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

NCT ID: NCT03472287

Last Updated: 2020-02-10

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1
• Total Enrollment: 11 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2018-05-18
• Study Completion Date: 2019-02-14

Brief 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

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Cohort 1 (Adolescents, Adults)

Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.

Group Type: EXPERIMENTAL

Diacerein 1% Ointment

Intervention Type: DRUG

Diacerein 1% Ointment administered topically

Cohort 2 (Children)

Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.

Group Type: EXPERIMENTAL

Diacerein 1% Ointment

Intervention Type: DRUG

Diacerein 1% Ointment administered topically

Interventions

Diacerein 1% Ointment

Diacerein 1% Ointment administered topically

Intervention Type: DRUG

Other Intervention Names

CCP-020

Eligibility Criteria

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.

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

• Minimum Eligible Age: 4 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Castle Creek Pharmaceuticals, LLC

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Mary Spellman

Role: STUDY_CHAIR

Castle Creek Pharmaceuticals, LLC

Locations

Stanford School of Medicine

Palo Alto, California, United States

Northwestern University Medical Center

Chicago, Illinois, United States

UNC Dermatology

Chapel Hill, North Carolina, United States

Countries

United States

Provided Documents

Document Type: Statistical Analysis Plan

View Document

Document Type: Study Protocol

View Document

Other Identifiers

CCP-020-101

Identifier Type: -

Identifier Source: org_study_id