A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

This study is for boys and girls with Fabry disease who are between 2 and 11 years old. A very important part of joining is that your child must have a specific type of genetic change (called an 'amenable GLA variant') that Migalastat is designed to work with. Your child should also have experienced at least one medical issue related to their Fabry disease.

Children can't join if they have severe kidney problems or are already on dialysis. Also, if your child has an allergy to Migalastat or similar medicines, or if they are currently taking other research medicines, they wouldn't be able to participate. If your child has had gene therapy, they also can't take part. Girls and boys who are able to have children must agree to use birth control during the study and for 30 days afterwards.

Additionally, if your child has recently received (within the last 14 days) or is currently receiving other specific Fabry disease treatments like enzyme replacement therapy (ERT), they would need to stop that treatment for the required time before joining.

If your child joins, they will take Migalastat as a capsule. The study involves two treatment periods totaling about 12 months, followed by an option to continue on the medicine in an extension phase. There will be no breaks in treatment between these active phases. If your child stops taking part at any time, they will have a safety check-up 30 days later.

Throughout the study, your child will have regular visits to the clinic for medical checks. This will include blood tests to measure the level of Migalastat in their body at certain times. These blood tests are done at specific points, including four samples over a 24-hour period within the first month, and then single samples at months 6 and 12. These tests help the doctors understand how the medicine is working and how safe it is. The total duration of active treatment for initial participation is about 12 months.