Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis

NCT ID: NCT00560287

Last Updated: 2010-03-29

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE4
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-01-31
• Study Completion Date: 2010-08-31

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

Conditions

Amyotrophic Lateral Sclerosis; Chronic Respiratory Failure

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: DOUBLE

Study Groups

1

Volume assist non-invasive ventilation

Group Type: ACTIVE_COMPARATOR

Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers

Intervention Type: DEVICE

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 ventilation

Intervention Type: DEVICE

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.

2

Pressure Assist mode

Group Type: ACTIVE_COMPARATOR

Non invasive ventilation delivered with one of the ventilator specifically designed for NIV and given to the patient by the home care providers

Intervention Type: DEVICE

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 ventilation

Intervention Type: DEVICE

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.

Interventions

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.

Intervention Type: 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.

Intervention Type: DEVICE

Other Intervention Names

Legendaire (Airox); Vivo (Breas); Elisee (Saime); Synchrony (Respironics); Legendaire (Airox); Vivo (Breas); Elisee (Saime); Synchrony (Respironics)

Eligibility Criteria

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

• Minimum Eligible Age: 20 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondazione Salvatore Maugeri

OTHER

Sponsor Role: lead

Responsible Party

Fondazione S.Maugerii

Principal Investigators

Stefano Nava

Role: PRINCIPAL_INVESTIGATOR

Fondazione Salvatore Maugeri

Stefano Nava, MD

Role: PRINCIPAL_INVESTIGATOR

Fondazione S.maugeri

Locations

Fondazione S.Maugeri

Pavia, , Italy

Site Status: RECRUITING

Respiratory Unit FSM

Pavia, , Italy

Site Status: NOT_YET_RECRUITING

Countries

Italy

Central Contacts

Stefano Nava, MD

Role: CONTACT

snava@fsm.it

0382 592 ext. 806

Franco Fanfulla, MD

Role: CONTACT

ffanfulla@fsm.it

0382 592 ext. 815

Facility Contacts

Stefano Nava, md

Role: primary

snava@fsm.it

o3825921

Stefano Nava, md

Role: backup

snava@fsm.it

03825921

Stefano Nava, md

Role: primary

snava@fsm.it

0382 592 ext. 806

Francesco Fanfulla, MD

Role: backup

ffanfulla@fsm.it

0382 592 ext. 815

Other Identifiers

190H09

Identifier Type: -

Identifier Source: secondary_id

190H09

Identifier Type: -

Identifier Source: org_study_id