Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex

This is a closed-label, randomized, double-blind, Phase I clinical study to assess the safety and tolerability of the investigational product (IP), TolaSure Gel, 5% w/w, in patients diagnosed with severe epidermolysis bullosa simplex (EBS, previously known as EBS-Dowling Meara). Each patient (5 to complete) will apply TolaSure Gel, 5% w/w and TolaSure Vehicle Gel once-daily to defined Treatment Areas and Suction Blister Areas. End of Study (EOS) will occur at the Principal Investigator's (PI's) discretion according to the resolution or lack of improvement of the Treatment Areas. The maximum time a patient could be in the study is 10 weeks. The average time frame for each patient to complete the study is expected to be 6-8 weeks.

The primary safety endpoint will be the incidence of treatment-emergent adverse events (TEAEs). Safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate).

Secondary efficacy endpoints will (1) assess wound severity within the Treatment Areas using an Investigator Global Assessment (IGA) scoring system, (2) examine under a microscope keratinocyte morphology and the incidence of keratin aggregates in Treatment Area skin biopsies, (3) determine the time required to induce a suction blister within the designated Suction Blister Areas, and (4) evaluate patient self-assessment of overall impression, pain, and itch within the Treatment Areas. The information obtained from these assessments will be used to examine the efficacy of TolaSure treatment in improving EBS symptoms.