Optimizing INITIation of Non-invasive Ventilation in ALS Patients

study id #: NCT05033951

condition: Amyotrophic Lateral Sclerosis

status: Not yet recruiting

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.

intervention: Standard care

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

last updated: February 04, 2022

start date: October 1, 2021

estimated completion: October 1, 2025

last updated: September 5, 2021

size / enrollment: 250

study description: First visit:

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

Pulmonary function:

FVC ( % predicted) upright and supine
capillary blood gas analysis, room air (pH, pCO2, HCO3-, pO2)
anamnestic questions (Borg scale 0-4)
Gender
Age
Percutaneous Endoscopic Gastrostomy (PEG)/ Percutaneous Radiological Gastrostomy (PRG) tube: yes/no
Medication
Weight/ BMI
Living situation
Civil status

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)
Medical history

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.

Cohort 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).

Pulmonary function:

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
Survival
Tracheostomy or not
Weight

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

Cohort 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).

Pulmonary function:

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
Survival
Tracheostomy or not
Weight
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.

primary outcomes:

• 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 months
  

secondary outcomes:

• Survival
  15 months
• Overall function
  15 months
• Forced vital capacity
  15 months
• Carbondioxide
  15 months
• Anamnestic questions
  15 months
• Bulbar function
  15 months
• Weight
  15 months
• General health Quality of Life in patients with severe respiratory insufficiency
  15 months
• General health Quality of Life
  15 months

inclusion criteria:

exclusion criteria: Criteria:

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

sponsor: University Medical Center Groningen

contacts: Rineke Jaspers Focks, MD, +31534875424, [email protected]

trial center locations: