This study is an international clinical trial for people with Epidermolysis Bullosa Simplex (EBS).
Participants will be randomly assigned to use either diacerein 1% ointment (AC-203) or a control ointment once daily for 8 weeks, without knowing which ointment they are using. After an additional 8-week period without treatment, all participants will use diacerein 1% ointment (AC-203) for 24 weeks. Participants will report outcomes, including pain, itching, and quality of life at each visit.
Anticipated enrolment close date:
Who can take part
Eligible participants
- At least 6 months of age
- Clinically diagnosed severe or intermediate EBS
- EBS lesions on at least 3% body surface area
You cannot take part if you:
- Have other clinically significant skin disease(s)
- Currently have cancer or a history of treatment for a cancer within the last 5 years
- Are pregnant or breastfeeding
What is involved for participants
- Participants will be in this study for up to 50 weeks (screening period up to 10 weeks).
- Involves approximately 10 hospital visits. Most of the study visits should take about one hour, however your first and second visit will be longer.
- Medical history, physical examination, height, and weight
- Provide blood and urine samples
- Perform ECG test
- Medical photography
Ethics
All research in Australia involving human participants is reviewed by an independent group called a Human Research Ethics Committee (HREC). The ethical aspects of this research study have been approved by the Royal Melbourne Hospital HREC.
This study is being carried out according to the National Statement on Ethical Conduct in Human Research (2007). This statement protects the interests of people who agree to participate in human research studies.