This clinical trial focuses on studying the long-term safety and tolerability of a medication called Lucerastat in adults with Fabry disease. Fabry disease is a rare genetic disorder that affects the body’s ability to break down a certain type of fat, leading to a buildup that can cause a variety of symptoms, including pain, kidney problems, and heart issues. Lucerastat, also known by its code name ACT-434964, is taken orally in the form of a hard capsule.

The purpose of this study is to observe how well patients tolerate Lucerastat over an extended period. Participants in the study will take Lucerastat daily, with a maximum dose of 2000 milligrams per day, for up to 72 weeks. Throughout the study, researchers will monitor participants for any side effects or adverse reactions to the medication. This study is open-label, meaning that both the participants and the researchers know that Lucerastat is being administered, and it is a single-arm study, which means all participants receive the same treatment without a comparison group.

By participating in this study, researchers aim to gather valuable information on the long-term effects of Lucerastat in managing Fabry disease. This information could help in understanding the potential benefits and risks of using Lucerastat as a treatment option for individuals living with this condition. The study is designed to ensure that participants’ health and safety are closely monitored throughout the trial period.