MedPath

Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis

Phase 4
Conditions
Amyotrophic Lateral Sclerosis
Chronic Respiratory Failure
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
Registration Number
NCT00560287
Lead Sponsor
Fondazione Salvatore Maugeri
Brief 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 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

Recruitment & Eligibility

Status
UNKNOWN
Sex
All
Target Recruitment
50
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
1Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providersVolume assist non-invasive ventilation
1Non invasive ventilationVolume assist non-invasive ventilation
2Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providersPressure Assist mode
2Non invasive ventilationPressure Assist mode
Primary Outcome Measures
NameTimeMethod
quality of life1 year
tolerance to NIV1 year
number of hours of NIV per day1 year
frequency of hospital admission1 year
frequency of changing the ventilator settings by the operator.1 year
Secondary Outcome Measures
NameTimeMethod
survival1 year
diurnal and nocturnal gas exchange1 year
Pulmonary Function Tests (PFTs).1 year

Trial Locations

Locations (2)

Fondazione S.Maugeri

🇮🇹

Pavia, Italy

Respiratory Unit FSM

🇮🇹

Pavia, Italy

© Copyright 2025. All Rights Reserved by MedPath