Obstructive Sleep ApneaREFRESHEDNCT06030349
Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea in Children With Down Syndrome
An observational study of Obstructive Sleep Apnea and Down Syndrome, run by Children's Health Ireland at 6 sites in 2 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 16 years
- Conditions the study is for
- Obstructive Sleep Apnea, Down Syndrome, Quality of Life
- What is being tested?
- Non-invasive ventilation
- Type of treatment
- Medical device
- How many people
- 200 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 28 Nov 2022to 28 Dec 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 16 years
- All sexes
- No healthy volunteers
- Child
Study population: Children with Down Syndrome referred for evaluation for suspected sleep disordered breathing
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
6 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
6 sites in 2 countries
United Kingdom4 sites, 4 recruiting
- Not yet recruiting
Royal Belfast Hospital for Sick Children
Belfast, BT12 6BA
Dara O'Donoghue
Dara O'Donoghue
- Not yet recruiting
Birmingham Children's Hospital
Birmingham, B4 6NH
Priti Kenia
Priti Kenia
- Not yet recruiting
Royal Hospital for Children & Young People
Edinburgh, EH16 4TJ
Florian Gahleitner
Florian Gahleitner
- Not yet recruiting
Southampton Children's Hospital
Southampton, SO16 6YD
Hazel Evans
Catherine M Hill
Ireland2 sites, 2 recruiting
- Recruiting
Trinity College Dublin
Dublin, D02 PN40
Ruth Monaghan
Elizabeth Nixon
- Recruiting
Children's Health Ireland
Dublin, D12 N512
Roy G Stone, 0035314282626 ext. 604, refreshed@olchc.ie
Sheila Javadpour
About this study
Official title: Real World Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea Syndrome in Children With Down Syndrome
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.
Read the 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.
- Being tested
- Non-invasive ventilation
- Medical subject headings
- Sleep Apnea, Obstructive, Down Syndrome, Behavior, Treatment Adherence and Compliance
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-invasive ventilationDevice
Non-invasive ventilation
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Device: Non-invasive ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: After 8 weeks of attempted initiation of non-invasive ventilation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2023
- Study start28 Nov 2022
- Primary completion28 Nov 2027 (estimated)
- Study completion28 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Obstructive Sleep ApneaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Health Ireland
- Sponsor type
- Other government
- 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
- Responsible party
- Principal investigator, Sheila Javadpour, Children's Health Ireland
- Investigators
- Sheila Javadpour, Principal investigator, Children's Health Ireland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06030349
- Sponsor's protocol ID
- REC137-22
- Acronym
- REFRESHED
- Status last verified
- Feb 2024
- First submitted
- 30 Apr 2023
- Last updated
- 12 Feb 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real World Clinical Outcomes From Treatment and Evaluation of Obstructive Sleep Apnoea Syndrome in Children With Down Syndrome
- ClinicalTrials.gov ID
- NCT06030349
- Acronym / sponsor ID
- REFRESHED / REC137-22
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- Children's Health IrelandOther government
- 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
- Study start
- 28 Nov 2022 (actual)
- Primary completion
- 28 Nov 2027 (estimated)
- Study completion
- 28 Dec 2027 (estimated)
- First posted
- 11 Sep 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
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
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Standard | – | Device: Non-invasive ventilationIn 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. |
| Advanced | – | Device: Non-invasive ventilationIn 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. |
Who may qualify
- Ages
- 6 months to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Children with Down Syndrome referred for evaluation for suspected sleep disordered breathing
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Non-invasive ventilationNon-invasive ventilation | Device | Advanced, Standard |
Primary outcome
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.
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.
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.
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.
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.
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.
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.
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Sheila Javadpour principal investigator
Children's Health Ireland
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Obstructive Sleep Apnea and Down Syndrome, run by Children's Health Ireland at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 12 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United Kingdom, Ireland. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Roy G Stone (0035314282626 ext. 604, refreshed@olchc.ie), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 8 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Feb 2024.