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

NCT06030349 | Recruiting

• 1 other identifier: REC137-22
• Study Type: observational
• Target: 200
• Locations: 2 countries
• Sites: 6

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.

Conditions

Obstructive Sleep Apnea; Down Syndrome; Quality of Life; Behavior; Adherence, Treatment

Outcome Measures

Primary Outcomes (8)

• 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.

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• 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.

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• 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.

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• To determine the effect of treatment of obstructive sleep apnoea with non-invasive ventilation on sleep behaviours in children with Down Syndrome > 3 years.

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• To determine the effect of treatment of obstructive sleep apnoea with non-invasive ventilation on sleep behaviours in children with Down Syndrome < 3 years.

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• 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

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• 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

  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.

  Prior to and following 8 weeks attempted initiation of non-invasive ventilation treatment

• Assessment of expectations, experiences and barriers experienced by families in the treatment of obstructive sleep apnoea syndrome with 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

  After 8 weeks of attempted initiation of non-invasive ventilation

Study Arms (2)

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

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

Interventions

Non-invasive ventilation DEVICE

Non-invasive ventilation

Advanced; Standard

Eligibility Criteria

• Age: 6 Months - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

Children with Down Syndrome referred for evaluation for suspected sleep disordered breathing

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Children's Health Ireland: lead
• University of Edinburgh: collaborator
• Birmingham Children's Hospital: collaborator
• Belfast Health and Social Care Trust: collaborator
• University Hospital Southampton NHS Foundation Trust: collaborator
• University of Dublin, Trinity College: collaborator

Study Sites (6)

Trinity College Dublin

Dublin, D02 PN40, Ireland

RECRUITING

Children's Health Ireland

Dublin, D12 N512, Ireland

RECRUITING

Royal Belfast Hospital for Sick Children

Belfast, BT12 6BA, United Kingdom

NOT YET RECRUITING

Birmingham Children's Hospital

Birmingham, B4 6NH, United Kingdom

NOT YET RECRUITING

Royal Hospital for Children & Young People

Edinburgh, EH16 4TJ, United Kingdom

NOT YET RECRUITING

Southampton Children's Hospital

Southampton, SO16 6YD, United Kingdom

NOT YET RECRUITING

MeSH Terms

Conditions

Sleep Apnea, Obstructive; Down Syndrome; Behavior; Treatment Adherence and Compliance

Interventions

Noninvasive Ventilation

Condition Hierarchy (Ancestors)

Sleep Apnea Syndromes; Apnea; Respiration Disorders; Respiratory Tract Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Health Behavior

Intervention Hierarchy (Ancestors)

Respiration, Artificial; Airway Management; Therapeutics; Respiratory Therapy

Study Officials

• Sheila Javadpour

  Children's Health Ireland

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Roy G Stone

CONTACT

0035314282626; refreshed@olchc.ie

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Lead for Respiratory Sleep Medicine

Study Record Dates

• First Submitted: April 30, 2023
• First Posted: September 11, 2023
• Study Start: November 28, 2022
• Primary Completion (Estimated): November 28, 2027
• Study Completion (Estimated): December 28, 2027
• Last Updated: February 12, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

IE (2); GB (4)