A Long-term Observational Study to Assess Skin Cancer Incidence in Patients With Dystrophic and Junctional Epidermolysis Bullosa Receiving Filsuvez

What this Trial Is About

Epidermolysis bullosa (EB) is a condition where collagen does not form properly, making skin fragile and prone to blisters. Patients with certain types of EB, especially dystrophic EB (DEB) and junctional EB (JEB), have a greatly increased risk of developing skin cancers such as squamous cell carcinoma, basal cell carcinoma, and malignant melanoma. This research aims to observe whether the use of Filsuvez, a topical gel, affects the likelihood of developing skin malignancies in these patients over time. This is a long-term observational study where patients with DEB or JEB will receive their usual standard of care, which may include treatment with Filsuvez or other therapies. Participants are not assigned treatments by the study; instead, their real-world treatment choices are monitored. The study collects data from clinical centers and existing EB registries in Europe and the United Kingdom. Patients will be observed for up to 5 years to track the development and nature of any skin cancers. During the study, researchers will collect information from both groups—those using Filsuvez and those not using it—to compare the incidence of skin malignancies. Assessments include regular monitoring through clinical visits and registry data, focusing on the first occurrence of skin cancers. The main outcome is the incidence of the first skin malignancy during the follow-up period. Participants' health and disease status will be tracked throughout the 5 years to better understand skin cancer risks related to different treatments for EB.

A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez