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

NCT06423573 | Recruiting DMC

• 1 other identifier: AEB-21
• Study Type: observational
• Target: 580
• Locations: 4 countries
• Sites: 5

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.

Conditions

Epidermolysis Bullosa, Dystrophic; Epidermolysis Bullosa, Junctional

Keywords

Filsuvez; Birch bark extract

Outcome Measures

Primary Outcomes (1)

• Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez

  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 Outcomes (1)

• Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez

  Up to 5 years

Study Arms (2)

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

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.

Interventions

Filsuvez DRUG

Topical gel

Also known as: Birch bark extract

Patients with dystrophic or junctional EB being treated with Filsuvez

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

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

You may not qualify if:

• None

Sponsors & Collaborators

• Amryt Pharma: lead
• Chiesi Farmaceutici S.p.A.: collaborator

Study Sites (5)

CHU de Toulouse, Hôpital Larrey

Toulouse, France

RECRUITING

Hospital of Skin and Venereal Diseases of Thessaloniki

Thessaloniki, Greece

RECRUITING

Hospital Clinic, Barcelona

Barcelona, Spain

RECRUITING

Hospital San Juan de Dios (Barcelona)

Barcelona, Spain

RECRUITING

Guy's and St Thomas' NHS Foundation Trust

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Collagen Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Skin Diseases, Vesiculobullous

Central Study Contacts

Chiesi Clinical Trials

CONTACT

+3905212791; clinicaltrials_info@chiesi.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 16, 2024
• First Posted: May 21, 2024
• Study Start: December 18, 2024
• Primary Completion (Estimated): November 30, 2031
• Study Completion (Estimated): August 1, 2032
• Last Updated: September 19, 2025

Record last verified: 2025-09

Locations

ES (2); GB (1); FR (1); GR (1)