Study of BEAM-301 for glycogen storage disease type Ia
Part of Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an experimental medicine called BEAM-301 for people with a specific form of glycogen storage disease type Ia (GSDIa). The goal is to see if it can help reduce dangerous low blood sugar episodes.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must be 18 years old or older.
- You must have glycogen storage disease type Ia and a specific genetic change called the R83C variant.
- You must have had at least one episode of very low blood sugar (below 60 mg/dL) in the last 2 years.
- You cannot have had a liver transplant or have liver cirrhosis.
- You cannot have a liver adenoma (growth) that is larger than 5 cm or one that is growing fast.
- You cannot have had any previous cell or gene therapy.
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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