Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Condition: Epidermolysis Bullosa; Epidermolysis Bullosa Simplex
MeSH terms: Epidermolysis Bullosa; Epidermolysis Bullosa Simplex
Intervention: diacerein 1% ointment
Intervention description: Topically apply study medication to lesions identified by the Investigator that require.
treatment
Intervention MeSH terms: Diacerein
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Intervention model: Single Group Assignment
Intervention model description: This is an international, multicenter, open-label, long term extension study evaluating the safety of topical Diacerein 1% Ointment for the treatment of subjects with EBS. At Baseline Visit, EBS subjects who participated the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) and who meet all the inclusion/exclusion criteria will be eligible to enroll in this study.
Masking: None (Open Label)
Masking description: Each treatment cycle will consist of 8 weeks on treatment, followed by 8 weeks off treatment with a maximum of 2 treatment cycles allowed for up to 1 year. Subjects should be assessed at minimum every 8 weeks for disease activity. Once a subject completes two cycles of treatment or reaches Week 52, the subject will be discharged from the study. Subjects may not start a new treatment cycle past Week 36 from Baseline Visit without prior sponsor approval. 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.
Enrollment: 51.0
Sex: All
Minimum age: 6 Months
Maximum age: 99 Years
Healthy volunteers: No
Keywords: Epidermolysis Bullosa (EB); Epidermolysis Bullosa Simplex (EBS)

Summary:

      The primary objective of this study is to evaluate the long term safety and tolerability of
      diacerein 1% ointment for 2 treatment cycles in subjects with EBS that previously
      participated in the CCP-020-301 or the CCP-020-101 studies.
    

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.