Clinical trial NCT04081116: Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough
Official title: The Optimal Settings of MI-E in Children With NMD and Weak Cough
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Neuromuscular Diseases in Children
Lead sponsor: Oslo University Hospital
Sponsor class: OTHER
First posted: 2019-09-09
Enrollment: 77
Sex: ALL
Minimum age (years): 6
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: Three different settings are tested on the same patients but in randomized order.
Primary purpose: TREATMENT

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: PCF in the MI-E circuit
timeFrame: 30 minutes.
measure: Patient reported comfort
timeFrame: Total time use 30 min
Secondary outcomes:
measure: Carbon dioxide
timeFrame: maximal time use is 30 min
measure: Oxygen
timeFrame: maximal time use is 30 min
measure: Hart rate
timeFrame: Total max 30 minutes (During three MI-E trials)
measure: Patient reported efficacy
timeFrame: The VAS is recorded after each of the three trials. Total time use 30 min
Other outcomes:
Locations:
city: Bergen
country: Norway
facility: Helse bergen HF
city: Oslo
country: Norway
facility: Oslo university hospital
city: Stavanger
country: Norway
facility: Stavanger university hospital
city: Trondheim
country: Norway
facility: St. Olav Trondheim university hospital
Study officials:
References:
citation: 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.