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) |
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Pages (from-to) | 21-33 |
Number of pages | 13 |
Journal | Neurodegenerative disease management |
Volume | 11 |
Issue number | 1 |
DOIs | |
State | Published - Feb 2021 |
Keywords
- Charcot-Marie-Tooth disease
- burden of illness
- international
- observational
- patient-reported outcomes
ASJC Scopus subject areas
- Medicine(all)