Pilot Study of a Self-Supporting Nasopharyngeal Airway in Hypotonia (ssNPA)

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

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.

Study Overview

• Status: Completed
• Conditions: Sleep Apnea, Obstructive; Child, Only; Hypotonia
• Intervention / Treatment: Device: ssNPA

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 2
• Phase: Not Applicable

Contacts and Locations

Study Locations

• United States
  • Michigan
    • Ann Arbor, Michigan, United States, 48109
      • C.S. Mott Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 3 years to 21 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: University of Michigan
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health (NIH)
• Investigators: Principal Investigator: David A Zopf, MD, University of Michigan

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2021
• Primary Completion (Actual): August 30, 2022
• Study Completion (Actual): August 30, 2022

Study Registration Dates

• First Submitted: April 13, 2021
• First Submitted That Met QC Criteria: April 13, 2021
• First Posted (Actual): April 15, 2021

Study Record Updates

• Last Update Posted (Actual): December 28, 2023
• Last Update Submitted That Met QC Criteria: December 13, 2023
• Last Verified: December 1, 2023

More Information

Terms related to this study

• Keywords: Obstructive sleep apnea; Tolerability; Hypotonia; Nasal airway
• Additional Relevant MeSH Terms: Nervous System Diseases; Respiratory Tract Diseases; Apnea; Respiration Disorders; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Neurologic Manifestations; Neuromuscular Manifestations; Sleep Apnea Syndromes; Sleep Apnea, Obstructive; Muscle Hypotonia
• Other Study ID Numbers: 1R61HL151952 (U.S. NIH Grant/Contract); R61HL151952 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: For this initial pilot of 5 participants there are no plans to share data.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: Yes