Epidermolysis Bullosa, DystrophicFOSteRNCT06423573
A Study to Assess the Incidence of Skin Cancers in Patients With Epidermolysis Bullosa Receiving Filsuvez
An observational study of Epidermolysis Bullosa, Dystrophic and Epidermolysis Bullosa, Junctional, run by Chiesi Farmaceutici S.p.A. at 12 sites in 6 countries.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Epidermolysis Bullosa, Dystrophic, Epidermolysis Bullosa, Junctional
- What is being tested?
- Filsuvez
- How many people
- 170 participants
- Where
- 12 sitesin 6 countries
- Study dates
- 18 Dec 2024to 1 Aug 2032
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- 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.
Who may be able to join
- Patients with a confirmed diagnosis of dystrophic EB or junctional EB
Who cannot join
- None
Trial locations and contacts
12 sites in 6 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 6 countries
United States6 sites, 6 recruiting
- Not yet recruiting
UCHealth / Children's Hospital Colorado
Aurora, Colorado, 80045
Emily Gorell, DO, 707-319-9269, emily.gorell@cuanschutz.edu
- Not yet recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Lara Wine Lee, MD, 843-792-3021, winelee@musc.edu
- Not yet recruiting
Cincinnati Children's
Cincinnati, Ohio, 45229
Kalyani Marathe, 513-636-2009, Kalyani.Marathe@cchmc.org
- Not yet recruiting
UF College of Medicine
Gainesville, Florida, 32606
Michael Lavery, MD, 352-594-1547, m.lavery@dermatology.med.ufl.edu
- Not yet recruiting
Lucile Packard Children's Hospital
Palo Alto, California, 94304
Joyce Teng, MD, 650-724-9627, jteng3@stanford.edu
- Not yet recruiting
Childrens Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Marissa Perman-Laskin, MD, 215-590-1930, permanm@chop.edu
Spain2 sites, 2 recruiting
- Recruiting
Hospital Clinic, Barcelona
Barcelona
Josep Riera Monroig, 93 227 54 00, jriera@clinic.cat
Josep Riera Monroig
- Recruiting
Hospital San Juan de Dios (Barcelona)
Barcelona
Asuncion Vicente, asuncion.vicente@sjd.es
Asuncion Vicente
France1 site, 1 recruiting
- Recruiting
CHU de Toulouse, Hôpital Larrey
Toulouse
Juliette Mazereeuw Hautier, 33 5 67 77 18 76, mazereeuw-hautier.j@chu-toulouse.fr
Juliette Mazereeuw Hautier
Germany1 site, 1 recruiting
- Recruiting
UMC Freiburg EB Centre
Freiburg im Breisgau, 79104
Cristina Has, MD, 4976127069920, cristina.has@uniklinik-freiburg.de
Greece1 site, 1 recruiting
- Recruiting
Hospital of Skin and Venereal Diseases of Thessaloniki
Thessaloniki
Dimitra Kiritsi, 0030-6983375771, dimkyritsi@auth.gr
Dimitra Kiritsi
United Kingdom1 site, 1 recruiting
- Recruiting
Guy's and St Thomas' NHS Foundation Trust
London
Jemima Mellerio, 44 20 7188 6399, jemima.mellerio@gstt.nhs.uk
Jemima Mellerio
About this study
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
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 a minimum of 3 years. The main purpose is to see if the use of Filsuvez affects the likelihood of developing skin malignancies in these patient populations.
Read the 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 clinical study centers in the European Union (EU), United Kingdom (UK) and the United States (US). Participants will be followed for a minimum of 3 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.
- Being tested
- Filsuvez
- Keywords
- Filsuvez, Birch bark extract
- Medical subject headings
- Epidermolysis Bullosa Dystrophica, Epidermolysis Bullosa, Junctional
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FilsuvezDrug
Also called Birch bark extract
Topical gel
| Study group | Role | What they receive |
|---|---|---|
| Patients with dystrophic or junctional EB being treated with Filsuvez | Patients in this cohort will be receiving treatment with Filsuvez for their EB. Receives: 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Minimum 3 years
Secondary outcomes (1)
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
Measured: Minimum 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2024
- Study start18 Dec 2024
- Primary completion30 Nov 2031 (estimated)
- Study completion1 Aug 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa, DystrophicStudies of the same condition, open and under way.
- More trials run by Chiesi Farmaceutici S.p.A.The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 170 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chiesi Farmaceutici S.p.A.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06423573
- Sponsor's protocol ID
- CLI-FILSVAA1-01
- Acronym
- FOSteR
- Status last verified
- Sep 2025
- First submitted
- 16 May 2024
- Last updated
- 28 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06423573
- Acronym / sponsor ID
- FOSteR / CLI-FILSVAA1-01
- Keywords
- Filsuvez, Birch bark extract
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 170 participants (estimated)
- Lead sponsor
- Chiesi Farmaceutici S.p.A.Industry (pharma, biotech, devices)
- Study start
- 18 Dec 2024 (actual)
- Primary completion
- 30 Nov 2031 (estimated)
- Study completion
- 1 Aug 2032 (estimated)
- First posted
- 21 May 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 a minimum of 3 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 clinical study centers in the European Union (EU), United Kingdom (UK) and the United States (US). Participants will be followed for a minimum of 3 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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with dystrophic or junctional EB being treated with Filsuvez | – | Drug: FilsuvezPatients in this cohort will be receiving treatment with Filsuvez for their EB. |
| 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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- 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.
Key inclusion criteria (1 listed)
- Patients with a confirmed diagnosis of dystrophic EB or junctional EB
Key exclusion criteria (1 listed)
- None
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FilsuvezAlso: Birch bark extractTopical gel | Drug | Patients with dystrophic or junctional EB being treated with Filsuvez |
Primary outcome
Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez
Time frame: Minimum 3 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
1 secondary outcome
Incidence of first skin malignancy during follow-up in EB patients not receiving Filsuvez
Time frame: Minimum 3 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2026.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Epidermolysis Bullosa, Dystrophic and Epidermolysis Bullosa, Junctional, run by Chiesi Farmaceutici S.p.A. at 12 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 28 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, Spain, France, Germany, Greece and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Chiesi Clinical Trials (+3905212791, clinicaltrials_info@chiesi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of first skin malignancy during follow-up in EB patients receiving Filsuvez (time frame: Minimum 3 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2026.