Optimización de la INICIACIÓN de la ventilación no invasiva en pacientes con ELA

ID de estudio #: NCT05033951

condición: La esclerosis lateral amiotrófica

Estado: Aún no está reclutando

Objective: The primary objective in this study is to identify which (pheno)type of ALS patient has the most benefit from NIV in improving quality of life.

Study population: Adult patients with ALS, PLS (Primary Lateral Sclerosis) of PSMA (Progressive Spinal Muscular Atrophy) in the Netherlands. Patients will be included during their first visit to one of the HMV centres in the Netherlands.

Main study parameters/endpoints: The main study parameter is change in Quality of Life (QoL) defined as change in ALS Assessment Questionnaire (ALSAQ-40) in patients with ALS after initiation of NIV.

Design: Multi-centre prospective cohort study consisting of 2 non-randomized cohorts, i.e. ALS patients who start NIV and ALS patients who do not start NIV at the time of inclusion in the present study.

Duration: The total duration of the study: 3,5 years. Inclusion period: 30 months. Follow up time after initiation of NIV: 9 months. Thereafter, 6 months will be used for data analysis.

Setting: Involvement of all (4) HMV centres in the Netherlands (Groningen, Maastricht, Rotterdam, Utrecht).

Procedure: 250 ALS patients will be included. During the first regular visit to the HMV centre patients will be asked to participate in the study. Data will be recorded during regular visits to the HMV centre.

intervención: Standard care

Resultados: https://clinicaltrials.gov/ct2/show/results/NCT05033951

última actualización: Febrero

fecha de inicio: 1 de octubre de 2021

finalización estimada: 1 de octubre de 2025

última actualización: 5 de septiembre de 2021

tamaño / inscripción: 250

descripción del estudio: Primera visita:

After informed consent the following data which are gathered as parts of usual care will be recorded in the case report form (CRF):

Función pulmonar:

FVC ( % predicted) upright and supine
capillary blood gas analysis, room air (pH, pCO2, HCO3-, pO2)
anamnestic questions (Borg scale 0-4)
Género sentido
Edad
Percutaneous Endoscopic Gastrostomy (PEG)/ Percutaneous Radiological Gastrostomy (PRG) tube: yes/no
Medicamentos
Weight/ BMI
Situación de vida
Estado civil

The following clinical characteristics from their medical record (from neurologist or rehabilitation specialist):

Date of diagnosis
Type of ALS at onset
ALSFRS-R at the time of diagnosis
Pulmonary function at the time of diagnosis
Cognitive status (ECAS, ALS-FTD-Q)
Historial clínico

Validated questionnaires (online):

Quality of life questionnaires (SF 36, ALSAQ-40)
Respiratory insufficiency questionnaire (SRI)
ALS FRS R (patient version)
Bulbar symptoms questionnaire (CNS BFS)

After the first visit patients will participate in one of the two cohorts (non-randomized):

Cohort 1: patients who start NIV in the first two months after the first visit to the HMV
Cohort 2: patients who do not start NIV in the first two months after the first visit to the HMV.

Cohorte 1:

Data will be recorded at the moment of initiation of NIV and 3, 6 and 9 months after initiation. Data will be recorded during regular visits (to the HMV centre or at the patients home).

Función pulmonar:

capillary blood gas, room air (pH, pCO2 (kPa/ mmHg), HCO3- (mmol/l), SaO2 (%))
anamnestic questions (Borg scale 0-4)
Nocturnal gas exchange by transcutaneous measurement, at the patients home - Mean tcpCO2 (kPa), Maximal tcpCO2 (kPa), mean oxygen saturation (%), lowest oxygen saturation (%)
Data from NIV equipment (compliance, modus, IPPA, EPAP, freq, mask type)

Use of NIV

hours a day/night
In case of no use: why not?
PEG/ PRG tube: yes/no
Supervivencia
Tracheostomy or not
Peso

Validated online questionnaires:

Quality of life questionnaires (SF 36, ALSAQ-40)
Respiratory insufficiency questionnaire (SRI)
ALS FRS R (patient version)
Bulbar symptoms questionnaire (CNS BFS)
Nocturnal gas exchange, transcutaneous, at the patients home

Cohorte 2:

Data will be recorded every 3 months until initiation of NIV or death or max 1,5 years after the first visit to the HMV.

Data will be recorded during regular visits (to the HMV centre or at the patients home).

Función pulmonar:

Capillary blood gas (pH, pCO2 (kPa/ mmHg), HCO3- (mmol/l), SaO2 (%))
anamnestic questions (Borg scale 0-4)
Nocturnal gas exchange by transcutaneous measurement, at the patients home - tcpCO2 (kPa), Maximal tcpCO2 (kPa), oxygen saturation (%), lowest oxygen saturation (%)
PEG/ PRG tube: yes/no
Supervivencia
Tracheostomy or not
Peso
Cognitive status (from medical record)
Reason for not starting NIV

Validated online questionnaires:

Quality of life questionnaires (SF 36, ALSAQ-40)
Respiratory insufficiency questionnaire (SRI)
ALS FRS R (patient version)
Bulbar symptoms questionnaire (CNS BFS)
Nocturnal gas exchange, transcutaneous, at the patients home

If a patient in cohort 2 will start with NIV, the patient will be transferred to cohort 1.

resultados primarios:

• Health related Quality of Life in patients with ALS
  Assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) Min score: 0 Max score: 160 Higher scores mean a worse outcome
• 15 meses
  

resultados secundarios:

• Supervivencia
  15 meses
• Overall function
  15 meses
• Capacidad vital forzada
  15 meses
• Dióxido de carbono
  15 meses
• Anamnestic questions
  15 meses
• Bulbar function
  15 meses
• Peso
  15 meses
• General health Quality of Life in patients with severe respiratory insufficiency
  15 meses
• General health Quality of Life
  15 meses

criterios de inclusión:

Criterio de exclusión: Criterios:

Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.
Embarazo

patrocinador: Centro Médico Universitario Groningen

Contactos: Rineke Jaspers Focks, MD, + 31534875424, r.jaspersfocks@roessingh.nl

ubicaciones del centro de prueba: