Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol
Introduction Amyotrophic lateral sclerosis (ALS) is a devastating illness that leads to muscle weakness and death usually within around 3 years of diagnosis. People with ALS (pwALS) often lose weight due to raised energy requirements and symptoms of the disease presenting significant challenges to t...
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BMJ Publishing Group
2025-05-01
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| author | Simon Waterhouse Chin Maguire Vanessa Halliday Cindy Cooper Elizabeth Coates Mike Bradburn Pamela J Shaw Ammar Al-Chalabi Tracey Anne Young Christopher J McDermott Wendy Baird Paul Norman Janet Cade Sean White Daniel Beever Gemma Hackney Elaine Scott David Alexander White Arron Peace Shamila Ditta Jonathan Woodward Naseeb Ezaydi Haris Stavroulakis |
| author_facet | Simon Waterhouse Chin Maguire Vanessa Halliday Cindy Cooper Elizabeth Coates Mike Bradburn Pamela J Shaw Ammar Al-Chalabi Tracey Anne Young Christopher J McDermott Wendy Baird Paul Norman Janet Cade Sean White Daniel Beever Gemma Hackney Elaine Scott David Alexander White Arron Peace Shamila Ditta Jonathan Woodward Naseeb Ezaydi Haris Stavroulakis |
| collection | DOAJ |
| description | Introduction Amyotrophic lateral sclerosis (ALS) is a devastating illness that leads to muscle weakness and death usually within around 3 years of diagnosis. People with ALS (pwALS) often lose weight due to raised energy requirements and symptoms of the disease presenting significant challenges to taking adequate oral diet, with those who lose more weight being at a greater chance of earlier death. There is also some evidence to suggest that a higher calorie diet may benefit the disease course in pwALS, but further research is needed.Methods and analysis Two armed, parallel group, superiority, open labelled, randomised controlled trial, with internal pilot, to assess the effectiveness of an early high calorie diet on functional outcomes in ALS, comprising two treatment arms: (1) standard care, (2) standard care with additional active management using the OptiCALS complex intervention to achieve a high calorie diet (initially randomised 1:1, then 1:2 following a protocol amendment). Using a food first approach, pwALS will be encouraged and supported to follow a diet that meets an individualised calorie target from food before prescribing oral nutritional supplements. 259 pwALS will be recruited from up to 20 ALS centres across the United Kingdom and Ireland and followed up for a period of 12 months. Primary outcome is functional change measured over 12 months, using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale. Secondary end points include measures of functional health, quality of life, calorie intake and weight, as well as time to gastrostomy and survival. A health economic analysis and process evaluation will also be undertaken. Participant recruitment is expected to complete in September 2025, and participant follow-up is expected to complete in September 2026. The results of this study are expected in March 2027.Ethics and dissemination The trial was approved by Greater Manchester—North West Research Ethics Committee, reference 20/NW/0334 on 8 September 2020. We will publish the study findings in peer-reviewed academic journals and present at local, national and international conferences where possible.Trial registration number ISRCTN30588041. |
| format | Article |
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| institution | DOAJ |
| issn | 2044-6055 |
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| publishDate | 2025-05-01 |
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| spelling | doaj-art-37e68cb237e24307ad5d890bb7eda0eb2025-08-20T03:21:31ZengBMJ Publishing GroupBMJ Open2044-60552025-05-0115510.1136/bmjopen-2024-096098Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol Simon Waterhouse0Chin Maguire1Vanessa Halliday2Cindy Cooper3Elizabeth Coates4Mike Bradburn5Pamela J Shaw6Ammar Al-Chalabi7Tracey Anne Young8Christopher J McDermott9Wendy Baird10Paul Norman11Janet Cade12Sean White13Daniel Beever14Gemma Hackney15Elaine ScottDavid Alexander White16Arron Peace17Shamila DittaJonathan WoodwardNaseeb Ezaydi18Haris Stavroulakis19Clinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKSheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKDivision of Neuroscience. School of Medicine and Population Health. Sheffield Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UKDepartment of Neuroscience, King’s College London, London, UKSheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKDivision of Neuroscience. School of Medicine and Population Health. Sheffield Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UKSheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKDepartment of Psychology, The University of Sheffield, Sheffield, UKSchool of Food Science and Nutrition, University of Leeds, Leeds, UKSheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKClinical Trials Research Unit, Sheffield Centre for Health and Related Research, The University of Sheffield, Sheffield, UKDivision of Neuroscience. School of Medicine and Population Health. Sheffield Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UKIntroduction Amyotrophic lateral sclerosis (ALS) is a devastating illness that leads to muscle weakness and death usually within around 3 years of diagnosis. People with ALS (pwALS) often lose weight due to raised energy requirements and symptoms of the disease presenting significant challenges to taking adequate oral diet, with those who lose more weight being at a greater chance of earlier death. There is also some evidence to suggest that a higher calorie diet may benefit the disease course in pwALS, but further research is needed.Methods and analysis Two armed, parallel group, superiority, open labelled, randomised controlled trial, with internal pilot, to assess the effectiveness of an early high calorie diet on functional outcomes in ALS, comprising two treatment arms: (1) standard care, (2) standard care with additional active management using the OptiCALS complex intervention to achieve a high calorie diet (initially randomised 1:1, then 1:2 following a protocol amendment). Using a food first approach, pwALS will be encouraged and supported to follow a diet that meets an individualised calorie target from food before prescribing oral nutritional supplements. 259 pwALS will be recruited from up to 20 ALS centres across the United Kingdom and Ireland and followed up for a period of 12 months. Primary outcome is functional change measured over 12 months, using the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale. Secondary end points include measures of functional health, quality of life, calorie intake and weight, as well as time to gastrostomy and survival. A health economic analysis and process evaluation will also be undertaken. Participant recruitment is expected to complete in September 2025, and participant follow-up is expected to complete in September 2026. The results of this study are expected in March 2027.Ethics and dissemination The trial was approved by Greater Manchester—North West Research Ethics Committee, reference 20/NW/0334 on 8 September 2020. We will publish the study findings in peer-reviewed academic journals and present at local, national and international conferences where possible.Trial registration number ISRCTN30588041.https://bmjopen.bmj.com/content/15/5/e096098.full |
| spellingShingle | Simon Waterhouse Chin Maguire Vanessa Halliday Cindy Cooper Elizabeth Coates Mike Bradburn Pamela J Shaw Ammar Al-Chalabi Tracey Anne Young Christopher J McDermott Wendy Baird Paul Norman Janet Cade Sean White Daniel Beever Gemma Hackney Elaine Scott David Alexander White Arron Peace Shamila Ditta Jonathan Woodward Naseeb Ezaydi Haris Stavroulakis Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol BMJ Open |
| title | Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol |
| title_full | Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol |
| title_fullStr | Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol |
| title_full_unstemmed | Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol |
| title_short | Randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention, OptiCALS, for patients with amyotrophic lateral sclerosis: study protocol |
| title_sort | randomised controlled trial with parallel process evaluation and health economic analysis to evaluate a nutritional management intervention opticals for patients with amyotrophic lateral sclerosis study protocol |
| url | https://bmjopen.bmj.com/content/15/5/e096098.full |
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