Amyotrophic Lateral SclerosisPhase 4NCT00560287
Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis
A Phase 4 study testing Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers and Non invasive ventilation in people with Amyotrophic Lateral Sclerosis and Chronic Respiratory Failure, run by Fondazione Salvatore Maugeri at 2 sites in Italy.
Trial summary
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular… Read more about this study
- Who is it for?
- Ages 20 to 80 years
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, Chronic Respiratory Failure
- What is being tested?
- Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers and Non invasive ventilation
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 2 sitesItaly
- Study dates
- Jan 2008to Aug 2010
- Phase
- Phase 4
- 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 20 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with the diagnosis of ALS and one of the following signs:
- Vital Capacity < 50% predicted
- A Maximal Inspiratory Pressure (MIP) < 60% predicted
- Polygraphic signs of nocturnal hypoventilation with daytime symptoms.
Who cannot join
- Life expectancy>12 months
- Any comorbidity
- Acute Respiratory Failure
Trial locations and contacts
2 sites in 1 country.
Italy2 sites, 2 recruiting
- Recruiting
Fondazione S.Maugeri
Pavia, 27100
Stefano Nava, md, o3825921, snava@fsm.it
Stefano Nava, md, 03825921, snava@fsm.it
- Not yet recruiting
Respiratory Unit FSM
Pavia, 27100
Stefano Nava, md, 0382 592 ext. 806, snava@fsm.it
Francesco Fanfulla, MD, 0382 592 ext. 815, ffanfulla@fsm.it
About this study
Official title: Role of Non-invasive Ventilation in Amyotrophic Lateral Sclerosis: Volume Versus Pressure Mode
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of the symptoms of chronic hypoventilation in the short term, and in two small studies survival was prolonged.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very likely that the operators need to frequently adjust the level of inspiratory pressure in an attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be avoided.
Read more
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent variations of the settings, since the provision of a fixed tidal volume may always guarantee and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
Read the detailed description
Background
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of the symptoms of chronic hypoventilation in the short term, and in two small studies survival was prolonged. As a matter of fact a recent Cochrane review stated that "long-term mechanical ventilation should be offered as a therapeutic option to patients with chronic respiratory failure due to neuromuscular diseases". Recurrent episodes of nocturnal desaturation especially during REM sleep may deeply influence the prognosis of patients affect by these disorders (i.e. ALS), and they are often associated with hypercapnia, so that the correction of nocturnal hypoventilation seems to be the major goal to achieve with the ventilatory treatment.
A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole.
Most of the studies performed in RTD disorders were performed using a pressure assisted mode (i.e Pressure Support Ventilation), because of the supposed better tolerance of the patients and easy of settings for the operators, even though this has never been scientifically proven.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very likely that the operators need to frequently adjust the level of inspiratory pressure in an attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be avoided.
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent variations of the settings, since the provision of a fixed tidal volume may always guarantee and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
Aim of the Project
The primary aims of the project are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
- Enrollment criteria: Patients with the diagnosis of ALS and one of the following signs: 1) Vital Capacity < 50% predicted 2) a Maximal Inspiratory Pressure (MIP) < 60% predicted 3) polygraphic signs of nocturnal hypoventilation with daytime symptoms.
- The patients will be randomized to receive NIV with one of the following modes: 1) Pressure Support Ventilation with the inspiratory pressure set according to the patient's tolerance in order to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg. The back-up rate will be set at 10 breaths/min. 2) Volume assist ventilation with a back-up rate of 10 breaths/min and a tidal volume set to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg
- Being tested
- Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers, Non invasive ventilation
- Keywords
- Amyotrophic Lateral Sclerosis, Non-invasive ventilation, Chronic Respiratory Failure
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providersDevice
Also called Legendaire (Airox), Vivo (Breas), Elisee (Saime), Synchrony (Respironics)
A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole.
Non invasive ventilationDevice
Also called Legendaire (Airox), Vivo (Breas), Elisee (Saime), Synchrony (Respironics)
A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole.
| Study group | Role | What they receive |
|---|---|---|
| 1 | Active comparator | Volume assist non-invasive ventilation Receives: Device: Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers; Device: Non invasive ventilation |
| 2 | Active comparator | Pressure Assist mode Receives: Device: Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers; Device: Non invasive ventilation |
What is being measured
The primary outcome is the main question the study is designed to answer.
quality of life
Measured: 1 year
tolerance to NIV
Measured: 1 year
number of hours of NIV per day
Measured: 1 year
frequency of hospital admission
Measured: 1 year
frequency of changing the ventilator settings by the operator.
Measured: 1 year
Secondary outcomes (3)
survival
Measured: 1 year
diurnal and nocturnal gas exchange
Measured: 1 year
Pulmonary Function Tests (PFTs).
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2007 (estimated)
- Study startJan 2008
- Primary completionAug 2009
- Study completionAug 2010 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies 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 4
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Care provider, Outcomes assessor
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fondazione Salvatore Maugeri
- Sponsor type
- University, hospital or other
- Investigators
- Stefano Nava, Principal investigator, Fondazione Salvatore MaugeriStefano Nava, MD, Principal investigator, Fondazione S.maugeri
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00560287
- Sponsor's protocol ID
- 190H09
- Other IDs
- 190H09
- Status last verified
- Mar 2010
- First submitted
- 16 Nov 2007
- Last updated
- 29 Mar 2010
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Mar 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Role of Non-invasive Ventilation in Amyotrophic Lateral Sclerosis: Volume Versus Pressure Mode
- ClinicalTrials.gov ID
- NCT00560287
- Acronym / sponsor ID
- 190H09
- Keywords
- Amyotrophic Lateral Sclerosis, Non-invasive ventilation, Chronic Respiratory Failure
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Fondazione Salvatore MaugeriUniversity, hospital or other
- Study start
- Jan 2008
- Primary completion
- Aug 2009 (actual)
- Study completion
- Aug 2010 (estimated)
- First posted
- 19 Nov 2007 (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.
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of the symptoms of chronic hypoventilation in the short term, and in two small studies survival was prolonged.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very likely that the operators need to frequently adjust the level of inspiratory pressure in an attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be avoided.
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent variations of the settings, since the provision of a fixed tidal volume may always guarantee and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
Detailed description
Background
Non-invasive mechanical ventilation (NIV) has been increasingly used as a treatment of chronic hypercapnic respiratory failure. Its use in patients affected by chronic obstructive pulmonary disorders is still controversial, while most of the studies performed in restrictive thoracic disorders (RTD), and in particular in neuromuscular patients, suggested alleviation of the symptoms of chronic hypoventilation in the short term, and in two small studies survival was prolonged. As a matter of fact a recent Cochrane review stated that "long-term mechanical ventilation should be offered as a therapeutic option to patients with chronic respiratory failure due to neuromuscular diseases". Recurrent episodes of nocturnal desaturation especially during REM sleep may deeply influence the prognosis of patients affect by these disorders (i.e. ALS), and they are often associated with hypercapnia, so that the correction of nocturnal hypoventilation seems to be the major goal to achieve with the ventilatory treatment.
A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole.
Most of the studies performed in RTD disorders were performed using a pressure assisted mode (i.e Pressure Support Ventilation), because of the supposed better tolerance of the patients and easy of settings for the operators, even though this has never been scientifically proven.
In the terminal phase of the disease, when the respiratory muscles became weaker it is very likely that the operators need to frequently adjust the level of inspiratory pressure in an attempt to guarantee an adequate tidal volume, so that alveolar hypoventilation may be avoided.
Theoretically the use of a volume assisted ventilation may overpass this problem of frequent variations of the settings, since the provision of a fixed tidal volume may always guarantee and adequate alveolar ventilation.
The primary aims of this multicenter randomized study are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
Aim of the Project
The primary aims of the project are to evaluate the clinical efficacy, the patients' tolerance and quality of life and the frequency of changing settings in a group of patients with SLS and initial chronic respiratory failure undergoing long-term NIV with Pressure Support Ventilation or Volume Assisted Ventilation.
- Enrollment criteria: Patients with the diagnosis of ALS and one of the following signs: 1) Vital Capacity < 50% predicted 2) a Maximal Inspiratory Pressure (MIP) < 60% predicted 3) polygraphic signs of nocturnal hypoventilation with daytime symptoms.
- The patients will be randomized to receive NIV with one of the following modes: 1) Pressure Support Ventilation with the inspiratory pressure set according to the patient's tolerance in order to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg. The back-up rate will be set at 10 breaths/min. 2) Volume assist ventilation with a back-up rate of 10 breaths/min and a tidal volume set to achieve an expired tidal volume > 6 ml/Kg and <8ml/Kg
Study design
- Primary purpose
- Supportive care
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (care provider, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1 | Active comparator | Device: Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers, Device: Non invasive ventilationVolume assist non-invasive ventilation |
| 2 | Active comparator | Device: Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers, Device: Non invasive ventilationPressure Assist mode |
Who may qualify
- Ages
- 20 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with the diagnosis of ALS and one of the following signs:
Key exclusion criteria (3 listed)
- Life expectancy>12 months
- Any comorbidity
- Acute Respiratory Failure
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 ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providersAlso: Legendaire (Airox), Vivo (Breas), Elisee (Saime), Synchrony (Respironics)A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole. | Device | 1, 2 |
| Non invasive ventilationAlso: Legendaire (Airox), Vivo (Breas), Elisee (Saime), Synchrony (Respironics)A very recent randomized controlled trial show that in ALS without severe bulbar dysfunction, NIV improves survival with maintenance of, and improvement in, quality of life, including sleep quality. The survival benefit from NIV in this group is much greater than that from currently available neuroprotective therapy, like riluzole. | Device | 1, 2 |
Primary outcome
quality of life
Time frame: 1 year
tolerance to NIV
Time frame: 1 year
number of hours of NIV per day
Time frame: 1 year
frequency of hospital admission
Time frame: 1 year
frequency of changing the ventilator settings by the operator.
Time frame: 1 year
3 secondary outcomes
survival
Time frame: 1 year
diurnal and nocturnal gas exchange
Time frame: 1 year
Pulmonary Function Tests (PFTs).
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2010.
Related trials for Amyotrophic Lateral Sclerosis
Not yet recruitingPhase 1
A Study of Single Ascending Doses of NI112 in Adults With Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS)
Recruiting
MindEx: A Novel, Multifocal, Cognitive Brain-Machine Interface System
Paralysis; Quadriplegic, Quadraplegia
RecruitingPhase 1/2
Study is to Assess the Safety and Tolerability of VTx-002 in Participants With ALS
Amyotrophic Lateral Sclerosis
RecruitingObservational study
Investigating Complex Neurodegenerative Disorders Related to Amyotrophic Lateral Sclerosis and Frontotemporal Dementia
Frontotemporal Dementia, Amyotrophic Lateral Sclerosis
Recruiting
Anticipated Patient and Caregiver Burden
Amyotrophic Lateral Sclerosis
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.
Stefano Nava principal investigator
Fondazione Salvatore Maugeri
Stefano Nava, MD principal investigator
Fondazione S.maugeri
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers and Non invasive ventilation in people with Amyotrophic Lateral Sclerosis and Chronic Respiratory Failure, run by Fondazione Salvatore Maugeri at 2 sites in Italy.
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 4 inclusion and 3 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 29 Mar 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Italy. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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 Stefano Nava, MD (0382 592 ext. 806, snava@fsm.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
quality of life (time frame: 1 year). The study lists 5 primary outcomes in all. 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 29 Mar 2010.