Status:
UNKNOWN
International GNE Myopathy Patient Registry
Lead Sponsor:
Newcastle University
Conditions:
GNE Myopathy
Hereditary Inclusion Body Myopathy
Eligibility:
All Genders
18+ years
Brief 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, l...
Detailed Description
GNE myopathy is an ultra- rare condition. Most of the knowledge is coming from case reports or small cohort observations. There is a need to more precisely understand the long-term disease course and ...
Eligibility Criteria
Inclusion
- Aged 18 years or older at the time of informed consent
- Clinical and/or genetic diagnosis of GNE myopathy (also known as HIBM, QSM, Inclusion Body Myopathy Type 2, DMRV, or Nonaka disease)
- Willing and able to provided electronic (or written) consent and comply with all study requirements.
Exclusion
- Under 18 years of age
Key Trial Info
Start Date :
March 1 2014
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2021
Estimated Enrollment :
430 Patients enrolled
Trial Details
Trial ID
NCT04009226
Start Date
March 1 2014
End Date
December 1 2021
Last Update
October 6 2020
Active Locations (1)
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1
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, United Kingdom, NE1 3BZ