Actively Recruiting
A Phase II, Placebo Controlled, Randomized, Double-Blinded Trial to Evaluate the Safety and Efficacy of TolaSure Gel in Epidermolysis Bullosa Simplex
Led by BioMendics, LLC · Updated on 2025-09-11
40
Participants Needed
2
Research Sites
39 weeks
Total Duration
On this page
Sponsors
B
BioMendics, LLC
Lead Sponsor
N
Northwestern University
Collaborating Sponsor
AI-Summary
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 TolaSure's potential to reduce blistering and improve symptoms in EBS over the course of 4 months of treatment plus 2 months of follow-up.
CONDITIONS
Brief Title
A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient is male or female aged 4 years or older.
- Diagnosed with generalized intermediate to severe epidermolysis bullosa simplex (EBS) or has a genetic mutation in K14 or K5 genes consistent with this condition.
- Currently experiencing an active blistering flare in target areas such as lower legs or torso covering about 2-3% of body surface area.
- Target lesion areas must not be infected or treated with topical antibiotics within 14 days.
- Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control during and for 3 months after the study.
- Patient must not be nursing.
- Laboratory values must be within normal or acceptable ranges.
- Patient must be in good general health without conditions that impair evaluation or increase risk.
- Must agree to avoid other topical therapies on target areas during the study.
- Patient or authorized representative must be willing and able to follow study procedures.
- Must provide informed consent or assent as approved by ethics committee.
You will not qualify if you...
- Use of any investigational drug or therapy within 30 days prior to study.
- Use of systemic steroids within 30 days or topical steroids within 14 days (except inhaled or ophthalmic).
- Use of systemic antibiotics within 7 days.
- Currently receiving chemotherapy or radiation therapy.
- Surgery within the past 2 weeks except minor or cosmetic procedures.
- Starting chronic medications less than 30 days before study drug.
- Current cancer treatment.
- History of severe nutritional deficiencies.
- Current systemic infection.
- HIV/AIDS diagnosis.
- Presence of non-EBS skin diseases or conditions that may interfere with study or increase risk.
- Illnesses or conditions likely to interfere with participation or completion.
- Inability to attend visits or comply with study procedures.
- Being a member of the investigational team or immediate family.
- Other reasons deemed unsuitable by the investigator.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 2 months
Participants apply either TolaSure Gel or placebo gel once daily to designated treatment areas during the first 2 months.
Weekly visits for assessments during treatment
Duration - Up to 2 additional months
Participants continue daily application of TolaSure Gel for an additional 2 months. Those initially on placebo switch to TolaSure Gel in this crossover phase.
Weekly visits for assessments during treatment
Duration - 6 months from start of treatment, with follow-up 2 months after last treatment
Participants stop treatment and complete end of study procedures, followed by a follow-up visit 2 months after treatment ends to monitor safety and outcomes.
1 follow-up visit after treatment completion
Trial Site Locations
Total: 2 locations
1
Stanford University School of Medicine, Dermatology Department
Palo Alto, California, United States, 94304
Actively Recruiting
2
NU Dermatolgy CTU
Chicago, Illinois, United States, 60611
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
CROSSOVER
Primary Purpose
TREATMENT
Number of Arms
2
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here