Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis

The recruitment status of this study is unknown because the information has not been verified recently.
Verified March 2010 by Fondazione Salvatore Maugeri.
Recruitment status was  Recruiting
Sponsor:
Information provided by:
Fondazione Salvatore Maugeri
ClinicalTrials.gov Identifier:
NCT00560287
First received: November 16, 2007
Last updated: March 26, 2010
Last verified: March 2010

November 16, 2007
March 26, 2010
January 2008
August 2009   (final data collection date for primary outcome measure)
  • quality of life [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • tolerance to NIV [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • number of hours of NIV per day [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • frequency of hospital admission [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • frequency of changing the ventilator settings by the operator. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
quality of life 2) tolerance to NIV 3) number of hours of NIV per day 4) frequency of hospital admission 5) frequency of changing the ventilator settings by the operator. [ Time Frame: 1 year ]
Complete list of historical versions of study NCT00560287 on ClinicalTrials.gov Archive Site
  • survival [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • diurnal and nocturnal gas exchange [ Time Frame: 1 year ] [ Designated as safety issue: No ]
  • Pulmonary Function Tests (PFTs). [ Time Frame: 1 year ] [ Designated as safety issue: No ]
1) survival 2) diurnal and nocturnal gas exchange e 3) Pulmonary Function Tests (PFTs). [ Time Frame: 1 year ]
Not Provided
Not Provided
 
Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis
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.

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.

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

Interventional
Phase 4
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Caregiver, Outcomes Assessor)
Primary Purpose: Supportive Care
  • Amyotrophic Lateral Sclerosis
  • Chronic Respiratory Failure
  • Device: Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers
    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.
    Other Names:
    • Legendaire (Airox)
    • Vivo (Breas)
    • Elisee (Saime)
    • Synchrony (Respironics)
  • Device: Non invasive ventilation
    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.
    Other Names:
    • Legendaire (Airox)
    • Vivo (Breas)
    • Elisee (Saime)
    • Synchrony (Respironics)
  • Active Comparator: 1
    Volume assist non-invasive ventilation
    Interventions:
    • 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
  • Active Comparator: 2
    Pressure Assist mode
    Interventions:
    • 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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
50
August 2010
August 2009   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • Life expectancy>12 months
  • Any comorbidity
  • Acute Respiratory Failure
Both
20 Years to 80 Years
No
Contact: Stefano Nava, MD 0382 592 ext 806 snava@fsm.it
Contact: Franco Fanfulla, MD 0382 592 ext 815 ffanfulla@fsm.it
Italy
 
NCT00560287
190H09, 190H09
Yes
Stefano Nava, Fondazione S.Maugerii
Fondazione Salvatore Maugeri
Not Provided
Principal Investigator: Stefano Nava Fondazione Salvatore Maugeri
Principal Investigator: Stefano Nava, MD Fondazione S.maugeri
Fondazione Salvatore Maugeri
March 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP