Diacerein for butterfly children

• Register: EUCTR
• Last refreshed on: 13 March 2017
• Main ID: EUCTR2013-002034-21-AT
• Date of registration: 01/08/2013
• Prospective Registration: Yes
• Primary sponsor: EB House Austria
• Public title: Diacerein for butterfly children
• Scientific title: Diacerein for the treatment of Epidermolysis bullosa simplex - Diacerein for EBS
• Date of first enrolment: 16/12/2013
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-002034-21
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: yes Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Austria

Contacts

• Name: EB House Austria
• Affiliation: EB House Austria

• Name: EB House Austria
• Affiliation: EB House Austria

Key inclusion & exclusion criteria

Inclusion criteria:
- Epidermolysis bullos simplex - type Dowling-Meara
- Mutations in either the keratin 14 or the keratin 5 gene, leading to protein aggregation
- Age: 6-19
Are the trial subjects under 18? yes
Number of subjects for this age range: 14
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- Other types of EB with mutations in the keratin 14 or teh keratin 5 gene.
- participation in another clinical trial at the same time
- known intolerance against diacerein, adjuvants (especially Tartrazine) or other substances with similar structures
- pregnancy or lactation
- relevant other illnesses (acute infections, ...)
- patients with impaired liver functions


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

Health Condition(s) or Problem(s) studied

Epidermolysis bullosa simplex - type Dowling-Meara

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

Intervention(s)

Product Name: SimpleCare
Pharmaceutical Form: Cream
INN or Proposed INN: DIACEREIN
CAS Number: 13739-02-1
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Cream
Route of administration of the placebo: Topical use (Noncurrent)

Primary Outcome(s)

Main Objective: Reduction of blister numbers on the treated body surface by 40% in the diacerein group in comparison to placebo within 4 weeks.

Timepoint(s) of evaluation of this end point: After 4 weeks

Secondary Objective: Time until the reaching of the initial blister number (+/-10%) during the follow-up phase.

Primary end point(s): Reduction of blister numbers in the treated area by 40% in comparison to placebo

Secondary Outcome(s)

Secondary end point(s): Time span until reaching the initial blister number (+/- 10%).

Timepoint(s) of evaluation of this end point: After 16 weeks.

Secondary ID(s)

Dia2014

Source(s) of Monetary Support

DEBRA Austria