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

Recruiting

• Conditions: Epidermolysis Bullosa, Dystrophic; Epidermolysis Bullosa, Junctional
• Interventions: Drug: Filsuvez

• Registration Number: NCT06423573
• Lead Sponsor: Amryt Pharma

Brief Summary

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.

Study Timeline

• Study First Submitted: 2024-05-16
• Study First Submitted QC: 2024-05-16
• Study First Posted: 2024-05-21
• Study Start: 2024-12-18
• Status Verified: 2025-03
• Last Update Submitted: 2025-03-13
• Last Update Posted: 2025-03-17
• Primary Completion: 2031-11-30
• Study Completion: 2032-08-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 580

Inclusion Criteria

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

Exclusion Criteria

• None

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients with dystrophic or junctional EB being treated with Filsuvez	Filsuvez	Patients in this cohort will be receiving treatment with Filsuvez for their EB.

Primary Outcome Measures

Name	Time	Method
Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez	Up to 5 years	Patients being treated with Filsuvez will be followed for occurrence of skin malignancies from the date of study enrolment until either the date of discontinuation (withdrawal of consent, withdrawal from the site or registry, physician decision, death, or lost to follow-up) or termination of the study

Secondary Outcome Measures

Name	Time	Method
Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez	Up to 5 years	Patients with EB who are not being treated with Filsuvez will be followed for occurrence of skin malignancies from the date of study enrolment until either the date of discontinuation (withdrawal of consent, withdrawal from the site or registry, physician decision, death, or lost to follow-up) or termination of the study

Trial Locations

Locations (1)

CHU de Toulouse, Hôpital Larrey; 🇫🇷 Toulouse, France