Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough

Study Overview
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Brief Summary
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Neuromuscular diseases (NMD) in children are severe, possibly life-threatening orphan conditions. The children are vulnerable and often subject to rapid deterioration of pulmonary function due to impaired ability to clear airway secretions. The use of mechanical cough augmentation with insufflation-exsufflation (MIE) is a strategy to treat and prevent. Possible major benefits are described, but optimal settings for best efficacy and comfort in children are not established. The project aim to improve the quality of the treatment with MIE in children with NMD and weak cough. On the basis of a bench study a clinical trial aims to examine the most effective MIE settings when used in stable state and when respiratory tract infections are present.

Detailed Description
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On the basis of a prevalence study and a bench study different settings will be studied in children with NMD and weak cough to examine if settings derived from the lung model, are optimal to increase the peak cough flow (PCF) and comfort measured by visual analogue scale (VAS) in children with NMD and weak cough.

Official Title
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The Optimal Settings of MI-E in Children With NMD and Weak Cough

Conditions
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Neuromuscular Diseases in Children

Intervention / Treatment
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* Other: Symmetric settings (high pressures/fast rate)
* Other: Asymmetric settings (Pi<Pe/Ti>Te)
* Other: Preinclusion settings


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed neuromuscular disease < 18 years Established use (> 3mnd) of MI-E. Reduced PCF PCF < 270 l/min (when > 12 years) < 5th percentiles for PCF 16 (when 4 - 12 years) Clinical indication (difficulty to clear secretions, audible weak cough, history of pneumonia or frequent or prolonged respiratory tract infections). Exclusion Criteria: age < 6 mnd obstructive lung disease (hyperinflation or emphysema on x-ray.

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Single Group Assignment
Interventional Model Description: Three different settings are tested on the same patients but in randomized order.
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MI-E testing symmetric settings<br>Symmetric settings is one of 3 different settings that will be tested on the same day but in randomized order. | Other: Symmetric settings (high pressures/fast rate)<br>* Describes settings on the MI-E device<br>Other: Asymmetric settings (Pi<Pe/Ti>Te)<br>* Lower insufflation pressures at longer times<br>Other: Preinclusion settings<br>* Settings in use at start of study<br>|
| Experimental: MI-E testing assymetric settings<br>Asymmetric settings is one of 3 different settings that will be tested on the same day but in randomized order. | Other: Symmetric settings (high pressures/fast rate)<br>* Describes settings on the MI-E device<br>Other: Asymmetric settings (Pi<Pe/Ti>Te)<br>* Lower insufflation pressures at longer times<br>Other: Preinclusion settings<br>* Settings in use at start of study<br>|
| Sham Comparator: Settings in use<br>Settings in use is one of 3 different settings that will be tested on the same day but in randomized order | Other: Symmetric settings (high pressures/fast rate)<br>* Describes settings on the MI-E device<br>Other: Asymmetric settings (Pi<Pe/Ti>Te)<br>* Lower insufflation pressures at longer times<br>Other: Preinclusion settings<br>* Settings in use at start of study<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| PCF in the MI-E circuit | Recording of maximal value produced by the MI-E device during Cough | 30 minutes. | 
| Patient reported comfort | Comfort rated on a Visual Analog Scale (VAS) (0-100) where 100 is very uncomfortable and 0 is very comfortable. | Total time use 30 min | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Carbon dioxide | Transcutaneous trend measurement of CO2 during data collection | maximal time use is 30 min | 
| Oxygen | Transcutaneous trend measurement of peripheral O2 during data collection | maximal time use is 30 min | 
| Hart rate | Transcutaneous trend measurement of hart rate during data collection | Total max 30 minutes (During three MI-E trials) | 
| Patient reported efficacy | Efficacy rated on a Visual Analog Scale (0-100) where 100 is not efficient at all and 0 is very efficient. | The VAS is recorded after each of the three trials. Total time use 30 min |