Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
Conditions
Keywords
Filsuvez, Birch bark extract
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.
Interventions
DRUGFilsuvez
Topical gel
Sponsors
Chiesi Farmaceutici S.p.A.
Amryt Pharma
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Patients with a confirmed diagnosis of dystrophic EB or junctional EB
Exclusion criteria
* None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
France, Greece, Spain, United Kingdom
Contacts
Primary ContactChiesi Clinical Trials
Outcome results
None listed