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Investigating the Experience of Living With Down Syndrome and Obstructive Sleep Apnea Syndrome (Stage 2)

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ClinicalTrials.gov Identifier: NCT04132999
Recruitment Status : Not yet recruiting
First Posted : October 21, 2019
Last Update Posted : October 22, 2019
Sponsor:
Collaborators:
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Children's Hospital Medical Center, Cincinnati
University of Pennsylvania
Information provided by (Responsible Party):
Children's Hospital of Philadelphia

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 Not Applicable

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.


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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 86 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: 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)
Estimated Study Start Date : March 2021
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : September 2024

Resource links provided by the National Library of Medicine


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.




Primary Outcome Measures :
  1. Effect of PAP adherence on quality of life and neurobehavioral outcomes [ Time Frame: 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).

  2. Efficacy of INT-PAP in promoting PAP adherence [ Time Frame: 6 months, and 12 months ]
    Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.

  3. Efficacy of CON in promoting PAP adherence [ Time Frame: 6 months, and 12 months ]
    Adherence to PAP expressed as percentage of nights used, and minutes of usage on nights used.

  4. 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 [ Time Frame: 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.



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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04132999


Locations
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United States, Pennsylvania
The Children's Hospital of Philadelphia Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Ignacio E Tapia, MD    267-426-5842    tapia@email.chop.edu   
Contact: Ruth Bradford    (267) 426-5747    bradford@email.chop.edu   
Sponsors and Collaborators
Children's Hospital of Philadelphia
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Children's Hospital Medical Center, Cincinnati
University of Pennsylvania

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Responsible Party: Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT04132999     History of Changes
Other Study ID Numbers: R61HL15125301 -- Stage 2
R61HL151253-01 ( U.S. NIH Grant/Contract )
First Posted: October 21, 2019    Key Record Dates
Last Update Posted: October 22, 2019
Last Verified: October 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Children's Hospital of Philadelphia:
Down Syndrome
Obstructive Sleep Apnea
PAP
Positive Airway Pressure
Additional relevant MeSH terms:
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Apnea
Sleep Apnea Syndromes
Sleep Apnea, Obstructive
Down Syndrome
Syndrome
Disease
Pathologic Processes
Respiration Disorders
Respiratory Tract Diseases
Signs and Symptoms, Respiratory
Signs and Symptoms
Sleep Disorders, Intrinsic
Dyssomnias
Sleep Wake Disorders
Nervous System Diseases
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn