Patient-reported impact of Charcot-Marie-Tooth disease: Protocol for a real-world digital lifestyle study

Florian P. Thomas; Mario Saporta; Shahram Attarian; Teresa Sevilla; Rafael Sivera Mascaró; Gian Maria Fabrizi; Filippo Genovese; Amy Gray; Simon Bull; Daniel Tanesse; Manuel Rego; Allison Moore; Courtney Hollett; Katia Monteiro; Xavier Paoli; Samuel Llewellyn; Mark Larkin; Youcef Boutalbi

doi:10.2217/nmt-2020-0044

Patient-reported impact of Charcot-Marie-Tooth disease: Protocol for a real-world digital lifestyle study

Florian P. Thomas, Mario Saporta, Shahram Attarian, Teresa Sevilla, Rafael Sivera Mascaró, Gian Maria Fabrizi, Filippo Genovese, Amy Gray, Simon Bull, Daniel Tanesse, Manuel Rego, Allison Moore, Courtney Hollett, Katia Monteiro, Xavier Paoli, Samuel Llewellyn, Mark Larkin, Youcef Boutalbi

Neurology

Research output: Contribution to journal › Article › peer-review

Abstract

Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and sensory neuropathy affecting the peripheral nervous system. This study will explore the real-world impact of CMT. The trial is a digital study of approximately 2000 people in 6 countries with CMT ≥18 years. Participants will use a smartphone application to check eligibility, provide consent and contribute data. The dataset will include a personal profile, covering demographics, lifestyle, diagnosis and treatment and a selection of validated generic and disease-specific instruments. Participants will provide data for up to 2 years. Data analysis will be conducted upon registration of the 1000th participant and at 12-month intervals from launch. This study is designed to help researchers and clinicians understand the real-world impact of CMT and the unmet needs of patients. ClinicalTrials.gov identifier: NCT03782883</ext-link.

Original language	English (US)
Pages (from-to)	21-33
Number of pages	13
Journal	Neurodegenerative disease management
Volume	11
Issue number	1
DOIs	https://doi.org/10.2217/nmt-2020-0044
State	Published - Feb 2021

Keywords

Charcot-Marie-Tooth disease
burden of illness
international
observational
patient-reported outcomes

ASJC Scopus subject areas

Medicine(all)

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10.2217/nmt-2020-0044

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Thomas, F. P., Saporta, M., Attarian, S., Sevilla, T., Mascaró, R. S., Fabrizi, G. M., Genovese, F., Gray, A., Bull, S., Tanesse, D., Rego, M., Moore, A., Hollett, C., Monteiro, K., Paoli, X., Llewellyn, S., Larkin, M., & Boutalbi, Y. (2021). Patient-reported impact of Charcot-Marie-Tooth disease: Protocol for a real-world digital lifestyle study. Neurodegenerative disease management, 11(1), 21-33. https://doi.org/10.2217/nmt-2020-0044

Patient-reported impact of Charcot-Marie-Tooth disease : Protocol for a real-world digital lifestyle study. / Thomas, Florian P.; Saporta, Mario; Attarian, Shahram; Sevilla, Teresa; Mascaró, Rafael Sivera; Fabrizi, Gian Maria; Genovese, Filippo; Gray, Amy; Bull, Simon; Tanesse, Daniel; Rego, Manuel; Moore, Allison; Hollett, Courtney; Monteiro, Katia; Paoli, Xavier; Llewellyn, Samuel; Larkin, Mark; Boutalbi, Youcef.

In: Neurodegenerative disease management, Vol. 11, No. 1, 02.2021, p. 21-33.

Research output: Contribution to journal › Article › peer-review

Thomas, FP, Saporta, M, Attarian, S, Sevilla, T, Mascaró, RS, Fabrizi, GM, Genovese, F, Gray, A, Bull, S, Tanesse, D, Rego, M, Moore, A, Hollett, C, Monteiro, K, Paoli, X, Llewellyn, S, Larkin, M & Boutalbi, Y 2021, 'Patient-reported impact of Charcot-Marie-Tooth disease: Protocol for a real-world digital lifestyle study', Neurodegenerative disease management, vol. 11, no. 1, pp. 21-33. https://doi.org/10.2217/nmt-2020-0044

Thomas, Florian P. ; Saporta, Mario ; Attarian, Shahram ; Sevilla, Teresa ; Mascaró, Rafael Sivera ; Fabrizi, Gian Maria ; Genovese, Filippo ; Gray, Amy ; Bull, Simon ; Tanesse, Daniel ; Rego, Manuel ; Moore, Allison ; Hollett, Courtney ; Monteiro, Katia ; Paoli, Xavier ; Llewellyn, Samuel ; Larkin, Mark ; Boutalbi, Youcef. / Patient-reported impact of Charcot-Marie-Tooth disease : Protocol for a real-world digital lifestyle study. In: Neurodegenerative disease management. 2021 ; Vol. 11, No. 1. pp. 21-33.

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abstract = "Charcot-Marie-Tooth disease (CMT) is a rare, chronic, progressive motor and sensory neuropathy affecting the peripheral nervous system. This study will explore the real-world impact of CMT. The trial is a digital study of approximately 2000 people in 6 countries with CMT ≥18 years. Participants will use a smartphone application to check eligibility, provide consent and contribute data. The dataset will include a personal profile, covering demographics, lifestyle, diagnosis and treatment and a selection of validated generic and disease-specific instruments. Participants will provide data for up to 2 years. Data analysis will be conducted upon registration of the 1000th participant and at 12-month intervals from launch. This study is designed to help researchers and clinicians understand the real-world impact of CMT and the unmet needs of patients. ClinicalTrials.gov identifier: NCT03782883",

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Patient-reported impact of Charcot-Marie-Tooth disease: Protocol for a real-world digital lifestyle study

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Keywords

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