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New treatment option for Epidermolysis Bullosa Simplex
Official title A Phase II, Placebo Controlled, Clinical Trial of Topical TolaSure Targeting Aggregated Mutant Keratin in Epidermolysis Bullosa Simplex
ClinicalTrials.gov ID: NCT07027345
What this study is testing
What is 5% TolaSure Topical Gel?
5% TolaSure Topical Gel is an investigational medicine, given as an once-daily treatment applied to the skin, being studied as a potential treatment for epidermolysis bullosa simplex.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance. You may not know which one you got.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 and older
You may be able to join if
- 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...
- 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...
- 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...
- 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...
You likely can't join if
- 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.
See the full eligibility 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
- Chicago, Illinois, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States; Chicago, Illinois, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.