Down SyndromeNCT04132999
Positive Airway Pressure (PAP) for Children With Down Syndrome (DS) and Obstructive Sleep Apnea Syndrome (OSAS)
A study testing Family-informed intervention and Standard Clinical Care in people with Down Syndrome and Obstructive Sleep Apnea, run by University of Miami at 3 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Down Syndrome, Obstructive Sleep Apnea
- What is being tested?
- Family-informed intervention and Standard Clinical Care
- Type of treatment
- Lifestyle or therapy programme
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 3 sitesUnited States
- Study dates
- 1 Mar 2021to 30 Sep 2025
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical referral for PAP initiation to treat OSAS
- Ages 6-18 years
- Children are able to cooperate with testing
- Have not used PAP for the past 3 years prior to enrollment
Who cannot join
- 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.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Miami
Miami, Florida, 33136
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: Positive Airway Pressure for the Treatment of the Obstructive Sleep Apnea Syndrome in Children With Down Syndrome (Stage 2)
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.
Read the 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.
- Conditions
- Down Syndrome, Obstructive Sleep Apnea
- Being tested
- Family-informed intervention, Standard Clinical Care
- Keywords
- Down Syndrome, Obstructive Sleep Apnea, PAP, Positive Airway Pressure
- Medical subject headings
- Down Syndrome, Sleep Apnea, Obstructive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Family-informed interventionBehavioural
Multiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team
Standard Clinical CareBehavioural
Support which is given as part of the standard clinical care for patients who are currently prescribed PAP.
| Study group | Role | What they receive |
|---|---|---|
| Family-informed intervention (INT) | Active comparator | Multiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team. Receives: Behavioral: Family-informed intervention |
| Standard Clinical Care | Active comparator | Support which is given as part of the standard clinical care for patients who are currently prescribed PAP. Receives: Behavioral: Standard Clinical Care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Nights Adherent to PAP Therapy from Initiation to 6 Months
PAP adherence will be assessed as the percentage of nights in which the participant is adherent to PAP therapy from initiation (T0) through 6 months (T6). This is calculated as the number of nights meeting adherence criteria divided by the total number of nights during the participant's study participation period, multiplied by 100.
Measured: 6 months
Secondary outcomes (4)
Number of Minutes of PAP Use on Nights Used
PAP adherence will be measured as the number of minutes of PAP use per night among nights with any recorded PAP use, calculated per participant from PAP initiation through the end of the 6-month intervention period.
Measured: 6 months
Change in PedsQL Total Score (Quality of Life)
Quality of life will be measured using the Pediatric Quality of Life Inventory (PedsQL) total score. The PedsQL is a 5-point scale ranging from 0 to 4(0 = Never, 4 = Always), with higher scores indicating better quality of life.
Measured: Baseline, 6 months, and 12 months
Change in Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) Parent Report Score
Executive functioning will be assessed using the Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) Parent Report. Scores are reported as standardized T-scores, which range from 30 to 120. Lower T-scores indicate better executive functioning, while higher T-scores indicate greater executive dysfunction.
Measured: Baseline, 6 months, and 12 months
Percentage of Nights Adherent to PAP Therapy from Initiation to 12 Months
PAP adherence will be assessed as the percentage of nights in which the participant is adherent to PAP therapy from initiation (T0) through 12 months (T12). This is calculated as the number of nights meeting adherence criteria divided by the total number of days during the participant's study participation period, multiplied by 100.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2019
- Study start1 Mar 2021
- Primary completion30 Sep 2025
- Study completion30 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Down SyndromeStudies of the same condition, open and under way.
- More trials run by University of MiamiThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- family-informed intervention (INT) vs standard clinical care (CON).
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Miami
- Sponsor type
- University, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Children's Hospital Medical Center, Cincinnati, University of Pennsylvania
- Responsible party
- Principal investigator, Ignacio Tapia, University of Miami
- Investigators
- Ignacio E. Tapia, M.D., M.S., Principal investigator, University of Miami
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04132999
- Sponsor's protocol ID
- 20230780
- Other IDs
- R61HL151253, R33HL151253
- Status last verified
- Aug 2025
- First submitted
- 10 Oct 2019
- Last updated
- 30 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Xanthopoulos MS, Byars K, Meinzen-Derr J, Ebensen A, Xiao R, Heubi C, Gurbani NS, Ishman SL, Bradford R, Hicks S, Redline S, Tapia IE. Standard medical care versus enhanced interdisciplinary care for implementation of positive airway pressure in youth with Down syndrome: a randomised controlled trial protocol. BMJ Open. 2025 Nov 23;15(11):e110990. doi: 10.1136/bmjopen-2025-110990.
PubMed 41285506
Study at a glance
From the registry record, last updated by the sponsor on 30 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Positive Airway Pressure for the Treatment of the Obstructive Sleep Apnea Syndrome in Children With Down Syndrome (Stage 2)
- ClinicalTrials.gov ID
- NCT04132999
- Acronym / sponsor ID
- 20230780
- Conditions
- Down Syndrome, Obstructive Sleep Apnea
- Keywords
- Down Syndrome, Obstructive Sleep Apnea, PAP, Positive Airway Pressure
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 27 participants (actual)
- Lead sponsor
- University of MiamiUniversity, hospital or other
- Collaborators
- National Institutes of Health (NIH), National Heart, Lung, and Blood Institute (NHLBI), Children's Hospital Medical Center, Cincinnati, University of Pennsylvania
- Study start
- 1 Mar 2021 (actual)
- Primary completion
- 30 Sep 2025 (actual)
- Study completion
- 30 Sep 2025 (actual)
- First posted
- 21 Oct 2019 (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.
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.
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
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- family-informed intervention (INT) vs standard clinical care (CON).
| Arm | Type | Receives |
|---|---|---|
| Family-informed intervention (INT) | Active comparator | Behavioral: Family-informed interventionMultiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team. |
| Standard Clinical Care | Active comparator | Behavioral: Standard Clinical CareSupport which is given as part of the standard clinical care for patients who are currently prescribed PAP. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Clinical referral for PAP initiation to treat OSAS
- Ages 6-18 years
- Children are able to cooperate with testing
- Have not used PAP for the past 3 years prior to enrollment
Key exclusion criteria (5 listed)
- 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.
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 |
|---|---|---|
| Family-informed interventionMultiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team | Behavioural | Family-informed intervention (INT) |
| Standard Clinical CareSupport which is given as part of the standard clinical care for patients who are currently prescribed PAP. | Behavioural | Standard Clinical Care |
Primary outcome
Percentage of Nights Adherent to PAP Therapy from Initiation to 6 Months
Time frame: 6 months
PAP adherence will be assessed as the percentage of nights in which the participant is adherent to PAP therapy from initiation (T0) through 6 months (T6). This is calculated as the number of nights meeting adherence criteria divided by the total number of nights during the participant's study participation period, multiplied by 100.
4 secondary outcomes
Number of Minutes of PAP Use on Nights Used
Time frame: 6 months
Change in PedsQL Total Score (Quality of Life)
Time frame: Baseline, 6 months, and 12 months
Change in Behavior Rating Inventory of Executive Function, Second Edition (BRIEF-2) Parent Report Score
Time frame: Baseline, 6 months, and 12 months
Percentage of Nights Adherent to PAP Therapy from Initiation to 12 Months
Time frame: 12 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Jun 2026.
- Derived from this study: PubMed 41285506Xanthopoulos MS, Byars K, Meinzen-Derr J, Ebensen A, Xiao R, Heubi C, Gurbani NS, Ishman SL, Bradford R, Hicks S, Redline S, Tapia IE. Standard medical care versus enhanced interdisciplinary care for implementation of positive airway pressure in youth with Down syndrome: a randomised controlled trial protocol. BMJ Open. 2025 Nov 23;15(11):e110990. doi: 10.1136/bmjopen-2025-110990.
- 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. This study is not recruiting at the moment.
Ignacio E. Tapia, M.D., M.S. principal investigator
University of Miami
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Family-informed intervention and Standard Clinical Care in people with Down Syndrome and Obstructive Sleep Apnea, run by University of Miami at 3 sites in United States.
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 4 inclusion and 5 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: finished, as of the sponsor's last update on 30 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Nights Adherent to PAP Therapy from Initiation to 6 Months (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04132999, which also lists 1 linked publication.
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 30 Jun 2026.