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

February 4, 2020 updated by: Castle Creek Pharmaceuticals, LLC

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)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

11

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • Palo Alto, California, United States, 94304
        • Stanford School of Medicine
    • Illinois
      • Chicago, Illinois, United States, 60654
        • Northwestern University Medical Center
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • UNC Dermatology

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

4 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Other
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Cohort 1 (Adolescents, Adults)
Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.
Drug: Diacerein 1% Ointment
Diacerein 1% Ointment administered topically
Other Names:
  • CCP-020
Experimental: Cohort 2 (Children)
Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.
Drug: Diacerein 1% Ointment
Diacerein 1% Ointment administered topically
Other Names:
  • CCP-020

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Detectable Plasma Concentrations of Diacerein and Rhein
Time Frame: Days 1-10, at select time points per protocol

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Castle Creek Pharmaceuticals, LLC

Investigators

  • Study Chair: Mary Spellman, Castle Creek Pharmaceuticals, LLC

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 18, 2018

Primary Completion (Actual)

January 31, 2019

Study Completion (Actual)

February 14, 2019

Study Registration Dates

First Submitted

March 14, 2018

First Submitted That Met QC Criteria

March 20, 2018

First Posted (Actual)

March 21, 2018

Study Record Updates

Last Update Posted (Actual)

February 10, 2020

Last Update Submitted That Met QC Criteria

February 4, 2020

Last Verified

February 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CCP-020-101

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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