International Observational Registry for Patients of All Ages With X-Linked Hypophosphatemia XLH

What this Trial Is About

Researchers are conducting an international, multicenter, prospective observational registry to study patients with X-Linked Hypophosphatemia XLH. This registry aims to collect detailed data on the natural history, treatment patterns, progression, and long-term outcomes of XLH in both adults and children. The study includes patients regardless of whether they are currently receiving treatment for XLH. Participants will be enrolled in a non-interventional setting without any additional treatments or procedures beyond their standard clinical care. Data will be collected retrospectively and prospectively using a web-based system. Physicians will update patient information during routine visits, with reminders to update data approximately every 12 months. Some sites may also participate in a post-authorization safety study PASS to monitor treatment-related adverse events. Participants will provide informed consent or assent according to age and national guidelines. The study team will collect baseline and ongoing clinical information, including treatment details and adverse events, over a period extending to 10 years. No fixed follow-up schedule is required, but data updates align with patients usual healthcare visits. Researchers will review adverse events related to treatment as a primary outcome measure throughout the study period.

Registry for Patients With X-Linked Hypophosphatemia