Impact of Early Non Invasive Ventilation in Amyotrophic Lateral Sclerosis (ALS) Patients

February 19, 2016 updated by: Eva Farrero Munoz, Hospital Universitari de Bellvitge

Impact of Early Non-invasive Ventilation in Amyotrophic Lateral Sclerosis Patients: a Randomized Controlled Trial

Timing of initiating domiciliary no invasive ventilation (NIV) in amyotrophic lateral sclerosis patients remains unclear. The hypothesis of the study is that the early use of NIV, in the initial phase of respiratory muscle weakness, improves the prognosis of ALS patients.

Principal objective: To evaluate the impact of early NIV in the survival of ALS patients.

Secondary objectives:To determine the effects from early NIV in the progression of respiratory muscle weakness. To analyze the impact of early NIV in the quality of life of ALS patients. To evaluate the correlation between the FVC and other parameters of respiratory evaluation (maximal inspiratory pressure (MIP), sniff nasal inspiratory pressure (SNP), nocturnal desaturation) and their utility in the early indication of the NIV. To evaluate the tolerance to the early NIV.

Methods: multicentric, randomized, open-label, controlled clinical trial with a parallel treatment design. Patients will be included when their FVC reaches the threshold of the 75% of the predicted value and will be randomized in: Group A: the NIV treatment will begin immediately and Group B: the NIV treatment will be started when patients fulfil at least one of the following criteria: (i) FVC < 50% predicted, (ii) orthopnea, and/or (iii) PaCO2 > 45 mmHg. Follow-up visits will be done every three months with pulmonary function test, nocturnal pulse oximetry, quality of life and quality of sleep tests, assessment of disease progression (ALSFSR-R scale), tolerance and compliance with NIV.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

ALS patients with early impairment of respiratory muscles (FVC around 75%) will be radomised to initiate NIV or conventional follow-up.

The NIV treatment will be initiated during a hospital with the model of ventilator VIVO 40 (BREAS Medical AD, Sweden) using a pressure support assisted/control mode, with a minimum pressure support 10 cm.

Initial NIV indication in both groups of patients will nocturnal. Patients will be defined as tolerant to NIV if they do not refer inconveniences and is able to asleep with NIV.

The efficacy of the intervention will be compared in both groups with survival until death or change to invasive tracheal ventilation as a prymary efficacy parameter.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
42

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
    • Barcelona
      • L'Hospitalet de Llobregat, Barcelona, Spain, 08907
        • Hospital Universitari Bellvitge

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 80 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Participant is willing and able to give informed consent for participation in the study
  • Definite ALS diagnosis according to El Escorial Criteria
  • Ability to understand and perform the pulmonary function test
  • FVC ≤ 75% (with FVC registry >75% documented within the six previous months)

Exclusion Criteria:

  • Major comorbidity (non-related with ALS) that can shorten life expectancy
  • Cognitive impairment that prevents the patient to understand and perform the study procedures including technically acceptable pulmonary function tests (FVC, MIP, SNP, PCF)
  • Patient refusal of NIV treatment
  • Previous respiratory or cardiac diseases with known impaired spirometry
  • Indication of NIV according to standard criteria (PaCO2 > 45 mmHg, FVC < 50%, orthopnea)
  • ALS with slow disease progression (more than 3 years)
  • Participation in another clinical trial

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: NONE

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
ACTIVE_COMPARATOR: early non invasive ventilation
Patients assigned to this arm will start non invasive ventilation with home pressure ventilator model Vivo 40 (BREAS Medical AB)immediately after randomization (when their FVC reaches the threshold of the 75% of the predicted value)
Device: Home pressure ventilator model Vivo 40 (BREAS Medical AB)
Nocturnal non invasive ventilation (Vivo 40)with minimum pressure support of 10 cm H2O (IPAP 14, EPAP 4). Parameters will be adjusted according to tolerance and to achieve PaCO2 < 45 mmHg and improve symptoms.
ACTIVE_COMPARATOR: standard
patients in this arm will start non invasive ventilation with home pressure ventilator model Vivo 40 (BREAS Medical AB) when they fulfil at least one of the following criteria: (i) FVC < 50% predicted, (ii) orthopnea, and/or (iii) PaCO2 > 45 mmHg.
Device: Home pressure ventilator model Vivo 40 (BREAS Medical AB)
Nocturnal non invasive ventilation (Vivo 40)with minimum pressure support of 10 cm H2O (IPAP 14, EPAP 4). Parameters will be adjusted according to tolerance and to achieve PaCO2 < 45 mmHg and improve symptoms.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
survival until death or tracheostomy
Time Frame: three years
To evaluate the impact of the early use of NIV in the survival function of the time until death or tracheostomy of ALS patients, compared with a standard initiation of NIV.
three years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
effects from early use of NIV in progression of respiratory muscle weakness
Time Frame: three years
To determine the effects from early use of NIV in progression of respiratory muscle weakness, measured by rate of decline in FVC
three years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Hospital Universitari de Bellvitge

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Fondo de Investigacion Sanitaria

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Eva Farrero, MD, Hospital Universitari Bellvitge

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 1, 2012

Primary Completion (ACTUAL)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2015

Study Completion (ACTUAL)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2015

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 13, 2012

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 16, 2012

First Posted (ESTIMATE)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 17, 2012

Study Record Updates

Last Update Posted (ESTIMATE)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
February 22, 2016

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 19, 2016

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
February 1, 2016

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • AC 111/11

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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