Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS) (NCT03154333) | Clinical Trial Compass
TerminatedPhase 2
Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)
Stopped: Futility
United States54 participantsStarted 2017-06-01
Plain-language summary
Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.
Who can participate
Age range4 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Key Inclusion Criteria:
* Subject is at least 4 years of age at Screening
* Subject has a documented genetic mutation consistent with EBS. Gene mutations acceptable for inclusion are as follows: KRT5, KRT14, PLEC1, TGM5, PKP1, DSP, FERMT1, EXPH5, DST, KLHL24.
* Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
* Localized: plantar and/or palmar areas
* Generalized: arms, legs, torso, hands and feet
* Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3
* Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study
* Subject is non-pregnant as confirmed by a negative urine pregnancy screen, non-lactating and is not planning for pregnancy during the study period
* If the subject is a woman of childbearing potential, agrees to use an approved effective method of birth control
* Subject is in good general health and free of any known disease state or physical condition which might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation
Key Exclusion Criteria:
* Subject has EBS lesions to be treated that are infected
* Subject has used any diacerein containing product within 6 months prior to Screening
* Subject has used systemic immunotherapy…
What they're measuring
1
Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area