Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Epidermolysis Bullosa, Epidermolysis Bullosa Simplex
About this trial
This is an interventional treatment trial for Epidermolysis Bullosa focused on measuring Epidermolysis Bullosa (EB), Epidermolysis Bullosa Simplex (EBS)
Eligibility Criteria
Key Inclusion Criteria:
- The subject is capable of understanding and complying with protocol requirements.
- The subject or the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
- Subject has a documented genetic mutation consistent with EBS.
- Subject has completed study CCP-020-301 or participated in study CCP-020-101.
- Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions
- If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, for the duration of the study.
- Subject is non-lactating and is not planning for pregnancy during the study period.
- Subject is willing and able to follow all study instructions and to attend all study visits.
Key Exclusion Criteria:
- Subject has EBS lesions to be treated that are infected
- Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline Visit.
- The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication.
- Subject has experienced a change in clinical status from the feeder study that puts the subject at undue risk to participate.
Sites / Locations
- Ann and Robert H. Lurie Children's Hospital of Chicago
- University of North Carolina (UNC) - Chapel Hill
- Children's Hospital of San Antonio
Arms of the Study
Arm 1
Experimental
Treatment period
Subjects with active lesions as determined Investigator's clinical assessment, will initiate a cycle of applying diacerein 1% ointment once-daily, study medication, at home to their EBS lesions for 8 weeks. Following the Treatment Period, subjects will go Off Treatment for 8 weeks using only investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens. As determined Investigator's clinical assessment, subjects may enter into another Treatment Period of 8 weeks. The duration of a subject's participation in the extension study may be as short as 32 weeks or as long as 52 weeks depending on the cycle initiation schedule for each individual subject.