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)
BioMendics, LLC
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.
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 patie…
Eligibility
- Age range
- 4+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * Patient is a male or female at least 4 years of age. * Patient has a documented diagnosis of generalize intermediate (previously Kӧbner) to severe (previously EBS-DM) autosomal dominant epidermolysis bullosa simplex (EBS) and/or genetic mutation in either the K14 or K5 genes consistent with generalized intermediate to severe EBS. The Investigator will determine patient eligibility based on historical phenotypic presentation of EBS symptoms along with genetic/diagnosis documentation in order to determine EBS severity. (If generalized intermediate to severe EBS is suspecte…
Interventions
- Drug5% TolaSure Topical Gel
TolaSure Topical Gel is applied once-daily to designated treatment areas for up to 4 months (Part 2 End of Study).
- DrugTopical Placebo Gel
Topical Placebo Gel is applied once-daily to designated treatment areas for up to 2 months (Part 1 End of Study).
Locations (2)
- Stanford University School of Medicine, Dermatology DepartmentPalo Alto, California
- NU Dermatolgy CTUChicago, Illinois