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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03472287
Recruitment Status : Completed
First Posted : March 21, 2018
Results First Posted : February 10, 2020
Last Update Posted : February 10, 2020
Sponsor:
Information provided by (Responsible Party):
Castle Creek Pharmaceuticals, LLC

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.

Condition or disease Intervention/treatment Phase
Epidermolysis Bullosa (EB) Epidermolysis Bullosa Simplex Dystrophic Epidermolysis Bullosa Junctional Epidermolysis Bullosa Drug: Diacerein 1% Ointment Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 11 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Other
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)
Actual Study Start Date : May 18, 2018
Actual Primary Completion Date : January 31, 2019
Actual Study Completion Date : February 14, 2019


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 Name: 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 Name: CCP-020




Primary Outcome Measures :
  1. 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.




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Ages Eligible for Study:   4 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03472287


Locations
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United States, California
Stanford School of Medicine
Palo Alto, California, United States, 94304
United States, Illinois
Northwestern University Medical Center
Chicago, Illinois, United States, 60654
United States, North Carolina
UNC Dermatology
Chapel Hill, North Carolina, United States, 27599
Sponsors and Collaborators
Castle Creek Pharmaceuticals, LLC
Investigators
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Study Chair: Mary Spellman Castle Creek Pharmaceuticals, LLC
  Study Documents (Full-Text)

Documents provided by Castle Creek Pharmaceuticals, LLC:
Statistical Analysis Plan  [PDF] January 7, 2019
Study Protocol  [PDF] February 7, 2018


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Responsible Party: Castle Creek Pharmaceuticals, LLC
ClinicalTrials.gov Identifier: NCT03472287    
Other Study ID Numbers: CCP-020-101
First Posted: March 21, 2018    Key Record Dates
Results First Posted: February 10, 2020
Last Update Posted: February 10, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Castle Creek Pharmaceuticals, LLC:
EB
EBS
DEB
JEB
Additional relevant MeSH terms:
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Epidermolysis Bullosa
Epidermolysis Bullosa Dystrophica
Epidermolysis Bullosa Simplex
Epidermolysis Bullosa, Junctional
Skin Abnormalities
Congenital Abnormalities
Skin Diseases, Genetic
Genetic Diseases, Inborn
Skin Diseases
Skin Diseases, Vesiculobullous
Collagen Diseases
Connective Tissue Diseases
Diacetylrhein
Anti-Inflammatory Agents