CompletedNot Applicable

Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough

Norway77 participantsStarted 2019-01-01

Plain-language 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.

Who can participate

Age range

6 Months – 18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

PCF in the MI-E circuit

Timeframe: 30 minutes.

Patient reported comfort

Timeframe: Total time use 30 min

Trial details

NCT IDNCT04081116

SponsorOslo University Hospital

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-02-28

View on ClinicalTrials.gov More Neuromuscular Diseases in Children trials