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

Launched by BIOMENDICS, LLC · Jun 16, 2025

Keywords

ClinConnect Summary

This clinical trial is studying a new gel called TolaSure to see if it can help people with a skin condition called Epidermolysis Bullosa Simplex (EBS), which causes the skin to be very fragile and blister easily. The study is looking at how safe and effective this gel is when applied to the skin daily, especially for those with moderate to severe forms of EBS. Both adults and children aged 4 years and older can take part, and about 40 participants will be involved. For the first two months, participants will either use the TolaSure gel or a placebo (a gel without the active medicine), and then everyone will use TolaSure for another two months. After finishing the treatment, there will be a follow-up check two months later, so the whole study lasts about six months.

To join the study, participants need to have a confirmed diagnosis of moderate to severe EBS and be currently experiencing a flare-up—meaning they have fresh blisters on certain parts of their body like their legs or torso. They must be in generally good health and able to follow the study instructions, including not using other skin treatments on the areas being treated during the study. Women who can become pregnant need to have a negative pregnancy test and agree to use reliable birth control. The study team will carefully check medical history and current health to make sure it’s safe to participate. If eligible, participants can expect to apply the gel daily to affected skin and have regular check-ins with the study team, some of which may be done remotely. This study offers a chance to try a new treatment that could reduce blistering and improve skin comfort for people living with EBS.

Gender

ALL

Eligibility criteria

• 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 suspected but not diagnosed or genetically confirmed, confirmatory testing will be performed).
• * Patient is actively flaring in one of the preferred target lesional areas (TLAs): 1) lower extremities (ideally below the knee and above the ankle or between the knee and top of the thigh) or 2) torso (excluding the groin and apocrine areas). The following skin conditions are required for treatment purposes:
• • A flare is defined as a minimum area of \~2-3% Body Surface Area (BSA) containing intact blisters (of varying size and number), and freshly ruptured blisters across 50% of the TLA (as assessed by the principal investigator (PI)). Skin erosions, keratoderma, fissures and/or erythema may also be present.
• • Patients will be permitted to treat their feet to assess plantar blister surface area but blistering on the feet is not a requirement for study inclusion.
• • TLA may not be infected (as assessed by PI) or have been treated with a topical antibiotic within 14 days.
• • If the patient is a woman of childbearing potential (WOCBP),
• • Has a negative urine pregnancy test.
• • Agrees to use an approved effective form of birth control with failure rates \<1% per year (e.g., implant, injectable, combined oral contraceptive, intrauterine contraceptive device, sexual abstinence, vasectomized partner) during participation in the study (and at least 3 months thereafter).
• • Is not nursing.
• • Patient's laboratory values (blood and urine) are within the range of normal or abnormal values are within normal levels for the disease and in the opinion of the PI the values are not clinically relevant for study participation.
• • Patient is in good, general health and free of any known disease state or physical condition which, in the investigator's opinion, might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation.
• • Over the duration of the study, the patient agrees to not use any other topical therapies and/or impregnated dressings within the TLAs (e.g., medicated cleansers, CBD oil, MediHoney, Silvadine cream 1%, topicals containing antimicrobials, keratin, and/or collagen, lipido-colloid or polymeric membrane dressings, and/or hydrogels).
• • Patient and/or legally appointed and authorized representative must be able and willing to follow study procedures and instructions in order to maintain compliance throughout the study period.
• • The patient or legally appointed and authorized representative must have read, understood and signed an Institutional Review Board/Ethics Committee (IRB/EC) approved Informed Consent or Assent Form.
• Exclusion Criteria:
• * Patient's use of prior or concomitant medication or medical treatments/procedures:
• • Any investigational drug or therapy within 30 days.
• • Systemic steroidal therapy within 30 days.
• • Topical steroidal therapy within 14 days (Note: inhaled and ophthalmic products containing steroids are allowed).
• • Systemic antibiotic therapy within 7 days.
• • Currently receiving chemotherapy or radiation.
• • Surgery within the previous 2 weeks (except for minor surgery, cosmetic or dental procedures as determined by the investigator).
• • Started to take chronic medications (NSAIDs, antihistamines, etc.) at least 30 days prior to starting study medication.
• * Patient's medical history includes:
• • Cancer that is currently undergoing treatment.
• • History of chronic and severe vitamin, mineral, or protein deficiency.
• • Current systemic infection.
• • HIV/AIDS.
• • Non-EBS skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, might put the patient at undue risk by study participation or interferes with the study medication application or the study assessments.
• • An illness (e.g., neurological, cardiovascular, respiratory, hepatic, renal, or metabolic disease), condition, or situation that in the opinion of the principal investigator is likely to interfere with the patient's participation in or completion of the study.
• • Factors present in the patient and/or his/her legal representative that could interfere with study compliance such as inability to attend scheduled study visits or to perform study protocol procedures.
• • Patient is a member of the investigational team or his/her immediate family.
• • Other unspecified reasons that, in the opinion of the Investigator, make the patient unsuitable for enrollment.