Home-Initiated NiPPV for Sleep Apnea

Overseen By Reshma Amin, MD

Age: < 18

Sex: Any

Trial Phase: Academic

Sponsor: The Hospital for Sick Children

Disqualifiers: Cardiac disease, Severe sleep apnea, others

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

Approved in 3 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 12 weeks

24 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new method for initiating the use of a special breathing machine, called NiPPV (Noninvasive Positive Pressure Ventilation), at home for children with complex medical needs who struggle to breathe at night. Typically, settings for this machine are adjusted during an overnight hospital stay. However, this trial tests whether starting the machine at home is equally safe and effective. Children aged 5-17 who have been newly prescribed NiPPV and have tried it while awake are suitable candidates for this study. The goal is to determine if starting the machine at home can improve comfort and quality of life for children and their families. As an unphased trial, this study provides families the chance to contribute to research that could simplify and enhance home care for children.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What prior data suggests that home initiation of NiPPV is safe for children with medical complexity?

Research shows that using a home breathing machine, known as noninvasive positive pressure ventilation (NiPPV), is generally safe. Studies have found that using NiPPV at home can reduce hospital visits and improve survival rates for people with conditions like COPD, a lung disease. For those with COPD, using a home breathing machine led to fewer hospital stays and less need for emergency care compared to not using one.

Although researchers conducted these studies with adults, they provide a good understanding of the safety of using NiPPV at home. Remote monitoring and regular check-ins with healthcare providers help adjust the machine settings over time to ensure comfort and effectiveness.

Since hospitals already use NiPPV, starting it at home aims to make life easier without sacrificing safety. Overall, current research supports that home NiPPV is well-tolerated, with few serious side effects reported. ^{1 2 3 4 5}

Why are researchers excited about this trial?

Researchers are excited about the Home Initiation of NiPPV for sleep apnea because it offers a more convenient and potentially more comfortable alternative to standard hospital-based initiation. Unlike the traditional method, which requires an overnight stay at a hospital for setting adjustments, this approach allows patients to start and adjust their therapy at home using remote telemonitoring and home overnight oximetries. This method not only saves time but may also better accommodate the patient's lifestyle, possibly improving adherence and comfort with the treatment.

What evidence suggests that home initiation of NiPPV is effective for sleep apnea?

This trial will compare two methods for initiating noninvasive positive pressure ventilation (NiPPV) for sleep apnea. Research has shown that using a home device like NiPPV can improve health outcomes compared to not using it. NiPPV assists breathing by gently pushing air through a mask. Studies have found that using NiPPV at home correlates with lower death rates in certain conditions, such as COPD, a lung disease. This suggests that starting NiPPV at home might be safe and beneficial for children with complex medical needs. Although more research is needed on home initiation, the potential benefits make it an option worth considering. ^{1 2 5 6 7}

Who Is on the Research Team?

Reshma Amin, MD

Principal Investigator

The Hospital for Sick Children

Are You a Good Fit for This Trial?

This trial is for children aged 5-17 with complex medical needs who have trouble breathing at night and are starting to use a NiPPV breathing machine. They must be able to tolerate an awake NiPPV trial, provide informed consent, and not be at high risk of complications or involved in other studies that could affect the outcome.

Inclusion Criteria

I am between 5 and 17 years old.

I have been newly prescribed non-invasive positive pressure ventilation.

I have successfully used a breathing support machine while awake.

Exclusion Criteria

I have heart issues that could worsen with certain breathing treatments.

Your doctor thinks using a specific type of breathing support might be risky for you, such as causing lung problems or breathing in food or liquid.

You are currently taking part in another study that may affect how well you use the NiPPV machine.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants are assigned to start NiPPV either at home or in the sleep laboratory, with settings titrated over time.

12 weeks

Visits at 1, 4, and 12 weeks for titration

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

In-hospital PSG

Participants in the home initiation group complete an in-hospital PSG after 12 weeks for final titration.

1 night

What Are the Treatments Tested in This Trial?

Interventions

• Home Initiation of NiPPV

Trial Overview The study tests initiating noninvasive positive pressure ventilation (NiPPV) at home versus in a sleep lab for children with medical complexity. It aims to determine if starting treatment at home is feasible and safe by randomly assigning participants to either setting.

How Is the Trial Designed?

Group I: Home Initiation of NiPPV Experimental Treatment 1 Intervention

NiPPV settings will be titrated during an awake NiPPV trial by the respiratory therapist. Once NiPPV equipment is obtained and NiPPV is used at home (time 0), further titration of the NiPPV settings will occur on an outpatient basis guided by remote telemonitoring and home overnight oximetries at 1, 4, and 12 weeks. Additional titration of NiPPV settings may be guided by reported participant symptoms and comfort, as per standard of care. Participants will complete an in-hospital PSG after 12 weeks, during which NiPPV settings are titrated during the night by a PSG technologist to determine optimal settings according to current international guidelines.

Group II: Standard of care Active Control 1 Intervention

Standard of care is NiPPV initiation during a one-night, in-hospital PSG, during which NiPPV settings are titrated during the night by a PSG technologist to determine optimal settings according to current international guidelines. Once NiPPV equipment is obtained and NiPPV is used at home (time 0), NiPPV setting changes may occur at 1, 4, and 12 weeks when participants are contacted by the respiratory therapist. Additional titration of NiPPV settings may be guided by reported participant symptoms and comfort, as per standard of care.

Home Initiation of NiPPV is already approved in European Union, United States, Canada for the following indications:

Approved in European Union as Noninvasive Positive Pressure Ventilation for:

• Chronic respiratory failure
• Neuromuscular diseases
• Severe thoracic cage abnormalities
• Obstructive sleep apnea

Approved in United States as Noninvasive Positive Pressure Ventilation for:

• Restrictive thoracic disorders
• Severe chronic obstructive pulmonary disease
• Central sleep apnea
• Complex sleep apnea
• Hypoventilation syndrome

Approved in Canada as Noninvasive Positive Pressure Ventilation for:

• Similar to those in the European Union and United States, specific indications may vary by province

Who Is Running the Clinical Trial?

The Hospital for Sick Children

Lead Sponsor

Trials

724

Recruited

6,969,000+

Published Research Related to This Trial

Nasal intermittent positive pressure ventilation (NIPPV) offers a noninvasive method to enhance respiratory support in neonates, showing advantages over traditional nasal continuous positive airway pressure (nCPAP) in both primary and post-extubation settings.

NIPPV has been found to effectively reduce severe apnea in premature infants, indicating its potential as a valuable option for neonatal respiratory care.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Research advances in neonatal nasal intermittent positive pressure ventilation]. Zhu, ZC., Zhou, JG., Chen, C.[2023]

In a study of 66 COPD patients over two years, adding noninvasive intermittent positive pressure ventilation (NIPPV) to pulmonary rehabilitation significantly improved health-related quality of life (HRQoL) and several other health metrics, despite not showing a significant difference in the Chronic Respiratory Questionnaire scores.

The addition of NIPPV also led to improvements in mood, dyspnea, gas exchange, exercise tolerance, and lung function, indicating that NIPPV can enhance the overall treatment outcomes for patients with chronic hypercapnic respiratory failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Two-year home-based nocturnal noninvasive ventilation added to rehabilitation in chronic obstructive pulmonary disease patients: a randomized controlled trial. Duiverman, ML., Wempe, JB., Bladder, G., et al.[2022]

Nasal intermittent positive pressure ventilation (NIPPV), specifically synchronized NIPPV (SNIPPV), significantly reduces the rate of extubation failure in preterm infants compared to nasal continuous positive airway pressure (NCPAP), with a relative risk of 0.21 and a number needed to treat of 3.

While NIPPV shows promise in reducing the frequency of apnoea of prematurity, further research is needed to fully understand its effectiveness in this area, as current studies indicate a trend favoring NIPPV but do not provide conclusive meta-analysis results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nasal continuous positive airway pressure versus nasal intermittent positive pressure ventilation for preterm neonates: a systematic review and meta-analysis. De Paoli, AG., Davis, PG., Lemyre, B.[2019]

In a study involving 8 healthy subjects, two-level nasal intermittent positive pressure ventilation (nIPPV) in controlled mode significantly increased effective minute ventilation (VE) compared to spontaneous unassisted breathing, especially during sleep.

The study suggests that using controlled mode with an inspiratory positive airway pressure (IPAP) of 15 cm H2O and a frequency of around 20 breaths per minute is more effective for increasing VE than using spontaneous mode, which showed less improvement and more respiratory rhythm instability.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effectiveness of controlled and spontaneous modes in nasal two-level positive pressure ventilation in awake and asleep normal subjects. Parreira, VF., Delguste, P., Jounieaux, V., et al.[2019]

In a study of 13 severely obese patients with obstructive sleep apnea who did not respond to CPAP, nocturnal nasal positive pressure ventilation (NIPPV) significantly improved daytime blood gas levels, reducing CO2 levels and increasing oxygen tension after just 7 to 18 days of treatment.

Short-term NIPPV not only enhanced overall ventilation but also allowed 9 out of 13 patients to successfully transition back to CPAP therapy, suggesting it can be an effective interim treatment for those with severe hypercapnic OSA who struggle with standard CPAP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Effects of short-term NIPPV in the treatment of patients with severe obstructive sleep apnea and hypercapnia. Piper, AJ., Sullivan, CE.[2019]

In a study of 55 preterm neonates with CPAP failure, 74% responded positively to nasal intermittent positive pressure ventilation (NIPPV), avoiding the need for intubation and mechanical ventilation.

NIPPV also resulted in a shorter duration of oxygen therapy (7.77 days) compared to conventional ventilation (9.28 days), suggesting it may be a more effective treatment option for these infants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Noninvasive positive pressure ventilation or conventional mechanical ventilation for neonatal continuous positive airway pressure failure. Badiee, Z., Nekooie, B., Mohammadizadeh, M.[2022]

In a study of 100 neonates with neonatal respiratory distress syndrome (NRDS), nasal intermittent positive pressure ventilation (NIPPV) was found to be more effective than nasal continuous positive airway pressure (NCPAP) in reducing CO2 retention and improving oxygenation within the first 6 hours of treatment.

NIPPV also resulted in significantly lower rates of second endotracheal intubation and reduced the need for additional pulmonary surfactant, indicating its potential to improve overall respiratory support outcomes in neonates with NRDS.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical application of nasal intermittent positive pressure ventilation in initial treatment of neonatal respiratory distress syndrome]. Fu, CH., Xia, SW.[2014]

Nasal intermittent positive pressure ventilation (NIPPV) significantly reduces the need for endotracheal ventilation and increases the success rate of extubation in neonates, based on a review of 14 randomized controlled trials involving 1052 infants.

NIPPV also decreases the incidence of apnea of prematurity and shows better outcomes, such as reduced rates of death and bronchopulmonary dysplasia, with no reported side effects associated with its use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nasal intermittent positive pressure ventilation versus nasal continuous positive airway pressure in neonates: a systematic review and meta-analysis. Tang, S., Zhao, J., Shen, J., et al.[2019]

In a trial involving 22 patients with difficulties weaning off conventional ventilation, 18 successfully transitioned to nasal intermittent positive pressure ventilation (NIPPV), allowing them to be discharged home after a median of 11 days.

NIPPV not only helped facilitate spontaneous breathing but also reduced the need for intensive care, with 16 patients remaining well after discharge, and 10 continuing NIPPV at home, demonstrating its efficacy in managing chronic respiratory insufficiency.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Nasal ventilation to facilitate weaning in patients with chronic respiratory insufficiency. Udwadia, ZF., Santis, GK., Steven, MH., et al.[2019]

In a study of 40 patients with acute respiratory failure, noninvasive positive pressure ventilation (NIPPV) was successful in 85% of cases, significantly improving blood pH and carbon dioxide levels shortly after treatment initiation.

NIPPV effectively reduced the need for invasive mechanical ventilation in a developing country, showing results comparable to those seen in developed nations, which suggests it could be a valuable resource-saving intervention in settings with limited medical resources.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An evaluation of the role of noninvasive positive pressure ventilation in the management of acute respiratory failure in a developing country. George, IA., John, G., John, P., et al.[2022]

Bilevel positive airway pressure (BPAP) devices have been developed to effectively treat complex sleep-related breathing disorders that do not respond to traditional CPAP therapy, enhancing patient care for conditions like obstructive sleep apnea (OSA).

Advanced BPAP therapies, including adaptive servoventilation and volume-assured pressure support, provide tailored noninvasive ventilation options to address various hypoventilation disorders and central sleep apnea syndromes, showcasing their versatility in managing complex respiratory issues.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Sleep-Related Breathing Disorders: When CPAP Is Not Enough. Selim, B., Ramar, K.[2022]

In a study involving six patients with chronic respiratory failure, nasal intermittent positive pressure ventilation (NIPPV) significantly improved arterial blood gas levels, reducing carbon dioxide (PaCO2) from an average of 68.3 Torr to 55.8 Torr and increasing oxygen saturation (SaO2) from 91.6% to 95.5%.

NIPPV was safely administered at home for durations ranging from 2 to 24 months without any hospitalizations due to exacerbations, demonstrating its effectiveness as a long-term, noninvasive treatment option for patients with respiratory failure.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Long-term artificial ventilation by nasal intermittent positive pressure ventilation; 6 cases of domiciliary assisted ventilation]. Sekino, H., Ohi, M., Chin, K., et al.[2006]

Citations

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Results - Noninvasive Positive Pressure Ventilation in ... - NCBI The introduction of non-invasive ventilation in the hospital can take up to two weeks. Respiratory Services (KQ4). We did not identify guidelines that provided ...

2. jamanetwork.com jamanetwork.com/journals/jama/fullarticle/2760390

Association of Home Noninvasive Positive Pressure ... Home NIPPV, compared with no device use, was significantly associated with better clinical outcomes and no significant difference in quality of life.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT05881031

NCT05881031 | Home Initiation of Noninvasive Positive ... This study will be the first step towards developing a study to evaluate if home NiPPV starts are effective. Starting NiPPV at home has the potential to improve ...

4. cms.gov cms.gov/Medicare/Coverage/DeterminationProcess/downloads/id108TA.pdf

Noninvasive Positive Pressure Ventilation in the Home Key messages. • In patients with COPD, home NIPPV as delivered by a BPAP device (compared to no device) was associated with lower mortality, ...

5. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC7042860/

Association of Home Noninvasive Positive Pressure ... Among patients with COPD and hypercapnia, home NIPPV, compared with no device use, was significantly associated with better clinical outcomes and no significant ...

6. ncbi.nlm.nih.gov ncbi.nlm.nih.gov/books/NBK575995/

Discussion - Noninvasive Positive Pressure Ventilation in the ... In patients with COPD, home BPAP (compared to no device) was associated with lower mortality, intubations, hospital admissions, and dyspnea. There was no change ...

7. cms.gov cms.gov/medicare-coverage-database/view/ncacal-decision-memo.aspx?proposed=N&ncaid=315

Noninvasive Positive Pressure Ventilation (NIPPV) in the ... The outcomes of interest included decreased mortality, decreased frequency of exacerbations requiring emergency room or hospital admission, increased time to ...