Neuromuscular Diseases in ChildrenNCT04081116
Mechanical Insufflation -Exsufflation in Children With NMD and Weak Cough
A study testing Symmetric settings (high pressures/fast rate), Asymmetric settings (Pi<Pe/Ti>Te) and Preinclusion settings in people with Neuromuscular Diseases in Children, run by Oslo University Hospital at 4 sites in Norway.
Trial 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. Read more about this study
- Who is it for?
- Ages 6 months to 18 years
- Conditions the study is for
- Neuromuscular Diseases in Children
- What is being tested?
- Symmetric settings (high pressures/fast rate), Asymmetric settings (Pi<Pe/Ti>Te) and Preinclusion settings
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 4 sitesNorway
- Study dates
- 1 Jan 2019to 28 Feb 2021
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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).
Who cannot join
- age < 6 mnd
- obstructive lung disease (hyperinflation or emphysema on x-ray.
Trial locations and contacts
4 sites in 1 country.
Norway4 sites
Helse bergen HF
Bergen
Oslo university hospital
Oslo
Stavanger university hospital
Stavanger
St. Olav Trondheim university hospital
Trondheim
About this study
Official title: 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.
Read the 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.
- Conditions
- Neuromuscular Diseases in Children
- Being tested
- Symmetric settings (high pressures/fast rate), Asymmetric settings (Pi<Pe/Ti>Te), Preinclusion settings
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Symmetric settings (high pressures/fast rate)Other
Describes settings on the MI-E device
Asymmetric settings (Pi<Pe/Ti>Te)Other
Lower insufflation pressures at longer times
Preinclusion settingsOther
Settings in use at start of study
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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 Receives: Other: Symmetric settings (high pressures/fast rate); Other: Asymmetric settings (Pi<Pe/Ti>Te); Other: Preinclusion settings |
What is being measured
The primary outcome is the main question the study is designed to answer.
PCF in the MI-E circuit
Recording of maximal value produced by the MI-E device during Cough
Measured: 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.
Measured: Total time use 30 min
Secondary outcomes (4)
Carbon dioxide
Transcutaneous trend measurement of CO2 during data collection
Measured: maximal time use is 30 min
Oxygen
Transcutaneous trend measurement of peripheral O2 during data collection
Measured: maximal time use is 30 min
Hart rate
Transcutaneous trend measurement of hart rate during data collection
Measured: 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.
Measured: The VAS is recorded after each of the three trials. Total time use 30 min
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Sep 2019
- Study start1 Jan 2019
- Primary completion28 Feb 2021
- Study completion28 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Neuromuscular Diseases in ChildrenStudies of the same condition, open and under way.
- More trials run by Oslo University HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Model description
- Three different settings are tested on the same patients but in randomized order.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 77 participants (actual)
Sponsor and investigators
- Lead sponsor
- Oslo University Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Brit Hov, Oslo University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04081116
- Sponsor's protocol ID
- 18/12375
- Status last verified
- Jan 2023
- First submitted
- 4 Feb 2019
- Last updated
- 20 Jan 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
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.
PubMed 33887060
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Optimal Settings of MI-E in Children With NMD and Weak Cough
- ClinicalTrials.gov ID
- NCT04081116
- Acronym / sponsor ID
- 18/12375
- Conditions
- Neuromuscular Diseases in Children
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- Oslo University HospitalUniversity, hospital or other
- Study start
- 1 Jan 2019 (actual)
- Primary completion
- 28 Feb 2021 (actual)
- Study completion
- 28 Feb 2021 (actual)
- First posted
- 9 Sep 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
- Model description
- Three different settings are tested on the same patients but in randomized order.
| Arm | Type | Receives |
|---|---|---|
| MI-E testing symmetric settings | Experimental | Other: Symmetric settings (high pressures/fast rate), Other: Asymmetric settings (Pi<Pe/Ti>Te), Other: Preinclusion settingsSymmetric settings is one of 3 different settings that will be tested on the same day but in randomized order. |
| MI-E testing assymetric settings | Experimental | Other: Symmetric settings (high pressures/fast rate), Other: Asymmetric settings (Pi<Pe/Ti>Te), Other: Preinclusion settingsAsymmetric settings is one of 3 different settings that will be tested on the same day but in randomized order. |
| Settings in use | Sham comparator | Other: Symmetric settings (high pressures/fast rate), Other: Asymmetric settings (Pi<Pe/Ti>Te), Other: Preinclusion settingsSettings in use is one of 3 different settings that will be tested on the same day but in randomized order |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Diagnosed neuromuscular disease < 18 years
- Established use (> 3mnd) of MI-E.
- Reduced PCF
- Clinical indication (difficulty to clear secretions, audible weak cough, history of pneumonia or frequent or prolonged respiratory tract infections).
Key exclusion criteria (2 listed)
- age < 6 mnd
- obstructive lung disease (hyperinflation or emphysema on x-ray.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Symmetric settings (high pressures/fast rate)Describes settings on the MI-E device | Other | MI-E testing assymetric settings, MI-E testing symmetric settings, Settings in use |
| Asymmetric settings (Pi<Pe/Ti>Te)Lower insufflation pressures at longer times | Other | MI-E testing assymetric settings, MI-E testing symmetric settings, Settings in use |
| Preinclusion settingsSettings in use at start of study | Other | MI-E testing assymetric settings, MI-E testing symmetric settings, Settings in use |
Primary outcome
PCF in the MI-E circuit
Time frame: 30 minutes.
Recording of maximal value produced by the MI-E device during Cough
Patient reported comfort
Time frame: Total time use 30 min
Comfort rated on a Visual Analog Scale (VAS) (0-100) where 100 is very uncomfortable and 0 is very comfortable.
4 secondary outcomes
Carbon dioxide
Time frame: maximal time use is 30 min
Oxygen
Time frame: maximal time use is 30 min
Hart rate
Time frame: Total max 30 minutes (During three MI-E trials)
Patient reported efficacy
Time frame: The VAS is recorded after each of the three trials. Total time use 30 min
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2023.
- Derived from this study: PubMed 33887060Morrow 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Symmetric settings (high pressures/fast rate), Asymmetric settings (Pi<Pe/Ti>Te) and Preinclusion settings in people with Neuromuscular Diseases in Children, run by Oslo University Hospital at 4 sites in Norway.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 20 Jan 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Norway. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PCF in the MI-E circuit (time frame: 30 minutes.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04081116, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2023.