Oral eliglustat for children with Gaucher disease
Part of Brain & nervous system, Genetic & congenital, Hormones & metabolism clinical trials.
This study tests an oral medication (eliglustat) for children with Gaucher disease types 1 and 3, replacing enzyme infusions. It may help maintain disease control with a simpler treatment.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 12 and 18 years old.
- You have been getting enzyme replacement therapy for at least 2 years and your blood counts, spleen, and liver are under control.
- You are not pregnant (if female) and agree to avoid grapefruit and grapefruit juice during the study.
- Your genetic test shows you are an extensive or intermediate metabolizer (CYP2D6).
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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