## Protocol Section
### Identification Module
**NCT ID:** NCT06423573

**Acronym:** FOSteR

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

**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

#### Organization Study ID Info
**ID:** AEB-21
#### Organization
**Class:** INDUSTRY
**Full Name:** Amryt Pharma
### Status Module
#### Completion Date
**Date:** 2032-08-01

**Type:** ESTIMATED

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-21

**Type:** ACTUAL

**Last Update Submit Date:** 2024-05-16

**Overall Status:** NOT_YET_RECRUITING

#### Primary Completion Date
**Date:** 2031-11-30

**Type:** ESTIMATED

#### Start Date
**Date:** 2024-11-30

**Type:** ESTIMATED

**Status Verified Date:** 2024-05

#### Study First Post Date
**Date:** 2024-05-21
**Type:** ACTUAL
**Study First Submit Date:** 2024-05-16
**Study First Submit QC Date:** 2024-05-16
### Sponsor Collaborators Module

#### Collaborators

**Class:** INDUSTRY

**Name:** Chiesi Farmaceutici S.p.A.

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Amryt Pharma

#### Responsible Party

**Type:** SPONSOR

### Oversight Module

**Is FDA Regulated Device:** False

**Is FDA Regulated Drug:** False

**Oversight Has DMC:** True

### Description Module

**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.

### Conditions Module

**Conditions:**

- Epidermolysis Bullosa, Dystrophic

- Epidermolysis Bullosa, Junctional

**Keywords:**

- Filsuvez

- Birch bark extract

### Design Module

#### Design Info

**Observational Model:** COHORT

**Time Perspective:** PROSPECTIVE

#### Enrollment Info

**Count:** 580

**Type:** ESTIMATED

**Study Type:** OBSERVATIONAL

### Arms Interventions Module

#### Arm Group 1

**Description:** Patients in this cohort will be receiving treatment with Filsuvez for their EB.

**Intervention Names:**

- Drug: Filsuvez

**Label:** Patients with dystrophic or junctional EB being treated with Filsuvez

#### Arm Group 2

**Description:** Patients in this cohort will be receiving treatment other than Filsuvez or no treatment at all for their EB.

**Label:** Patients with dystrophic or junctional EB not being treated with Filsuvez

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Patients with dystrophic or junctional EB being treated with Filsuvez

**Description:** Topical gel

**Name:** Filsuvez

**Other Names:**

- Birch bark extract

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** 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

**Measure:** Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez

**Time Frame:** Up to 5 years

#### Secondary Outcomes

**Description:** 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

**Measure:** Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez

**Time Frame:** Up to 5 years

### Eligibility Module

**Eligibility Criteria:** Inclusion Criteria:

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

Exclusion Criteria:

* None

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**Standard Ages:**

- CHILD

- ADULT

- OLDER_ADULT

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

### Contacts Locations Module

#### Central Contacts

**Contact 1:**

**Email:** clinicaltrials_info@chiesi.com

**Name:** Chiesi Clinical Trials

**Phone:** +3905212791

**Role:** CONTACT

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000012868

- Term: Skin Abnormalities

- ID: D000000013

- Term: Congenital Abnormalities

- ID: D000012873

- Term: Skin Diseases, Genetic

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000012871

- Term: Skin Diseases

- ID: D000012872

- Term: Skin Diseases, Vesiculobullous

- ID: D000003095

- Term: Collagen Diseases

- ID: D000003240

- Term: Connective Tissue Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC17

- Name: Skin and Connective Tissue Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC04

- Name: Neoplasms

- Abbrev: BC05

- Name: Musculoskeletal Diseases

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M7976

- Name: Epidermolysis Bullosa

- Relevance: HIGH

- As Found: Epidermolysis Bullosa

- ID: M18587

- Name: Epidermolysis Bullosa, Junctional

- Relevance: HIGH

- As Found: Epidermolysis Bullosa, Junctional

- ID: M15681

- Name: Skin Neoplasms

- Relevance: LOW

- As Found: Unknown

- ID: M18586

- Name: Epidermolysis Bullosa Dystrophica

- Relevance: HIGH

- As Found: Epidermolysis Bullosa, Dystrophic

- ID: M12

- Name: Congenital Abnormalities

- Relevance: LOW

- As Found: Unknown

- ID: M15672

- Name: Skin Abnormalities

- Relevance: LOW

- As Found: Unknown

- ID: M15674

- Name: Skin Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M15676

- Name: Skin Diseases, Genetic

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M15675

- Name: Skin Diseases, Vesiculobullous

- Relevance: LOW

- As Found: Unknown

- ID: M15045

- Name: Rheumatic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6323

- Name: Collagen Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M6464

- Name: Connective Tissue Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T2098

- Name: Epidermolysis Bullosa

- Relevance: HIGH

- As Found: Epidermolysis Bullosa

- ID: T3164

- Name: Junctional Epidermolysis Bullosa

- Relevance: HIGH

- As Found: Epidermolysis Bullosa, Junctional

- ID: T1991

- Name: Dystrophic Epidermolysis Bullosa

- Relevance: HIGH

- As Found: Epidermolysis Bullosa, Dystrophic

### Condition Browse Module - Meshes

- ID: D000004820

- Term: Epidermolysis Bullosa

- ID: D000016109

- Term: Epidermolysis Bullosa, Junctional

- ID: D000016108

- Term: Epidermolysis Bullosa Dystrophica

### Misc Info Module

- Version Holder: 2024-05-31
