A Phase 2 interventional study of Filsuvez in Epidermolysis Bullosa Simplex, sponsored by Stanford University. Not yet recruiting at 1 site in United States. Open to participants aged 6 Months and older. Per ClinicalTrials.gov, last updated 2026-11-05.
Sponsored by Stanford University · Phase 2, Interventional, and Treatment
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.
20 studies on the registry are indexed under Epidermolysis Bullosa Simplex; 5 are open to participants now.
This study's planned enrollment of 10 is below the median of 19 across 17 interventional studies indexed under Epidermolysis Bullosa Simplex.
Browse Epidermolysis Bullosa Simplex studies →Stanford University is the lead sponsor of 2,168 studies on the registry; 431 are open to participants now.
Of its 267 completed or terminated interventional studies of FDA-regulated products, 207 (78%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
The following skin conditions are required:
If the subject is a woman of (WOCBP) (Need to verify current labeling on use during pregnancy)
Exclusion Criteria:
Subject's use of prior or concomitant medication or medical treatments/procedures:
Subject's medical history includes:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
All the participants will apply Filsuvez about 3% BSA for 3 months and a follow-up visit 2 months later.
Drug: Filsuvez
All the participants will apply Filsuvez about 3% BSA for 3 months.
Change in Blister surface area reduction of the target treatment areas
Clinical photography will be used to assess changes in disease severity within the TLA and recurrence of blistering by measuring blister surface area in the TLA over time.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in EBSdart scores
Epidermolysis Bullosa Simplex Disease Activity and Response Tool (EBSdart) grades blistering and erosions/ulcerations in all body regions and fissures and keratoderma on the palms and soles, including the area affected. IGA regional subscores are recorded separately for the palms, soles, trunk, upper extremities, and lower extremities, using palm- and sole-specific criteria where applicable. A score range from 0-99, where higher scores indicate severe condition.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Palmoplantar Blister Surface Area
Clinical photography will be used to assess changes in disease severity within the Palmoplantar blister surface area and recurrence of blistering by measuring blister surface area in the Palmoplantar blister surface area over time.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Pain Score in Target Lesional Areas
Using the adult or pediatric Patient-Reported Outcomes Measurement Information System (PROMIS) Measure form, peak pain during IP application will be reported on a weekly basis. Pain will be compared between baseline and at the end of treatment. The appropriate PROMIS Pain Interference Short Form will be administered based on the participant's age: the Adult Pain Interference Short Form 8a, the Pediatric Pain (8yo+) Interference Short Form 8a, or the Parent Proxy Pain (6m-8yo) Interference Short Form 8a. Each item is scored on a 5-point Likert scale (1-5), where higher scores indicate greater pain interference with daily activities (1 = Not at All; 5 = Very Much).
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Itch Score in Target Lesional Area
Using the adult or pediatric PROMIS Measure form (8 yo +), itch characteristics will be reported on a weekly basis to track trends in how itch impacts daily living in Birch triterpenes at baseline versus 3 mo post treatment. The appropriate PROMIS Pain Interference Short Form will be administered based on the participant's age: the Adult Itch Interference Short Form 8a, the Pediatric Itch Interference Short Form 8a, or the Parent Proxy Itch (6m-8yo) Interference Short Form 8a. Each item is scored on a 5-point Likert scale (1-5), where higher scores indicate greater pain interference with daily activities (1 = Never; 5 = Almost always).
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Quality of Life
Participant's quality of life metrics will be reported on a weekly basis to track trends at baseline versus 3 mo post Birch Triterpenes treatment. The Quality of Life in Epidermolysis Bullosa (QOLEB) questionnaire is a disease-specific instrument consisting of 17 items. Each item is scored on a 4-point scale (0-3), yielding a total score ranging from 0 to 51, with higher scores indicating poorer quality of life and greater disease burden.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Modified Foot Function Index(mFFI)
Participant's mFFI will be reported on a biweekly basis to track changes with Birch Triterpenes. The Modified Foot Function Index (mFFI) consists of 27 items that assess foot pain, disability, and activity limitation. Each item is rated on an 11-point numeric rating scale (0-10), where 0 indicates no pain/no difficulty/Not at all and 10 indicates the worst pain imaginable or greatest difficulty/all the time. Total scores range from 0 to 270, with higher scores representing worse foot function and greater disability.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Safety: Incidence of treatment-emergent adverse events (TEAEs)
Safety monitoring will include clinical chemistry tests, physical examinations (including assessment of TLA), and vital sign measurements. These assessments along with AE reviews will identify potential TEAEs. Safety monitoring will include clinical laboratory testing (blood and urine) and urine pregnancy testing (as appropriate) only at Visit 1/Screening and visit 4/EOS.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Palmoplantar Blister Surface Area- EBSDart scores
EBSdart grades blistering and erosions/ulcerations in all body regions and fissures and keratoderma on the palms and soles, including the area affected. IGA regional subscores are recorded separately for the palms, soles, trunk, upper extremities, and lower extremities, using palm- and sole-specific criteria where applicable. A score range from 0-99.
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Plan to share: No
No publications or documents are linked to this record.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Birch triterpenes
Epidermolysis Bullosa Simplex →