To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)

Not Recruiting

Trial ID: NCT03472287

Purpose

A pharmacokinetic (PK) study in 16-20 EB subjects to be allocated to two cohorts. Cohort 1 to include 8-10 subjects (ages 12 yrs and older); Cohort 2 to include 8-10 subjects (ages 6 months-11 yrs, inclusive). Cohort 2 only included subjects 4 yrs and older. Serial PK blood sampling collected on Days 1 and 10. Analyses were performed to determine the concentrations of diacerein and rhein.

Official Title

A Multi-Center Study to Evaluate the Pharmacokinetics of Diacerein and Rhein and the Safety of Diacerein After Maximum Use, Topical Administration of CCP-020 (Diacerein 1% Ointment) to Patients With Epidermolysis Bullosa (EB)

Eligibility


Key Inclusion Criteria:

   - At least 12 years of age (Cohort 1) or at least 6 months of age to 11 years, inclusive
   (Cohort 2) at screening. For US only: at least 4 years of age to 11 years, inclusive
   (Cohort 2)

   - The subject must weigh at least 9 kg (19.8 lbs) at Screening.

   - Subject has a documented genetic mutation consistent with EB.

   - 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.

Key Exclusion Criteria:

   - Subject has EB lesions where drug will be applied that are infected

   - Subject has used any diacerein containing product within 1 month prior to Visit 1

   - Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior
   to dosing.

   - 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

   - Subject has participated in an investigational drug trial in which administration of
   an investigational study medication occurred within 30 days prior to dosing

Intervention(s):

drug: Diacerein 1% Ointment

Not Recruiting

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Joyce Teng, MD