Portal de investigación
Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study.
Fecha de publicación:
Fecha Ahead of Print:
Autores de IIS La Fe
Participantes ajenos a IIS La Fe
- Thomas, Florian P.
- Saporta, Mario
- Attarian, Shahram
- Mascaro, 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
Grupos
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.
Datos de la publicación
- ISSN/ISSNe:
- 1758-2024, 1758-2032
- Tipo:
- Review
- Páginas:
- 21-33
- PubMed:
- 33086913
- Factor de Impacto:
- 0,487 SCImago ℠
- Cuartil:
- Q3 SCImago ℠
Neurodegenerative Disease Management TAYLOR & FRANCIS LTD
Documentos
- No hay documentos
Filiaciones
Keywords
- Charcot-Marie-Tooth disease, burden of illness, international, observational, patient-reported outcomes
Cita
Thomas FP,Saporta M,Attarian S,Sevilla T,Mascaro 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. Patient-reported impact of Charcot-Marie-Tooth disease: protocol for a real-world digital lifestyle study. Neurodegener. Dis. Manag. 2021. 11(1):p. 21-33.