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

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.

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