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Status unknownNCT05033951INITIALSUpdated May 16, 2024

Optimizing INITIation of Non-invasive Ventilation in ALS Patients

An observational study in Amyotrophic Lateral Sclerosis, sponsored by University Medical Center Groningen. Status unknown at 1 site in Netherlands. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-05-16.

Sponsored by University Medical Center Groningen · Observational

The sponsor has not verified this record recently (last verified May 2024), so the status shown — last known as Recruiting — may be out of date.
Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
250
Ages
18 Years and older
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Amyotrophic Lateral Sclerosis

Keywords

  • Noninvasive ventilation
  • Quality of life
03

In context

Amyotrophic Lateral Sclerosis

1,000 studies on the registry are indexed under Amyotrophic Lateral Sclerosis; 284 are open to participants now.

This study's planned enrollment of 250 is above the median of 103 across 272 observational studies indexed under Amyotrophic Lateral Sclerosis.

Browse Amyotrophic Lateral Sclerosis studies →

Lead sponsor

University Medical Center Groningen is the lead sponsor of 625 studies on the registry; 174 are open to participants now.

Browse University Medical Center Groningen studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

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

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

Exclusion Criteria:

  • Use of NIV or invasive ventilation (tracheostomy) at time of first visit to a HMV centre.
  • Pregnancy
05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
250 participants (estimated)
Target follow-up
42 Months
Patient registry
Yes

Groups and cohorts

  • Cohort 1

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

    Other: Standard care

  • Cohort 2

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

    Other: Standard care

Interventions

  • OtherStandard care

    Both groups: standard care+ questionnaires

06

What researchers measure

Primary outcomes

  1. 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

    Time frame: 15 months

Secondary outcomes

  1. Survival

    Determine the effect of NIV on survival in patients with different ALS phenotypes.

    Time frame: 15 months

  2. 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

    Time frame: 15 months

  3. Forced vital capacity

    Pulmonary function based on FVC Min score: 0% Max score: 100% Lower scores mean a worse outcome

    Time frame: 15 months

  4. Carbondioxide

    Pulmonary function based on carbondioxide pCO2 in capillary bloodgas Min score: 4 kPa Max score: 9 kPa Higher score means a worse outcome

    Time frame: 15 months

  5. Anamnestic questions

    Pulmonary function based on anamnestic questions, Borg scale Min score: 0 Max score: 40 Higher score means a worse outcome

    Time frame: 15 months

  6. 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

    Time frame: 15 months

  7. Weight

    Weight in kg Min score: 40 Max score 130 Lower scores mean a worse outcome

    Time frame: 15 months

  8. 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

    Time frame: 15 months

  9. 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

    Time frame: 15 months

07

Study locations

1 of 1 sites recruiting
  • University medical centre Groningen
    Groningen, 9700RB, Netherlands
    • Peter wijkstra, MD PhD · Contact
    Recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Aug 2022 (actual). Primary completion is estimated for Apr 2025, 32 months after the start.

Of 131 non-industry observational studies under Amyotrophic Lateral Sclerosis, half reached primary completion within 65 months of starting (the median), counting those still running; a quarter within 30 and three quarters within 125. This study's planned 32 months is below that.

Counting every sponsor, half of 141 studies reached it within 61 months; a quarter within 29 and three quarters within 114.

How these figures are worked out, and which studies are left out of them.

Registered
Jul 7, 2021
First posted
Sep 5, 2021
StartActual
Aug 1, 2022
Primary completionEst.
Apr 1, 2025
CompletionEst.
Oct 1, 2025
Last update
May 16, 2024
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on May 16, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT05033951
Lead sponsor
University Medical Center Groningen
Responsible party
Peter Wijkstra (Prof. Dr. P.J. Wijkstra, University Medical Center Groningen) — Principal investigator
First posted
Sep 5, 2021
Start date
Aug 1, 2022
Primary completion
Apr 1, 2025 (estimated)
Completion
Oct 1, 2025 (estimated)
Last update
May 16, 2024

Study contacts

Rineke Jaspers Focks, MD
Contact
r.jaspersfocks@roessingh.nl
+31534875424
Peter Wijkstra, MD, PhD
Contact
p.j.wijkstra@umcg.nl
+31503613200

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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