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Recruiting NCT06423573

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

Observational Epidermolysis Bullosa, Dystrophic Epidermolysis Bullosa, Junctional

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Filsuvez.
Who it may be relevant to
Registry conditions: Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional. Basic parameters: No limits · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
France, Greece, Spain, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Official title

A Long-term Non-interventional Study to Assess the Incidence of Skin Malignancies in Patients With Dystrophic and Junctional Epidermolysis Bullosa Receiving Treatment With Filsuvez

Overview

In patients with epidermolysis bullosa (EB), collagen does not form properly, so their skin is very fragile and blisters easily. Such patients are also at greatly increased risk of developing skin cancers. Filsuvez is a topical gel used to promote healing of skin lesions in patients with certain types of EB. In this observational study, patients with either dystrophic EB (DEB) or junctional EB (JEB) will receive standard of care treatment, whether Filsuvez or something else, and will be followed for up to 5 years. The main purpose is to see if the use of Filsuvez affects the likelihood of developing skin malignancies in these patient populations.

Detailed description

Some forms of EB are associated with a greatly increased incidence of aggressive skin and mucosal squamous cell carcinoma (SCC), as well as increased incidence of other skin malignancies including basal cell carcinoma (BCC) and malignant melanoma (MM). This long-term non-interventional study will assess all types of skin malignancies (SCC, BCC, and MM) in DEB and JEB patients. It is an observational study of real-world treatment practices, in which patients will receive standard of care therapy whatever that may entail. Data will be obtained from two sources: clinical study centers in the European Union (EU) and the United Kingdom (UK), and pre-existing EB registries in the EU. Participants will be followed for up to 5 years, and information about the development and nature of skin malignancies will be collected over this time period both from patients who are taking and those who are not taking Filsuvez.

Interventions

  • Drug Filsuvez
    Topical gel

Primary outcome measures

  • Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez [Time frame: Up to 5 years]
Secondary outcome measures (1)
  • Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez [Time frame: Up to 5 years]

Eligibility criteria

Inclusion criteria

  • Patients with a confirmed diagnosis of dystrophic EB or junctional EB

Exclusion criteria

  • None

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model
Cohort

Study locations

Spain · 2 centers
  • Hospital Clinic, Barcelona — Barcelona
  • Hospital San Juan de Dios (Barcelona) — Barcelona
France · 1 center
  • CHU de Toulouse, Hôpital Larrey — Toulouse
Greece · 1 center
  • Hospital of Skin and Venereal Diseases of Thessaloniki — Thessaloniki
United Kingdom · 1 center
  • Guy's and St Thomas' NHS Foundation Trust — London

Identifiers

NCT: NCT06423573 · AEB-21

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗