PAP for Children With DS and OSAS

Sponsor

Children's Hospital of Philadelphia (Other)

Overall Status

Recruiting

CT.gov ID

NCT04132999

Collaborator

National Institutes of Health (NIH) (NIH), National Heart, Lung, and Blood Institute (NHLBI) (NIH), Children's Hospital Medical Center, Cincinnati (Other), University of Pennsylvania (Other)

Enrollment

Locations

Arms

42.1

Anticipated Duration (Months)

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Down Syndrome Obstructive Sleep Apnea	Behavioral: Family-informed intervention Behavioral: Standard Clinical Care	N/A

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 Design

Study Type:

Interventional

Anticipated Enrollment :

86 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

family-informed intervention (INT) vs standard clinical care (CON).family-informed intervention (INT) vs standard clinical care (CON).

Masking:

Triple (Participant, Care Provider, Investigator)

Primary Purpose:

Treatment

Official Title:

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

Actual Study Start Date :

Mar 1, 2021

Anticipated Primary Completion Date :

Sep 1, 2024

Anticipated Study Completion Date :

Sep 1, 2024

Arms and Interventions

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.

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.

Outcome Measures

Primary Outcome Measures

Effect of PAP adherence on quality of life and neurobehavioral outcomes [Baseline, 6 months, and 12 months]
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).
Efficacy of INT-PAP in promoting PAP adherence [6 months, and 12 months]
Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.
Efficacy of CON in promoting PAP adherence [6 months, and 12 months]
Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.
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 [Baseline, 6 months, and 12 months]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio	United States	45229
2	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104