Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex
ConditionEpidermolysis Bullosa Simplex
Trial statusNot yet recruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age6+
SponsorStanford University
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
Filsuvez
DrugAll the participants will apply Filsuvez about 3% BSA for 3 months.
Treatment groups
10 Participants
are divided into 1 treatment groupGroup A: FilsuvezExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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).2
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).Secondary outcomes
1
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).2
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).3
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).4
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).Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: