Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex

NCT07756138 | Not Yet Recruiting Phase 2 FDA Drug

• 1 other identifier: IRB-87068
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Epidermolysis Bullosa Simplex

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

Secondary Outcomes (7)

• Change in Palmoplantar Blister Surface Area

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

• Change in Pain Score in Target Lesional Areas

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

• Change in Itch Score in Target Lesional Area

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

• Change in Quality of Life

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

• Change in Modified Foot Function Index(mFFI)

  From enrollment to the end of treatment at Week12 and Week20 (EOS).

Study Arms (1)

Filsuvez

EXPERIMENTAL

All the participants will apply Filsuvez about 3% BSA for 3 months and a follow-up visit 2 months later.

Drug: Filsuvez

Interventions

Filsuvez DRUG

All the participants will apply Filsuvez about 3% BSA for 3 months.

Filsuvez

Eligibility Criteria

• Age: 6 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Stanford University School of Medicine

Palo Alto, California, 94304, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Simplex

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous

Study Officials

• Joyce Teng, MD, PhD, FAAD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of dermatology, Stanford University

Study Record Dates

• First Submitted: July 24, 2026
• First Posted: August 10, 2026
• Study Start (Estimated): October 1, 2026
• Primary Completion (Estimated): July 2, 2027
• Study Completion (Estimated): June 18, 2028
• Last Updated: August 10, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)