Continued access to study treatment for Fabry disease participants
Part of Brain & nervous system, Genetic & congenital, Heart & circulation, Hormones & metabolism clinical trials.
This trial gives patients with Fabry disease who took part in a previous study the chance to continue receiving the study treatment, lucerastat, after that study ends. It aims to provide uninterrupted care while longer-term safety and effects are monitored.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You must have Fabry disease.
- You must have participated in the previous study (ID-069A302) of this treatment.
- You must still be eligible for continued treatment in the opinion of your doctor.
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 study tests a new medicine called lucerastat to see how well it works and how safe it is. It is for adult men with Fabry disease who have not been treated before. The medicine may help reduce the buildup of fatty substances in cells and improve symptoms.
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 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 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 study tests an enzyme replacement therapy (agalsidase alfa) for Fabry disease in Chinese children and adults. It's an observational study that follows patients who receive the treatment as part of their regular care.
This study is a national registry that collects health and medical information from people with Fabry disease. It helps researchers better understand the condition and plan future care and studies.
Hear when a new Fabry Disease trial opens
We’ll email you when one opens — at most once a week, no account needed, unsubscribe anytime.