Study of migalastat for children with Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Your child must be between 2 and under 12 years old at the start of the study
- Your child must have a confirmed Fabry disease diagnosis and a specific gene change that works with migalastat
- Your child must not have received enzyme replacement therapy for at least 14 days before the study begins
- Your child must have at least one sign or past sign of Fabry disease, like pain or lab abnormality
- Your child must not have severe kidney problems or be on dialysis
- Your child cannot be pregnant or breastfeeding, and if sexually active, must use effective birth control
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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