RecruitingPhase 2

A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex

United States40 participantsStarted 2025-09-11

Plain-language summary

This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and older) diagnosed with localized, intermediate, or severe epidermolysis bullosa simplex (EBS). Each patient (40 to complete) will be enrolled in the study and will be randomized to receive either TolaSure Gel or a topical Placebo for daily application for 2-months. After 2-months, all patients will receive TolaSure Gel to daily apply for an additional 2-months. A remote follow-up visit will occur 2-months after the end of study. Total time in the study is 6-months. Localized patients will be applying study medication to their feet, with the option to treat their hands as well throughout the study. Intermediate and severe EBS patients will be applying study medication to randomized treatment area(s) (a minimum of \~2-3% Body Surface Area (BSA)), with the option to treat their feet as well throughout the study.

Who can participate

Age range

2 Years

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: * Patient is a male or female at least 2 years of age. * Patient has a documented diagnosis and confirmed clinical history of localized (previously known as Weber-Cockayne), intermediate (previously Kӧbner), or severe (previously EBS-Dowling Meara) autosomal dominant EBS and/or a genetic mutation in either the KRT14 or KRT5 gene consistent with localized, intermediate, or severe EBS. The Investigator will determine patient eligibility based on historical phenotypic presentation of EBS symptoms along with genetic/diagnosis documentation to determine EBS severity. (If localized, intermediate, or severe EBS is suspected but not diagnosed or genetically confirmed, confirmatory testing will be performed). * For localized EBS participants: * Patient must have at least moderate plantar disease activity, defined as ≥15% involvement of the plantar surface of at least one foot, as assessed by clinical examination or standardized imaging, with evidence of active disease (e.g., blisters, erosions, or crusting). * For treatment, the Target Lesional Area (TLA) will consist of daily treating the feet (tops and bottoms) which is approximately 7% body surface area (BSA). Localized EBS subjects will also have the option to treat their hands (tops and bottoms), which is approximately an additional 6% BSA. Palmoplantar evaluation is separate from the intermediate to severe TLA-treated regions per protocol. * For intermediate to severe EBS participants: Patient is active…

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

Change in Blister Surface Area of the Designated Treatment Area(s)

Timeframe: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)

Trial details

NCT IDNCT07027345

SponsorBioMendics, LLC

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2027-01

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