Epidermolysis Bullosa SimplexPhase 1TAMESNCT05062070
Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex
A Phase 1 study testing TolaSure Topical Gel in people with Epidermolysis Bullosa Simplex, run by BioMendics, LLC at 1 site in United States.
Trial summary
TolaSure is a topical gel for the promotion of accelerated wound healing. 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 epidermolysis bullosa simplex (i.e., EBS-Dowling Meara). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Epidermolysis Bullosa Simplex
- What is being tested?
- TolaSure Topical Gel
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- 5 May 2022to 22 Jun 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient is a male or female at least 18 years of age.
- Patient has a documented genetic mutation in either the keratin 14 (K14) or keratin 5 (K5) gene and that their EBS diagnosis and/or specific mutation are consistent with severe EBS (previously EBS-DM). (If severe EBS is suspected but not genetically confirmed, confirmatory testing will be performed).
- Patient possesses the four required skin areas:
- Treatment Areas:
- Must have two (2) Treatment Areas defined as an IGA score ≥ 3. Treatment Areas may have defined erosions, erythema, and ulcerations.
- Must be comparable in size with an area of approximately 300-400 cm^2 per area. Individually, a treatment area may be continuous or non-contiguous and must contain newly-formed blisters/erosions. Ideally, the two Treatment Areas on the patient should not be adjacent to each other and should be located bilaterally and roughly symmetrically on the trunk, abdomen, or upper or lower extremities.
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- May not be infected (as assessed by the Principal Investigator) or have been treated with a topical antibiotic within 14 days.
- May not be located on palms, soles, scalp, groin, and any other areas that the Principal Investigator believes there will be difficulty in applying treatments and bandaging or assessing outcome measures.
- Suction Blister Areas (SBAs):
- Must have two (2) areas of non-wounded and visually uninvolved skin, defined as an IGA score of zero (0).
- Must be comparable in size (approximately 50 cm^2)
- 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 Principal Investigator 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 wounds or which exposes the patient 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 Treatment Areas or Suction Blister Areas (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 must be able and willing to follow study procedures and instructions in order to maintain compliance throughout the study period.
- Patient has been informed, has read and understood the subject informed consent form, and has given written informed consent.
Who cannot join
- Patient's use of prior or concomitant medication or medical treatments/procedures:
- Any investigational drug 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.
- Systemic diuretics or cardiac glycosides within 30 days.
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- Currently receiving chemotherapy or radiation.
- Patient has undergone stem cell transplant or gene therapy for the treatment of inherited EB.
- Surgery within the previous 3 months (except for minor cosmetic or dental procedures).
- Patient's medical history includes:
- Cancer that is currently undergoing treatment.
- History of 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 subject 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 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.
- History of drug or alcohol abuse (as defined by the Investigator).
- Patient's use of smoking/vaping tobacco products.
- Other unspecified reasons that, in the opinion of the Investigator, make the subject unsuitable for enrollment.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine, Dermatology Department
Redwood City, California, 94063
About this study
Official title: A Phase I/II, Closed Label, Randomized, Pilot Study for the Safety and Efficacy of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex (TAMES)
TolaSure is a topical gel for the promotion of accelerated wound healing. 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 epidermolysis bullosa simplex (i.e., EBS-Dowling Meara). A total of 10, severe EBS patients, males and females ages 18 years and older, will be enrolled. Patients will apply TolaSure and Vehicle Gel once-daily for a maximum of 10 weeks.
Read the detailed description
This is a closed-label, randomized, double-blind, Phase I clinical study to assess the safety and tolerability of the investigational product (IP), TolaSure Gel, 5% w/w, in patients diagnosed with severe epidermolysis bullosa simplex (EBS, previously known as EBS-Dowling Meara). Each patient (5 to complete) will apply TolaSure Gel, 5% w/w and TolaSure Vehicle Gel once-daily to defined Treatment Areas and Suction Blister Areas. End of Study (EOS) will occur at the Principal Investigator's (PI's) discretion according to the resolution or lack of improvement of the Treatment Areas. The maximum time a patient could be in the study is 10 weeks. The average time frame for each patient to complete the study is expected to be 6-8 weeks.
The primary safety endpoint will be the incidence of treatment-emergent adverse events (TEAEs). Safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate).
Secondary efficacy endpoints will (1) assess wound severity within the Treatment Areas using an Investigator Global Assessment (IGA) scoring system, (2) examine under a microscope keratinocyte morphology and the incidence of keratin aggregates in Treatment Area skin biopsies, (3) determine the time required to induce a suction blister within the designated Suction Blister Areas, and (4) evaluate patient self-assessment of overall impression, pain, and itch within the Treatment Areas. The information obtained from these assessments will be used to examine the efficacy of TolaSure treatment in improving EBS symptoms.
- Conditions
- Epidermolysis Bullosa Simplex
- Being tested
- TolaSure Topical Gel, Vehicle Topical Gel
- Keywords
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, EBS, Severe EBS, EBS Dowling-Meara, Skin Diseases, Suction Blister, Skin Abnormalities, Keratin, Aggregate, Mutant, EBS-DM
- 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.
TolaSure Topical GelDrug
Also called Active Ingredient BM-3103
TolaSure Topical Gel is applied once-daily for up to 10 weeks to designated Treatment Area and Suction Blister Area.
Vehicle Topical GelDrug
Also called TolaSure Vehicle Gel
TolaSure Vehicle Gel is applied once-daily for up to 10 weeks to designated Treatment Area and Suction Blister Area.
| Study group | Role | What they receive |
|---|---|---|
| 5 Percent TolaSure Topical Gel | Active comparator | 5% (w/w) TolaSure Gel Receives: Drug: TolaSure Topical Gel |
| Topical Vehicle Gel | Placebo comparator | TolaSure Vehicle Gel Receives: Drug: Vehicle Topical Gel |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Safety monitoring will include clinical chemistry tests, physical examinations (including assessment of Treatment and Suction Blister Areas), and vital sign measurements. These assessments along with AE reviews will identify potential TEAEs.
Measured: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)
Secondary outcomes (6)
Investigator Global Assessment (IGA)
The Principal Investigator will use the IGA scale to score the average overall severity of the EBS wounds within the Treatment Areas, considered together. Changes from baseline in IGA scores for the TolaSure-treated and Vehicle-treated Treatment Areas will be analyzed.
Measured: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)
Keratin Aggregate Counts and Cell Morphology Assessment
Biopsies from perilesional skin in each of the Treatment Areas are examined to determine a change from baseline in the number of keratin aggregate positive basal and suprabasal keratinocytes using transmission electron microscopy (TEM) and/or light microscopy.
Measured: Day 1, During Study (optional, estimated Week 3-Week 6), End of Study (maximum 10 weeks)
Suction Blister Time
A suction blister is raised within the Suction Blister Areas and the time to blister formation is recorded in seconds. Changes from baseline suction blister times in the TolaSure-treated and Vehicle-treated Suction Blister Areas will be analyzed.
Measured: Day 1 and End of Study (maximum 10 weeks)
Patient's-reported Overall Impression Score of Treatment Areas
Change from baseline in the patient-reported overall impression score of both the TolaSure-treated and Vehicle-treated Treatment Areas will be calculated.
Measured: Day 1, then Weekly until End of Study (maximum 10 weeks)
Patient's-reported Pain Score of Treatment Areas
Change from baseline in the patient-reported pain numerical rating scale (NRS) of both the TolaSure-treated and Vehicle-treated Treatment Areas will be calculated.
Measured: Day 1, then Weekly until End of Study (maximum 10 weeks)
Patient's-reported Itch Score of Treatment Areas
Change from baseline in the patient-reported itch numerical rating scale (NRS) of both the TolaSure-treated and Vehicle-treated Treatment Areas will be calculated.
Measured: Day 1, then Weekly until End of Study (maximum 10 weeks)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2021
- Study start5 May 2022
- Primary completion22 Jun 2023
- Study completion22 Jun 2023
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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMendics, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05062070
- Sponsor's protocol ID
- TAMES-01
- Acronym
- TAMES
- Status last verified
- Sep 2023
- First submitted
- 21 Sep 2021
- Last updated
- 11 Sep 2023
- 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 11 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Closed Label, Randomized, Pilot Study for the Safety and Efficacy of TolaSure Gel, 5% w/w Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex (TAMES)
- ClinicalTrials.gov ID
- NCT05062070
- Acronym / sponsor ID
- TAMES / TAMES-01
- Conditions
- Epidermolysis Bullosa Simplex
- Keywords
- Epidermolysis Bullosa, Epidermolysis Bullosa Simplex, EBS, Severe EBS, EBS Dowling-Meara, Skin Diseases, Suction Blister, Skin Abnormalities
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- BioMendics, LLCIndustry (pharma, biotech, devices)
- Study start
- 5 May 2022 (actual)
- Primary completion
- 22 Jun 2023 (actual)
- Study completion
- 22 Jun 2023 (actual)
- First posted
- 30 Sep 2021 (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.
TolaSure is a topical gel for the promotion of accelerated wound healing. 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 epidermolysis bullosa simplex (i.e., EBS-Dowling Meara). A total of 10, severe EBS patients, males and females ages 18 years and older, will be enrolled. Patients will apply TolaSure and Vehicle Gel once-daily for a maximum of 10 weeks.
Detailed description
This is a closed-label, randomized, double-blind, Phase I clinical study to assess the safety and tolerability of the investigational product (IP), TolaSure Gel, 5% w/w, in patients diagnosed with severe epidermolysis bullosa simplex (EBS, previously known as EBS-Dowling Meara). Each patient (5 to complete) will apply TolaSure Gel, 5% w/w and TolaSure Vehicle Gel once-daily to defined Treatment Areas and Suction Blister Areas. End of Study (EOS) will occur at the Principal Investigator's (PI's) discretion according to the resolution or lack of improvement of the Treatment Areas. The maximum time a patient could be in the study is 10 weeks. The average time frame for each patient to complete the study is expected to be 6-8 weeks.
The primary safety endpoint will be the incidence of treatment-emergent adverse events (TEAEs). Safety monitoring will include physical exams, vital sign measurements, clinical laboratory testing (blood and urinalysis), and urine pregnancy testing (as appropriate).
Secondary efficacy endpoints will (1) assess wound severity within the Treatment Areas using an Investigator Global Assessment (IGA) scoring system, (2) examine under a microscope keratinocyte morphology and the incidence of keratin aggregates in Treatment Area skin biopsies, (3) determine the time required to induce a suction blister within the designated Suction Blister Areas, and (4) evaluate patient self-assessment of overall impression, pain, and itch within the Treatment Areas. The information obtained from these assessments will be used to examine the efficacy of TolaSure treatment in improving EBS symptoms.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 5 Percent TolaSure Topical Gel | Active comparator | Drug: TolaSure Topical Gel5% (w/w) TolaSure Gel |
| Topical Vehicle Gel | Placebo comparator | Drug: Vehicle Topical GelTolaSure Vehicle Gel |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (20 listed)
- Patient is a male or female at least 18 years of age.
- Patient has a documented genetic mutation in either the keratin 14 (K14) or keratin 5 (K5) gene and that their EBS diagnosis and/or specific mutation are consistent with severe EBS (previously EBS-DM). (If severe EBS is suspected but not genetically confirmed, confirmatory testing will be performed).
- Patient possesses the four required skin areas:
- If the patient is a woman of childbearing potential (WOCBP),
- 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 Principal Investigator the values are not clinically relevant for study participation.
Key exclusion criteria (21 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.
- History of drug or alcohol abuse (as defined by the Investigator).
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 |
|---|---|---|
| TolaSure Topical GelAlso: Active Ingredient BM-3103TolaSure Topical Gel is applied once-daily for up to 10 weeks to designated Treatment Area and Suction Blister Area. | Drug | 5 Percent TolaSure Topical Gel |
| Vehicle Topical GelAlso: TolaSure Vehicle GelTolaSure Vehicle Gel is applied once-daily for up to 10 weeks to designated Treatment Area and Suction Blister Area. | Drug | Topical Vehicle Gel |
Primary outcome
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Time frame: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)
Safety monitoring will include clinical chemistry tests, physical examinations (including assessment of Treatment and Suction Blister Areas), and vital sign measurements. These assessments along with AE reviews will identify potential TEAEs.
6 secondary outcomes
Investigator Global Assessment (IGA)
Time frame: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)
Keratin Aggregate Counts and Cell Morphology Assessment
Time frame: Day 1, During Study (optional, estimated Week 3-Week 6), End of Study (maximum 10 weeks)
Suction Blister Time
Time frame: Day 1 and End of Study (maximum 10 weeks)
Patient's-reported Overall Impression Score of Treatment Areas
Time frame: Day 1, then Weekly until End of Study (maximum 10 weeks)
Patient's-reported Pain Score of Treatment Areas
Time frame: Day 1, then Weekly until End of Study (maximum 10 weeks)
Patient's-reported Itch Score of Treatment Areas
Time frame: Day 1, then Weekly until End of Study (maximum 10 weeks)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Sep 2023.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing TolaSure Topical Gel in people with Epidermolysis Bullosa Simplex, run by BioMendics, LLC at 1 site 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 20 inclusion and 21 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: finished, as of the sponsor's last update on 11 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment-Emergent Adverse Events (TEAEs) (time frame: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)). 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 11 Sep 2023.