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

This study is currently recruiting participants.
Verified July 2012 by Hospital Universitari de Bellvitge
Sponsor:
Collaborator:
Fondo de Investigacion Sanitaria
Information provided by (Responsible Party):
Eva Farrero Munoz, Hospital Universitari de Bellvitge
ClinicalTrials.gov Identifier:
NCT01641965
First received: July 13, 2012
Last updated: July 19, 2012
Last verified: July 2012
History of Changes
  Purpose

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.


Condition Intervention
Amyotrophic Lateral Sclerosis
 Device: Home pressure ventilator model Vivo 40 (BREAS Medical AB)

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Impact of Early Non-invasive Ventilation in Amyotrophic Lateral Sclerosis Patients: a Randomized Controlled Trial

Resource links provided by NLM:

U.S. FDA Resources

Further study details as provided by Hospital Universitari de Bellvitge:

Primary Outcome Measures:
  • survival until death or tracheostomy [ Time Frame: three years ] [ Designated as safety issue: No ]
    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.


Secondary Outcome Measures:
  • effects from early use of NIV in progression of respiratory muscle weakness [ Time Frame: three years ] [ Designated as safety issue: No ]
    To determine the effects from early use of NIV in progression of respiratory muscle weakness, measured by rate of decline in FVC


Estimated Enrollment: 72
Study Start Date: April 2012
Estimated Primary Completion Date: April 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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.

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01641965

Contacts
Contact: Eva Farrero, MD 0034 93 2607576 efarrero@bellvitgehospital.cat

Locations
Spain
Hospital Universitari Bellvitge Recruiting
L'Hospitalet de Llobregat, Barcelona, Spain, 08907
Contact: Eva Farrero, MD     0034 93 2607576     efarrero@bellvitgehospital.cat    
Sub-Investigator: Nuria Gonzalez, MD            
Sponsors and Collaborators
Hospital Universitari de Bellvitge
Fondo de Investigacion Sanitaria
  More Information

No publications provided

Responsible Party: Eva Farrero Munoz, Medical Doctor, Hospital Universitari de Bellvitge
ClinicalTrials.gov Identifier: NCT01641965     History of Changes
Other Study ID Numbers: AC 111/11
Study First Received: July 13, 2012
Last Updated: July 19, 2012
Health Authority: Spain: Ethics Committee

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
Spinal Cord Diseases
Central Nervous System Diseases
Nervous System Diseases
 Neurodegenerative Diseases
TDP-43 Proteinopathies
Neuromuscular Diseases
Proteostasis Deficiencies
Metabolic Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on June 18, 2013