An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12-month Treatment with Migalastat in Pediatric Subjects (aged 2 to < 12 years) with Fabry Disease and Amenable GLA Variants

To join this study, children need to be between 2 and under 12 years old and have Fabry disease. Importantly, they must have a specific type of genetic change (doctors call them 'amenable GLA variants') that the medicine, migalastat, is designed to work with.

Children of any gender can take part. The study team will review each child's health and medical history very carefully to make sure the study is a good fit for them and that it's safe for them to participate. They'll also check if there are any other reasons why a child might not be able to join, such as other serious health conditions or taking certain medications.

It's worth remembering that these are just the basic requirements. A doctor involved in the study will be able to tell you all the detailed specific things that would make a child eligible or not eligible.

If your child takes part in this study, they will receive the study medicine, Migalastat Hydrochloride, as a dispersible tablet for 12 months. This means it's a tablet that can be dissolved in liquid. Throughout the study, your child will have regular visits to the clinic. At these visits, doctors and nurses will closely monitor their health. This will include checking their blood and urine, taking vital signs like blood pressure and heart rate, doing physical examinations, and measuring their weight and height. They’ll also have tests like heart scans (echocardiograms) and electrocardiograms (ECGs) to check their heart health. For older girls (8 years and up) and boys (9 years and up), their growth and development will also be checked. After the 12 months of treatment, there will be follow-up appointments to see how your child is doing. The total duration of active participation, including follow-up, will be longer than just the 12 months of taking the medicine.