Study of ATX-01 for Myotonic Dystrophy Type 1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This trial tests an experimental drug called ATX-01 for people with myotonic dystrophy type 1 (DM1), a condition that causes muscle weakness and stiffness. The study aims to see if ATX-01 can help reduce symptoms like muscle stiffness (myotonia) and improve daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have a confirmed diagnosis of myotonic dystrophy type 1 (DM1) with a genetic test showing an expanded CTG repeat in the DMPK gene.
- You need to be able to walk 10 meters (about 33 feet) without using a cane, walker, or braces. Ankle-foot orthoses (supports for the foot and ankle) are allowed.
- You must have grip myotonia (difficulty letting go after gripping something) lasting more than 3 seconds.
- You cannot have a form of DM1 that was present at birth (congenital DM1).
- You cannot have taken mexiletine or other medications for muscle stiffness within the last 3 weeks.
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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