Optimizing INITIation of Non-invasive Ventilation in ALS Patients

NCT ID: NCT05033951

Last Updated: 2024-05-16

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 250 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-08-01
• Study Completion Date: 2025-10-01

Brief 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.

Detailed 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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Cohort 1

Patients who start NIV in the first two months after the first visit to the HMV

Standard care

Intervention Type: OTHER

Both groups: standard care+ questionnaires

Cohort 2

Patients who do not start NIV in the first two months after the first visit to the HMV.

Standard care

Intervention Type: OTHER

Both groups: standard care+ questionnaires

Interventions

Standard care

Both groups: standard care+ questionnaires

Intervention Type: OTHER

Eligibility Criteria

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

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

Sponsors

University Medical Center Groningen

OTHER

Sponsor Role: lead

Responsible Party

Peter Wijkstra

Prof. Dr. P.J. Wijkstra

Responsibility Role: PRINCIPAL_INVESTIGATOR

Locations

University medical centre Groningen

Groningen, , Netherlands

Site Status: RECRUITING

Countries

Netherlands

Central Contacts

Rineke Jaspers Focks, MD

Role: CONTACT

r.jaspersfocks@roessingh.nl

+31534875424

Peter Wijkstra, MD, PhD

Role: CONTACT

p.j.wijkstra@umcg.nl

+31503613200

Facility Contacts

Peter wijkstra, MD PhD

Role: primary

Other Identifiers

202000124

Identifier Type: -

Identifier Source: org_study_id