Safety study of AMP-101 for DOK7 congenital myasthenia
Part of Brain & nervous system, Genetic & congenital clinical trials.
This study tests a new medicine called AMP-101 for people with a rare genetic condition called DOK7 congenital myasthenia, which causes muscle weakness and tiredness. The goal is to see if AMP-101 is safe and if it helps improve muscle strength and daily function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You have a confirmed diagnosis of moderate to severe DOK7 congenital myasthenia syndrome, based on genetic testing and symptoms like muscle weakness in the shoulders, hips, or thighs, and trouble with exercise.
- You are willing to stop certain medications (like 3,4-DAP or pyridostigmine) at least 1 month before the study starts, if you are taking them.
- If you are on oral salbutamol, you must be stable on the same dose for about 6 months during the study, or if you stopped it, you must have been off it for at least 1 month before the study.
- You are not pregnant or breastfeeding, and if you can become pregnant, you agree to use birth control during the study.
- You do not have active infections (like HIV, hepatitis B or C, or certain viruses) and have not used corticosteroids within the last 14 days.
- You have not had prior gene therapy and do not have severe liver or breathing problems.
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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