RECRUITING

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

Conditions

Epidermolysis Bullosa Simplex Epidermolysis Bullosa EBS Severe EBS EBS Dowling-Meara EBS Koebner Skin Diseases Keratin Aggregate Mutant EBS-DM Keratin Disorder Skin Abnormalities generalized intermediate

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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)

Quick Facts

Study Start:2025-09

Study Completion:2026-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT07027345

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:4 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion 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.	* 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.

Inclusion Criteria

Exclusion 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.

* 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.

Contacts and Locations

Study Contact

Karen McGuire, PhD

CONTACT

330-651-2140

kmcguire@biomendics.com

Study Locations (Sites)

Stanford University School of Medicine, Dermatology Department

Palo Alto, California, 94304

United States

NU Dermatolgy CTU

Chicago, Illinois, 60611

United States

Collaborators and Investigators

Sponsor: BioMendics, LLC

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-09

Study Completion Date2026-09

Study Record Updates

Study Start Date2025-09

Study Completion Date2026-09

Terms related to this study

Keywords Provided by Researchers

Epidermolysis Bullosa
Epidermolysis Bullosa Simplex
EBS
Severe EBS
EBS Dowling-Meara
EBS Koebner
Skin Diseases
Keratin
Aggregate
Mutant
EBS-DM
Keratin Disorder
Skin Abnormalities
generalized
intermediate

Additional Relevant MeSH Terms

Epidermolysis Bullosa Simplex