Optimizing INITIation of Non-invasive Ventilation in ALS Patients

NCT05033951 | Recruiting DMC

• 1 other identifier: 202000124
• Study Type: observational
• Target: 250
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

Noninvasive ventilation; Quality of life

Outcome Measures

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

  15 months

Secondary Outcomes (9)

• Survival

  15 months

• Overall function

  15 months

• Forced vital capacity

  15 months

• Carbondioxide

  15 months

• Anamnestic questions

  15 months

Study Arms (2)

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

Standard care OTHER

Both groups: standard care+ questionnaires

Cohort 1; Cohort 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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

You may qualify if:

• 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.

You may not qualify if:

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

Sponsors & Collaborators

• University Medical Center Groningen: lead

Study Sites (1)

University medical centre Groningen

Groningen, 9700RB, Netherlands

RECRUITING

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

Standard of Care

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Quality Indicators, Health Care; Quality of Health Care; Health Services Administration; Health Care Quality, Access, and Evaluation

Central Study Contacts

Rineke Jaspers Focks, MD

CONTACT

+31534875424; r.jaspersfocks@roessingh.nl

Peter Wijkstra, MD, PhD

CONTACT

+31503613200; p.j.wijkstra@umcg.nl

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 42 Months
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Dr. P.J. Wijkstra

Study Record Dates

• First Submitted: July 7, 2021
• First Posted: September 5, 2021
• Study Start: August 1, 2022
• Primary Completion: April 1, 2025
• Study Completion: October 1, 2025
• Last Updated: May 16, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

NL (1)