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

NCT03472287 | Completed Phase 1 Results FDA Drug

• 1 other identifier: CCP-020-101
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 3

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.

Conditions

Epidermolysis Bullosa (EB); Epidermolysis Bullosa Simplex; Dystrophic Epidermolysis Bullosa; Junctional Epidermolysis Bullosa

Keywords

EB; EBS; DEB; JEB

Outcome Measures

Primary Outcomes (1)

• Detectable Plasma Concentrations of Diacerein and Rhein

  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

Study Arms (2)

Cohort 1 (Adolescents, Adults)

EXPERIMENTAL

Adolescent and adult subjects with EB (aged 12 years and older) received diacerein 1% ointment daily for 10 days.

Drug: Diacerein 1% Ointment

Cohort 2 (Children)

EXPERIMENTAL

Children with EB (aged 4 to 11 years, inclusive) received diacerein 1% ointment daily for 10 days.

Drug: Diacerein 1% Ointment

Interventions

Diacerein 1% Ointment DRUG

Diacerein 1% Ointment administered topically

Also known as: CCP-020

Cohort 1 (Adolescents, Adults); Cohort 2 (Children)

Eligibility Criteria

• Age: 4 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Castle Creek Pharmaceuticals, LLC: lead

Study Sites (3)

Stanford School of Medicine

Palo Alto, California, 94304, United States

Location

Northwestern University Medical Center

Chicago, Illinois, 60654, United States

Location

UNC Dermatology

Chapel Hill, North Carolina, 27599, United States

Location

MeSH Terms

Conditions

Epidermolysis Bullosa; Epidermolysis Bullosa Simplex; Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa, Junctional

Interventions

diacerein; Ointments

Condition Hierarchy (Ancestors)

Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Skin Diseases; Skin and Connective Tissue Diseases; Skin Diseases, Vesiculobullous; Collagen Diseases; Connective Tissue Diseases

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Results Point of Contact

• Title: Dr. Mary Spellman/Chief Medical Officer
• Organization: Castle Creek Pharmaceuticals, LLC

mspellman@castlecreekpharma.com

862 286 0400

Study Officials

• Mary Spellman

  Castle Creek Pharmaceuticals, LLC

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 14, 2018
• First Posted: March 21, 2018
• Study Start: May 18, 2018
• Primary Completion: January 31, 2019
• Study Completion: February 14, 2019
• Last Updated: February 10, 2020
• Results First Posted: February 10, 2020

Record last verified: 2020-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)