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

NCT01641965 | Completed DMC

• 1 other identifier: AC 111/11
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Amyotrophic Lateral Sclerosis

Outcome Measures

Primary Outcomes (1)

• survival until death or tracheostomy

  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 Outcomes (1)

• effects from early use of NIV in progression of respiratory muscle weakness

  three years

Study Arms (2)

early non invasive ventilation

ACTIVE COMPARATOR

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)

standard

ACTIVE COMPARATOR

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)

Interventions

Home pressure ventilator model Vivo 40 (BREAS Medical AB) DEVICE

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.

early non invasive ventilation; standard

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Hospital Universitari de Bellvitge: lead
• Fondo de Investigacion Sanitaria: collaborator

Study Sites (1)

Hospital Universitari Bellvitge

L'Hospitalet de Llobregat, Barcelona, 08907, Spain

Location

Related Publications (1)

• Sarasate M, Gonzalez N, Cordoba-Izquierdo A, Prats E, Gonzalez-Moro JMR, Marti S, Lujan M, Calle M, Anton A, Povedano M, Farrero E. Impact of Early Non-Invasive Ventilation in Amyotrophic Lateral Sclerosis: A multicenter Randomized Controlled Trial. J Neuromuscul Dis. 2023;10(4):627-637. doi: 10.3233/JND-221658.

  PMID: 37212068 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Eva Farrero, MD

  Hospital Universitari Bellvitge

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Medical Doctor

Study Record Dates

• First Submitted: July 13, 2012
• First Posted: July 17, 2012
• Study Start: April 1, 2012
• Primary Completion: December 1, 2015
• Study Completion: December 1, 2015
• Last Updated: February 22, 2016

Record last verified: 2016-02

Data Sharing

• IPD Sharing: Will not share

Locations

ES (1)