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

• Status: Completed
• Trial end date: 2019-02-14
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Castle Creek Pharmaceuticals, LLC
• Treatments: Diacetylrhein

Criteria

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