Observational Study Tracking the Natural History and Progression of Fucosidosis with or without Prior Stem Cell Transplant

What this Trial Is About

Researchers are conducting an observational study to better understand fucosidosis, a rare disease, its symptoms, and how it changes over time. The study aims to gather detailed information about the diseases natural history and progression, including patients who have or have not undergone stem cell transplant. There is currently no approved treatment for fucosidosis, so this study focuses on observing the condition without introducing new medications. The study is divided into two parts Part A involves collecting past medical data from up to 57 participants, and Part B involves following up with up to 31 participants prospectively, many of whom may also be in Part A. Participants will continue to receive their usual medical care throughout the study, with no additional treatments or interventions provided by the study team. Participants will provide medical history and health information that will be reviewed over time. The main focus is to track disease progression in individuals untreated by investigational products for up to four years. This includes gathering retrospective data and ongoing prospective observations to aid future research and potential treatment development. The study is expected to run until January 2031.

A Long-Term Observational Study of Patients With Fucosidosis