The primary objective of this study is to evaluate the long-term safety and tolerability of Diacerein 1% Ointment for 2 treatment cycles in subjects with EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101.
ID
Source
Brief title
Condition
- Epidermal and dermal conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
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.
Secondary outcome
-
Background 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, and is associated with
significant morbidity and mortality. EBS is both a pediatric and an adult
disease that tends to affect younger patients most severely. Diacerein 1%
Ointment is a topical ointment containing diacerein (4,5-bis[acetyloxy]-9,10-
dihydro-9,10dioxo-2-anthracene carboxylic acid, also known as diacetyl
rhein), a highlypurified anthraquinone derivative, and is being developed for
the treatment of EBS. Diacerein in the topical formulation is hydrolyzed to
rhein in the epidermis and dermis following administration. Diacerein and rhein
have been shown to inhibit the in vitro and in vivo production and activity of
interleukin-1* (IL-1*) and other proinflammatory cytokines. IL-1* is a pro
inflammatory cytokine that has been linked to a number of inflammatory and
autoimmune diseases, including rheumatoid arthrtis (RA), OA, hemophilic
arthropathy, gouty arthritis, type 2 diabetes mellitus (T2DM), diabetic
nephropathy (DN), and EBS. In vitro and in vivo animal studie have shown that
both diacerein and its active metabolite rhein inhibit the produiction and
activity of pro-inflammatory and procatabolic cytokines such as IL-1 and IL-6,
and the expression of inducible nitric oxide synthase (iNOS) and tumor necrosis
factor-* (TNF-*).
Study objective
The primary objective of this study is to evaluate the long-term safety and
tolerability of Diacerein 1% Ointment for 2 treatment cycles in subjects with
EBS that were previously enrolled in studies CCP-020-301 or CCP-020-101.
Study design
The study enrolls subjects from two feeder studies, CCP-020-301 and
CCP-020-101. Subjects that enroll will, after a screening, undergo a 16 week
cycle if they have developed blisters, in which they will be on the study drug
for the first 8 weeks, and off the study drug for the next 8 weeks. This cycle
can be done a maximum of two times, and subjects cannot start a new cycle after
week 36.
Intervention
Subjects who are enrolled will apply diacerein 1% ointment daily for 8 weeks in
a 16 week cycle.
Study burden and risks
Risks: Possible side effects of the study medication
Burden: Blood draws, instructions on study drug, filling in questionnaires
Rivium 2de straat 55
Capelle aan den IJssel 2909 LG
NL
Rivium 2de straat 55
Capelle aan den IJssel 2909 LG
NL
Listed location countries
Age
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. Subjects who participated in the
CCP-020-301 or the CCP-020-101 study are eligible to be rolled into the
CCP-020-302 open label extension study, regardless of their completion status
on the feeder study. , 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.
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.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2017-003757-41-NL |
| CCMO | NL64012.042.17 |