NCT07027345

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

A Phase II, Closed Label, Placebo Controlled, Randomized, Double-Blinded Clinical Trial to Evaluate the Efficacy and Safety of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex (TAMES)

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 40 (estimated)
• Sponsor: BioMendics, LLC · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (4 years of age and older) diagnosed with generalized intermediate to 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. 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.

Detailed description

This is a closed-label, Phase II clinical study to assess the efficacy, safety and tolerability of the investigational product (IP), topical TolaSure Gel, 5% w/w, in adults and pediatric patients diagnosed with generalized intermediate to severe epidermolysis bullosa simplex (EBS). TolaSure Gel, 5% w/w and Placebo tubes will be placed into kits where all labels (tubes and kit labels) will be blinded for drug content. Kits will be bundled (based on expected amount of study gel usage) and distributed randomly to patients \[Goal of patient population, 1:1 M:F and approximately 70% pediatric patients\]. Each patient (40 to complete) will be enrolled in the study and once a blistering flare is confirmed by the clinical study team, and the patient will be randomized to receive either TolaSure Gel, 5% w/w or Placebo (50:50 chance). For Part 1 of the study, daily application of randomized treatment for 2 months will be applied to selected Target Lesional Areas (TLAs; a minimum area of approximately 2-3% Body Surface Area (BSA)). If a TLA resolves completely the patient will continue to treat designated area(s). Additionally, patients have the option to daily treat their feet. For Part 2 of the study, a single arm crossover will then allow those patients that received Placebo to go on TolaSure Gel, 5% w/w for a subsequent 2 months. The patients already in the TolaSure Gel, 5% w/w cohort will continue with daily IP application for an additional 2 months as well. Again, patients have the option to continue to treat their feet. At Part 2 End of Study (EOS), patients will cease IP application and EOS procedures will be completed. Patients can have optional perilesional biopsies taken from a treated and untreated area to evaluate changes in ultrastructural pathology. A patient follow-up visit will occur after 6 months (2-months after last treatment application). The primary endpoint will assess changes in disease severity within the TLAs and recurrence of blistering using clinical imaging to assess blister surface area over time. The secondary efficacy endpoints will evaluate blistering on the feet, subject self-assessments including pain, itch, modified Foot Function Index (mFFI), and Quality of Life (QoL) assessment. Safety endpoints will include the incidence of treatment-emergent adverse events (TEAEs). In order to provide adequate assurance of subject safety, safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate). These safety assessments in addition to adverse event (AE) reviews will be sufficient to identify potential TEAEs. The information obtained from these assessments will be utilized to examine the efficacy of TolaSure treatment in ameliorating EBS symptoms as well as the accuracy of the proposed mechanism of action (MOA) of TolaSure for this disease indication.

Conditions

• Epidermolysis Bullosa Simplex

Interventions

Type	Name	Description
DRUG	5% TolaSure Topical Gel	TolaSure Topical Gel is applied once-daily to designated treatment areas for up to 4 months (Part 2 End of Study).
DRUG	Topical Placebo Gel	Topical Placebo Gel is applied once-daily to designated treatment areas for up to 2 months (Part 1 End of Study).

Timeline

• Start date: 2025-09-01
• Primary completion: 2025-12-01
• Completion: 2026-09-01
• First posted: 2025-06-18
• Last updated: 2025-09-11

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study