Phase 1
N=11
To Evaluate the Pharmacokinetic of Diacerein and Rhein After Maximum Use in Patients With Epidermolysis Bullosa (EB)
Epidermolysis Bullosa (EB) · Epidermolysis Bullosa Simplex · Dystrophic Epidermolysis Bullosa · Junctional Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT03472287 ↗Enrolled (actual)
11
Serious AEs
0.0%
Results posted
Feb 2020
Primary outcome: Primary: Detectable Plasma Concentrations of Diacerein and Rhein — 3; 2; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 1
- Interventions
- Diacerein 1% Ointment (Drug)
- Age
- Pediatric, Adult, Older Adult · 4+ yrs
- Sex
- All
- Sponsor
- Castle Creek Pharmaceuticals, LLC
- Primary completion
- Jan 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Detectable Plasma Concentrations of Diacerein and Rhein |
3; 2; 0; 0; 1; 1 | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT03472287). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.