Child, Only, Hypotonia, Sleep Apnea, Obstructive
Conditions
Keywords
Obstructive sleep apnea, Hypotonia, Nasal airway, Tolerability
Brief summary
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
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.
Interventions
DEVICEssNPA
The ssNPA is a self-supporting nasopharyngeal airway, a stent that is a non-surgical alternative to treat severe OSA
Sponsors
National Institutes of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
University of Michigan
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 21 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Ability to Tolerate Measured by Percent of Participants With Protocol Goal | 8 weeks | 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). |
| Comfort as Measured by Percent of Participants With Protocol Objective Score | 8 weeks | 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). |
| Ease of Use as Indicated by Percent of Participants Whose Parents Responded Favorably to Device Use, With Protocol Goal | 8 weeks | Score of \>=5 on Likert scale 1-10 (higher score is better). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Snoring | 8 weeks | 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 |
| Insertion Protocol Optimization as Measured by Percent of Participants Who Found Insertion Relatively Easy | 8 weeks | Percent of participants who reported a score of \<= 5 on Likert scale of 1-10, where lower score indicates easier insertion. |
| Change in Daytime Sleepiness as Shown by Number of Participants Who Had a Reduction in Their Epworth Sleepiness Scale Score | 8 weeks | Reduction in Epworth Sleepiness Scale score (0-24; lower score is better); |
| Sleep Quality, as Measured by Number of Participants With Parent Identified Improved Sleep | 8 weeks | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| ssNPA self-supporting nasopharyngeal airway to be used nightly for approximately 8 weeks.
ssNPA: The ssNPA is a self-supporting nasopharyngeal airway, a stent that is a non-surgical alternative to treat severe OSA | 2 |
| Total | 2 |
Baseline characteristics
| Characteristic | ssNPA |
|---|---|
| Age, Categorical <=18 years | 2 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 2 Participants |
| Region of Enrollment United States | 2 participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
Primary
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).
Time frame: 8 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ssNPA | Ability to Tolerate Measured by Percent of Participants With Protocol Goal | 50 percent of participants |
Primary
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).
Time frame: 8 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ssNPA | Comfort as Measured by Percent of Participants With Protocol Objective Score | 100 percent of participants |
Primary
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).
Time frame: 8 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ssNPA | Ease of Use as Indicated by Percent of Participants Whose Parents Responded Favorably to Device Use, With Protocol Goal | 100 percent of participants |
Secondary
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);
Time frame: 8 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ssNPA | Change in Daytime Sleepiness as Shown by Number of Participants Who Had a Reduction in Their Epworth Sleepiness Scale Score | 1 Participants |
Secondary
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.
Time frame: 8 weeks
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| ssNPA | Insertion Protocol Optimization as Measured by Percent of Participants Who Found Insertion Relatively Easy | 100 percent of participants |
Secondary
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.
Time frame: 8 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| ssNPA | Sleep Quality, as Measured by Number of Participants With Parent Identified Improved Sleep | 2 Participants |
Secondary
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
Time frame: 8 weeks
Population: One participant was an inconsistent user of the device, therefore 8 week survey data is not available. Since Clinicaltrials.gov's purpose is not to show individual level results, the results for the consistent user are not shown here.