Clinical trial NCT05881031: Home Initiation of Noninvasive Positive Pressure Ventilation in Children With Medical Complexity
Official title: Single Site Feasibility and Safety Study of Home Initiation of Noninvasive Positive Pressure Ventilation in Children With Medical Complexity
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: Sleep-Disordered Breathing
Keywords: noninvasive ventilation
home mechanical ventilation
Lead sponsor: The Hospital for Sick Children
Sponsor class: OTHER
First posted: 2023-05-30
Enrollment: 24
Sex: ALL
Minimum age (years): 60
Maximum age (years): 215
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: This will be a 12-week, open-label feasibility study of a pragmatic, two-arm parallel-group, randomized controlled trial with 1:1 allocation of children newly initiating NiPPV that compares home NiPPV initiation to usual care of initiating NiPPV in a one-night in-hospital polysomnogram.
Primary purpose: TREATMENT

Brief summary: 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.

Detailed description: Children with medical complexity (CMC) are increasingly prescribed noninvasive positive pressure ventilation (NiPPV) for chronic respiratory failure. In our clinical experience, patients and families would prefer NiPPV initiation in the home environment but this is not standard of care. This proposal will evaluate the feasibility and safety of an innovative model of care for NiPPV initiation in the home environment utilizing remote telemonitoring compared to usual care which is in-hospital polysomnography (PSG) laboratory-based initiation.

The incidence of CMC requiring NiPPV is exponentially growing. NiPPV effectively corrects abnormal gas exchange, improves sleep quality, and reduces symptoms of chronic respiratory failure. The use of NiPPV has been associated with increased survival and improved health-related quality of life (HRQOL). To ensure effectiveness, NiPPV must be used for all periods of sleep and settings must be individually-titrated. Unfortunately, poor adherence results in many CMC being undertreated for chronic respiratory failure. Successful NiPPV initiation is critical because early negative experiences are commonly reported barriers to adherence6 and early usage predicts longer term use. Standard of care is the initiation and titration of NiPPV during a one-night in-hospital PSG. A PSG is challenging for CMC and families due to their medical fragility and lasting effects of travel and a disrupted routine, the significant amount of equipment and supplies that must be brought to the study as well as the financial implications and additional psychosocial stress for caregivers. In addition, negative experiences with NiPPV may be exacerbated by introduction of the therapy in an unfamiliar PSG laboratory with burdensome monitoring. Furthermore, a one-night PSG provides limited sleep data that may not be representative of dynamic and variable sleeping patterns that occur in the comfort of one's home.

Recent technological advances in remote NiPPV monitoring coupled with a shift to virtual care models has enabled a patient and family centered opportunity to bypass the PSG laboratory and initiate NiPPV at home. Remote NiPPV monitoring extends data collection beyond discrete health care encounters and provides real-time data on adherence and efficacy that can be wirelessly transmitted to facilitate setting adjustment by clinicians. Home NiPPV initiation is increasingly being requested by patients and caregivers. The potential benefits of this innovative care model include increasing patient and family empowerment, improving the delivery of high quality patient-centred care at home, and optimizing NiPPV use. Although there is growing data in the adult population to support home NiPPV initiation via telemonitoring, this cannot be simply extrapolated to CMC. There are key differences in the etiology and manifestation of chronic respiratory failure in CMC compared to adults. Furthermore, family caregivers play an important role in NiPPV acceptance and adherence.

Canadian guidelines acknowledge the lack of evidence for the setting in which NiPPV is initiated. Home NiPPV initiation in CMC is a novel model of care that may result in large health system impacts, but requires a feasibility study before embarking on a large-scale trial.

Eligibility criteria: Inclusion Criteria:

1. Age 5-17 years old
2. Newly prescribed NiPPV
3. Tolerated awake NiPPV trial
4. Provides informed consent

Exclusion Criteria:

1. Cardiac disease at risk of hemodynamic instability with NiPPV initiation (eg cardiac dysfunction (ejection fraction \<45%), pulmonary hypertension (mean pulmonary artery pressure ≥ 20 mmHg on right heart catheterization or suggestive echocardiogram findings in the opinion of a pediatric cardiologist), or single ventricle)
2. At high risk of complications with NiPPV in the opinion of the child's physician (eg pneumothorax and aspiration risk)
3. Severe sleep disordered breathing with peak CO2 ≥ 60mmHg or apnea-hypopnea index (AHI)≥ 30/hr (AHI measures the number of respiratory events per hour)
4. Participation in concurrent research study that may affect NiPPV adherence (proposed primary outcome of full study)
5. Exclusion of study participants if the caregiver or participant is not English speaking

Primary outcomes:
measure: Recruitment and retention rate
timeFrame: 2 years
measure: Proportion of eligible patients randomized
timeFrame: 2 years
measure: Proportion of patients that crossover based on safety criteria
timeFrame: 2 years
Secondary outcomes:
measure: Mean minutes of nightly NiPPV usage
timeFrame: 12 weeks
measure: Proxy-reported Psychological Scale (percentage rank)
timeFrame: 12 weeks
measure: Proxy-reported Psychological Scale (percentage rank)
timeFrame: 4 weeks
measure: Proxy-reported Psychological Scale (T-value)
timeFrame: 12 weeks
measure: Proxy-reported Psychological Scale (T-value)
timeFrame: 4 weeks
measure: Self-Reported Psychological Scale (percentage rank)
timeFrame: 12 weeks
measure: Self-Reported Psychological Scale (percentage rank)
timeFrame: 4 weeks
measure: Self-Reported Psychological Scale (T-value)
timeFrame: 12 weeks
measure: Self-Reported Psychological Scale (T-value)
timeFrame: 4weeks
measure: Caregiver reported Sense of Mastery
timeFrame: 12 weeks
measure: Caregiver reported Sense of Mastery
timeFrame: 4 weeks
measure: Patient and family study experience, preference of intervention and barriers
timeFrame: 12 weeks
measure: Difference in expiratory positive airway pressure based on home NiPPV titration and PSG titration in the intervention arm
timeFrame: 12 weeks
measure: Difference in inspiratory positive airway pressure based on home NiPPV titration and PSG titration in the intervention arm
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
Study officials:
affiliation: The Hospital for Sick Children
name: Reshma Amin, MD
role: PRINCIPAL_INVESTIGATOR
References: