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

Conditions

• Epidermolysis Bullosa Simplex

Eligibility Criteria

Sex

ALL

Age

4 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date

Sep 30, 2025

Status verified

Sep 2025

Primary completion

Dec 31, 2025

Completion

Sep 30, 2026

Study Design

Enrollment

40 participants (estimated)

Allocation

RANDOMIZED

Intervention model

CROSSOVER

Primary purpose

TREATMENT

Arms

• Active Comparator: 5% TolaSure Topical Gel
  5% (w/w) TolaSure Gel
• Placebo Comparator: Topical Placebo Gel
  Placebo Control Gel

Primary Outcome Measure

Change in Blister Surface Area of the Designated Treatment Area(s) [ Time Frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months) ]

Locations (2)

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