An International Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)

• Register: EUCTR
• Last refreshed on: 9 November 2020
• Main ID: EUCTR2017-003757-41-NL
• Date of registration: 06/12/2017
• Prospective Registration: Yes
• Primary sponsor: Castle Creek Pharmaceuticals, LLC
• Public title: An International Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)
• Scientific title: An International, Multicenter, Open-label, Long Term Extension Study Evaluating the Safety of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS)
• Date of first enrolment: 11/04/2018
• Target sample size: 80
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003757-41
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, France, Germany, Israel, Italy, Netherlands, United Kingdom, United States

Contacts

• Name: Vasiliki Iassonidou
• Affiliation: Medpace Germany GmbH

• Name: Vasiliki Iassonidou
• Affiliation: Medpace Germany GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
1. In the opinion of the Investigator, the subject is capable of understanding and complying with protocol requirements.
2. The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent/assent form and any required privacy authorization prior to the initiation of any study procedures.
3. Subject has a documented genetic mutation consistent with EBS.
4. Subject has completed study CCP-020-301 or participated in study CCP-020-101.
5. Subject/caregiver agrees to report use of any topical therapies applied to EBS lesions (e.g. medicated cleansers, bleach cleansers, bleach baths, topical antiseptics, topical disinfectants, etc.).
6. If the subject is a woman of childbearing potential, she has a negative urine pregnancy test and agrees to use an approved effective method of birth control, as defined by this protocol, for the duration of the study.
7. Subject is non-lactating and is not planning for pregnancy during the study period.
8. Subject is willing and able to follow all study instructions and to attend all study visits.

Are the trial subjects under 18? yes
Number of subjects for this age range: 44
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 20
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion criteria:
1. Subject has EBS lesions to be treated that are infected (i.e., EBS lesions that require topical antibiotic therapy to treat an infection).
2. Subject has evidence of a systemic infection or has used systemic antibiotics within 7 days prior to Baseline.
3. The subject was discontinued from the feeder study due to an adverse event judged to be related or possibly related to the study medication.
4. Subject has experienced a change in clinical status from the feeder study that, in the investigator’s opinion, puts the subject at undue risk to participate.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

Epidermolysis Bullosa Simplex (EBS)
MedDRA version: 20.0 Level: PT Classification code 10014989 Term: Epidermolysis bullosa System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: Diacerein
Product Code: Diacerein
Pharmaceutical Form: Ointment
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Current Sponsor code: CCP-020
Other descriptive name: Diacerein
Concentration unit: % (W/W) percent weight/weight
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)

Main Objective: The primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101.

Secondary Objective: None

Primary 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: For each cycle: start day; start day + 8 weeks; start day + 16 weeks

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Not applicable

Secondary end point(s): None

Secondary ID(s)

CCP-020-302

131 384

2017-003757-41-GB

Source(s) of Monetary Support

Castle Creek Pharmaceuticals, LLC

Ethics review

Status: Approved
Approval date: 11/04/2018
Contact:

Results

• Results available: Yes
• Date Posted: 17/10/2020
• Date Completed: 17/04/2020
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2017-003757-41/results