Optimizing INITIation of Non-invasive Ventilation in ALS Patients

Amyotrophic Lateral Sclerosis Clinical Trial

— INITIALS  

Official title:

Optimizing INITIation of Non-invasive Ventilation in ALS Patients

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05033951
• Other study ID #: 202000124
• Status: Recruiting
• Start date: August 1, 2022
• Est. completion date: October 1, 2025

Study information

• Verified date: May 2024
• Source: University Medical Center Groningen
• Contact: Rineke Jaspers Focks, MD
• Phone: +31534875424
• Email: r.jaspersfocks[@]roessingh.nl
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 250
• Est. completion date: October 1, 2025
• Est. primary completion date: April 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Age > 18 years

• Diagnosis of ALS, PLS or PSMA

• Ability to give informed consent.

• Ability to fill in the questionnaires independently or with assistance of a caregiver.

Exclusion Criteria:

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

• Pregnancy

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Intervention

Other:
• Standard care
Both groups: standard care+ questionnaires

Locations

Country	Name	City	State
Netherlands	University medical centre Groningen	Groningen

Sponsors (1)

Lead Sponsor	Collaborator
University Medical Center Groningen

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Survival	Determine the effect of NIV on survival in patients with different ALS phenotypes.	15 months
Secondary	Overall function	Overall function decline based on the ALS Functional Rating Scale Revised (ALS-FRS-R).; Min score: 0 Max score: 48 Higher scores mean a worse outcome	15 months
Secondary	Forced vital capacity	Pulmonary function based on FVC Min score: 0% Max score: 100% Lower scores mean a worse outcome	15 months
Secondary	Carbondioxide	Pulmonary function based on carbondioxide pCO2 in capillary bloodgas Min score: 4 kPa Max score: 9 kPa Higher score means a worse outcome	15 months
Secondary	Anamnestic questions	Pulmonary function based on anamnestic questions, Borg scale Min score: 0 Max score: 40 Higher score means a worse outcome	15 months
Secondary	Bulbar function	Bulbar function based on questionnaire Center for Neurological Study Bulbar Functional Scale (CNS BFS).; Min score: 21 Max score: 105 Higher scores means a worse outcome	15 months
Secondary	Weight	Weight in kg Min score: 40 Max score 130 Lower scores mean a worse outcome	15 months
Secondary	General health Quality of Life in patients with severe respiratory insufficiency	Assessed by severe respiratory insufficiency questionnaire (SRI) Min score: 49 Max score: 245 Higher scores mean worse outcome	15 months
Secondary	General health Quality of Life	Assessed by in Short Form Health Survey (SF-36) Min score: 45 Max score: 198 Higher score means a worse outcome	15 months