Optimizing INITIation of Non-invasive Ventilation in ALS Patients (INITIALS)

May 14, 2024 updated by: Peter Wijkstra, University Medical Center Groningen

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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 Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
250

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Netherlands
      • Groningen, Netherlands, 9700RB
        • Recruiting
        • University Medical Centre Groningen
        • Contact:
          • Peter wijkstra, MD PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years and older (Adult, Older Adult)

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Cohort 1
Patients who start NIV in the first two months after the first visit to the HMV
Other: Standard care
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.
Other: Standard care
Both groups: standard care+ questionnaires

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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
15 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Survival
Time Frame: 15 months
Determine the effect of NIV on survival in patients with different ALS phenotypes.
15 months
Overall function
Time Frame: 15 months

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
Forced vital capacity
Time Frame: 15 months
Pulmonary function based on FVC Min score: 0% Max score: 100% Lower scores mean a worse outcome
15 months
Carbondioxide
Time Frame: 15 months
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
Anamnestic questions
Time Frame: 15 months
Pulmonary function based on anamnestic questions, Borg scale Min score: 0 Max score: 40 Higher score means a worse outcome
15 months
Bulbar function
Time Frame: 15 months

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
Weight
Time Frame: 15 months
Weight in kg Min score: 40 Max score 130 Lower scores mean a worse outcome
15 months
General health Quality of Life in patients with severe respiratory insufficiency
Time Frame: 15 months
Assessed by severe respiratory insufficiency questionnaire (SRI) Min score: 49 Max score: 245 Higher scores mean worse outcome
15 months
General health Quality of Life
Time Frame: 15 months
Assessed by in Short Form Health Survey (SF-36) Min score: 45 Max score: 198 Higher score means a worse outcome
15 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University Medical Center Groningen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 1, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 1, 2025

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
October 1, 2025

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 7, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
September 5, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 16, 2024

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 14, 2024

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 202000124

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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