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

Epidermolysis Bullosa, Junctional Clinical Trial

— FOSteR  

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06423573
• Other study ID #: AEB-21
• Status: Not yet recruiting
• Start date: November 30, 2024
• Est. completion date: August 1, 2032

Study information

• Verified date: May 2024
• Source: Amryt Pharma
• Contact: Chiesi Clinical Trials
• Phone: +3905212791
• Email: clinicaltrials_info[@]chiesi.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 580
• Est. completion date: August 1, 2032
• Est. primary completion date: November 30, 2031
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

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

Exclusion Criteria:

• None

Related Conditions & MeSH terms

• Epidermolysis Bullosa
• Epidermolysis Bullosa Dystrophica
• Epidermolysis Bullosa, Junctional

Intervention

Drug:
• Filsuvez
Topical gel

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Amryt Pharma	Chiesi Farmaceutici S.p.A.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez	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