Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex
United States10 participantsStarted 2026-10
Plain-language summary
This pilot study will look at whether the topical Birch Triterpenes (Filsuvez) gel is safe and helpful for adults and children 6 months of age and older who have moderate to severe epidermolysis bullosa simplex (EBS). The study will examine whether the gel can reduce the number of blisters, help wounds heal faster, and improve comfort by reducing pain and itching and making daily activities easier.
Who can participate
Age range
6 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Subject has a documented diagnosis and confirmed clinical history of EBS. The Investigator will determine subject eligibility based on historical phenotypic presentation of EBS symptoms along with genetic/diagnosis documentation in order to determine EBS severity.
* The following skin conditions are required:
* A minimum area of \~3% BSA containing active blisters/erosions, and freshly ruptured blisters across 50% of the TLA (as assessed by the PI). Skin erosions, keratoderma, fissures and/or erythema may also be present.
* Subjects will be permitted to treat their feet to assess plantar blister surface area but blistering on the feet is not a requirement for study inclusion.
* TLA may not be infected (as assessed by PI) or have been treated with a topical antibiotic within 14 days.
* If the subject is a woman of childbearing potential (WOCBP) (Need to verify current labeling on use during pregnancy)
* Has a negative urine pregnancy test at baseline.
* Agrees to use an approved effective form of birth control with failure rates \<1% per year (e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomized partner) during participation in the study.
* Is not nursing.
* Subject's laboratory values (blood and urine) are within the range of normal or abnormal values are within normal levels for the disease and in the opinion of the PI the values are not clinically relevant for study pa…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Change in Blister surface area reduction of the target treatment areas
Timeframe: From enrollment to the end of treatment at Week12 and Week20 (EOS).
2
Change in EBSdart scores
Timeframe: From enrollment to the end of treatment at Week12 and Week20 (EOS).