Amyotrophic Lateral SclerosisINITIALSNCT05033951
Optimizing INITIation of Non-invasive Ventilation in ALS Patients
An observational study of Amyotrophic Lateral Sclerosis, run by University Medical Center Groningen at 1 site in Netherlands.
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- How many people
- 250 participants
- Where
- 1 siteNetherlands
- Study dates
- 1 Aug 2022to 1 Oct 2025
- How long will I take part?
- 42 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Patients must be diagnosed with Amyotrophic Lateral Sclerosis (ALS), Progressive Spinal Muscular Atrophy (PSMA) or Primary Lateral Sclerosis (PLS) by a neurologist. The patients will be included (after signing informed consent) during the first visit to one of the HMV centres in the Netherlands. Patients must to be able to participate and at least 18 years old
Who may be able to join
- 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.
Who cannot join
- Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.
- Pregnancy
Trial locations and contacts
1 site in 1 country.
Netherlands1 site, 1 recruiting
- Recruiting
University medical centre Groningen
Groningen, 9700RB
Peter wijkstra, MD PhD
About this study
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.
Read more
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.
Read the 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
- Being tested
- Standard care
- Keywords
- Noninvasive ventilation, Quality of life
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard careOther
Both groups: standard care+ questionnaires
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1 | Patients who start NIV in the first two months after the first visit to the HMV Receives: Other: Standard care | |
| Cohort 2 | Patients who do not start NIV in the first two months after the first visit to the HMV. Receives: Other: Standard care |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 15 months
Secondary outcomes (9)
Survival
Determine the effect of NIV on survival in patients with different ALS phenotypes.
Measured: 15 months
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
Measured: 15 months
Forced vital capacity
Pulmonary function based on FVC Min score: 0% Max score: 100% Lower scores mean a worse outcome
Measured: 15 months
Carbondioxide
Pulmonary function based on carbondioxide pCO2 in capillary bloodgas Min score: 4 kPa Max score: 9 kPa Higher score means a worse outcome
Measured: 15 months
Anamnestic questions
Pulmonary function based on anamnestic questions, Borg scale Min score: 0 Max score: 40 Higher score means a worse outcome
Measured: 15 months
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
Measured: 15 months
Weight
Weight in kg Min score: 40 Max score 130 Lower scores mean a worse outcome
Measured: 15 months
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
Measured: 15 months
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
Measured: 15 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2021
- Study start1 Aug 2022
- Primary completion1 Apr 2025 (estimated)
- Study completion1 Oct 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- More trials run by University Medical Center GroningenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 250 participants (estimated)
- Target duration
- 42 Months
Sponsor and investigators
- Lead sponsor
- University Medical Center Groningen
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Peter Wijkstra, University Medical Center Groningen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05033951
- Sponsor's protocol ID
- 202000124
- Acronym
- INITIALS
- Status last verified
- May 2024
- First submitted
- 7 Jul 2021
- Last updated
- 16 May 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Optimizing INITIation of Non-invasive Ventilation in ALS Patients
- ClinicalTrials.gov ID
- NCT05033951
- Acronym / sponsor ID
- INITIALS / 202000124
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- Noninvasive ventilation, Quality of life
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 250 participants (estimated)
- Lead sponsor
- University Medical Center GroningenUniversity, hospital or other
- Study start
- 1 Aug 2022 (actual)
- Primary completion
- 1 Apr 2025 (estimated)
- Study completion
- 1 Oct 2025 (estimated)
- First posted
- 5 Sep 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 42 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cohort 1 | – | Other: Standard carePatients who start NIV in the first two months after the first visit to the HMV |
| Cohort 2 | – | Other: Standard carePatients who do not start NIV in the first two months after the first visit to the HMV. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Patients must be diagnosed with Amyotrophic Lateral Sclerosis (ALS), Progressive Spinal Muscular Atrophy (PSMA) or Primary Lateral Sclerosis (PLS) by a neurologist. The patients will be included (after signing informed consent) during the first visit to one of the HMV centres in the Netherlands. Patients must to be able to participate and at least 18 years old
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (2 listed)
- Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.
- Pregnancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Standard careBoth groups: standard care+ questionnaires | Other | Cohort 1, Cohort 2 |
Primary outcome
Health related Quality of Life in patients with ALS
Time frame: 15 months
Assessed by Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40) Min score: 0 Max score: 160 Higher scores mean a worse outcome
9 secondary outcomes
Survival
Time frame: 15 months
Overall function
Time frame: 15 months
Forced vital capacity
Time frame: 15 months
Carbondioxide
Time frame: 15 months
Anamnestic questions
Time frame: 15 months
Bulbar function
Time frame: 15 months
Weight
Time frame: 15 months
General health Quality of Life in patients with severe respiratory insufficiency
Time frame: 15 months
General health Quality of Life
Time frame: 15 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Amyotrophic Lateral Sclerosis, run by University Medical Center Groningen at 1 site in Netherlands.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 16 May 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Rineke Jaspers Focks, MD (+31534875424, r.jaspersfocks@roessingh.nl), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Health related Quality of Life in patients with ALS (time frame: 15 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 May 2024.