Pilot Study of a Self-Supporting Nasopharyngeal Airway in Hypotonia

Study Overview
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Brief Summary
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Children with hypotonic upper airway obstruction have a high prevalence of severe obstructive sleep apnea, which if not treated has significant clinical consequence. Available treatment approaches, such as surgery and positive airway pressure, show limited efficacy and adherence. The multidisciplinary team has developed and now proposes to further test a non-surgical, well-tolerated nasopharyngeal airway device that in initial patients has resolved even extremely severe obstructive sleep apnea, and improved patient and family quality of life.

Detailed Description
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There is a critical need for safe and effective treatment options for persistent obstructive sleep apnea (OSA) in patients with Hypotonic Upper Airway Obstruction (HUAO). HUAO encompass conditions such as cerebral palsy, hypoxic encephalopathy, syndromic tone anomalies, and neuromuscular disorders, and typically share a similar pattern of multisite upper airway collapse. OSA is characterized by recurrent episodes of partial or complete upper airway obstruction during sleep with associated arousals and/ or oxygen desaturations. Of hypotonic patients with symptoms of sleep disordered breathing, one quarter have moderate OSA, and more than half have severe OSA. Thus, not only are patients more likely to have OSA, but it is likely to be much more severe. Currently available treatment options, ranging from palliative care to tracheostomy, often fail to fully meet the needs of these patients. The multidisciplinary team has developed a dramatically effective non-surgical nasopharyngeal airway stent that has demonstrated good tolerability in hypotonic patients. This initial phase will test an enhanced version of the device for acceptability and tolerability. Critically, insertion, adherence, and compliance protocols will be optimized for preparation of the full trial. This record originally included the Secondary Outcome Measure Device Design. This Secondary Outcome Measure was removed during the results reporting process upon advisement of ClinicalTrials.gov reviewers, as it was not a directly health-related outcome.

Official Title
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Pilot Study of Novel Nasopharyngeal Airway Device for Treating Upper Airway Obstruction in Pediatric Hypotonia

Conditions
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Sleep Apnea, Obstructive, Hypotonia, Child, Only

Intervention / Treatment
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* Device: ssNPA


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children with HUAO: This includes those who newly diagnosed with OSA. These children will undergo overnight polysomnography to determine the presence of OSA (AHI>10 or AHI>5 with nocturnal hypoxemia defined as SpO2 nadir <=75%). Obstructive sleep apnea on polysomnogram with AHI>=10 Presence of at least one symptom of OSA (such as snoring 3 or more nights per week, daytime sleepiness, or hyperactive/inattentive behaviors) Post adeonotonsillectomy or those with contraindications to tonsillectomy. obstructive sleep apnea on polysomnogram with AHI>=10 Tonsil size 2+ or smaller Parent/caregivers willing and able to provide informed consent and child willing and able to provide assent, where appropriate. Exclusion Criteria: AHI ≤10 on polysomnogram without hypoxemia or AHI<5 with hypoxemia - any reason why ssNPA may not be suitable Any medical reason why ssNPA therapy may not be suitable Active COVID 19 infections ETCO2 or TCO2 values >60 mmHg for >10% of sleep time on PSG Psychiatric, medical, or social factors likely to invalidate assessments, make adherence with ssNPA highly unlikely or make local follow-up at 8 weeks unfeasible. Some psychiatric conditions may be provoked or exacerbated by OSA, and those most commonly implicated - Attention Deficit/Hyperactivity Disorder, Conduct Disorder, and Oppositional Defiant Disorder - will not be exclusions. However, more pervasive conditions such as severe autism will be excluded Presence of supraglottic airway collapse or more distal airway stenosis or collapse (for example glottic, subglottic stenosis, or concern for distal airway stenosis or malacia) Moderate/severe tracheobroncomalacia Need for anticoagulative therapy Bleeding disorder Restrictive thoracic disorders

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ssNPA<br>self-supporting nasopharyngeal airway to be used nightly for approximately 8 weeks. | Device: ssNPA<br>* The ssNPA is a self-supporting nasopharyngeal airway, a stent that is a non-surgical alternative to treat severe OSA<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Ability to Tolerate Measured by Percent of Participants With Protocol Goal | Protocol objective was to achieve a score of >=7 in at least 60% of participants. Score of >=7 on Likert scale 0-10 (higher score is better). | 8 weeks | 
| Comfort as Measured by Percent of Participants With Protocol Objective Score | Protocol objective was to achieve a score of >=6 in at least 60% of participants. Score of >=6 on Likert scale 0-10 (higher score is better). | 8 weeks | 
| Ease of Use as Indicated by Percent of Participants Whose Parents Responded Favorably to Device Use, With Protocol Goal | Score of >=5 on Likert scale 1-10 (higher score is better). | 8 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Snoring | Less frequent snoring, as measured by scores of 1 or 2 on a question that read: While using the device my child's snoring occurred: 1 much less often; 2: less often; 3: unchanged; 4: more often; 5: much more | 8 weeks | 
| Change in Daytime Sleepiness as Shown by Number of Participants Who Had a Reduction in Their Epworth Sleepiness Scale Score | Reduction in Epworth Sleepiness Scale score (0-24; lower score is better); | 8 weeks | 
| Sleep Quality, as Measured by Number of Participants With Parent Identified Improved Sleep | Number of participants whose parents' response on the sleep quality improvement question were >=7. Change in sleep quality was reported on a 1-10 scale, where 1 was greatest worsening of sleep and 10 was most improved sleep. | 8 weeks | 
| Insertion Protocol Optimization as Measured by Percent of Participants Who Found Insertion Relatively Easy | Percent of participants who reported a score of <= 5 on Likert scale of 1-10, where lower score indicates easier insertion. | 8 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Obstructive sleep apnea, Hypotonia, Nasal airway, Tolerability