Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
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|ClinicalTrials.gov Identifier: NCT03389308|
Recruitment Status : Enrolling by invitation
First Posted : January 3, 2018
Last Update Posted : February 7, 2018
|Condition or disease||Intervention/treatment||Phase|
|Epidermolysis Bullosa Simplex||Drug: Diacerein 1% Ointment||Phase 2|
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 Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or the CCP-020-101 PK study (feeder studies) without major Clinical Safety Report (CSR) reportable protocol deviations and who meet all the inclusion/exclusion criteria will be eligible to complete 2 treatment cycles of CCP-020.
Each treatment cycle will consist of 8 weeks on treatment (once-daily, at home study medication applications) followed by 8 weeks off treatment (only Investigator approved bland, non-medicated emollient/moisturizer, routine cleansing products and sunscreens) with a maximum of 2 treatment cycles allowed for up to 52 weeks.
At Baseline Visit (corresponding to the final study visit of the feeder study), the investigator will perform a clinical assessment and determine if any of the subject's lesions require treatment (up to 30% BSA). If the subject has active lesions as determined by the Investigator's clinical assessment, the subject will initiate a cycle of once-daily, at home study medication application to their EBS lesions for 8 weeks (treatment period) followed by an 8-week period where no treatment will be administered. Subjects presenting with no active lesions (as determined by the Investigator's clinical assessment) will not begin treatment and instead, will be instructed to return to the clinic for re-evaluation in 8 weeks or upon worsening of EBS lesions, whichever happens first.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||80 participants|
|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 completed the CCP-020-301 double-blind safety and efficacy study or the CCP-020-101 PK study (feeder studies) without major Clinical Safety Report (CSR) reportable protocol deviations 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.|
|Official Title:||An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex (EBS)|
|Actual Study Start Date :||December 1, 2017|
|Estimated Primary Completion Date :||November 15, 2019|
|Estimated Study Completion Date :||December 15, 2019|
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.
Drug: Diacerein 1% Ointment
Topically apply study medication to lesions identified by the Investigator that require.
Other Name: Diacerein
- Evaluate Safety and Tolerability of Diacerein 1% Ointment in Terms of the Occurrence of Adverse Events in Subjects with EBS [ Time Frame: From Baseline Visit to 16 weeks, not to exceed 52 weeks cyling between treatment and off treatment periods ]The primary objective of this study is to evaluate the long term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101. Safety will be evaluated in terms of the occurrence of Adverse Events. An adverse event is defined as any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and/or unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational medicinal product, whether or not related to the investigational medicinal product. All adverse events, including observed or volunteered problems, complaints, or symptoms, are to be recorded on the appropriate Case Report Form.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03389308
|United States, Illinois|
|Ann and Robert H. Lurie Children's Hospital of Chicago|
|Chicago, Illinois, United States, 60611|
|United States, North Carolina|
|University of North Carolina (UNC) - Chapel Hill|
|Chapel Hill, North Carolina, United States, 27516|
|United States, Texas|
|Children's Hospital of San Antonio|
|San Antonio, Texas, United States, 78218|
|Study Director:||Amir Tavakkol, MD||Castle Creek Pharmaceuticals|