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

Dystrophic Epidermolysis Bullosa Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03472287
• Other study ID #: CCP-020-101
• Status: Completed
• Phase: Phase 1
• Start date: May 18, 2018
• Est. completion date: February 14, 2019

Study information

• Verified date: February 2020
• Source: Castle Creek Pharmaceuticals, LLC
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: February 14, 2019
• Est. primary completion date: January 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 4 Years and older

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

Related Conditions & MeSH terms

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

Intervention

Drug:
• Diacerein 1% Ointment
Diacerein 1% Ointment administered topically

Locations

Country	Name	City	State
United States	UNC Dermatology	Chapel Hill	North Carolina
United States	Northwestern University Medical Center	Chicago	Illinois
United States	Stanford School of Medicine	Palo Alto	California

Sponsors (1)

Lead Sponsor	Collaborator
Castle Creek Pharmaceuticals, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Detectable Plasma Concentrations of Diacerein and Rhein	Bioanalytical analyses were performed to determine concentrations levels of diacerein and rhein in plasma using validated bioanalytical methods.; For Cohort 1, blood samples were taken at pre-dose and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose.; For Cohort 2, blood samples were taken at pre-dose and 1, 2, 4, 6, and 8 hours post-dose.; Trough PK samples were collected on any 2 available days from Days 3 through 9 for Cohort 1 only.; Summary statistics for each scheduled time were only reported if at least 50% of subjects had quantifiable concentrations.	Days 1-10, at select time points per protocol