Safety and Efficacy of Diacerein 1% Ointment for Subjects With Epidermolysis Bullosa Simplex (EBS)

NCT03154333 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: CCP-020-301
• Study Type: interventional
• Target: 54
• Locations: 7 countries
• Sites: 21

Brief Summary

Epidermolysis bullosa simplex (EBS) is a rare genetic skin disease characterized by fragility of the skin and mucous membranes resulting in painful blisters and erosions after minor trauma. The purpose of this study is to compare the efficacy of diacerein 1% ointment to vehicle ointment when applied once-daily for 8 weeks in subjects with EBS.

Conditions

Epidermolysis Bullosa Simplex

Keywords

Epidermolysis; bullosa; simplex; EBS

Outcome Measures

Primary Outcomes (1)

• Proportion of Subjects Who Achieved ≥ 60% Reduction in Body Surface Area (BSA) of EBS Lesions Within Assessment Area

  Analysis of the proportion of subjects who achieved a ≥60% reduction in Body Surface Area (BSA) of EBS lesions within Assessment Area from Baseline to Week 8

  Baseline to Week 8

Secondary Outcomes (1)

• The Proportion of Subjects Who Achieved Success on the Investigator's Global Assessment (IGA)

  Baseline to Week 8

Study Arms (2)

diacerein 1% ointment

EXPERIMENTAL

diacerein 1% ointment will be used for 8 weeks

Drug: diacerein 1% ointment

vehicle ointment

PLACEBO COMPARATOR

vehicle ointment will be used for 8 weeks

Drug: A placebo ointment

Interventions

diacerein 1% ointment DRUG

diacerein 1% ointment administered topically

Also known as: CCP-020

diacerein 1% ointment

A placebo ointment DRUG

vehicle ointment administered topically

vehicle ointment

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:

• Subject is at least 4 years of age at Screening
• Subject has an Assessment Area of EBS lesions to be treated, that is ≥2% body surface area (BSA) and the EBS lesions are in one or both of the following body areas:
• Localized: plantar and/or palmar areas
• Generalized: arms, legs, torso, hands and feet
• Subject's EBS lesions in the Assessment Area have an Investigator's Global Assessment (IGA) score of ≥3
• Subject/caregiver agrees to not use any topical therapies other than the study medication that might influence the status of the EBS lesions during the duration of the study
• Subject is non-pregnant as confirmed by a negative urine pregnancy screen, non-lactating and is not planning for pregnancy during the study period
• If the subject is a woman of childbearing potential, agrees to use an approved effective method of birth control
• Subject is in good general health and free of any known disease state or physical condition which might impair evaluation of the EBS lesions or which exposes the subject to an unacceptable risk by study participation

You may not qualify if:

• Subject has EBS lesions to be treated that are infected
• Subject has used any diacerein containing product within 6 months prior to Screening
• Subject has used systemic immunotherapy or cytotoxic chemotherapy within 60 days prior to Screening
• Subject has used systemic steroidal therapy or has used topical steroidal therapy on the EBS lesions to be treated within 30 days prior to Baseline
• Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Screening
• Subject is currently using systemic analgesics and/or anti-histamine therapy, for treatment of EBS lesions unless on a stable regimen (i.e., the same dosing regimen) for at least 4 weeks prior to Screening
• Subject has used any systemic diuretics or cardiac glycosides or any systemic product that might put the subject at undue risk
• Subject has used any topical product containing allantoin on the EBS lesions to be treated within 30 days prior to Screening
• Subject has a current malignancy, or a history of treatment for a malignancy within 2 years prior to Screening
• Subject currently has diabetes mellitus (HbA1c ≥6.5%) or controlled diabetes (HbA1c \< 6.5%)
• Subject has a history of cardiac, hepatic (ALT and or AST \>2x ULN, Total bilirubin \>1.5x ULN at Screening), or renal disease (eGFR\<30 ml/min/1.73 m\^2)
• Subject has a non-EBS skin disease or condition (e.g., sunburn) that might put the subject at undue risk by study participation or interferes with the study medication application or the study assessments

Sponsors & Collaborators

• Castle Creek Pharmaceuticals, LLC: lead

Study Sites (22)

Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Ann and Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

University of Minnesota

Minneapolis, Minnesota, 55455, United States

Location

University of Missouri Healthcare

Columbia, Missouri, 65212, United States

Location

Stony Brook University Medical Center

Stony Brook, New York, 11790, United States

Location

University of North Carolina - Chapel Hill

Chapel Hill, North Carolina, 27516, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425-5780, United States

Location

Children's Hospital of San Antonio ; Texas Dermatology and Laser Specialists

San Antonio, Texas, 78218, United States

Location

Premier Specialists Pty Ltd; The Church

Kogarah, New South Wales, 2217, Australia

Location

EB House Austria

Salzburg, A-5020, Austria

Location

Hopital Saint Louis

Paris, Cedex, 75010, France

Location

Hopital Necker-Enfants Malades

Paris, Cedex, 75015, France

Location

CHU de NICE - Hopital de l'Archet II - Service de Dermatologie

Nice, 06202, France

Location

University Medical Center Freiburg

Freiburg im Breisgau, 79104, Germany

Location

Tel Aviv Sourasky Medical Center

Tel Aviv, 6423906, Israel

Location

University Medical Center Groningen

Groningen, AB20;30,001, Netherlands

Location

Great Ormond Street Hospital

London, England, WC1N 3JH, United Kingdom

Location

St. Thomas' Hospital - St Johns Institute of Dermatology

London, SE1 7EH, United Kingdom

Location

MeSH Terms

Conditions

Epidermolysis Bullosa Simplex

Interventions

diacerein; Ointments

Condition Hierarchy (Ancestors)

Epidermolysis Bullosa; 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

Intervention Hierarchy (Ancestors)

Dosage Forms; Pharmaceutical Preparations

Results Point of Contact

• Title: Dr. Mary Spellman
• Organization: Castle Creek Pharmaceuticals, LLC

mspellman@castlecreekpharma.com

862 286 0400

Study Officials

• Mary Spellman, MD

  Castle Creek Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 10, 2017
• First Posted: May 16, 2017
• Study Start: June 1, 2017
• Primary Completion: October 15, 2018
• Study Completion: October 31, 2018
• Last Updated: November 5, 2019
• Results First Posted: November 5, 2019

Record last verified: 2019-10

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); US (12); IL (1); NL (1); FR (3); DE (1); GB (2)