Epidermolysis Bullosa SimplexPhase 2(TAMES)NCT07027345
A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex
A Phase 2 study testing 5% TolaSure Topical Gel in people with Epidermolysis Bullosa Simplex, run by BioMendics, LLC at 2 sites in United States.
Trial summary
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and older) diagnosed with localized, intermediate, or severe epidermolysis bullosa simplex (EBS). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex
- What is being tested?
- 5% TolaSure Topical Gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 2 sitesUnited States
- Study dates
- 11 Sep 2025to Sep 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient is a male or female at least 2 years of age.
- Patient has a documented diagnosis and confirmed clinical history of localized (previously known as Weber-Cockayne), intermediate (previously Kӧbner), or severe (previously EBS-Dowling Meara) autosomal dominant EBS and/or a genetic mutation in either the KRT14 or KRT5 gene consistent with localized, intermediate, or severe EBS. The Investigator will determine patient eligibility based on historical phenotypic presentation of EBS symptoms along with genetic/diagnosis documentation to determine EBS severity. (If localized, intermediate, or severe EBS is suspected but not diagnosed or genetically confirmed, confirmatory testing will be performed).
- For localized EBS participants:
- Patient must have at least moderate plantar disease activity, defined as ≥15% involvement of the plantar surface of at least one foot, as assessed by clinical examination or standardized imaging, with evidence of active disease (e.g., blisters, erosions, or crusting).
- For treatment, the Target Lesional Area (TLA) will consist of daily treating the feet (tops and bottoms) which is approximately 7% body surface area (BSA). Localized EBS subjects will also have the option to treat their hands (tops and bottoms), which is approximately an additional 6% BSA. Palmoplantar evaluation is separate from the intermediate to severe TLA-treated regions per protocol.
- For intermediate to severe EBS participants: 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:
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- A TLA is defined as a contiguous or non-contiguous region totaling up to ~15-20% body surface area (BSA).
- 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 and is evaluated separately per protocol.
- For all patients (with localized, intermediate, and severe EBS), the 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States2 sites, 2 recruiting
- Recruiting
NU Dermatolgy CTU
Chicago, Illinois, 60611
NU Dermatolgy CTU, 312-227-6817, NUderm-research@northwestern.edu
- Recruiting
Stanford University School of Medicine, Dermatology Department
Palo Alto, California, 94304
Thomas Buschbacher, 650-313-0354, tbusch@stanford.edu
About this 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)
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and older) diagnosed with localized, intermediate, or 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. Localized patients will be applying study medication to their feet, with the option to treat their hands as well throughout the study. Intermediate and severe EBS 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.
Read the detailed 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 localized, intermediate, or 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 patients].
Each patient (40 to complete) will be enrolled in the study and once a blistering flare is confirmed by the clinical study team, and the patient will be randomized to receive either TolaSure Gel, 5% w/w or Placebo (50:50 chance).
For Part I of the study, participants apply the randomized study treatment once daily for 2 months to a defined Target Lesional Area (TLA). The TLA and clinical presentation required for enrollment for each EBS subtype is described below.
For localized EBS subjects, the TLA will consist of daily treating the feet (tops and bottoms) which is approximately 7% body surface area (BSA). At the Baseline Visit, localized EBS subjects must have at least moderate plantar disease activity, defined as ≥15% involvement of the plantar surface of at least one foot, as assessed by clinical examination or standardized imaging, with evidence of active disease (e.g., blisters, erosions, or crusting). Localized EBS subjects will also have the option to treat their hands (tops and bottoms), which is approximately an additional 6% BSA. Palmoplantar evaluations for localized EBS subjects are separate from the intermediate to severe EBS subjects' TLA-treated regions per protocol.
For intermediate to severe EBS subject, the TLA can be a contiguous or non-contiguous region totaling up to approximately 15-20% BSA, excluding palmoplantar surfaces, groin, and apocrine areas. At the subject Baseline Visit, intermediate to severe EBS subject's TLA must consist of a 2-3% BSA active flaring sub-region consisting mainly of intact or freshly ruptured blisters. Subjects have the option to treat their feet, but plantar evaluation for intermediate to severe EBS subjects is separate from the TLA per protocol.
Study drug (TolaSure Gel, 5% w/w or Placebo Gel) is applied to the entire TLA, not just the symptomatic regions (e.g., blistering, keratoderma, erosions, crusting). If symptoms resolve, treatment continues daily on all assigned TLAs.
For Part 2 of the study, a single arm crossover will then allow those subjects that received Placebo to go on TolaSure Gel, 5% w/w for a subsequent 2 months. The subjects already in the TolaSure Gel, 5% w/w cohort will continue with daily IP application for an additional 2 months as well.
At End of Study (EOS), subjects will cease IP application and EOS procedures will be completed. A subject follow-up visit will occur after 6 months.
The primary endpoint will assess changes in disease severity within the TLAs (excluding palmoplantar surfaces) and recurrence of blistering using clinical imaging of blister surface area over time. The secondary efficacy endpoints will evaluate blistering on the feet, subject self-assessments including pain, itch, modified Foot Function Index (mFFI), and Quality of Life (QoL) assessment. An exploratory endpoint will examine blistering and keratoderma of localized EBS subjects that treat their hands.
Safety endpoints will include the incidence of treatment-emergent adverse events (TEAEs). In order to provide adequate assurance of subject safety, safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate). These safety assessments in addition to AE reviews will be sufficient to identify potential TEAEs.
The information obtained from these assessments will be utilized to examine the efficacy of TolaSure treatment in ameliorating EBS symptoms as well as the accuracy of the proposed mechanism of action (MOA) of TolaSure for this disease indication.
- Conditions
- Epidermolysis Bullosa Simplex
- Being tested
- 5% TolaSure Topical Gel, Topical Placebo Gel
- Keywords
- 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
- Medical subject headings
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Skin Diseases, Skin Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
5% TolaSure Topical GelDrug
TolaSure Topical Gel is applied once-daily to designated treatment areas for up to 4 months (Part 2 End of Study).
Topical Placebo GelDrug
Topical Placebo Gel is applied once-daily to designated treatment areas for up to 2 months (Part 1 End of Study).
| Study group | Role | What they receive |
|---|---|---|
| 5% TolaSure Topical Gel | Active comparator | 5% (w/w) TolaSure Gel Receives: Drug: 5% TolaSure Topical Gel |
| Topical Placebo Gel | Placebo comparator | Placebo Control Gel Receives: Drug: Topical Placebo Gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Blister Surface Area of the Designated Treatment Area(s)
Clinical photography will be used to assess changes in disease severity within the target lesional area (TLA) and recurrence of blistering by measuring blister surface area in the designated TLA over time.
Measured: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Secondary outcomes (6)
Plantar Blister Surface Area Reduction
Plantar blister surface area changes over time will be examined using clinical photography for localized EBS patients that treat their feet and those intermediate to severe EBS subjects that opt-in to treat their feet.
Measured: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Pain Score in Target Lesional Areas During Treatment Application
Using the adult or pediatric PROMIS® Measure form, peak pain during treatment application will be reported on a weekly basis until Part 2 End of Study (EOS, 4-months), with a possible assessment at the 6-month follow up visit. Pain trends in TolaSure Gel, 5% w/w and Placebo groups will be compared. The following PROMIS® Measure forms will be used depending on the patient's age: The Adult Pain Interference-Short Form 8a, Pediatric Pain Interference-Short Form 8a, and Parent Proxy Pain Interference-Short Form 8a. These scales are from 1 to 5 and relate to how much pain interferes with daily activities, with 1 indicating Never/Not at All and 5 indicating Almost Always/Very Much. For 2-4 year-old subjects, worst pain Numeric Rating Scale (NRS) questionnaire will be reported by caregivers/guardians. Pain NRS is from 0 to 10 with 0=no pain and 10=worst pain imaginable.
Measured: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Itch Score in Target Lesional Area
Using the adult or pediatric PROMIS® Measure form, itch characteristics will be reported on a weekly basis until Part 2 End of Study (EOS, 4-months), with a possible assessment at the 6-month follow up visit. Itch trends in TolaSure Gel, 5% w/w and Placebo groups will be compared. The following PROMIS® Measure forms will be used depending on the patient's age: The Adult Itch Interference-Short Form 8a, Pediatric Itch-Short Form 8a (PIQ-C), and Parent Proxy Itch-Short Form 8a (PIQ-C). These scales are from 1 to 5 and relate to how much itch interferes with daily activities and the level of itch, with 1 indicating Never/No Itch and 5 indicating Almost Always/Very Severe. For 2-4 year-old subjects, worst scratch/itch Numeric Rating Scale (WSI-NRS) questionnaire will be reported by caregivers/guardians. WSI-NRS is from 0 to 10 with 0=no itching or scratching and 10=worst imaginable itching or scratching.
Measured: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Quality of Life (QoL)
Patient's quality of life (QoL) metrics, highlighting symptoms and daily living concerns of EBS patients, will be reported at Visit 2/ Baseline, Part 1 End of Study (EOS), and Part 2 EOS, with a possible assessment at the 6-month follow up visit. This QoL assessment will be given to patients in a questionnaire format. The questions focus on how EBS blistering areas affect daily physical and social activities as well as mental and emotional health. The scale range is from 0 to 10, with 0 being not at all affected and 10 being severely affected. QoL metrics in TolaSure Gel, 5% w/w and Placebo control groups will be examined.
Measured: Day 1 (Baseline), Part 1 End of Study (2-months), Part 2 End of Study (4-months)
Subject-reported Modified Foot Function Index (mFFI)
Patient's modified Foot Function Index (mFFI), is a questionnaire about foot pathology as it relates to pain, disability, and/or activity restrictions. Questions relating to pain are on a 0 to 10 scale with 0 being no pain at all and 10 being the worst pain imaginable. Daily activity scores also range from 0 to 10 with 0 being Not at All/Not Difficult at All and 10 being All the Time/So Difficult. The mFFI questionnaire will be reported at Visit 2/ Baseline, Part 1 End of Study (EOS), and Part 2 EOS, with a possible assessment at the 6-month follow up visit. mFFI metrics in TolaSure Gel, 5% w/w and Placebo control groups will be examined.
Measured: Day 1 (Baseline), Part 1 End of Study (2-months), Part 2 End of Study (4-months)
Incidence of treatment-emergent adverse events (TEAEs)
Safety monitoring will include clinical laboratory testing (blood and urine) and urine pregnancy testing (as appropriate) only at Visit 1/Screening and Part 2 EOS, physical examinations (including assessment of treatment areas), and vital sign measurements at baseline and bi-weekly until Part 1 EOS and Part 2 EOS. These assessments along with AE reviews will identify potential TEAEs.
Measured: Day 1 (Baseline) and bi-weekly until End of Study (maximum 4-months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jun 2025
- Study start11 Sep 2025
- Primary completionJan 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Epidermolysis Bullosa SimplexStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Model description
- Part 1 of the study (2-months total) will involve a randomized, double-blinded treatment assignment model where one arm will receive active, and the other arm will receive placebo. Part 2 of the study (2-months total) will involve a single arm crossover where the placebo arm will crossover and receive active. The active arm will continue treating with active. During this part of the study the treatment labels will still be blinded.
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMendics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Northwestern University, Ann & Robert H Lurie Children's Hospital of Chicago, Stanford University, Lucile Packard Children's Hospital
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07027345
- Sponsor's protocol ID
- TAMES-02
- Acronym
- (TAMES)
- Status last verified
- Aug 2026
- First submitted
- 9 Jun 2025
- Last updated
- 13 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT07027345
- Acronym / sponsor ID
- (TAMES) / TAMES-02
- Conditions
- Epidermolysis Bullosa Simplex
- Keywords
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, EBS, Severe EBS, EBS Dowling-Meara, EBS Koebner, Skin Diseases, Keratin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- BioMendics, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Northwestern University, Ann & Robert H Lurie Children's Hospital of Chicago, Stanford University, Lucile Packard Children's Hospital
- Study start
- 11 Sep 2025 (actual)
- Primary completion
- Jan 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 18 Jun 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase II clinical study will assess the efficacy, safety and tolerability of topical TolaSure Gel in adults and pediatric patients (2 years of age and older) diagnosed with localized, intermediate, or 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. Localized patients will be applying study medication to their feet, with the option to treat their hands as well throughout the study. Intermediate and severe EBS 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.
Detailed 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 localized, intermediate, or 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 patients].
Each patient (40 to complete) will be enrolled in the study and once a blistering flare is confirmed by the clinical study team, and the patient will be randomized to receive either TolaSure Gel, 5% w/w or Placebo (50:50 chance).
For Part I of the study, participants apply the randomized study treatment once daily for 2 months to a defined Target Lesional Area (TLA). The TLA and clinical presentation required for enrollment for each EBS subtype is described below.
For localized EBS subjects, the TLA will consist of daily treating the feet (tops and bottoms) which is approximately 7% body surface area (BSA). At the Baseline Visit, localized EBS subjects must have at least moderate plantar disease activity, defined as ≥15% involvement of the plantar surface of at least one foot, as assessed by clinical examination or standardized imaging, with evidence of active disease (e.g., blisters, erosions, or crusting). Localized EBS subjects will also have the option to treat their hands (tops and bottoms), which is approximately an additional 6% BSA. Palmoplantar evaluations for localized EBS subjects are separate from the intermediate to severe EBS subjects' TLA-treated regions per protocol.
For intermediate to severe EBS subject, the TLA can be a contiguous or non-contiguous region totaling up to approximately 15-20% BSA, excluding palmoplantar surfaces, groin, and apocrine areas. At the subject Baseline Visit, intermediate to severe EBS subject's TLA must consist of a 2-3% BSA active flaring sub-region consisting mainly of intact or freshly ruptured blisters. Subjects have the option to treat their feet, but plantar evaluation for intermediate to severe EBS subjects is separate from the TLA per protocol.
Study drug (TolaSure Gel, 5% w/w or Placebo Gel) is applied to the entire TLA, not just the symptomatic regions (e.g., blistering, keratoderma, erosions, crusting). If symptoms resolve, treatment continues daily on all assigned TLAs.
For Part 2 of the study, a single arm crossover will then allow those subjects that received Placebo to go on TolaSure Gel, 5% w/w for a subsequent 2 months. The subjects already in the TolaSure Gel, 5% w/w cohort will continue with daily IP application for an additional 2 months as well.
At End of Study (EOS), subjects will cease IP application and EOS procedures will be completed. A subject follow-up visit will occur after 6 months.
The primary endpoint will assess changes in disease severity within the TLAs (excluding palmoplantar surfaces) and recurrence of blistering using clinical imaging of blister surface area over time. The secondary efficacy endpoints will evaluate blistering on the feet, subject self-assessments including pain, itch, modified Foot Function Index (mFFI), and Quality of Life (QoL) assessment. An exploratory endpoint will examine blistering and keratoderma of localized EBS subjects that treat their hands.
Safety endpoints will include the incidence of treatment-emergent adverse events (TEAEs). In order to provide adequate assurance of subject safety, safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate). These safety assessments in addition to AE reviews will be sufficient to identify potential TEAEs.
The information obtained from these assessments will be utilized to examine the efficacy of TolaSure treatment in ameliorating EBS symptoms as well as the accuracy of the proposed mechanism of action (MOA) of TolaSure for this disease indication.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- Part 1 of the study (2-months total) will involve a randomized, double-blinded treatment assignment model where one arm will receive active, and the other arm will receive placebo. Part 2 of the study (2-months total) will involve a single arm crossover where the placebo arm will crossover and receive active. The active arm will continue treating with active. During this part of the study the treatment labels will still be blinded.
| Arm | Type | Receives |
|---|---|---|
| 5% TolaSure Topical Gel | Active comparator | Drug: 5% TolaSure Topical Gel5% (w/w) TolaSure Gel |
| Topical Placebo Gel | Placebo comparator | Drug: Topical Placebo GelPlacebo Control Gel |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (19 listed)
- Patient is a male or female at least 2 years of age.
- Patient has a documented diagnosis and confirmed clinical history of localized (previously known as Weber-Cockayne), intermediate (previously Kӧbner), or severe (previously EBS-Dowling Meara) autosomal dominant EBS and/or a genetic mutation in either the KRT14 or KRT5 gene consistent with localized, intermediate, or severe EBS. The Investigator will determine patient eligibility based on historical phenotypic presentation of EBS symptoms along with genetic/diagnosis documentation to determine EBS severity. (If localized, intermediate, or severe EBS is suspected but not diagnosed or genetically confirmed, confirmatory testing will be performed).
- For localized EBS participants:
- For intermediate to severe EBS participants: 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:
- For all patients (with localized, intermediate, and severe EBS), the TLA may not be infected (as assessed by PI) or have been treated with a topical antibiotic within 14 days.
Key exclusion criteria (18 listed)
- Patient's use of prior or concomitant medication or medical treatments/procedures:
- Patient's medical history includes:
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 5% TolaSure Topical GelTolaSure Topical Gel is applied once-daily to designated treatment areas for up to 4 months (Part 2 End of Study). | Drug | 5% TolaSure Topical Gel |
| Topical Placebo GelTopical Placebo Gel is applied once-daily to designated treatment areas for up to 2 months (Part 1 End of Study). | Drug | Topical Placebo Gel |
Primary outcome
Change in Blister Surface Area of the Designated Treatment Area(s)
Time frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Clinical photography will be used to assess changes in disease severity within the target lesional area (TLA) and recurrence of blistering by measuring blister surface area in the designated TLA over time.
6 secondary outcomes
Plantar Blister Surface Area Reduction
Time frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Pain Score in Target Lesional Areas During Treatment Application
Time frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Itch Score in Target Lesional Area
Time frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)
Subject-reported Quality of Life (QoL)
Time frame: Day 1 (Baseline), Part 1 End of Study (2-months), Part 2 End of Study (4-months)
Subject-reported Modified Foot Function Index (mFFI)
Time frame: Day 1 (Baseline), Part 1 End of Study (2-months), Part 2 End of Study (4-months)
Incidence of treatment-emergent adverse events (TEAEs)
Time frame: Day 1 (Baseline) and bi-weekly until End of Study (maximum 4-months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2026.
- BioMendics, LLCLink provided by the sponsor.
- Stanford Medicine, Dermatology, Current EB StudiesLink provided by the sponsor.
- Northwestern Medicine, Department of Dermatology Clinical TrialsLink provided by the sponsor.
- Individual participant dataSharing planned: No
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RecruitingPhase 2
A Study to Evaluate ENERGI-F703EB Cream in Epidermolysis Bullosa
Epidermolysis Bullosa (EB), Junctional Epidermolysis Bullosa
Not yet recruitingPhase 2
Filsuvez in Moderate-to-Severe Epidermolysis Bullosa Simplex
Epidermolysis Bullosa Simplex
Active, not recruitingPhase 2/3
An International, Multicenter, Randomized, Double-Blind, Parallel Group, Vehicle-Controlled, Phase 2/3 Study With Open-Label Extension Evaluating the Efficacy and Safety of Diacerein 1% Ointment for the Treatment of Generalized Epidermolysis Bullosa Simplex (EBS)
Generalized Epidermolysis Bullosa Simplex
RecruitingPhase 2
A 20-Week Study Assessing the Efficacy of Apremilast in Patients with EB Simplex Generalized
Epidermolysis Bullosa Simplex, Genodermatosis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Stanford University School of Medicine, Dermatology Department
Palo Alto, United States
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing 5% TolaSure Topical Gel in people with Epidermolysis Bullosa Simplex, run by BioMendics, LLC at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 19 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Blister Surface Area of the Designated Treatment Area(s) (time frame: Day 1 (Baseline) and weekly until End of Study (2-months and maximum 4-months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Aug 2026; AzTrials last refreshed its copy on 26 Oct 2026.