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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 : June 26, 2018
Sponsor:
Information provided by (Responsible Party):
Castle Creek Pharmaceuticals, LLC

Tracking Information
First Submitted Date  ICMJE December 19, 2017
First Posted Date  ICMJE January 3, 2018
Last Update Posted Date June 26, 2018
Actual Study Start Date  ICMJE December 1, 2017
Estimated Primary Completion Date November 15, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 2, 2018)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03389308 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex
Official Title  ICMJE 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)
Brief 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 were previously enrolled in studies CCP-020-301 or CCP-020-101.
Detailed 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 Visit 1, EBS subjects who completed the CCP-020-301 double-blind safety and efficacy study or participated in the CCP-020-101 PK study (feeder studies) 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  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE 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 has completed 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.
Primary Purpose: Treatment
Condition  ICMJE Epidermolysis Bullosa Simplex
Intervention  ICMJE Drug: Diacerein 1% Ointment

Topically apply study medication to lesions identified by the Investigator that require.

treatment

Other Name: Diacerein
Study Arms  ICMJE 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.

Intervention: Drug: Diacerein 1% Ointment
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Enrolling by invitation
Estimated Enrollment  ICMJE
 (submitted: January 2, 2018)
80
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 15, 2019
Estimated Primary Completion Date November 15, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • In the opinion of the Investigator, the subject is capable of understanding and complying with protocol requirements.
  • The subject or, when applicable, 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 (e.g. medicated cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.).
  • 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, as defined by this protocol, 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.

Exclusion Criteria:

  • Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require topical antibiotic therapy to treat an infection).
  • 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, in the investigator's opinion, puts the subject at undue risk to participate.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 99 Years   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03389308
Other Study ID Numbers  ICMJE CCP-020-302
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Castle Creek Pharmaceuticals, LLC
Study Sponsor  ICMJE Castle Creek Pharmaceuticals, LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Amir Tavakkol, MD Castle Creek Pharmaceuticals
PRS Account Castle Creek Pharmaceuticals, LLC
Verification Date February 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP