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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)

NCT01784679 · Status: COMPLETED · Type: OBSERVATIONAL · Enrollment: 319

Last updated 2018-04-27

No results posted yet for this study

Summary

HIBM 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.

Conditions

  • Hereditary Inclusion Body Myopathy
  • GNE Myopathy
  • Nonaka Disease
  • Quadriceps Sparing Myopathy (QSM)
  • Distal Myopathy With Rimmed Vacuoles (DMRV)

Sponsors & Collaborators

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-04-05
Primary Completion
2017-11-30
Completion
2017-11-30

Countries

  • United States
  • Bulgaria
  • Canada
  • France
  • United Kingdom

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01784679 on ClinicalTrials.gov