Expanded access to migalastat for Fabry disease
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This expanded access program provides migalastat (a pill) to people with Fabry disease who have a specific genetic mutation and cannot use standard enzyme replacement therapy. It's an option for those who need treatment and have no other alternatives.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are at least 2 years old
- Your GLA gene test shows a mutation that should respond to migalastat (the study drug)
- You have strong symptoms or complications from Fabry disease that need treatment
- You cannot receive enzyme replacement therapy (ERT) or cannot get access to it
- You are not scheduled for a kidney or other organ transplant
- You are willing to take the study drug and share your health data
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
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.
This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.
This study follows pregnant and breastfeeding women with Fabry disease (and their infants) to better understand what happens during pregnancy and early life. You may or may not be taking migalastat, and the goal is to gather real-world information that could help future care.
This trial tests if enzyme replacement therapy works well and is safe for Fabry disease. It is for people who are already prescribed this treatment.
This trial tests a one-time gene therapy called AMT-191 for men with classic Fabry disease who are still having symptoms despite enzyme replacement therapy. The goal is to see if it can reduce pain and stomach problems and improve overall health.
This Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.
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