Calendario›RARE›GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)

Ensayo de Ultragenyx Pharmaceutical Inc. en Hereditary Inclusion Body Myopathy · GNE Myopathy · Nonaka Disease · Quadriceps Sparing Myopathy (QSM) · Distal Myopathy With Rimmed Vacuoles (DMRV) (del registro, en inglés).

Más ensayos de: Miopatía por GNE.

Fase

no aplica

Estado

Completado

Participantes

319

previstos

Centros

5

Fin del objetivo primario

nov 2017

fecha real

Qué significa cada fase y cada estado.

Estudio observacional. Comenzó en abr 2013.

Qué mide

Characterize HIBM disease presentation and progression over time using relevant clinical assessments of muscle strength and function. (del registro, en inglés) (3 años)

Cómo lo describe el promotor

En el documentoEn inglés, del registroHIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and ultimately a wheelchair-bound state. The rate of progression is gradual and variable over the course of 10-20 years or longer. There is a need to better understand the disease-specific features of HIBM to heighten disease awareness; facilitate early diagnosis; identify patients; expand knowledge of the clinical presentation, progression and variation of the disease; identify and validate biomarkers and other efficacy measures; inform on the design and interpretation of clinical studies of investigational products; and eventually to optimize patient management.

Más ensayos de RARE

Todos los de RARE, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01784679), actualizada en abr 2018.