Long-term safety study of del-desiran for DM1
Part of Bones, joints & muscles, Brain & nervous system, Genetic & congenital clinical trials.
This study tests whether the drug del-desiran continues to be safe and effective for people with DM1 (myotonic dystrophy type 1) after they complete an earlier study. You'll receive the drug and have regular check-ups to see how you're doing.
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).
- If you're joining the fixed-dose part, you need significant hand myotonia (muscle stiffness in your hands).
- You cannot be pregnant, breastfeeding, or planning to become pregnant during the study.
- You must agree to use birth control as required.
- Your diabetes must be well-controlled, and you cannot have had heart failure in the last 3 months.
- Your body mass index (BMI) must be 35 or lower at the start of 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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