Study of UX016 for people with GNE myopathy
Part of Bones, joints & muscles, Brain & nervous system clinical trials.
This study tests a new medicine called UX016 for people with GNE myopathy, a rare genetic muscle disease that causes weakness in the hands and legs. The trial aims to see if the medicine is safe and whether it can help improve muscle strength. It is the first time this medicine is being tested in humans.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have GNE myopathy (also known as hereditary inclusion body myopathy or Nonaka myopathy) confirmed by a genetic test.
- You must be able to walk at least 20 meters (about 65 feet) without help, but using a cane or walker is allowed.
- Your biceps strength on the dominant side must be less than 80% of normal, as measured by a handheld device.
- You must be willing to have needle muscle biopsies of your thigh.
- You must not have taken certain supplements (like ManNAc or sialic acid) or IVIG in the last 60 days, and you must avoid them during the study.
- If you are female and could become pregnant, or if you are male, you must agree to use effective birth control during the study.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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