Effects of Long Term Ventilation Support on the Quality of Life of ALS Patients and Their Families (ALS-LTMV)

Amyotrophic lateral sclerosis (ALS) is a serious rapidly progressive disease of the nervous system. The average survival from the time of diagnosis is two to three years. The patient physical and psychological sufferings in ALS are immense, and apart from Riluzole, there is no effective treatment. Care of advanced ALS have an estimated cost of 4-8 million NOK per year. Perhaps the most challenging topic of ALS care is the decision to extend ventilation support into the stages of disease that require treatment both during day and night. In these cases, treatment is clearly life-sustaining and although quality of life may be maintained, the burden of caregiving imposed upon family or health care workers is huge, regardless of tracheostomy (TIV) or non-invasive (NIV) modality.

The present study is a longitudinal questionnaire study in Norway measuring overall quality of life, health-related quality of life, and disease-specific quality of life in ALS patients, partners and children before and after the introduction of life sustaining ventilation support. The investigators aim to increase the knowledge on how life-sustaining ventilation support with NIV or TIV affects the quality of life in ALS patients, life partners and children. The results from the study may provide crucial information for clinicians and patients on one of the most difficult ethical issues of ALS treatment. The investigators anticipate that this information will facilitate a shared decision making processes, weighing benefits and disadvantages in a wider perspective.

Study Overview

• Status: Recruiting
• Conditions: Nervous System Diseases; Neurodegenerative Diseases; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Spinal Cord Diseases; TDP-43 Proteinopathies
• Intervention / Treatment: Device: Long term mechanical ventilation support; Device: No long term mechanical ventilation support

• Study Type: Interventional
• Enrollment (Estimated): 200
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Ole-Bjørn Tysnes; Phone Number: + 47 55975063; Email: ole-bjorn.tysnes@helse-bergen.no
• Study Contact Backup: Name: Tale Litlere Bjerknes; Phone Number: +47 55975045; Email: tale.litlere.bjerknes@helse-bergen.no

Study Locations

• Norway
  • Bergen, Norway
    • Recruiting
    • Haukeland University Hospital
    • Contact: Ole-Bjørn Tysnes; Phone Number: +4755975063; Email: ole-bjorn.tysnes@helse-bergen.no
  • Lørenskog, Norway
    • Recruiting
    • Akershus University Hospital
    • Principal Investigator: Ola Nakken
    • Contact: Ola Nakken
  • Oslo, Norway
    • Recruiting
    • Oslo University Hospital
    • Principal Investigator: Angelina Maniaol
    • Contact: Angelina Maniaol
  • Sarpsborg, Norway, 1714
    • Not yet recruiting
    • Østfold Hospital Kalnes
    • Contact: Sophie Cranner Seip, MD; Phone Number: +47 69 86 00 00; Email: Sophie.Cranner.Seip@so-hf.no
  • Stavanger, Norway
    • Recruiting
    • Stavanger University Hospital
    • Principal Investigator: Katrin Ruth Schlüter
    • Contact: Katrin Ruth Schlüter
  • Tromsø, Norway
    • Recruiting
    • Universitetssykehuset Nord-Norge
    • Contact: Agnethe Eltoft, MD; Phone Number: +4777627075; Email: agnethe.eltoft@unn.no
    • Contact: Monica Vold, MD; Email: monica.linnea.vold@unn.no
    • Principal Investigator: Agnethe Eltoft, MD
    • Principal Investigator: Monica Vold, MD
  • Trondheim, Norway
    • Recruiting
    • St. Olavs Hospital
    • Contact: Eivind Brønstad, MD PhD
    • Principal Investigator: Eivind Brønstad, MD PhD
  • Nordland
    • Bodø, Nordland, Norway, 8005
      • Recruiting
      • Nordland Hospital Bodø
      • Contact: Ane Sjulstad, MD; Phone Number: +47 75571000; Email: Ane.Skaare.Sjulstad@nordlandssykehuset.no
  • Vest Agder
    • Kristiansand, Vest Agder, Norway
      • Recruiting
      • Sørlandet Hospital Trust
      • Contact: Hildegunn Øverlie, MD; Phone Number: + 47 38073000; Email: hildegunn.overlie@sshf.no

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 8 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: Yes

Description

Inclusion criteria for patients:

1. A clinical diagnosis of probable ALS according to the revised El Escorial criteria
2. Progression of the illness leading the consulting physician to offer treatment with LTMV
3. Can communicate in Norwegian

Inclusion criteria for partners of ALS patients:

1. Partner of a patient with ALS with progression of the illness leading the consulting physician to offer treatment with LTMV
2. Can communicate in Norwegian

Inclusion criteria for children:

1. Children from 8 years and older having a parent who suffers from ALS with progression of the illness leading the consulting physician to offer treatment with LTMV
2. Can communicate in Norwegian

Exclusion criteria for patients, partners and children of ALS patients:

1. Potential participants with cognitive impairment or dementia.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: ALS patients that choose life prolonging treatment with LTMV and their families	Device: Long term mechanical ventilation support; Patients that choose life prolonging treatment with long term mechanical ventilation support
Active Comparator: ALS patients that decline life prolonging treatment with LTMV and their families	Device: No long term mechanical ventilation support; Patients that decline life prolonging treatment with long term mechanical ventilation support

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall quality of life assessed by the "Quality of Life Scale"	"Quality of Life Scale" total score (range 16-112). Higher score indicates better quality of life.	At inclusion
Overall quality of life assessed by the "Quality of Life Scale"	"Quality of Life Scale" total score (range 16-112). Higher score indicates better quality of life.	3 months after inclusion
Overall quality of life assessed by the "Quality of Life Scale"	"Quality of Life Scale" total score (range 16-112). Higher score indicates better quality of life.	9 months after inclusion
Overall quality of life assessed by the "Quality of Life Scale"	"Quality of Life Scale" total score (range 16-112). Higher score indicates better quality of life.	15 months after inclusion
Overall quality of life assessed by the "Quality of Life Scale"	"Quality of Life Scale" total score (range 16-112). Higher score indicates better quality of life.	21 months after inclusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Health-related quality of life assessed by the "EQ-5D-5L"	"EQ-5D-5L" index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life.	At inclusion
Health-related quality of life assessed by the "EQ-5D-5L"	"EQ-5D-5L" index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life.	3 months after inclusion
Health-related quality of life assessed by the "EQ-5D-5L"	"EQ-5D-5L" index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life.	9 months after inclusion
Health-related quality of life assessed by the "EQ-5D-5L"	"EQ-5D-5L" index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life.	15 months after inclusion
Health-related quality of life assessed by the "EQ-5D-5L"	"EQ-5D-5L" index score and EQ-VAS score (range 0-100) where higher scores indicates better health-related quality of life.	21 months after inclusion
Disease-specific quality of life assessed by "Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5)"	"ALSAQ-5" score (range 0-100), with 0 reflecting the best health state	At inclusion
Disease-specific quality of life assessed by "Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5)"	"ALSAQ-5" score (range 0-100), with 0 reflecting the best health state	3 months after inclusion
Disease-specific quality of life assessed by "Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5)"	"ALSAQ-5" score (range 0-100), with 0 reflecting the best health state	9 months after inclusion
Disease-specific quality of life assessed by "Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5)"	"ALSAQ-5" score (range 0-100), with 0 reflecting the best health state	15 months after inclusion
Disease-specific quality of life assessed by "Amyotrophic lateral sclerosis assessment scale - 5 items (ALSAQ-5)"	"ALSAQ-5" score (range 0-100), with 0 reflecting the best health state	21 months after inclusion
Health-related quality of life assessed by "Severe Respiratory Insufficiency Questionnaire"	"Severe Respiratory Insufficiency Questionnaire" score (range 0-100), with 100 reflecting the best health state.	At inclusion
Health-related quality of life assessed by "Severe Respiratory Insufficiency Questionnaire"	"Severe Respiratory Insufficiency Questionnaire" score (range 0-100), with 100 reflecting the best health state.	3 months after inclusion
Health-related quality of life assessed by "Severe Respiratory Insufficiency Questionnaire"	"Severe Respiratory Insufficiency Questionnaire" score (range 0-100), with 100 reflecting the best health state.	9 months after inclusion
Health-related quality of life assessed by "Severe Respiratory Insufficiency Questionnaire"	"Severe Respiratory Insufficiency Questionnaire" score (range 0-100), with 100 reflecting the best health state.	15 months after inclusion
Health-related quality of life assessed by "Severe Respiratory Insufficiency Questionnaire"	"Severe Respiratory Insufficiency Questionnaire" score (range 0-100), with 100 reflecting the best health state.	21 months after inclusion
Health-related quality of life assessed by "Kidsscreen-27"	"Kidsscreen-27" score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life.	At inclusion
Health-related quality of life assessed by "Kidsscreen-27"	"Kidsscreen-27" score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life.	3 months after inclusion
Health-related quality of life assessed by "Kidsscreen-27"	"Kidsscreen-27" score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life.	9 months after inclusion
Health-related quality of life assessed by "Kidsscreen-27"	"Kidsscreen-27" score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life.	15 months after inclusion
Health-related quality of life assessed by "Kidsscreen-27"	"Kidsscreen-27" score where mean (±SD) scores of 50 ± 10 define normality for children and adolescents aged 8-18 years across Europe. Higher scores indicate a better health-related quality of life.	21 months after inclusion
Caregiver burden assessed by "Zarit Burden Interview"	"Zarit Burden Interview" total score (range 0-88 where a higher score indicates greater burden).	At inclusion
Caregiver burden assessed by "Zarit Burden Interview"	"Zarit Burden Interview" total score (range 0-88 where a higher score indicates greater burden).	3 months after inclusion
Caregiver burden assessed by "Zarit Burden Interview"	"Zarit Burden Interview" total score (range 0-88 where a higher score indicates greater burden).	9 months after inclusion
Caregiver burden assessed by "Zarit Burden Interview"	"Zarit Burden Interview" total score (range 0-88 where a higher score indicates greater burden).	15 months after inclusion
Caregiver burden assessed by "Zarit Burden Interview"	"Zarit Burden Interview" total score (range 0-88 where a higher score indicates greater burden).	21 months after inclusion

Collaborators and Investigators

• Sponsor: Haukeland University Hospital
• Collaborators: Oslo University Hospital; University Hospital of North Norway; Helse Stavanger HF; St. Olavs Hospital; University Hospital, Akershus; Nordland Hospital, Bodø; Hospital of Southern Norway Kristiansand; Østfold Hospital Kalnes
• Investigators: Principal Investigator: Ole-Bjørn Tysnes, Haukeland University Hospital

Study record dates

Study Major Dates

• Study Start (Actual): April 21, 2023
• Primary Completion (Estimated): August 21, 2032
• Study Completion (Estimated): August 21, 2032

Study Registration Dates

• First Submitted: January 30, 2023
• First Submitted That Met QC Criteria: February 21, 2023
• First Posted (Actual): February 27, 2023

Study Record Updates

• Last Update Posted (Estimated): August 28, 2025
• Last Update Submitted That Met QC Criteria: August 22, 2025
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: Quality of life; Health related quality of life; Overall quality of life; Disease specific quality of life; Ventilation support; Non-invasive ventilation support; Invasive ventilation support
• Additional Relevant MeSH Terms: Central Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Proteostasis Deficiencies; Nutritional and Metabolic Diseases; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Nervous System Diseases; Neurodegenerative Diseases; TDP-43 Proteinopathies; Spinal Cord Diseases
• Other Study ID Numbers: 492102

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
• product manufactured in and exported from the U.S.: No