Home Initiation of Noninvasive Positive Pressure Ventilation in Children With Medical Complexity

NCT05881031 | Recruiting

• 1 other identifier: 1000080480
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Sleep-Disordered Breathing

Keywords

noninvasive ventilation; home mechanical ventilation

Outcome Measures

Primary Outcomes (3)

• Recruitment and retention rate

  Feasibility outcome. The recruitment rate is the proportion of eligible people recruited to the trial. This is the number of people randomised divided by the number of people eligible. The retention rate is the proportion of randomised people who complete follow up at 3 months (end of trial).

  2 years

• Proportion of eligible patients randomized

  Feasibility outcome. Proportion of eligible participants who are recruited to the study and randomized

  2 years

• Proportion of patients that crossover based on safety criteria

  Safety outcome. Proportion of patients in the intervention arm who crossover to the control arm based on pre-specified monitoring criteria

  2 years

Secondary Outcomes (14)

• Mean minutes of nightly NiPPV usage

  12 weeks

• Proxy-reported Psychological Scale (percentage rank)

  12 weeks

• Proxy-reported Psychological Scale (percentage rank)

  4 weeks

• Proxy-reported Psychological Scale (T-value)

  12 weeks

• Proxy-reported Psychological Scale (T-value)

  4 weeks

Study Arms (2)

Standard of care

NO 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

EXPERIMENTAL

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.

Other: Home Initiation of NiPPV

Interventions

Home Initiation of NiPPV OTHER

Initiation of NiPPV in the home environment through the utilization of an awake NiPPV titration, oximetries, and telemonitoring.

Home Initiation of NiPPV

Eligibility Criteria

• Age: 60 Months - 215 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 5-17 years old
• Newly prescribed NiPPV
• Tolerated awake NiPPV trial
• Provides informed consent

You may not qualify if:

• 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)
• At high risk of complications with NiPPV in the opinion of the child's physician (eg pneumothorax and aspiration risk)
• Severe sleep disordered breathing with peak CO2 ≥ 60mmHg or apnea-hypopnea index (AHI)≥ 30/hr (AHI measures the number of respiratory events per hour)
• Participation in concurrent research study that may affect NiPPV adherence (proposed primary outcome of full study)

Sponsors & Collaborators

• The Hospital for Sick Children: lead

Study Sites (1)

The Hospital for Sick Children

Toronto, Ontario, M5G1X8, Canada

RECRUITING

MeSH Terms

Conditions

Sleep Apnea Syndromes

Condition Hierarchy (Ancestors)

Apnea; Respiration Disorders; Respiratory Tract Diseases; Sleep Disorders, Intrinsic; Dyssomnias; Sleep Wake Disorders; Nervous System Diseases

Study Officials

• Reshma Amin, MD

  The Hospital for Sick Children

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Reshma Amin, MD

CONTACT

(416) 813-7654; Reshma.Amin@sickkids.ca

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Model Details: 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.

Study Record Dates

• First Submitted: May 1, 2023
• First Posted: May 30, 2023
• Study Start: June 21, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2027
• Last Updated: May 7, 2026

Record last verified: 2026-05

Locations

CA (1)