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Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative

BACKGROUND: Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson’s disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach. OBJECTIVE: To provide an up-to-date inventory of OM for d...

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Autores principales: Gonzalez-Robles, Cristina, Weil, Rimona S., van Wamelen, Daniel, Bartlett, Michèle, Burnell, Matthew, Clarke, Caroline S., Hu, Michele T., Huxford, Brook, Jha, Ashwani, Lambert, Christian, Lawton, Michael, Mills, Georgia, Noyce, Alastair, Piccini, Paola, Pushparatnam, Kuhan, Rochester, Lynn, Siu, Carroll, Williams-Gray, Caroline H., Zeissler, Marie-Louise, Zetterberg, Henrik, Carroll, Camille B., Foltynie, Thomas, Schrag, Anette
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10578294/
https://www.ncbi.nlm.nih.gov/pubmed/37545260
http://dx.doi.org/10.3233/JPD-230051
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author Gonzalez-Robles, Cristina
Weil, Rimona S.
van Wamelen, Daniel
Bartlett, Michèle
Burnell, Matthew
Clarke, Caroline S.
Hu, Michele T.
Huxford, Brook
Jha, Ashwani
Lambert, Christian
Lawton, Michael
Mills, Georgia
Noyce, Alastair
Piccini, Paola
Pushparatnam, Kuhan
Rochester, Lynn
Siu, Carroll
Williams-Gray, Caroline H.
Zeissler, Marie-Louise
Zetterberg, Henrik
Carroll, Camille B.
Foltynie, Thomas
Schrag, Anette
author_facet Gonzalez-Robles, Cristina
Weil, Rimona S.
van Wamelen, Daniel
Bartlett, Michèle
Burnell, Matthew
Clarke, Caroline S.
Hu, Michele T.
Huxford, Brook
Jha, Ashwani
Lambert, Christian
Lawton, Michael
Mills, Georgia
Noyce, Alastair
Piccini, Paola
Pushparatnam, Kuhan
Rochester, Lynn
Siu, Carroll
Williams-Gray, Caroline H.
Zeissler, Marie-Louise
Zetterberg, Henrik
Carroll, Camille B.
Foltynie, Thomas
Schrag, Anette
author_sort Gonzalez-Robles, Cristina
collection PubMed
description BACKGROUND: Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson’s disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach. OBJECTIVE: To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials. METHODS: As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives’ input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory. RESULTS: An extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages. CONCLUSION: We present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges.
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spelling pubmed-105782942023-10-17 Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative Gonzalez-Robles, Cristina Weil, Rimona S. van Wamelen, Daniel Bartlett, Michèle Burnell, Matthew Clarke, Caroline S. Hu, Michele T. Huxford, Brook Jha, Ashwani Lambert, Christian Lawton, Michael Mills, Georgia Noyce, Alastair Piccini, Paola Pushparatnam, Kuhan Rochester, Lynn Siu, Carroll Williams-Gray, Caroline H. Zeissler, Marie-Louise Zetterberg, Henrik Carroll, Camille B. Foltynie, Thomas Schrag, Anette J Parkinsons Dis Consensus Paper BACKGROUND: Multi-arm, multi-stage (MAMS) platform trials can accelerate the identification of disease-modifying treatments for Parkinson’s disease (PD) but there is no current consensus on the optimal outcome measures (OM) for this approach. OBJECTIVE: To provide an up-to-date inventory of OM for disease-modifying PD trials, and a framework for future selection of OM for such trials. METHODS: As part of the Edmond J Safra Accelerating Clinical Trials in Parkinson Disease (EJS ACT-PD) initiative, an expert group with Patient and Public Involvement and Engagement (PPIE) representatives’ input reviewed and evaluated available evidence on OM for potential use in trials to delay progression of PD. Each OM was ranked based on aspects such as validity, sensitivity to change, participant burden and practicality for a multi-site trial. Review of evidence and expert opinion led to the present inventory. RESULTS: An extensive inventory of OM was created, divided into: general, motor and non-motor scales, diaries and fluctuation questionnaires, cognitive, disability and health-related quality of life, capability, quantitative motor, wearable and digital, combined, resource use, imaging and wet biomarkers, and milestone-based. A framework for evaluation of OM is presented to update the inventory in the future. PPIE input highlighted the need for OM which reflect their experience of disease progression and are applicable to diverse populations and disease stages. CONCLUSION: We present a range of OM, classified according to a transparent framework, to aid selection of OM for disease-modifying PD trials, whilst allowing for inclusion or re-classification of relevant OM as new evidence emerges. IOS Press 2023-09-08 /pmc/articles/PMC10578294/ /pubmed/37545260 http://dx.doi.org/10.3233/JPD-230051 Text en © 2023 – The authors. Published by IOS Press https://creativecommons.org/licenses/by/4.0/This is an open access article distributed under the terms of the Creative Commons Attribution (CC BY 4.0) License (https://creativecommons.org/licenses/by/4.0/) .
spellingShingle Consensus Paper
Gonzalez-Robles, Cristina
Weil, Rimona S.
van Wamelen, Daniel
Bartlett, Michèle
Burnell, Matthew
Clarke, Caroline S.
Hu, Michele T.
Huxford, Brook
Jha, Ashwani
Lambert, Christian
Lawton, Michael
Mills, Georgia
Noyce, Alastair
Piccini, Paola
Pushparatnam, Kuhan
Rochester, Lynn
Siu, Carroll
Williams-Gray, Caroline H.
Zeissler, Marie-Louise
Zetterberg, Henrik
Carroll, Camille B.
Foltynie, Thomas
Schrag, Anette
Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title_full Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title_fullStr Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title_full_unstemmed Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title_short Outcome Measures for Disease-Modifying Trials in Parkinson’s Disease: Consensus Paper by the EJS ACT-PD Multi-Arm Multi-Stage Trial Initiative
title_sort outcome measures for disease-modifying trials in parkinson’s disease: consensus paper by the ejs act-pd multi-arm multi-stage trial initiative
topic Consensus Paper
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10578294/
https://www.ncbi.nlm.nih.gov/pubmed/37545260
http://dx.doi.org/10.3233/JPD-230051
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