Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough

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

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.

Study Overview

• Status: Completed
• Conditions: Neuromuscular Diseases in Children
• Intervention / Treatment: Other: Symmetric settings (high pressures/fast rate); Other: Asymmetric settings (PiTe); Other: Preinclusion settings

Detailed Description

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.

• Study Type: Interventional
• Enrollment (Actual): 77
• Phase: Not Applicable

Contacts and Locations

Study Locations

• Norway
  • Bergen, Norway
    • Helse Bergen HF
  • Oslo, Norway
    • Oslo University Hospital
  • Stavanger, Norway
    • Stavanger University Hospital
  • Trondheim, Norway
    • St. Olav Trondheim university hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Single Group Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: MI-E testing symmetric settings; 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); Describes settings on the MI-E device; Other: Asymmetric settings (PiTe); Lower insufflation pressures at longer times; Other: Preinclusion settings; Settings in use at start of study
Experimental: MI-E testing assymetric settings; 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); Describes settings on the MI-E device; Other: Asymmetric settings (PiTe); Lower insufflation pressures at longer times; Other: Preinclusion settings; Settings in use at start of study
Sham Comparator: Settings in use; 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); Describes settings on the MI-E device; Other: Asymmetric settings (PiTe); Lower insufflation pressures at longer times; Other: Preinclusion settings; Settings in use at start of study

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Oslo University Hospital

Publications and helpful links

General Publications

• Morrow B, Argent A, Zampoli M, Human A, Corten L, Toussaint M. Cough augmentation techniques for people with chronic neuromuscular disorders. Cochrane Database Syst Rev. 2021 Apr 22;4(4):CD013170. doi: 10.1002/14651858.CD013170.pub2.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2019
• Primary Completion (Actual): February 28, 2021
• Study Completion (Actual): February 28, 2021

Study Registration Dates

• First Submitted: February 4, 2019
• First Submitted That Met QC Criteria: September 6, 2019
• First Posted (Actual): September 9, 2019

Study Record Updates

• Last Update Posted (Actual): January 20, 2023
• Last Update Submitted That Met QC Criteria: January 19, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Neuromuscular Diseases
• Other Study ID Numbers: 18/12375

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No