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

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
Externally published	Yes

Keywords

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

ASJC Scopus subject areas

Medicine(all)

Access to Document

10.2217/nmt-2020-0044

Cite this

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.

@article{c52af750232243abb18bdba9c699b30b,

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

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",

keywords = "Charcot-Marie-Tooth disease, burden of illness, international, observational, patient-reported outcomes",

author = "Thomas, {Florian P.} and Mario Saporta and Shahram Attarian and Teresa Sevilla and Mascar{\'o}, {Rafael Sivera} and Fabrizi, {Gian Maria} and Filippo Genovese and Amy Gray and Simon Bull and Daniel Tanesse and Manuel Rego and Allison Moore and Courtney Hollett and Katia Monteiro and Xavier Paoli and Samuel Llewellyn and Mark Larkin and Youcef Boutalbi",

note = "Publisher Copyright: {\textcopyright} 2021 ",

year = "2021",

month = feb,

doi = "10.2217/nmt-2020-0044",

language = "English (US)",

volume = "11",

pages = "21--33",

journal = "Neurodegenerative disease management",

issn = "1758-2024",

publisher = "Future Medicine Ltd.",

number = "1",

}

TY - JOUR

T1 - Patient-reported impact of Charcot-Marie-Tooth disease

T2 - Protocol for a real-world digital lifestyle study

AU - Thomas, Florian P.

AU - Saporta, Mario

AU - Attarian, Shahram

AU - Sevilla, Teresa

AU - Mascaró, Rafael Sivera

AU - Fabrizi, Gian Maria

AU - Genovese, Filippo

AU - Gray, Amy

AU - Bull, Simon

AU - Tanesse, Daniel

AU - Rego, Manuel

AU - Moore, Allison

AU - Hollett, Courtney

AU - Monteiro, Katia

AU - Paoli, Xavier

AU - Llewellyn, Samuel

AU - Larkin, Mark

AU - Boutalbi, Youcef

PY - 2021/2

Y1 - 2021/2

N2 - 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

AB - 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

KW - Charcot-Marie-Tooth disease

KW - burden of illness

KW - international

KW - observational

KW - patient-reported outcomes

UR - http://www.scopus.com/inward/record.url?scp=85098235736&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85098235736&partnerID=8YFLogxK

U2 - 10.2217/nmt-2020-0044

DO - 10.2217/nmt-2020-0044

M3 - Article

C2 - 33086913

AN - SCOPUS:85098235736

VL - 11

SP - 21

EP - 33

JO - Neurodegenerative disease management

JF - Neurodegenerative disease management

SN - 1758-2024

IS - 1

ER -

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

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this