Duchenne Muscular DystrophyNCT01981915
Optimum Insufflation Capacity in NMD
A study testing IPPB and LIAM in people with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy, run by Universität Duisburg-Essen at 1 site in Germany.
Trial summary
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for an efficient cough function. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy, Spinal Muscular Atrophy, Chronic Respiratory Insufficiency
- What is being tested?
- IPPB and LIAM
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteGermany
- Study dates
- Jan 2011to Mar 2013
- 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 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- neuromuscular disorder
- respiratory insufficiency
- use of home mechanical ventilation
Who cannot join
- acute illness
- history of pneumothorax
Trial locations and contacts
1 site in 1 country.
Germany1 site
University of Essen, Children's Hospital, Dep. of Pediatric Pulmonology
Essen, North Rhine-Westphalia, 45122
About this study
Official title: Optimum Insufflation Capacity and Peak Cough Flow Augmentation in Patients With Neuromuscular Disease
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for an efficient cough function. The restricted cough function allows secretion to accumulate, which in turn causes narrowing of the airway lumen and makes ventilation of the neuromuscular patient even more difficult. The patient's susceptibility to infection increases again and the vicious circle repeats itself. Severe secretion retention may even lead to ventilator failure. Effective secretion and cough management instead reduces the risk for stay in hospital. Therefore, secretion and cough management is a mandatory part of the therapeutic concept for treating patients with neuromuscular disease.
The therapeutic efficacy of the Lung Insufflation Assist Maneuver(LIA) integrated in the ventilator VENTIlogic LS-plus manufactured by Weinmann GmbH+Co KG was studied in a pilot study carried out by the Dep. for Pediatric Pulmonology and Sleep Medicine at the University Hospital of Essen/Germany in cooperation with Research \& Development at Weinmann GmbH \&Co KG, Germany . The objective of the pilot study was to examine the therapeutic efficacy of LIAM as a cough support function in patients with neuromuscular disease and indications for mechanical ventilation.
We hypothesized that i) a certain insufflation maneuver pressure may be optimal to achieve the highest individual peak cough flow and ii) that this pressure is below the pressure needed to achieve the maximum insufflation capacity. We define the lowest insufflation capacity at which the best individual PCF can be achieved as optimum insufflation capacity (OIC). The study was performed using two different techniques in order to demonstrate that findings are not dependent on maneuver details but are rather based on effects of maneuver pressure. The protocol was limited to techniques which do not require breath stacking: i) insufflation with an Intermittend Positive Pressure (IPPB) device and ii) with the VENTIlogic LS using LIAM.
- Being tested
- IPPB, LIAM
- Keywords
- Cough augmentation, Peak cough flow, hyperinsufflation
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IPPBProcedure or surgery
Also called Intermittent Positive Pressure Breathing = IPPB
Lung insufflation with positive pressure using IPPB up from 10 to 40 mbar in 5 mbar increments and consecutive measurement of the insufflation volume
LIAMProcedure or surgery
Also called LIAM = Lung Insufflation Assist Maneuver
Lung insufflation using the Lung Insufflation Assist maneuver of the Ventilogic LS mechanical ventilator (Weinmann Germany) with positive pressure up from 10 to 40 mbar in 5 mbar increments and consecutive measurement of the insufflation volume
| Study group | Role | What they receive |
|---|---|---|
| Lung Insufflation Volume | Experimental | Measurement of the lung volume after hyperinsufflation with positive pressure by IPPB or LIAM Receives: Procedure: IPPB; Procedure: LIAM |
| Peak Cough Flow | Experimental | Measurement of the peak cough flow after hyperinsufflation with positive pressure by IPPB or LIAM Receives: Procedure: IPPB; Procedure: LIAM |
What is being measured
The primary outcome is the main question the study is designed to answer.
insufflation capacity
Lung volume was measured during spontaneous breathing and after a lung insufflation assist maneuver.
Measured: change of lung volume with the procedure; during hospital stay on average 3 days
Secondary outcomes (1)
Peak cough flow
Peak cough flow was measured during spontaneous breathing and after a lung insufflation assist maneuver.
Measured: change of peak cough flow with the procedure; during hospital stay on average 3 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Nov 2013 (estimated)
- Study startJan 2011
- Primary completionFeb 2013
- Study completionMar 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universität Duisburg-Essen
- Sponsor type
- University, hospital or other
- Collaborators
- Weinmann Geräte für Medizin GmbH + Co. KG
- Responsible party
- Principal investigator, Uwe Mellies, Universität Duisburg-Essen
- Investigators
- Uwe Mellies, MD, Principal investigator, University of Essen, Germany
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01981915
- Sponsor's protocol ID
- OIC2013
- Status last verified
- Nov 2013
- First submitted
- 11 Oct 2013
- Last updated
- 13 Nov 2013
- Results posted
- No
- Data monitoring committee
- No
Publications
Dohna-Schwake C, Ragette R, Teschler H, Voit T, Mellies U. Predictors of severe chest infections in pediatric neuromuscular disorders. Neuromuscul Disord. 2006 May;16(5):325-8. doi: 10.1016/j.nmd.2006.02.003. Epub 2006 Apr 18.
PubMed 16621559Ishikawa Y, Bach JR, Komaroff E, Miura T, Jackson-Parekh R. Cough augmentation in Duchenne muscular dystrophy. Am J Phys Med Rehabil. 2008 Sep;87(9):726-30. doi: 10.1097/PHM.0b013e31817f99a8.
PubMed 18716484Bach JR, Mahajan K, Lipa B, Saporito L, Goncalves M, Komaroff E. Lung insufflation capacity in neuromuscular disease. Am J Phys Med Rehabil. 2008 Sep;87(9):720-5. doi: 10.1097/PHM.0b013e31817fb26f.
PubMed 18716483Hahneberger RW. Applanation tonometry in the conscious cynomolgus monkey (Macaca fascicularis). Acta Ophthalmol (Copenh). 1976 Jul;54(3):311-9. doi: 10.1111/j.1755-3768.1976.tb01260.x.
PubMed 821306
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimum Insufflation Capacity and Peak Cough Flow Augmentation in Patients With Neuromuscular Disease
- ClinicalTrials.gov ID
- NCT01981915
- Acronym / sponsor ID
- OIC2013
- Keywords
- Cough augmentation, Peak cough flow, hyperinsufflation
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Universität Duisburg-EssenUniversity, hospital or other
- Collaborators
- Weinmann Geräte für Medizin GmbH + Co. KG
- Study start
- Jan 2011
- Primary completion
- Feb 2013 (actual)
- Study completion
- Mar 2013 (actual)
- First posted
- 13 Nov 2013 (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.
Patients with underlying neuromuscular disorder (NMD) often suffer from weakness in the inspiratory and expiratory muscles. Consequently they do not have the strength to generate the minimum flow of 160 to 300 liters/minute for an efficient cough function. The restricted cough function allows secretion to accumulate, which in turn causes narrowing of the airway lumen and makes ventilation of the neuromuscular patient even more difficult. The patient's susceptibility to infection increases again and the vicious circle repeats itself. Severe secretion retention may even lead to ventilator failure. Effective secretion and cough management instead reduces the risk for stay in hospital. Therefore, secretion and cough management is a mandatory part of the therapeutic concept for treating patients with neuromuscular disease.
The therapeutic efficacy of the Lung Insufflation Assist Maneuver(LIA) integrated in the ventilator VENTIlogic LS-plus manufactured by Weinmann GmbH+Co KG was studied in a pilot study carried out by the Dep. for Pediatric Pulmonology and Sleep Medicine at the University Hospital of Essen/Germany in cooperation with Research \& Development at Weinmann GmbH \&Co KG, Germany . The objective of the pilot study was to examine the therapeutic efficacy of LIAM as a cough support function in patients with neuromuscular disease and indications for mechanical ventilation.
We hypothesized that i) a certain insufflation maneuver pressure may be optimal to achieve the highest individual peak cough flow and ii) that this pressure is below the pressure needed to achieve the maximum insufflation capacity. We define the lowest insufflation capacity at which the best individual PCF can be achieved as optimum insufflation capacity (OIC). The study was performed using two different techniques in order to demonstrate that findings are not dependent on maneuver details but are rather based on effects of maneuver pressure. The protocol was limited to techniques which do not require breath stacking: i) insufflation with an Intermittend Positive Pressure (IPPB) device and ii) with the VENTIlogic LS using LIAM.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lung Insufflation Volume | Experimental | Procedure: IPPB, Procedure: LIAMMeasurement of the lung volume after hyperinsufflation with positive pressure by IPPB or LIAM |
| Peak Cough Flow | Experimental | Procedure: IPPB, Procedure: LIAMMeasurement of the peak cough flow after hyperinsufflation with positive pressure by IPPB or LIAM |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- neuromuscular disorder
- respiratory insufficiency
- use of home mechanical ventilation
Key exclusion criteria (2 listed)
- acute illness
- history of pneumothorax
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 |
|---|---|---|
| IPPBAlso: Intermittent Positive Pressure Breathing = IPPBLung insufflation with positive pressure using IPPB up from 10 to 40 mbar in 5 mbar increments and consecutive measurement of the insufflation volume | Procedure or surgery | Lung Insufflation Volume, Peak Cough Flow |
| LIAMAlso: LIAM = Lung Insufflation Assist ManeuverLung insufflation using the Lung Insufflation Assist maneuver of the Ventilogic LS mechanical ventilator (Weinmann Germany) with positive pressure up from 10 to 40 mbar in 5 mbar increments and consecutive measurement of the insufflation volume | Procedure or surgery | Lung Insufflation Volume, Peak Cough Flow |
Primary outcome
insufflation capacity
Time frame: change of lung volume with the procedure; during hospital stay on average 3 days
Lung volume was measured during spontaneous breathing and after a lung insufflation assist maneuver.
1 secondary outcome
Peak cough flow
Time frame: change of peak cough flow with the procedure; during hospital stay on average 3 days
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 13 Nov 2013.
- Background: PubMed 16621559Dohna-Schwake C, Ragette R, Teschler H, Voit T, Mellies U. Predictors of severe chest infections in pediatric neuromuscular disorders. Neuromuscul Disord. 2006 May;16(5):325-8. doi: 10.1016/j.nmd.2006.02.003. Epub 2006 Apr 18.
- Background: PubMed 18716484Ishikawa Y, Bach JR, Komaroff E, Miura T, Jackson-Parekh R. Cough augmentation in Duchenne muscular dystrophy. Am J Phys Med Rehabil. 2008 Sep;87(9):726-30. doi: 10.1097/PHM.0b013e31817f99a8.
- Background: PubMed 18716483Bach JR, Mahajan K, Lipa B, Saporito L, Goncalves M, Komaroff E. Lung insufflation capacity in neuromuscular disease. Am J Phys Med Rehabil. 2008 Sep;87(9):720-5. doi: 10.1097/PHM.0b013e31817fb26f.
- Background: PubMed 821306Hahneberger RW. Applanation tonometry in the conscious cynomolgus monkey (Macaca fascicularis). Acta Ophthalmol (Copenh). 1976 Jul;54(3):311-9. doi: 10.1111/j.1755-3768.1976.tb01260.x.
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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.
Uwe Mellies, MD principal investigator
University of Essen, Germany
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing IPPB and LIAM in people with Duchenne Muscular Dystrophy and Spinal Muscular Atrophy, run by Universität Duisburg-Essen at 1 site in Germany.
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 3 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 13 Nov 2013. 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 Germany. 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?
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?
insufflation capacity (time frame: change of lung volume with the procedure; during hospital stay on average 3 days). 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 NCT01981915, which also lists 4 linked publications.
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 13 Nov 2013.