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

NCT ID: NCT01641965

Last Updated: 2016-02-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: NA
• Total Enrollment: 42 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2012-04-30
• Study Completion Date: 2015-12-31

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.

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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)

Group Type: ACTIVE_COMPARATOR

Home pressure ventilator model Vivo 40 (BREAS Medical AB)

Intervention Type: 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.

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.

Group Type: ACTIVE_COMPARATOR

Home pressure ventilator model Vivo 40 (BREAS Medical AB)

Intervention Type: 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.

Interventions

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.

Intervention Type: DEVICE

Eligibility 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Fondo de Investigacion Sanitaria

OTHER

Sponsor Role: collaborator

Hospital Universitari de Bellvitge

OTHER

Sponsor Role: lead

Responsible Party

Eva Farrero Munoz

Medical Doctor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Eva Farrero, MD

Role: PRINCIPAL_INVESTIGATOR

Hospital Universitari Bellvitge

Locations

Hospital Universitari Bellvitge

L'Hospitalet de Llobregat, Barcelona, Spain

Countries

Spain

References

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.

Reference Type: DERIVED

PMID: 37212068 (View on PubMed)

Other Identifiers

AC 111/11

Identifier Type: -

Identifier Source: org_study_id