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International GNE Myopathy Patient Registry

NCT04009226 · Status: UNKNOWN · Type: OBSERVATIONAL · Enrollment: 430

Last updated 2020-10-06

No results posted yet for this study

Summary

GNE myopathy, an ultra-rare disease, 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 understand the world wide epidemiology of this ultra-rare condition, better understand a long-term disease course and the progression of disease-specific features, support translational research by evaluating burden illness and support clinical research recruitment. Therefore, the study will longitudinally collect information via an online patient registry platform.

Conditions

  • GNE Myopathy
  • Hereditary Inclusion Body Myopathy

Interventions

OTHER

Patient Registry

Participants who have volunteered to participate will complete various questionnaires relating to their condition.

Sponsors & Collaborators

  • Newcastle University

    lead OTHER

Principal Investigators

  • Volker Straub, MD, PhD · John Walton Muscular Dystrophy Research Centre

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-03-31
Primary Completion
2021-12-31
Completion
2021-12-31

Countries

  • United Kingdom

Study Locations

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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 NCT04009226 on ClinicalTrials.gov