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

September 18, 2025 updated by: Amryt Pharma

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Estimated)

580

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
      • Toulouse, France
        • Recruiting
        • CHU de Toulouse, Hôpital Larrey
        • Contact:
        • Principal Investigator:
          • Juliette Mazereeuw Hautier
  • Greece
      • Thessaloniki, Greece
        • Recruiting
        • Hospital Of Skin And Venereal Diseases of Thessaloniki
        • Contact:
        • Principal Investigator:
          • Dimitra Kiritsi
  • Spain
      • Barcelona, Spain
        • Recruiting
        • Hospital Clinic, Barcelona
        • Contact:
        • Principal Investigator:
          • Josep Riera Monroig
      • Barcelona, Spain
        • Recruiting
        • Hospital San Juan de Dios (Barcelona)
        • Contact:
        • Principal Investigator:
          • Asuncion Vicente
  • United Kingdom
      • London, United Kingdom
        • Recruiting
        • Guy's and St Thomas' NHS Foundation Trust
        • Contact:
        • Principal Investigator:
          • Jemima Mellerio

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with dystrophic EB or junctional EB, whether they are receiving Filsuvez, receiving another treatment for EB, or not receiving any treatment.

Description

Inclusion Criteria:

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

Exclusion Criteria:

  • None

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with dystrophic or junctional EB being treated with Filsuvez
Patients in this cohort will be receiving treatment with Filsuvez for their EB.
Drug: Filsuvez
Topical gel
Other Names:
  • Birch bark extract
Patients with dystrophic or junctional EB not being treated with Filsuvez
Patients in this cohort will be receiving treatment other than Filsuvez or no treatment at all for their EB.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez
Time Frame: 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
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez
Time Frame: 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
Up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Amryt Pharma

Collaborators

Chiesi Farmaceutici S.p.A.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 18, 2024

Primary Completion (Estimated)

November 30, 2031

Study Completion (Estimated)

August 1, 2032

Study Registration Dates

First Submitted

May 16, 2024

First Submitted That Met QC Criteria

May 16, 2024

First Posted (Actual)

May 21, 2024

Study Record Updates

Last Update Posted (Estimated)

September 19, 2025

Last Update Submitted That Met QC Criteria

September 18, 2025

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AEB-21

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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