Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolys… (NCT05062070) | Clinical Trial Compass
CompletedPhase 1
Safety and Efficacy of Topical TolaSure Targeting Aggregated Mutant Keratin in Severe Epidermolysis Bullosa Simplex
United States6 participantsStarted 2022-05-05
Plain-language 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). 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.
Who can participate
Age range18 Years
SexALL
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Inclusion Criteria:
* 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.
* 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 c…
What they're measuring
1
Incidence of Treatment-Emergent Adverse Events (TEAEs)
Timeframe: Day 1, Day 2, then Weekly until End of Study (maximum 10 weeks)