: 267-426-5842; Email: xanthop@chop.edu

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
• Naive to PAP treatment

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
Effect of PAP adherence on quality of life and neurobehavioral outcomes	PedsQL score and measures of executive functioning will be compared in the 2 groups. PedsQL is a 5-point scale ranging from 0-4 (0 = Never, 4 = Always).	Baseline, 6 months, and 12 months
Efficacy of INT-PAP in promoting PAP adherence	Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.	6 months, and 12 months
Efficacy of CON in promoting PAP adherence	Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.	6 months, and 12 months
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	Patient-centered outcomes and family-relevant outcomes - identified during the R61 phase of the study (Stage 1) - will be compared between the intervention and control groups. These are qualitative measures, and require interviews and further analysis to be established.	Baseline, 6 months, and 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

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2021
• Primary Completion (Estimated): September 1, 2024
• Study Completion (Estimated): September 1, 2024

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 (Estimated): April 15, 2024
• Last Update Submitted That Met QC Criteria: April 12, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Keywords: Obstructive Sleep Apnea; Down Syndrome; Positive Airway Pressure; PAP
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Respiratory Tract Diseases; Respiration Disorders; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Signs and Symptoms, Respiratory; Intellectual Disability; Abnormalities, Multiple; Chromosome Disorders; Sleep Apnea Syndromes; Sleep Apnea, Obstructive; Syndrome; Apnea; Down Syndrome
• Other Study ID Numbers: 20230780; R61HL151253-01 (U.S. NIH Grant/Contract); R33HL151253 (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