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Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea in Children With Down Syndrome (REFRESHED)

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ClinicalTrials.gov Identifier: NCT06030349
Recruitment Status : Recruiting
First Posted : September 11, 2023
Last Update Posted : February 12, 2024
Sponsor:
Collaborators:
University of Edinburgh
Birmingham Children's Hospital
Belfast Health and Social Care Trust
University Hospital Southampton NHS Foundation Trust
University of Dublin, Trinity College
Information provided by (Responsible Party):
Sheila Javadpour, Children's Health Ireland

Study Description
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:

The goal of this observational study is to learn about the use of non-invasive ventilation for treatment of obstructive sleep apnoea syndrome in children with Down Syndrome. The main questions it aims to answer are:

  • What is the impact of non-invasive ventilation on sleep behaviours and quality of life?
  • What barriers are faced by children and their families in establishing tolerance to non-invasive ventilation?

Participants will be asked to complete questionnaires before and after starting treatment. Researchers will compare this data with the results of sleep studies and non-invasive ventilator downloads recorded as part of standard medical care. A sub-group of up to 20 participants will be invited to take part in 45-60 minute interviews exploring expectations, experiences and barriers encountered during non-invasive ventilation therapy.


Condition or disease Intervention/treatment
Obstructive Sleep Apnea Down Syndrome Quality of Life Behavior Adherence, Treatment Device: Non-invasive ventilation

Detailed Description:

Our aim is to examine the impact of non-invasive ventilation for treatment of obstructive sleep apnoea syndrome in children with Down Syndrome on behavioural and quality of life outcomes and to establish the specific barriers experienced in establishing adherence to treatment.

Information from clinical assessment, sleep studies and device downloads recorded as part of standard care will be combined with questionnaire data to assess for changes in behaviour and quality of life. All participants will undergo questionnaire assessment of barriers experienced. A sub-group of up to 20 participants will be invited to take part in 45-60 minute semi-structured interviews exploring expectations, experiences and barriers encountered.

Study Design
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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Real World Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea Syndrome in Children With Down Syndrome
Actual Study Start Date : November 28, 2022
Estimated Primary Completion Date : November 28, 2027
Estimated Study Completion Date : December 28, 2027

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
Standard
In addition to data from clinical assessment, sleep studies and non-invasive ventilator downloads recorded as part of standard care participants will undergo questionnaire assessment of behaviour, quality of life and barriers to tolerating treatment. Participants aged 3 years and above will receive the Child Sleep Habits Questionnaire, Obstructive Sleep Apnoea-18 questionnaire and Adherence Barriers to CPAP (Continuous Positive Airway Pressure) Questionnaire. Participants aged 6 months to 3 years will receive the Brief Infant Sleep Questionnaire, Infant Toddler Quality of Life - Short Form 47, Obstructive Sleep Apnoea -18 Caregiver Concern Domain and a modified version of the Adherence Barriers to CPAP Questionnaire.
 Device: Non-invasive ventilation
Non-invasive ventilation
Advanced
In addition to all elements of the standard testing group, a sub-group of up to 20 participants will be invited to take part in 45-60 minute semi-structured interviews exploring expectations, experiences and barriers encountered.
 Device: Non-invasive ventilation
Non-invasive ventilation


Outcome Measures
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Primary Outcome Measures :
  1. To determine the effect of treatment of obstructive sleep apnoea syndrome with non-invasive ventilation on quality of life in children with Down Syndrome > 3 years. [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Change in Obstructive Sleep Apnoea - 18 scores (Range 0- 126, Higher scores indicating worse quality of life) from baseline will be used to evaluate for improvement in sleep related breathing specific quality of life in children >3 years. Sleep disturbance, physical suffering, emotional distress, daytime problems and caregiver concern subscales will also be evaluated for change.

  2. To determine the effect of treatment of obstructive sleep apnoea syndrome with non-invasive ventilation on quality of life in children with Down Syndrome < 3 years. [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Change in Infant Toddler Quality of Life - Short Form 47 scores (Range 0 - 100, Higher scores indicating greater quality of life) from baseline will be used to evaluate for improvement in sleep related breathing specific quality of life in children < 3 years. Infant and parent subscales will also be evaluated for change.

  3. To determine the effect of treatment of obstructive sleep apnoea syndrome with non-invasive ventilation on caregiver concerns regarding sleep disordered breathing for caregivers of children with Down Syndrome < 3 years. [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Change in Caregiver-concern domain of the Obstructive Sleep Apnoea - 18 (Range 0 - 28, Higher scores indicating worse quality of life) from baseline will be used to evaluate for change in caregiver concern regarding sleep disordered breathing.

  4. To determine the effect of treatment of obstructive sleep apnoea with non-invasive ventilation on sleep behaviours in children with Down Syndrome > 3 years. [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Change in Child Sleep Habits Questionnaire scores (Range 0 - 97, Higher scores indicating greater sleeping difficulties) from baseline will be used to evaluate for overall improvement in sleep behaviours in children > 3 years. Bedtime resistance, sleep onset delay, sleep duration, sleep anxiety, night waking, parasomnia, sleep disordered breathing and daytime sleepiness subscales will also be evaluated for change.

  5. To determine the effect of treatment of obstructive sleep apnoea with non-invasive ventilation on sleep behaviours in children with Down Syndrome < 3 years. [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Change in Brief Infant Sleep Questionnaire scores (Range 0 - 100, Lower scores indicating greater sleeping difficulties) from baseline will be used to evaluate for overall improvement in sleep behaviours in children < 3 years. Infant sleep, parent perception and parent behaviour subscales will also be evaluated for change.

  6. To establish the specific barriers to adherence faced by children > 3 years with Down Syndrome and their caregivers when starting non-invasive ventilation for the treatment of obstructive sleep apnoea syndrome [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Quantitative assessment of barriers encountered in establishing adherence will be evaluated using the Continuous Positive Airway Pressure Questionnaire (Range 29-145, Higher scores indicating greater barriers to adherence) for children > 3 years. Behaviours/belief/environment, emotional and physical subscales will also be evaluated for change.

  7. To establish the specific barriers to adherence faced by children < 3 years with Down Syndrome and their caregivers when starting non-invasive ventilation for the treatment of obstructive sleep apnoea syndrome [ Time Frame: Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment ]
    Quantitative assessment of barriers encountered in establishing adherence will be evaluated using the Modified Continuous Positive Airway Pressure Questionnaire (Range 27 - 135, Higher scores indicating greater barriers to adherence) for children < 3 years. This is a non-validated questionnaire developed by our research group.

  8. Assessment of expectations, experiences and barriers experienced by families in the treatment of obstructive sleep apnoea syndrome with non-invasive ventilation [ Time Frame: After 8 weeks of attempted initiation of non-invasive ventilation ]
    Qualitative assessment of expectations, experiences and barriers experienced during non-invasive ventilation treatment will be explored through semi-structured interviews to add nuance to the outcomes around efficacy of treatment, quality of life, behaviour, and treatment adherence


Eligibility Criteria
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Ages Eligible for Study:   6 Months to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Children with Down Syndrome referred for evaluation for suspected sleep disordered breathing
Criteria

Inclusion Criteria:

  • Individuals with a confirmed diagnosis of Down Syndrome where a clinical decision has been made to initiate respiratory support with non-invasive ventilation.
  • Obstructive Apnoea Hypopnoea Index > 2 episodes/hour or where sleep disordered breathing symptoms occur in combination with an Apnoea Hypopnoea Index > 1 episode/hr. - English language proficiency.
  • Age ≥ 4 months at the commencement of therapy.

Exclusion Criteria:

  • Individuals not willing to comply with study procedures or assessments.
  • Individuals for whom ventilatory support devices are already prescribed and those with artificial airways.
  • Individuals on clinical trials of investigational support therapies.
Contacts and Locations
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

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): NCT06030349


Contacts
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Contact: Roy G Stone 0035314282626 ext 604 refreshed@olchc.ie

Locations
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Ireland
Trinity College Dublin Recruiting
Dublin, Ireland, D02 PN40
Contact: Ruth Monaghan         
Principal Investigator: Elizabeth Nixon         
Sub-Investigator: Ruth Monaghan         
Children's Health Ireland Recruiting
Dublin, Ireland, D12 N512
Contact: Roy G Stone    0035314282626 ext 604    refreshed@olchc.ie   
Principal Investigator: Sheila Javadpour         
Sub-Investigator: Roy G Stone         
Sub-Investigator: Lisa Farrell         
Principal Investigator: Anne-Marie Casey         
United Kingdom
Royal Belfast Hospital for Sick Children Not yet recruiting
Belfast, United Kingdom, BT12 6BA
Contact: Dara O'Donoghue         
Principal Investigator: Dara O'Donoghue         
Sub-Investigator: Barbara Maxwell         
Sub-Investigator: Ruth Harte         
Birmingham Children's Hospital Not yet recruiting
Birmingham, United Kingdom, B4 6NH
Contact: Priti Kenia         
Principal Investigator: Priti Kenia         
Royal Hospital for Children & Young People Not yet recruiting
Edinburgh, United Kingdom, EH16 4TJ
Contact: Florian Gahleitner         
Principal Investigator: Florian Gahleitner         
Southampton Children's Hospital Not yet recruiting
Southampton, United Kingdom, SO16 6YD
Contact: Hazel Evans         
Contact: Catherine M Hill         
Principal Investigator: Hazel Evans         
Principal Investigator: Catherine M Hill         
Sponsors and Collaborators
Children's Health Ireland
University of Edinburgh
Birmingham Children's Hospital
Belfast Health and Social Care Trust
University Hospital Southampton NHS Foundation Trust
University of Dublin, Trinity College
Investigators
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Principal Investigator: Sheila Javadpour Children's Health Ireland
More Information
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Responsible Party: Sheila Javadpour, Clinical Lead for Respiratory Sleep Medicine, Children's Health Ireland
ClinicalTrials.gov Identifier: NCT06030349    
Other Study ID Numbers: REC137-22
First Posted: September 11, 2023    Key Record Dates
Last Update Posted: February 12, 2024
Last Verified: February 2024
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
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
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