A study has been initiated to evaluate the pharmacokinetics and safety of CCP-020 (Diacerein 1% Ointment) after maximum use in people living with Epidermolysis Bullosa.
CCP-020 is an investigational topical diacerein 1% ointment being developed by Castle Creek Pharmaceuticals. It is a potentially disease-modifying therapy that may block an important inflammatory signaling pathway associated with EB, which could strengthen epidermal tissue and support healing.
This new multi-center study is expected to enroll 16-20 people living with EB ranging from infants and children ages six months to 11 years old, and adolescents and adults ages 12 years old and up. It is important to note that U.S. study locations will only enroll people who are ages four years old and up.
People who enroll in the study will be asked to provide blood samples over the course of 10 days while receiving treatment. There is a weight requirement of at least 9 kg (19.8 lbs) at screening, and you need to have a documented genetic mutation consistent with EB to enroll. A blood or saliva sample can be collected for genetic confirmation unless this genetic confirmation has been previously performed. People with localized EB lesions on the hands or feet, or generalized lesions on arms, legs, and torso are encouraged to enroll as long as the lesions cover greater than 2% of the total body surface area.
Phoenix Children's Hospital, Stanford School of Medicine, Northwestern University Medical Center, University of Minnesota and University of North Carolina Dermatology are planning to participate in the United States, while Great Ormond Street Hospital is participating in the United Kingdom, Hopital Universitaire, Necker-Enfants-Malades is participating in France and University Medical Center Groningen is participating in the Netherlands
- Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form.
- Subject is male or female at least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive (Cohort 2) at screening. US only: subjects must be at least 4 years of age to 11 years, inclusive (Cohort 2) at time of consent.
- The subject must weigh at least 9 kg (19.8 lbs) at Screening.
- Subject has a documented genetic mutation consistent with EB. A blood or saliva sample will be collected for genetic confirmation if no documented gene mutation data is available.
- Subject has EB lesions on ≥ 2% body surface area (BSA) and the EB lesions are in the following body areas:
- Localized: plantar and/or palmar areas
- Generalized: arms, legs, torso, hands and feet.
- Please click the link below for exclusion criteria.