NCT03472287

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

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)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Castle Creek Pharmaceuticals, LLC · Industry
• Sex: All
• Age: 4 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This was an open-label, single-period study that intended to include subjects with EB consisting of infants/ children (ages 6 months to 11 years, inclusive) and adolescents/ adults (ages 12 years and older). The study included 2 cohorts. Cohort 1 was to include adolescent/adult subjects with EB (ages 12 years and older); Cohort 2 was to include infants/children with EB (ages 4 to 11 years, inclusive). For both cohorts, the study intended to enroll subjects with epidermolysis bullosa simplex (EBS), dystrophic epidermolysis bullosa (DEB), and junctional epidermolysis bullosa (JEB) with lesions encompassing \>/=2% BSA. The diacerein application area was \>/=5% BSA and included lesioned and non-lesioned skin (if lesions accounted for \<5% BSA); however, topical administration was \</=30% BSA. On Days 1 and 10, the topical dose of study drug was applied by study staff, and blood was sampled for PK analyses of diacerein and rhein pre-dose and through 8 hours post-application. On Days 2 to 9, study drug was applied to the application area(s) at home. For Cohort 1, trough PK samples were collected on any 2 available days from Days 3 through 9, and study drug was applied after blood draw, while for Cohort 2 trough PK analyses were not performed.

Conditions

• Epidermolysis Bullosa (EB)
• Epidermolysis Bullosa Simplex
• Dystrophic Epidermolysis Bullosa
• Junctional Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	Diacerein 1% Ointment	Diacerein 1% Ointment administered topically

Timeline

• Start date: 2018-05-18
• Primary completion: 2019-01-31
• Completion: 2019-02-14
• First posted: 2018-03-21
• Last updated: 2020-02-10
• Results posted: 2020-02-10

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study