Neuromuscular DiseaseLIAMNCT02288299
Long-Term Effect of LIAM on Respiratory Performance in NIV Patients Suffering From Neuromuscular Disease
A study testing non invasive mechanical ventilation in people with Neuromuscular Disease, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
Trial summary
The long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in home non-invasively ventilated (NIV) patients suffering from neuromuscular disease will be assessed in a prospective, randomized, cross over, open label study Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neuromuscular Disease
- What is being tested?
- non invasive mechanical ventilation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 1 siteFrance
- Study dates
- 29 Oct 2015to 31 Dec 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- o neuromuscular disease ( progressive muscular dystrophy, for example; Duchenne muscular dystrophy, spinal muscular atrophy)
- age ≥ 18 years
- indication to NIV (ongoing ventilation or new patients)
- vital capacity ≤ 50% pred
- peak cough flow < 270 l/min
- "LIAM Responder" = increased insufflation capacity (≥ + 50%) and Peak Cough Flow (PCF ≥+ 50%) with LIAM
Who cannot join
- o acute respiratory failure (respiratory acidosis)
- home treatment by instrumental cough assistance in the preceding 12 months
- ongoing medical treatment of the neuromuscular disease (for ex: corticosteroids in Duchenne disease, enzyme therapy in Pompe disease)
- previous pneumothorax
- plan of legal protection
- pregnant or breastfeeding women
- failure to cooperate
- no affiliation to a social security scheme
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Home ventilation Unit , Raymond Poincaré hospital
Garches, Paris Area, 92380
David Orlikowski, MD, PhD, 33147107776, david.orlikowski@rpc.aphp.fr
Sandra Pottier, CRA, 33 1 47 10 44 69, sandra.pottier@rpc.aphp.fr
About this study
Official title: Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease
The long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in home non-invasively ventilated (NIV) patients suffering from neuromuscular disease will be assessed in a prospective, randomized, cross over, open label study
Read the detailed description
In advanced neuromuscular disorders, respiratory complications represent the main cause of morbidity and mortality. Beside chronic respiratory insufficiency, necessitating a ventilatory support, mostly performed by non-invasive ventilation (NIV), cough is impaired due to the muscle weakness, and respiratory physiotherapy becomes an essential part of the management.
Various techniques have been proposed to improve lung recruitment and cough in neuromuscular patients. The assisted techniques based on a positive pressure insufflation maneuver have shown an improvement in physiological variables on the short term, but there is to date no good-quality prospective study allowing to evaluate the long term efficacy of mechanical cough assistance devices in neuromuscular patients.
We designed a randomized, cross over, open label study to assess the long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in NIV patients suffering from neuromuscular disease.
- Conditions
- Neuromuscular Disease
- Being tested
- non invasive mechanical ventilation
- Keywords
- Instrumental cough assistance, positive pressure insufflation maneuver, home mechanical ventilation, prospective evaluation
- Medical subject headings
- Neuromuscular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
non invasive mechanical ventilationDevice
instrumental increase of inspiratory capacity and cough
| Study group | Role | What they receive |
|---|---|---|
| non invasive mechanical ventilation | Experimental | Patients suffering from neuromuscular disease with NIV indication and cough inefficiency Receives: Device: non invasive mechanical ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
Measured: 15 minutes
Secondary outcomes (3)
spontaneous vital capacity
spontaneous vital capacity change at 3 months
Measured: 15 minutes
peak flow
peak cough flow (spontaneous / assisted) change at 3 months
Measured: 15 minutes
optoelectronic plethysmography
change at 3 months in the distribution in ventilation (assessed by optoelectronic plethysmography), spontaneous/assisted
Measured: 1 hour
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Nov 2014 (estimated)
- Study start29 Oct 2015
- Primary completion31 Dec 2021 (estimated)
- Study completion31 Dec 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuromuscular DiseaseStudies of the same condition, open and under way.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- David Orlikowski, MD, PhD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02288299
- Sponsor's protocol ID
- 2014-A01005-42
- Acronym
- LIAM
- Status last verified
- Feb 2019
- First submitted
- 5 Nov 2014
- Last updated
- 18 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Long-Term Effect of LIAM (Lung Insufflation Assist Maneuver) on Respiratory Performance in Non-invasive Ventilated Patients Suffering From Neuromuscular Disease
- ClinicalTrials.gov ID
- NCT02288299
- Acronym / sponsor ID
- LIAM / 2014-A01005-42
- Conditions
- Neuromuscular Disease
- Keywords
- Instrumental cough assistance, positive pressure insufflation maneuver, home mechanical ventilation, prospective evaluation
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Centre d'Investigation Clinique et Technologique 805University, hospital or other
- Study start
- 29 Oct 2015 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 31 Dec 2021 (estimated)
- First posted
- 11 Nov 2014 (estimated)
- 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.
The long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in home non-invasively ventilated (NIV) patients suffering from neuromuscular disease will be assessed in a prospective, randomized, cross over, open label study
Detailed description
In advanced neuromuscular disorders, respiratory complications represent the main cause of morbidity and mortality. Beside chronic respiratory insufficiency, necessitating a ventilatory support, mostly performed by non-invasive ventilation (NIV), cough is impaired due to the muscle weakness, and respiratory physiotherapy becomes an essential part of the management.
Various techniques have been proposed to improve lung recruitment and cough in neuromuscular patients. The assisted techniques based on a positive pressure insufflation maneuver have shown an improvement in physiological variables on the short term, but there is to date no good-quality prospective study allowing to evaluate the long term efficacy of mechanical cough assistance devices in neuromuscular patients.
We designed a randomized, cross over, open label study to assess the long-term effect of LIAM (Lung Insufflation Assist Maneuver) on respiratory performance in NIV patients suffering from neuromuscular disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| non invasive mechanical ventilation | Experimental | Device: non invasive mechanical ventilationPatients suffering from neuromuscular disease with NIV indication and cough inefficiency |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- o neuromuscular disease ( progressive muscular dystrophy, for example; Duchenne muscular dystrophy, spinal muscular atrophy)
Key exclusion criteria (8 listed)
- o acute respiratory failure (respiratory acidosis)
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 |
|---|---|---|
| non invasive mechanical ventilationinstrumental increase of inspiratory capacity and cough | Device | non invasive mechanical ventilation |
Primary outcome
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
Time frame: 15 minutes
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up
3 secondary outcomes
spontaneous vital capacity
Time frame: 15 minutes
peak flow
Time frame: 15 minutes
optoelectronic plethysmography
Time frame: 1 hour
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2019.
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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.
David Orlikowski, MD, PhD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing non invasive mechanical ventilation in people with Neuromuscular Disease, run by Centre d'Investigation Clinique et Technologique 805 at 1 site in France.
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 8 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: status not confirmed, as of the sponsor's last update on 18 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact David Orlikowski, MD, PhD (+33(0)147107777, david.orlikowski@rpc.aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in the maximal assisted vital capacity (maximal insufflation capacity) between the begin of the treatment period and the 3-months follow up (time frame: 15 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 18 Nov 2019; AzTrials last refreshed its copy on 7 Nov 2026.