Family-Informed Intervention for Obstructive Sleep Apnea in Children with Down Syndrome

Recruiting at 2 trial locations

Age: < 65

Sex: Any

Trial Phase: Academic

Sponsor: University of Miami

Disqualifiers: Leukemia, Severe heart disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: 6

Duration: 12 months

86 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new method to assist children with Down Syndrome who have obstructive sleep apnea (OSA), a condition that causes breathing problems during sleep. Researchers compare a family-informed intervention, which includes additional support and guidance from a psychologist, to the usual care children receive. The goal is to determine if this extra help makes a difference over a year. Children referred for their first use of a CPAP machine to treat OSA and who can participate in testing may be suitable for this study. As an unphased trial, this study offers families the opportunity to contribute to innovative research that could improve care for children with Down Syndrome and OSA.

Will I have to stop taking my current medications?

The trial information does not specify whether participants need to stop taking their current medications.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that starting treatment early for obstructive sleep apnea in children with Down Syndrome can improve their social skills and learning. This treatment is generally easy for children to handle. Although detailed information about side effects specific to this family-focused approach is lacking, involving families suggests there might be fewer negative effects. These treatments typically focus on providing advice and support rather than medication, often resulting in fewer safety concerns.

In this trial, the family-focused approach includes several in-person meetings and phone calls with a psychologist and their team. This method helps families and caregivers manage the condition without invasive procedures. Therefore, the safety of this approach likely resembles other supportive therapies, which usually carry minimal risk. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the family-informed intervention for obstructive sleep apnea in children with Down syndrome because it offers a personalized approach that goes beyond just standard clinical care. Unlike typical treatments that mainly focus on using Positive Airway Pressure (PAP) devices, this intervention incorporates multiple face-to-face visits and telephone calls with a PAP psychologist and team. This comprehensive support system aims to engage the entire family, potentially improving adherence to PAP therapy and overall outcomes for children. By focusing on family involvement, this approach could lead to better management of obstructive sleep apnea, addressing not just the symptoms but also the unique challenges faced by children with Down syndrome and their families.

What evidence suggests that this trial's treatments could be effective for obstructive sleep apnea in children with Down Syndrome?

This trial will compare a family-informed intervention with standard clinical care for managing obstructive sleep apnea (OSA) in children with Down syndrome. Research has shown that involving families is crucial for managing OSA. Families enhance the success of treatments like positive airway pressure (PAP) therapy. Studies have found that when families participate, children adhere to their treatment plans better, leading to improved health. Caregivers' support in using PAP devices is especially crucial, as it ensures consistent use of the treatment at home. Although the family-focused approach is still under study, involving families appears promising for enhancing treatment effectiveness for these children. ^{2 4 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children aged 6-18 with Down Syndrome and obstructive sleep apnea who haven't used PAP treatment before. They must be able to do tests and have a clinical referral for PAP. It's not for kids in foster care, those treated with PAP already, or with major illnesses like leukemia or severe heart disease.

Inclusion Criteria

I have never received PAP treatment.

My child can follow instructions for tests.

I am referred for a CPAP machine to treat my sleep apnea.

Exclusion Criteria

I am a caregiver and I do not speak English well enough for medical assessments.

My child was previously treated with PAP.

Children who are in the care of the foster system.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive family-informed intervention (INT) or standard clinical care to promote PAP adherence

12 months

Multiple face-to-face visits, telephone calls, and in-person visits

Follow-up

Participants are monitored for PAP adherence, quality of life, and neurobehavioral outcomes

12 months

Assessments at baseline, 6 months, and 12 months

What Are the Treatments Tested in This Trial?

Interventions

• Family-informed intervention
• Standard Clinical Care

Trial Overview The study compares the effectiveness of a family-informed intervention versus standard clinical care over twelve months in treating sleep apnea in children with Down Syndrome.

How Is the Trial Designed?

Group I: Family-informed intervention (INT) Active Control 1 Intervention

Multiple face-to-face visits, telephone calls and-person visits with the PAP psychologist and team.

Group II: Standard Clinical Care Active Control 1 Intervention

Support which is given as part of the standard clinical care for patients who are currently prescribed PAP.

Family-informed intervention is already approved in United States for the following indications:

Approved in United States as Family-informed intervention for:

• Improving adherence to PAP therapy in children with Down syndrome and obstructive sleep apnea

Who Is Running the Clinical Trial?

University of Miami

Lead Sponsor

Trials

976

Recruited

423,000+

Children's Hospital of Philadelphia

Lead Sponsor

Trials

749

Recruited

11,400,000+

Children's Hospital Medical Center, Cincinnati

Collaborator

Trials

844

Recruited

6,566,000+

University of Pennsylvania

Collaborator

Trials

2,118

Recruited

45,270,000+

National Heart, Lung, and Blood Institute (NHLBI)

Collaborator

Trials

3,987

Recruited

47,860,000+

National Institutes of Health (NIH)

Collaborator

Trials

2,896

Recruited

8,053,000+

Published Research Related to This Trial

Among 954 children with Down syndrome, 39.1% were diagnosed with sleep problems, but only 47.7% underwent necessary overnight sleep studies (polysomnography) to confirm these issues.

Of those diagnosed with sleep problems, 81.2% received appropriate interventions, indicating that while treatment matched diagnoses, there is a need for improved screening and referral practices, especially considering age and gender disparities in treatment rates.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Use of Sleep Evaluations and Treatments in Children with Down Syndrome. Esbensen, AJ., Beebe, DW., Byars, KC., et al.[2018]

Level II home sleep apnea testing (HSAT) is feasible and well-accepted among youth with Down syndrome, with 73.7% of parents preferring it over traditional in-lab polysomnography.

HSAT demonstrated good accuracy in detecting moderate to severe obstructive sleep apnea (OSA), with a sensitivity of 81% and specificity of 75%, making it a viable alternative for evaluating OSA in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Feasibility and performance of home sleep apnea testing in youth with Down syndrome. Cielo, CM., Kelly, A., Xanthopoulos, M., et al.[2023]

Adeno-tonsillectomy (T&A) in children with Down syndrome (DS) led to some improvement in obstructive sleep apnea (OSA) parameters, but these improvements were less significant compared to non-DS children, indicating a limited efficacy of the procedure for DS patients.

While 27% of DS children experienced enough improvement to avoid further treatment after T&A, a significant 73% still required additional interventions like CPAP or BiPAP for ongoing OSA, highlighting the need for continued management in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of adeno-tonsillectomy on polysomnography patterns in Down syndrome children with obstructive sleep apnea: a comparative study with children without Down syndrome. Shete, MM., Stocks, RM., Sebelik, ME., et al.[2022]

Individuals with Down syndrome (DS) have a higher prevalence and severity of obstructive sleep apnea (OSA), necessitating routine screening for all DS patients due to the potential for serious health consequences.

Polysomnography is the gold standard for diagnosing OSA in DS, and treatment options include adenotonsillectomy and CPAP, with emerging therapies like hypoglossal nerve stimulation showing promise for difficult cases.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Obstructive sleep apnea in patients with Down syndrome: current perspectives. Simpson, R., Oyekan, AA., Ehsan, Z., et al.[2020]

Children with Down syndrome (DS) showed significantly greater adherence to noninvasive ventilation (NIV) for obstructive sleep apnea (OSA) compared to non-DS children, with an average usage of 78.95% of days versus 72.11%.

Despite better adherence, children with DS experienced poorer clinical outcomes, indicated by a higher apnea-hypopnoea index (AHI) and greater leakage from the NIV system, suggesting that while they use the device more consistently, issues like excessive leakage may reduce its effectiveness.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and adherence of noninvasive ventilation treatment in children with Down syndrome. MacDonagh, L., Farrell, L., O'Reilly, R., et al.[2021]

In a study of 19 young patients with Down syndrome, 79% were found to have obstructive sleep apnea (OSA), indicating a high prevalence of this condition in this population.

The study revealed significant associations between OSA and factors such as higher body mass index, age, and poor sleep quality, highlighting the need for further research to improve treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Prospective polysomnographic analysis of obstructive sleep apnea in down syndrome. Dyken, ME., Lin-Dyken, DC., Poulton, S., et al.[2013]

In a study of five children with Down syndrome suffering from obstructive sleep apnea, it was found that their altered anatomy contributed to the condition, particularly affecting the oropharynx, cranium, and midface.

A modified pharyngopalatal surgical approach was effective in relieving upper airway obstruction in these patients, suggesting that traditional tonsillectomy and adenoidectomy may not be sufficient for this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Obstructive sleep apnea in Down syndrome children: a surgical approach. Strome, M.[2022]

Obstructive sleep apnea (OSA) is common in children, particularly those with Down syndrome and other comorbidities, highlighting the need for family-focused evaluations and innovative diagnostic methods.

Therapeutic options for treating OSA in children are limited, but recent studies suggest that hypoglossal nerve stimulation may be a promising treatment, alongside traditional positive airway pressure methods.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

What's New in Pediatric Obstructive Sleep Apnea? Cielo, CM., Tapia, IE.[2023]

Parents of children with Down Syndrome often normalize sleep difficulties, viewing them as a typical challenge of parenting, which can lead to underreporting and inadequate treatment of these issues.

Healthcare professionals frequently reinforce this normalization, resulting in insufficient care and missed opportunities for referrals to specialized sleep services, highlighting the need for increased awareness and proactive approaches in addressing sleep disorders in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Parents' Experiences of Having a Child with Down Syndrome and Sleep Difficulties. Chawla, JK., Cooke, E., Miguel, MC., et al.[2023]

Citations

1. withpower.com withpower.com/trial/phase-sleep-apnea-syndromes-2-2021-c5b0c

Family-Informed Intervention for Obstructive Sleep Apnea in ... The research highlights the importance of family-focused approaches in managing obstructive sleep apnea (OSA) in children with Down syndrome, as families play a ...

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11387522/

Early detection and treatment of obstructive sleep apnoea in ... The aim of our study was to evaluate the effect of early OSA treatment in infants with DS on neurocognitive development and behaviour.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05508971

Treatment of Obstructive Sleep Apnea With Personalized ... The investigators' primary hypothesis is that a personalized surgical intervention based on DISE findings will be more effective in treating OSA in children ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT06043440?term=AREA%5BBasicSearch%5D(SB5)&rank=9

NCT06043440 | Down Syndrome Obstructive Sleep Apnea This will be a randomized, single-blind 6-month Phase-2 clinical trial that compares the impact of oxygen therapy during sleep on measures of cognition, ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10330846/

Caregiver Experiences Helping Children with Down ... Caregiver experiences helping children with Down syndrome use positive airway pressure to treat obstructive sleep apnea.

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11417314/

Early intervention for obstructive sleep apnoea in Down ... Early diagnosis and management of obstructive sleep apnoea may offer long-term benefits such as improvements in social-emotional development, learning, and ...

7. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC5039052/

Use of Sleep Evaluations and Treatments in Children with ... Data were obtained from electronic health records from 2009–2013 for a retrospective cohort of 954 children with DS, ages 5–21 years during the time sampled.

8. mdpi.com mdpi.com/2075-4426/13/1/71

Obstructive Sleep Apnoea in Children with Down Syndrome A comprehensive evaluation of obstructive sleep apnoea (OSA) may allow for the development of more efficient management of Down syndrome (DS).