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 journalArticlepeer-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 languageEnglish (US)
Pages (from-to)21-33
Number of pages13
JournalNeurodegenerative disease management
Volume11
Issue number1
DOIs
StatePublished - Feb 2021
Externally publishedYes

Keywords

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

ASJC Scopus subject areas

  • Medicine(all)

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