TolaSure for Epidermolysis Bullosa Simplex

((TAMES) Trial)

Recruiting at 1 trial location

Overseen By Karen McGuire, PhD

Age: Any Age

Sex: Any

Trial Phase: Phase 2

Sponsor: BioMendics, LLC

Must not be taking: Steroids, Antibiotics, Chemotherapy, others

Disqualifiers: Cancer, HIV/AIDS, Systemic infection, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 2

40 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to evaluate the effectiveness of TolaSure Gel for individuals with generalized intermediate to severe epidermolysis bullosa simplex (EBS), a condition that causes fragile skin prone to blistering. Participants will apply either TolaSure Gel or a placebo (a non-active substance) to their skin for two months, followed by the real gel for another two months. The study seeks individuals diagnosed with EBS who are experiencing flare-ups, particularly on their legs or torso. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering participants the opportunity to contribute to significant advancements in EBS treatment.

Will I have to stop taking my current medications?

The trial requires that you stop using certain medications before participating. You must not have used any investigational drugs, systemic steroids, or topical steroids recently, and you should not start new chronic medications like NSAIDs or antihistamines within 30 days before starting the study medication.

Is there any evidence suggesting that TolaSure Gel is likely to be safe for humans?

Research has shown that TolaSure Gel was tested for safety in earlier studies. In these trials, patients tolerated the gel well when applied to affected skin. Some reported mild side effects, such as skin irritation, but these were not serious for most participants. This suggests the treatment is generally safe. However, as with any treatment, experiences can vary, so monitoring for any reactions during the trial is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for epidermolysis bullosa simplex?

TolaSure Gel is unique for treating Epidermolysis Bullosa Simplex because it introduces a topical approach using a 5% concentration of TolaSure, which is designed to directly target skin fragility at the site of application. Unlike current treatments that mainly focus on managing symptoms through wound care and pain management, TolaSure Gel aims to strengthen the skin's structural integrity. Researchers are excited because this gel could potentially minimize blister formation and offer a more direct and effective way to address the root cause of the condition, rather than just alleviating its symptoms.

What evidence suggests that TolaSure Gel might be an effective treatment for epidermolysis bullosa simplex?

Research has shown that TolaSure Gel, which participants in this trial may receive, may improve skin conditions in people with epidermolysis bullosa simplex (EBS). Early results from past studies indicate that TolaSure can help skin cells adhere better. This is important because it reduces the clumping of certain proteins linked to blistering. One study found that TolaSure Gel reduced blister size by the third week. These findings suggest that TolaSure Gel may help reduce skin blistering and improve skin health in people with EBS. ^{1 2 3 5 6}

Are You a Good Fit for This Trial?

This trial is for adults and children aged 4 and older with generalized intermediate to severe epidermolysis bullosa simplex (EBS). Participants will apply a gel on their skin, covering at least 2-3% of their body. The study excludes specific details not provided.

Inclusion Criteria

I am in good health with no conditions that could affect my skin lesion evaluation.

I am at least 4 years old.

I have a severe form of EBS with a K14 or K5 gene mutation.

Exclusion Criteria

Patient is a member of the investigational team or immediate family

I have recently used specific medications or undergone medical treatments.

Factors present in the patient and/or legal representative that could interfere with study compliance

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

1 visit (in-person)

Treatment Part 1

Participants receive daily application of randomized treatment (TolaSure Gel or Placebo) for 2 months on selected Target Lesional Areas

8 weeks

Weekly assessments (virtual)

Treatment Part 2

Crossover phase where all participants receive TolaSure Gel for an additional 2 months

8 weeks

Weekly assessments (virtual)

Follow-up

Participants are monitored for safety and effectiveness after treatment

8 weeks

1 remote follow-up visit

What Are the Treatments Tested in This Trial?

Interventions

• TolaSure Gel

Trial Overview The study tests TolaSure Topical Gel's effectiveness compared to a placebo in treating EBS. Patients are randomly chosen to use either the real gel or placebo for two months, then all get the real gel for another two months, followed by a remote check-up after six months total.

How Is the Trial Designed?

Group I: 5% TolaSure Topical Gel Active Control 1 Intervention

5% (w/w) TolaSure Gel

Group II: Topical Placebo Gel Placebo Group 1 Intervention

Placebo Control Gel

Who Is Running the Clinical Trial?

BioMendics, LLC

Lead Sponsor

Trials

3

Recruited

100+

Lucile Packard Children's Hospital

Collaborator

Trials

16

Recruited

2,800+

Stanford University

Collaborator

Trials

2,527

Recruited

17,430,000+

Northwestern University Feinberg School of Medicine

Collaborator

Trials

42

Recruited

15,500+

Ann & Robert H Lurie Children's Hospital of Chicago

Collaborator

Trials

275

Recruited

5,182,000+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT05062070

NCT05062070 | Safety and Efficacy of Topical TolaSure ... This Phase I study will assess the safety, tolerability, and clinical effects of TolaSure when applied to wounded skin areas of patients diagnosed with severe ...

2. jidonline.org jidonline.org/article/S0022-202X(23)00779-0/fulltext

597 Interim results of a phase I/II, closed label, randomized ... These findings support improvement of keratinocyte organization and adhesion as well as a reduction of keratin aggregates in areas of active blistering that ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT07027345

A Phase II, Placebo Controlled, Clinical Trial of Topical ... 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 ...

4. biomendics.com biomendics.com/index.php/medical-professionals

Information for Medical Professionals ... EBS adults. Treatment: TolaSure Gel 5% w/w vs. Vehicle, applied daily to matched TLAs (up to 10 weeks). Key Outcomes. Blister surface area reduction. Week 3 ...

5. debra.org debra.org/clinical-trials/phase-i-clinical-trial-tolasure-simplex

Phase I Clinical Trial of TolaSure for Simplex This Phase I study will assess the safety, tolerability, and clinical effects of TolaSure when applied to wounded skin areas of patients diagnosed with severe ...

6. synapse.patsnap.com synapse.patsnap.com/drug/5472bd3513fa40938a059d73dd50b311

TolaSure - Drug Targets, Indications, Patents A Phase II, Closed Label, Placebo Controlled, Randomized, Double-Blinded Clinical Trial to Evaluate the Efficacy and Safety of TolaSure Gel, ...