Epidermolysis Bullosa SimplexPhase 2FILS-EBSNCT07756138
Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex
A Phase 2 study testing Filsuvez in people with Epidermolysis Bullosa Simplex, run by Stanford University at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 months and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex
- What is being tested?
- Filsuvez
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Dec 2026to 31 Jul 2028
- Phase
- Phase 2
- 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 6 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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)
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- 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 participation.
- Subject is in good, general health and free of any known disease state or physical condition which, in the investigator's opinion, might interfere evaluation of the EBS lesions during study or which exposes the subject to an unacceptable risk by study participation.
- Over the duration of the study, the subject agrees to not use any other topical therapies and/or impregnated dressings within the TLAs (e.g., medicated cleansers, CBD oil, MediHoney, Silvadene cream 1%, topicals containing antimicrobials, keratin, and/or collagen, lipid-colloid or polymeric membrane dressings, and/or hydrogels).
- Subject and/or legally appointed and authorized representative must be able and willing to follow study procedures and instructions to maintain compliance throughout the study period.
- The subject, parent, or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent and Assent Form if applicable.
Who cannot join
- Subject's use of prior or concomitant medication or medical treatments/procedures:
- Any investigational drug or therapy within 30 days.
- Systemic steroidal therapy within 30 days.
- Topical steroidal therapy in TLA within 14 days (Note: inhaled and ophthalmic products containing steroids are allowed).
- Systemic antibiotic therapy within 7 days.
- Currently receiving chemotherapy or radiation.
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- Surgery within the previous 2 weeks (except for minor surgery, cosmetic or dental procedures as determined by the investigator).
- Started to take chronic medications (NSAIDs, antihistamines, etc.) at least 30 days prior to starting study medication.
- Subject's medical history includes:
- Cancer that is currently undergoing treatment.
- Current systemic or ongoing skin infection.
- HIV/AIDS.
- Non-EBS skin disease (e.g., psoriasis, atopic dermatitis, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments.
- An illness (e.g., neurological, cardiovascular, respiratory, hepatic, renal, or metabolic disease), condition, or situation that in the opinion of the principal investigator is likely to interfere with the subject's participation in or completion of the study.
- Factors present in the subject and/or his/her legal representative that could interfere with study compliance such as inability to attend scheduled study visits or to perform study protocol procedures.
- Subject is a member of the investigational team or his/her immediate family.
- Other unspecified reasons that, in the opinion of the Investigator, make the subject unsuitable for enrollment.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site
Stanford University School of Medicine
Palo Alto, California, 94304
Joyce M. C. Teng, MD PhD
Chaw-Ning Lee, MD PHD
About this study
Official title: A Pilot Trial to Evaluate the Efficacy and Safety of Filsuvez in the Treatment of Moderate-to-Severe Epidermolysis Bullosa Simplex
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.
- Conditions
- Epidermolysis Bullosa Simplex
- Being tested
- Filsuvez
- Medical subject headings
- Epidermolysis Bullosa Simplex
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FilsuvezDrug
All the participants will apply Filsuvez about 3% BSA for 3 months.
| Study group | Role | What they receive |
|---|---|---|
| Filsuvez | Experimental | All the participants will apply Filsuvez about 3% BSA for 3 months and a follow-up visit 2 months later. Receives: Drug: Filsuvez |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Secondary outcomes (7)
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.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Aug 2026
- Study start15 Dec 2026 (estimated)
- Primary completion15 Jan 2027 (estimated)
- Study completion31 Jul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa SimplexStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Joyce Teng, Stanford University
- Investigators
- Joyce Teng, MD, PhD, FAAD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07756138
- Sponsor's protocol ID
- IRB-87068
- Acronym
- FILS-EBS
- Status last verified
- Jun 2026
- First submitted
- 24 Jul 2026
- Last updated
- 5 Nov 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Trial to Evaluate the Efficacy and Safety of Filsuvez in the Treatment of Moderate-to-Severe Epidermolysis Bullosa Simplex
- ClinicalTrials.gov ID
- NCT07756138
- Acronym / sponsor ID
- FILS-EBS / IRB-87068
- Conditions
- Epidermolysis Bullosa Simplex
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- 15 Dec 2026 (estimated)
- Primary completion
- 15 Jan 2027 (estimated)
- Study completion
- 31 Jul 2028 (estimated)
- First posted
- 10 Aug 2026 (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.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Filsuvez | Experimental | Drug: FilsuvezAll the participants will apply Filsuvez about 3% BSA for 3 months and a follow-up visit 2 months later. |
Who may qualify
- Ages
- 6 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (14 listed)
- 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:
- If the subject is a woman of childbearing potential (WOCBP) (Need to verify current labeling on use during pregnancy)
- 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 participation.
- Subject is in good, general health and free of any known disease state or physical condition which, in the investigator's opinion, might interfere evaluation of the EBS lesions during study or which exposes the subject to an unacceptable risk by study participation.
Key exclusion criteria (17 listed)
- Subject's use of prior or concomitant medication or medical treatments/procedures:
- Subject's medical history includes:
- Factors present in the subject and/or his/her legal representative that could interfere with study compliance such as inability to attend scheduled study visits or to perform study protocol procedures.
- Subject is a member of the investigational team or his/her immediate family.
- Other unspecified reasons that, in the opinion of the Investigator, make the subject unsuitable for enrollment.
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 |
|---|---|---|
| FilsuvezAll the participants will apply Filsuvez about 3% BSA for 3 months. | Drug | Filsuvez |
Primary outcome
Change in Blister surface area reduction of the target treatment areas
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
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.
Change in EBSdart scores
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
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.
7 secondary outcomes
Change in Palmoplantar Blister Surface Area
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Pain Score in Target Lesional Areas
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Itch Score in Target Lesional Area
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Quality of Life
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Modified Foot Function Index(mFFI)
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Safety: Incidence of treatment-emergent adverse events (TEAEs)
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
Change in Palmoplantar Blister Surface Area- EBSDart scores
Time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).
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 5 Nov 2026.
- Individual participant dataSharing planned: No
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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.
Joyce Teng, MD, PhD, FAAD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Filsuvez in people with Epidermolysis Bullosa Simplex, run by Stanford University at 1 site in United States.
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 14 inclusion and 17 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: opening soon, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Blister surface area reduction of the target treatment areas (time frame: From enrollment to the end of treatment at Week12 and Week20 (EOS).). The study lists 2 primary outcomes in all. 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 5 Nov 2026.