A Programme for Amyotrophic Lateral Sclerosis Care in Europe

NCT03081338 | Unknown

• 1 other identifier: STH18567
• Study Type: observational
• Target: 115
• Locations: 1 country
• Sites: 1

Brief Summary

Amyotrophic Lateral Sclerosis (ALS) or else known as Motor Neurone Disease (MND) is a rapidly progressive fatal neurological disease that strikes in the prime of life, and for which there is no treatment. The principal aim of management is to maintain quality of life and reduce the symptoms of the disease. This requires a multidisciplinary approach using best practice for symptom alleviation, including innovation approaches towards maximising quality of life. The purpose of this study is to use existing information drawn from partner countries into a system of care that is available to people with amyotrophic lateral sclerosis at the correct time, in the correct format and in a cost effective manner. This will be achieved by collecting details of patient and carer experiences across all stages of from diagnosis to end of life, including decision making in the terminal stages of the disease. A health economic analysis will help to identify the overall costs of disease management, provide models of increased efficiency that preserve and maximize quality of life, and begin to develop novel health economic measurement tools for terminal neurological illness. The completed project will provide a user-friendly best practice programme for amyotrophic lateral sclerosis that can be modified for management of other related degenerative diseases of the nervous system.

Conditions

ALS (Amyotrophic Lateral Sclerosis); MND (Motor Neurone DIsease)

Keywords

ALS, MND, Care, Patient journey

Outcome Measures

Primary Outcomes (1)

• Calculation of quality-adjusted life years (QALYs) that are used to inform economic evaluations of health care interventions using the EuroQoL validated EQ-5D-5L questionnaire.

  Two potential service changes will be identified through examination of the patient journey and through the review of discordance with existing best practice guidelines. Key areas of interest are likely to be focussed around the organisation and delivery of services for gastrostomy, non-invasive ventilation, management of cognitive and behavioural impairment, and the involvement of specialised palliative care teams. The precise methods used will be determined on consideration of the types of intervention and types of data available, but are likely to include case-matching and/or more sophisticated statistical methods such as logistic regression or propensity scoring.

  4 years

Study Arms (1)

Study cohort

A mix of incident and prevalent patient cases will be included in order to capture patients at different stages of the disease trajectory.

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

A mix of incident and prevalent cases will be included in order to capture patients at different stages of the disease trajectory. The total study population will contain 600 patients (300 newly diagnosed and 300 prevalent).

You may qualify if:

• A diagnosis of definite, probable, laboratory supported or possible ALS according to the El Escorial criteria.

You may not qualify if:

• Patient cognitive impairment to the extent that they cannot complete an informed consent process

Sponsors & Collaborators

• Sheffield Teaching Hospitals NHS Foundation Trust: lead

Study Sites (1)

Sheffield Institute for Translational Neuroscience

Sheffield, South Yorkshire, S10 2HQ, United Kingdom

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2015
• First Posted: March 16, 2017
• Study Start: May 1, 2015
• Primary Completion: March 31, 2018
• Study Completion: March 31, 2018
• Last Updated: August 8, 2017

Record last verified: 2017-08

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1)