Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough

NCT04081116 | Completed

• 1 other identifier: 18/12375
• Study Type: interventional
• Target: 77
• Locations: 1 country
• Sites: 4

Brief Summary

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.

Conditions

Neuromuscular Diseases in Children

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (4)

• Carbon dioxide

  maximal time use is 30 min

• Oxygen

  maximal time use is 30 min

• Hart rate

  Total max 30 minutes (During three MI-E trials)

• Patient reported efficacy

  The VAS is recorded after each of the three trials. Total time use 30 min

Study Arms (3)

MI-E testing symmetric settings

EXPERIMENTAL

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); Other: Asymmetric settings (Pi<Pe/Ti>Te); Other: Preinclusion settings

MI-E testing assymetric settings

EXPERIMENTAL

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); Other: Asymmetric settings (Pi<Pe/Ti>Te); Other: Preinclusion settings

Settings in use

SHAM COMPARATOR

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); Other: Asymmetric settings (Pi<Pe/Ti>Te); Other: Preinclusion settings

Interventions

Symmetric settings (high pressures/fast rate) OTHER

Describes settings on the MI-E device

MI-E testing assymetric settings; MI-E testing symmetric settings; Settings in use

Asymmetric settings (Pi<Pe/Ti>Te) OTHER

Lower insufflation pressures at longer times

MI-E testing assymetric settings; MI-E testing symmetric settings; Settings in use

Preinclusion settings OTHER

Settings in use at start of study

MI-E testing assymetric settings; MI-E testing symmetric settings; Settings in use

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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).

You may not qualify if:

• age \< 6 mnd
• obstructive lung disease (hyperinflation or emphysema on x-ray.

Sponsors & Collaborators

• Oslo University Hospital: lead

Study Sites (4)

Helse bergen HF

Bergen, Norway

Location

Oslo university hospital

Oslo, Norway

Location

Stavanger university hospital

Stavanger, Norway

Location

St. Olav Trondheim university hospital

Trondheim, Norway

Location

Related Publications (1)

• 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.

  PMID: 33887060 DERIVED

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Participant, care provider, investigator and outcomes assessor are blinded from the order of the settings tested. Only a technician change settings and know the order.
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Physiotherapist

Model Details: Three different settings are tested on the same patients but in randomized order.

Study Record Dates

• First Submitted: February 4, 2019
• First Posted: September 9, 2019
• Study Start: January 1, 2019
• Primary Completion: February 28, 2021
• Study Completion: February 28, 2021
• Last Updated: January 20, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

NO (4)