Oxygen therapy for children with Down Syndrome and sleep apnea

Randomized Control Trial of Oxygen Therapy in Children and Adolescents With Down Syndrome and Obstructive Sleep Apnea

This study is testing whether giving extra oxygen during sleep can help improve thinking skills and overall health in children with Down Syndrome who have sleep apnea.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 230 (estimated)
• Ages: 5 Years to 18 Years
• Sex: All
• Sponsor: Brigham and Women's Hospital (Academic / other)
• Locations: 7 sites (Los Angeles, California and 6 other locations)
• Trial ID: NCT06043440 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to evaluate the effects of oxygen supplementation during sleep on cognitive function and other health outcomes in children with Down Syndrome who have moderate to severe Obstructive Sleep Apnea. It is a randomized, single-blind study lasting six months, where participants will undergo various assessments including polysomnography, neurocognitive testing, and echocardiography. The study will recruit children from sleep clinics, Down syndrome clinics, and local associations, focusing on those who have not responded to standard treatments like adenotonsillectomy.

Who should consider this trial

Good fit: Ideal candidates are children aged 5 to 17.9 years with moderate to severe obstructive sleep apnea and a favorable response to oxygen therapy.

Not a fit: Patients who have clinically significant hypoxia or those who have already undergone adenotonsillectomy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could enhance cognitive function and overall quality of life for children with Down Syndrome suffering from sleep apnea.

How similar studies have performed: Other studies have explored oxygen therapy in similar contexts, but this specific approach in children with Down Syndrome and sleep apnea is relatively novel.

Eligibility criteria

Inclusion Criteria:

1. Ages 5.0 to 17.9 years at the time of screening
2. Children with OSA and obstructive apnea hypopnea index (OAHI) ≥5/hour.
3. Absence of clinically significant hypoxia defined as oxygen saturation \<88% for 5 minutes or episodic desaturation to 60% as these levels would otherwise identify children eligible to routinely receive oxygen.
4. Favorable response to oxygen therapy (allowing randomization) will be defined as follows:

   1. Oxygen saturation nadir \>92% and
   2. Decrease in obstructive index \< 5 / hour or by \> 50% from screening PSG
   3. Reaching an optimum oxygen flow which is defined as the flow that achieves the lowest level of AHI with maximum CO2 level less than 65 mmHg observed for 5 consecutive minutes and or an increase in CO2 by less than 15 points above baseline. The above criteria are observed while the patient spends a minimal of 30 minutes in the supine position and at least one cycle of rapid eye movement (REM) sleep.
   4. Oxygen flow required does not exceed 3.0 LPM and does not exceed a FiO2 \> 40 %.
5. Willingness to comply with all study procedures and available for duration of study.
6. At baseline the participant attempts to perform the neuropsychological tests

Exclusion Criteria:

1. Current CPAP use with documented compliance(\> 4 hrs/ night; \> 70% of nights).
2. Oxygen saturation \< 90% at rest during wakefulness.
3. Chronic daytime or nighttime use of supplemental oxygen.
4. Smoker in the child's bedroom.
5. Unrepaired congenital heart disease.
6. Moderate to severe pulmonary hypertension requiring treatment with oxygen and or pulmonary vasodilator.
7. Unable to participate in a PSG.
8. Individuals who develop alveolar hypoventilation with oxygen as previously defined.
9. Other severe chronic diseases determined by their provider as making them poor study candidates.
10. Enrolled or planning to enroll in another study that may conflict with protocol requirements or confound results in this trial.
11. Documented clinically significant untreated hypothyroidism
12. Children with adenotonsillar hypertrophy who are candidates for adenotonsillectomy and parents agree to the surgery.

Where this trial is running

Los Angeles, California and 6 other locations

• Children's Hospital of Los Angeles — Los Angeles, California, United States (Recruiting)
• University of Michigan, Ann Arbor Hospital — Ann Arbor, Michigan, United States (Recruiting)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (Recruiting)
• Rainbow Babies and Children's Hospital, Case Medical Center — Cleveland, Ohio, United States (Recruiting)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (Recruiting)
• East Virginia Medical Center — Norfolk, Virginia, United States (Recruiting)
• Seattle Children's Hospital — Seattle, Washington, United States (Recruiting)

Study contacts

• Principal investigator: Susan Redline — Brigham and Women's Hospital
• Study coordinator: Oltion Sina

Conditions Down Syndrome; Obstructive Sleep Apnea