Clinical trial NCT03472287: To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Epidermolysis Bullosa (EB)
Epidermolysis Bullosa Simplex
Dystrophic Epidermolysis Bullosa
Junctional Epidermolysis Bullosa
Keywords: EB
EBS
DEB
JEB
Lead sponsor: Castle Creek Pharmaceuticals, LLC
Sponsor class: INDUSTRY
First posted: 2018-03-21
Enrollment: 11
Sex: ALL
Minimum age (years): 4
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: OTHER

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.

Eligibility 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

Primary outcomes:
measure: Detectable Plasma Concentrations of Diacerein and Rhein
timeFrame: Days 1-10, at select time points per protocol
Secondary outcomes:
Other outcomes:
Locations:
city: Palo Alto
country: United States
facility: Stanford School of Medicine
state: California
city: Chicago
country: United States
facility: Northwestern University Medical Center
state: Illinois
city: Chapel Hill
country: United States
facility: UNC Dermatology
state: North Carolina
Study officials:
affiliation: Castle Creek Pharmaceuticals, LLC
name: Mary Spellman
role: STUDY_CHAIR
References: