Epidermolysis Bullosa
Conditions
Interventions
All subjects will be dosed open label with CCP-020 (Diacerein 1% ointment)
daily for 10 days.
Sponsors
Castle Creek Pharmaceuticals, LLC
Eligibility
Age
2 Years to 99 Years
Inclusion criteria
Inclusion criteria: 1. Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form. 2. 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. 3. The subject must weigh at least 9 kg (19.8 lbs) at Screening. 4. 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. 5. Subject has EB lesions on * 2% body surface area (BSA) and the EB lesions are in the following body areas: a. Localized: plantar and/or palmar areas b. Generalized: arms, legs, torso, hands and feet. 6. Subject/caregiver agrees to not apply any other topical products to the application area during the treatment period 7. 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. 8. Subject is non-pregnant, non-lactating and is not planning for pregnancy during the study period 9. Subject is in good general health and free of any known disease state or physical condition which, in the investigator*s opinion, which exposes the subject to an unacceptable risk by study participation. 10. Subject is willing and able to follow all study instructions and to attend all study visits.
Exclusion criteria
Exclusion criteria: 1. Subject has EB lesions where drug will be applied that are infected (i.e., EB lesions that require anti-microbial therapy to treat an infection) 2. Subject has used any diacerein containing product within 1 month prior to Visit 1 3. Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to dosing. 4. Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EB lesions in the application area within 14 days prior to dosing (Note: inhaled, nasal sprays, and ophthalmic products containing steroids are allowed) 5. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to dosing 6. Subject has used any systemic diuretics or cardiac glycosides or any systemic product that, in the opinion of the investigator, might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments within 30 days prior to dosing 7. Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to dosing (Note: does not include non-melanoma skin cancer) 8. Subject currently has diabetes mellitus (HbA1c *6.5%) Note: controlled diabetes (HbA1c 2x ULN, Total bilirubin >1.5x ULN at Visit 1), or renal disease (eGFR
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Derived plasma PK parameters (Cmax, Tmax and AUC), if available, for diacerein and rhein will be summarized by treatment using descriptive statistics (sample size, arithmetic and geometric mean, CV%, SD of the arithmetic mean, median, minimum, and maximum) | — |
Secondary
| Measure | Time frame |
|---|---|
| - | — |
Countries
Netherlands
Outcome results
None listed