Long Term Open-label Study Evaluating Safety of Diacerein 1% Ointment Topical Formulation in Subjects With Epidermolysis Bullosa Simplex

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)

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.

Study Overview

• Status: Completed
• Conditions: Epidermolysis Bullosa; Epidermolysis Bullosa Simplex
• Intervention / Treatment: Drug: diacerein 1% ointment

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: Interventional
• Enrollment (Actual): 51
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Ann and Robert H. Lurie Children's Hospital of Chicago
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27516
      • University of North Carolina (UNC) - Chapel Hill
  • Texas
    • San Antonio, Texas, United States, 78218
      • Children's Hospital of San Antonio

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 months to 99 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

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. treatment; Other Names: CCP-020

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Treatment-emergent Adverse Events	Number and percentage of participants with any treatment-emergent adverse event.	up to 52 weeks

Collaborators and Investigators

• Sponsor: Castle Creek Pharmaceuticals, LLC
• Investigators: Study Chair: Mary Spellman, MD, Castle Creek Pharmaceuticals

Study record dates

Study Major Dates

• Study Start (Actual): December 1, 2017
• Primary Completion (Actual): April 17, 2020
• Study Completion (Actual): April 17, 2020

Study Registration Dates

• First Submitted: December 19, 2017
• First Submitted That Met QC Criteria: January 2, 2018
• First Posted (Actual): January 3, 2018

Study Record Updates

• Last Update Posted (Actual): September 16, 2020
• Last Update Submitted That Met QC Criteria: August 26, 2020
• Last Verified: August 1, 2020

More Information

Terms related to this study

• Keywords: Epidermolysis Bullosa (EB); Epidermolysis Bullosa Simplex (EBS)
• Additional Relevant MeSH Terms: Skin Diseases; Congenital Abnormalities; Genetic Diseases, Inborn; Skin Diseases, Genetic; Skin Diseases, Vesiculobullous; Skin Abnormalities; Epidermolysis Bullosa; Epidermolysis Bullosa Simplex; Anti-Inflammatory Agents; Diacetylrhein
• Other Study ID Numbers: CCP-020-302

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No