What is TolaSure studying for epidermolysis bullosa simplex?

TolaSure is being studied to evaluate its ability to reduce blistering and improve symptoms in people with epidermolysis bullosa simplex (EBS). This clinical trial aims to assess the gel's safety and effectiveness in both adults and children with moderate to severe forms of EBS. The research is sponsored by BioMendics, LLC to better understand this treatment option.

Who may be eligible for this epidermolysis bullosa simplex clinical trial?

Children aged 4 years and older and adults diagnosed with moderate to severe epidermolysis bullosa simplex may be eligible. Both males and females can participate. Important factors include having active blister flares in specific skin areas, not using certain medications or topical treatments recently, and being in good general health. Please review eligibility criteria for more details.

Is there any cost to participate in this epidermolysis bullosa simplex clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not always covered. Participants should plan accordingly for any costs related to getting to and from study visits.

Actively Recruiting

Phase 2

Age: 4Years +

All Genders

ID07027345

Phase II Randomized, Double-Blind Trial of Topical TolaSure Gel for Treating Generalized Intermediate to Severe Epidermolysis Bullosa Simplex in Children and Adults

Led by BioMendics, LLC · Updated on 2025-09-11

Participants Needed

Research Sites

39 weeks

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the effectiveness, safety, and tolerability of a topical gel called TolaSure in adults and children aged 4 years and older with generalized intermediate to severe epidermolysis bullosa simplex EBS. This Phase II clinical study involves 40 patients who will be randomly assigned to receive either TolaSure Gel or a placebo applied daily for 2 months. After this period, all patients will receive TolaSure Gel for another 2 months, followed by a remote follow-up 2 months after treatment ends, totaling 6 months of participation. Participants will apply the study gel to selected areas with active blistering covering approximately 2-3% of their body surface area, with the option to treat their feet throughout the study. The study begins with a randomized, double-blinded phase where patients receive either TolaSure or placebo daily. After 2 months, the placebo group switches to TolaSure for an additional 2 months, while the original TolaSure group continues treatment. Optional skin biopsies may be taken to evaluate tissue changes. During the study, patients will be monitored weekly for changes in blister surface area, especially in treated areas and on the feet. They will also report pain, itch, foot function, and quality of life measures. Safety will be closely followed through physical exams, vital signs, lab tests, and adverse event reporting. The study aims to better understand TolaSures potential to reduce blistering and improve symptoms in EBS over the course of 4 months of treatment plus 2 months of follow-up.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 4Years +

All Genders

Eligibility Criteria

You may qualify if you...

Patient is male or female aged 4 years or older.
Diagnosed with generalized intermediate to severe epidermolysis bullosa simplex (EBS) or has a genetic mutation in K14 or K5 genes consistent with this condition.
Currently experiencing an active blistering flare in target areas such as lower legs or torso covering about 2-3% of body surface area.
Target lesion areas must not be infected or treated with topical antibiotics within 14 days.
Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during and for 3 months after the study.
Patient must not be nursing.
Laboratory values must be within normal or acceptable ranges.
Patient must be in good general health without conditions that impair evaluation or increase risk.
Must agree to avoid other topical therapies on target areas during the study.
Patient or authorized representative must be willing and able to follow study procedures.
Must provide informed consent or assent as approved by ethics committee.

You will not qualify if you...

Use of any investigational drug or therapy within 30 days prior to study.
Use of systemic steroids within 30 days or topical steroids within 14 days (except inhaled or ophthalmic).
Use of systemic antibiotics within 7 days.
Currently receiving chemotherapy or radiation therapy.
Surgery within the past 2 weeks except minor or cosmetic procedures.
Starting chronic medications less than 30 days before study drug.
Current cancer treatment.
History of severe nutritional deficiencies.
Current systemic infection.
HIV/AIDS diagnosis.
Presence of non-EBS skin diseases or conditions that may interfere with study or increase risk.
Illnesses or conditions likely to interfere with participation or completion.
Inability to attend visits or comply with study procedures.
Being a member of the investigational team or immediate family.
Other reasons deemed unsuitable by the investigator.

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