Home-Initiated NiPPV for Sleep Apnea

Recruiting in Palo Alto (17 mi)

Age: < 18

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Academic

Recruiting

Sponsor: The Hospital for Sick Children

Disqualifiers: Cardiac disease, Severe sleep apnea, others

No Placebo Group

Approved in 3 Jurisdictions

Trial Summary

What is the purpose of this trial?

Children with medical complexity (CMC) often have trouble breathing at night and need to use a breathing machine. This breathing machine is called noninvasive positive pressure ventilation (NiPPV). The use of NiPPV has been shown to improve quality of life and survival in children. Before it is used, NiPPV must first be tested to see what the correct 'machine settings' are for each child. This is usually done in the sleep laboratory at the hospital during a one-night stay. However, sleep studies in the hospital are disruptive and hard for CMC and their families because of the new environment and limited access to the equipment, supplies, comfort items and the routine their child has at home. Patients and families would prefer to start NiPPV at home but there needs to be more research on this to make sure it is possible and safe. This study will evaluate a new model of care to start NiPPV in the home. CMC aged 5-17 years old and starting NiPPV will be assigned at random, like a coin toss, to start NiPPV in the home or to start NiPPV in the sleep laboratory. The investigators will assess the feasibility and safety of the two ways to start NiPPV. 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 the use of NiPPV (ie early adherence predicts long-term use) resulting in both medical benefits as well as improved quality of life for CMC and their families.

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 data supports the effectiveness of the treatment Home Initiation of NiPPV for Sleep Apnea?

Research shows that noninvasive positive pressure ventilation (NIPPV) can help manage complex sleep-related breathing disorders and chronic respiratory insufficiency, suggesting it may be effective for sleep apnea. Bilevel positive airway pressure (BIPAP), a form of NIPPV, is used when continuous positive airway pressure (CPAP) is not enough, indicating its potential benefit for sleep apnea treatment. ^{1 2 3 4 5}

Is home-initiated noninvasive positive pressure ventilation (NiPPV) generally safe for humans?

Research shows that noninvasive positive pressure ventilation (NIPPV) has been studied for safety in both newborns and adults with conditions like respiratory distress and chronic obstructive pulmonary disease (COPD). These studies suggest that NIPPV is generally safe for use in humans. ^{3 6 7 8 9}

How does the treatment Home Initiation of NiPPV for Sleep Apnea differ from other treatments for this condition?

Home Initiation of NiPPV (Noninvasive Positive Pressure Ventilation) is unique because it provides a noninvasive way to support breathing by delivering air pressure through a mask, which can be more effective for patients who do not respond well to standard CPAP (Continuous Positive Airway Pressure) therapy. It adjusts the pressure during inhalation and exhalation, making it suitable for complex cases of sleep apnea and other breathing disorders. ^{1 2 10 11 12}

Research Team

Eligibility Criteria

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.

I or my caregiver can communicate in English.

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.

Trial Timeline

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

Treatment Details

Interventions

• Home Initiation of NiPPV (Behavioural Intervention)

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.

Participant Groups

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 Canada for the following indications:

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+

Dr. Ronald D. Cohn

The Hospital for Sick Children

Chief Executive Officer since 2019

MD from University of Düsseldorf, Germany

Dr. Lennox Huang

The Hospital for Sick Children

Chief Medical Officer since 2016

MD from McGill University

Findings from Research

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 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]

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 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]

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]

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) 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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Sleep-Related Breathing Disorders: When CPAP Is Not Enough. [2022]

2. England pubmed.ncbi.nlm.nih.gov

Nasal ventilation to facilitate weaning in patients with chronic respiratory insufficiency. [2019]

3. Norway 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. [2019]

4. United States pubmed.ncbi.nlm.nih.gov

Effectiveness of controlled and spontaneous modes in nasal two-level positive pressure ventilation in awake and asleep normal subjects. [2019]

5. India 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. [2022]

6. Iran pubmed.ncbi.nlm.nih.gov

Noninvasive positive pressure ventilation or conventional mechanical ventilation for neonatal continuous positive airway pressure failure. [2022]

7. China pubmed.ncbi.nlm.nih.gov

[Clinical application of nasal intermittent positive pressure ventilation in initial treatment of neonatal respiratory distress syndrome]. [2014]

8. England 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. [2022]

9. India pubmed.ncbi.nlm.nih.gov

Nasal intermittent positive pressure ventilation versus nasal continuous positive airway pressure in neonates: a systematic review and meta-analysis. [2019]

10. China pubmed.ncbi.nlm.nih.gov

[Research advances in neonatal nasal intermittent positive pressure ventilation]. [2023]

11. United States pubmed.ncbi.nlm.nih.gov

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

12. Japan pubmed.ncbi.nlm.nih.gov

[Long-term artificial ventilation by nasal intermittent positive pressure ventilation; 6 cases of domiciliary assisted ventilation]. [2006]