Clinical trial NCT05382494: Intranasal Steroid as Medical Therapy For Sleep-Disordered Breathing in Children
Official title: Efficacy of Intranasal Steroid for Children With Sleep-Disordered Breathing Non-Responsive to Initial Treatment With Intranasal Saline: A Randomized Trial
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE4
Conditions: Sleep Disorder; Breathing-Related
Snoring
Obstructive Sleep Apnea of Child
Sleep Disorders in Children
Tonsillar Hypertrophy
Adenoidal Disorder
Lead sponsor: Murdoch Childrens Research Institute
Sponsor class: OTHER
Collaborators: name: Monash Health
name: Royal Children's Hospital
First posted: 2022-05-19
Enrollment: 130
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Multicentre, double-blind, randomised control trial
Primary purpose: TREATMENT

Brief summary: MIST+ is studying a nasal spray to see if it will reduce the need for surgery for snoring. Children aged 3-12 are invited to take part. Snoring affects up to 10% of children and can cause sleeping problems and concentration or behavioural issues in the daytime. Currently the most common treatment for snoring is surgery to remove the tonsils and/or adenoids, however many children wait a long time to see a specialist. This research is trying to find if nasal sprays can help children with snoring, and whether this can reduce the need for surgery.

Detailed description: MIST+ is a multi centre, double-blind, placebo controlled trial. Children 3-12 years of age, who do not respond to a run-in phase of 6 weeks of normal saline intranasal spray to treat sleep disordered breathing, will be randomised 1:1 to a treatment phase of either intranasal corticosteroid (investigational product) or normal saline (placebo). Participants will receive treatment for 6 weeks and receive follow up at at 12 weeks, 6, and 12 months.

Eligibility criteria: Inclusion Criteria:

Each participant must meet all of the following criteria to be enrolled in this trial:

* Is between the ages of 3 and 12 years inclusive at the time of randomisation
* Has symptoms of Sleep Disordered Breathing (SDB) as determined by a Brouillette score ≥ -1 on telehealth/phone screening
* Has a legally acceptable representative capable of understanding the informed consent document and providing consent on the participant's behalf.

Exclusion Criteria:

Participants meeting any of the following criteria will be excluded from the study:

* Has a BMI over the 97th centile for age and gender
* Has a history of tonsillectomy and/or adenoidectomy
* Has a prior diagnosis of craniofacial, neuromuscular, syndromic or defined genetic disorders
* Has a history of haemorrhagic diathesis or recurrent (daily) or severe epistaxis
* Has a history of nasal surgery or trauma which has not fully healed
* Has active tonsillitis or nasal infection (must be resolved prior to randomisation)
* Is assessed to have stertor (snoring) while awake at rest
* Has a known hypersensitivity to the study drug or its formulation
* Has used oral, intravenous, or intranasal steroids in the past 6 weeks. (Inhaled steroids for asthma will be allowed concomitantly during the study)
* Daily use of antihistamine or decongestant nasal sprays
* Is known to require systemic steroids prior to the completion of the study treatment phase
* Has had treatment with any other investigational drug within 6 months prior to randomisation
* Is unable to provide consent without the aid of an interpreter.
* In the opinion of the Investigator may be unable to follow the protocol

Primary outcomes:
measure: The proportion of participants with resolution of significant Sleep Disordered Breathing (SDB) symptoms as defined by the parent completed Brouillette questionnaire </=-1 at 6 weeks
timeFrame: 6 weeks
Secondary outcomes:
measure: The proportion of participants with resolution of significant Sleep Disordered Breathing (SDB) symptoms as defined by the parent completed Brouillette questionnaire < -1 at 12 weeks
timeFrame: 12 weeks
measure: An improvement of score in parent completed Pediatric Sleep Questionnaire-sleep disordered breathing subscale (PSQ-SDB subscale) at week 6 when compared with baseline measured at the start of the intervention period (week 0)
timeFrame: 6 weeks
measure: An improvement of score in parent completed Obstructive Sleep Apnoea-18 (OSA-18) questionnaire at week 6 when compared with baseline measured at the start of intervention period (week 0).
timeFrame: 6 weeks
measure: An improvement of score in parent completed Pediatric Quality of Life Inventory (PedsQL) at week 6 when compared with baseline measured at the start of the intervention period (week 0).
timeFrame: 6 weeks
measure: An improvement of score in parent completed Strengths and Difficulties Questionnaire (SDQ) at week 6 when compared with baseline measured at the start of the intervention period (week 0).
timeFrame: 6 weeks
measure: An improvement of score in parent completed Glasgow Children's Benefit Inventory (GCBI) at week 6 when compared with baseline measured at the start of the intervention period (week 0).
timeFrame: 6 weeks
measure: Proportion of parent responders who think their child needs surgery to remove their tonsils or adenoids (T&A) at 6 weeks
timeFrame: 6 weeks
measure: Proportion of parent responders who think their child needs surgery to remove their tonsils or adenoids (T&A) at 12 weeks
timeFrame: 12 weeks
measure: Proportion of parent responders who think their child needs surgery to remove their tonsils or adenoids (T&A) at 6 months
timeFrame: 6 months
measure: Proportion of parent responders who would be happy to proceed with tonsils and adenoids surgery (T&A) if recommended to them at 6 weeks
timeFrame: 6 weeks
measure: Proportion of parent responders who would be happy to proceed with tonsils and adenoids (T&A) surgery if recommended to them at 12 weeks
timeFrame: 12 weeks
measure: Proportion of parent responders who would be happy to proceed with tonsils and adenoids (T&A) surgery if recommended to them at 6 months
timeFrame: 6 months
measure: Proportion of parent responders who would be happy to proceed with tonsils and adenoids (T&A) surgery if recommended to them at 12 months
timeFrame: 12 months
measure: Proportion of parents who think their child still needs a review by a hospital specialist at 6 weeks
timeFrame: 6 weeks
measure: Proportion of parents who think their child still needs a review by a hospital specialist at 12 weeks
timeFrame: 12 weeks
measure: Proportion of parents who think their child still needs a review by a hospital specialist at 6 months
timeFrame: 6 months
measure: Proportion of parents who think their child still needs a review by a hospital specialist at 12 months
timeFrame: 12 months
measure: Proportion of parents who would be happy to have their child taken off the hospital clinic waiting list at 6 weeks
timeFrame: 6 weeks
measure: Proportion of parents who would be happy to have their child taken off the hospital clinic waiting list at 12 weeks
timeFrame: 12 weeks
measure: Proportion of parents who would be happy to have their child taken off the hospital clinic waiting list at 6 months
timeFrame: 6 months
measure: Proportion of parents who would be happy to have their child taken off the hospital clinic waiting list at 12 months
timeFrame: 12 months
measure: Parent satisfaction with run-in phase and treatment phase therapy as an alternative to tonsillectomy and/or adenoidectomy at 6 weeks (Likert Scale)
timeFrame: 6 weeks
measure: Parent satisfaction with run-in phase and treatment phase therapy as an alternative to tonsillectomy and/or adenoidectomy at 12 weeks (Likert Scale)
timeFrame: 12 weeks
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) defined by Ear Nose Throat (ENT) surgery at 6 months
timeFrame: 6 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing defined by ENT surgery at 12 months
timeFrame: 12 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on symptoms of SDB reported in parent-completed Brouillette questionnaire at 6 months
timeFrame: 6 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on symptoms of SDB reported in parent-completed Brouillette questionnaire at 12 months.
timeFrame: 12 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on symptoms of SDB reported in parent-completed Pediatric Quality of Life Inventory (PedsQL) at 6 months
timeFrame: 6 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on symptoms of SDB reported in parent-completed Pediatric Quality of Life Inventory (PedsQL) at 12 months
timeFrame: 12 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on treatment for Sleep Disordered Breathing (SDB) at 6 months
timeFrame: 6 months
measure: Progression to Tonsillectomy and/or Adenoidectomy for Sleep Disordered Breathing (SDB) based on treatment for Sleep Disordered Breathing (SDB) at 12 months.
timeFrame: 12 months
measure: Number of adverse events (AEs) throughout the treatment phase
timeFrame: 6 weeks
measure: Number of adverse events (AEs) within the first week of the treatment
timeFrame: 1 week
measure: Compliance of medical therapy measured by weight of sent and returned bottles of medication
timeFrame: -6 weeks and 6 weeks (start of run-in phase to end of treatment phase)
measure: Clinical factors at baseline that are associated with response to interventions, based on statistical analysis
timeFrame: 6 weeks
Other outcomes:
Locations:
city: Clayton
country: Australia
facility: Monash Children's Hospital
state: Victoria
city: Parkville
country: Australia
facility: Royal Children's Hospital / Murdoch Children's Research Institute
state: Victoria
Study officials:
affiliation: Murdoch Children&amp;#39;s Research Institute
name: Kirsten Perrett
role: PRINCIPAL_INVESTIGATOR
References: