Epidermolysis Bullosa (EB) MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Diacerein
Product Code: CCP-020
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020
Concentration unit: % (W/W) percent weig
Sponsors
Castle Creek Pharmaceuticals, LLC
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Subject/caregiver is able to comprehend and willing to sign an Informed Consent and/or Assent Form. 2. Subject has a documented genetic mutation consistent with EB. 3. Subject has EB lesions on = 2% body surface area (BSA) and the EB lesions are in the following body areas: 4. Subject/caregiver agrees to not apply any other topical products to the application area during the treatment period 5. Subject is non-pregnant, non-lactating and is not planning for pregnancy during the study period 6. Subject is in good general health and free of any known disease state or physical condition which, in the investigator’s opinion, which exposes the subject to an unacceptable risk by study participation. Are the trial subjects under 18? yes Number of subjects for this age range: 15 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 5 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Subject has EB lesions where drug will be applied that are infected (i.e., EB lesions that require anti-microbial therapy to treat an infection) 2. Subject has an active non-EB skin disease (e.g., psoriasis, atopic dermatitis, eczema, sun damage, etc.), or condition (e.g., sunburn) that, in the opinion of the investigator, would put the subject at undue risk by study participation or would interfere with the study medication application or the study assessments 3. Subject has a history of sensitivity to any of the ingredients in the study medication 4. Subject has participated in an investigational drug trial in which administration of an investigational study medication occurred within 30 days prior to dosing
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to characterize the single-dose and steady- state pharmacokinetics (PK) of diacerein and its active metabolite, rhein, after topical application of CCP-020 (diacerein 1% ointment) under maximum use conditions in adult, adolescent and in infants/children with EB.;Secondary Objective: The secondary objective of the study is to assess the safety and tolerability of single-dose and steady-state topical application of CCP-020 (diacerein 1% ointment) in patients with EB.;Primary end point(s): Pharmacokinetics (PK) of diacerein will be evaluated measuring the amount of CCP-020 (diacerein 1% ointment) and active metabolite, rhein, in blood samples collected pre-dose and post-dose.;Timepoint(s) of evaluation of this end point: At every visit when blood in drawn, pre-dose PK will be performed. At Day 1 and Day 10, full-PK analysis (both pre-dose and post-dose) will also be performed | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety will be evaluated in terms of the occurrence of AEs and changes in clinical laboratory parameters, clinical examination findings, vital signs, weight, and urine measurements.;Timepoint(s) of evaluation of this end point: Safety is evaluated throughout the duration of the study, Day 1 to the follow-up call on Day 24. | — |
Countries
France, Germany, Netherlands, United Kingdom, United States
Contacts
Public ContactMary Elizabeth Scott
Medpace
Outcome results
None listed