CastleCreek Extension

• Research type

  Research Study

• Full 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)

• IRAS ID

  237889

• Contact name

  Jemima Mellerio

• Contact email

  Jemima.mellerio@kcl.ac.uk

• Sponsor organisation

  Castle Creek Pharmaceuticals, LLC

• Eudract number

  2017-003757-41

• Clinicaltrials.gov Identifier

  131,384, IND

• Duration of Study in the UK

  2 years, 0 months, 1 days

• Research summary

  The study drug, Diacerein 1% ointment is an investigational product being studied for the treatment of Epidermolysis bullosa simplex (EBS). EBS is a rare, genetic skin disease characterised 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.

  The study is an extension study to the CCP-020-301 and CCP-020-101 parent studies. The study is being done to look at:
  - Long-term safety and tolerability of Diacerein 1% Ointment for the treatment of EBS for participants previously enrolled in the CCP-020-301 or CCP-020-101.
  Diacerein in the topical formulation is broken down into “rhein” within the skin, which is then absorbed into the blood stream. Rhein has shown to reduce the production of interleukin-1β (IL-1β), a protein associated with inflammation. This is known as a ‘pro-inflammatory cytokine’. It has been found that Rhein works differently from conventional ‘Non-Steroidal Anti-Inflammatory (NSAIDs)’ drugs. This means it could be beneficial to those with EBS for who current treatment is not working.

  It is expected that up to 80 participants in 18-22 international investigation centres (of at least 4 years of age) in the US, Europe, Israel and Australia will be recruited to participate in this study, however the actual number of participants with EBS is dependent on the final number of participants that complete the CCP-020-301 or CCP-020-101. Participants who complete the aforementioned studies and who meet the inclusion criteria will be given the opportunity to complete two treatment cycles. Each treatment cycle will consist of 8 weeks on treatment followed by 8 weeks off treatment with a maximum of two treatment cycles allowed for up to 52 weeks.

• REC name

  North West - Liverpool Central Research Ethics Committee

• REC reference

  17/NW/0710

• Date of REC Opinion

  19 Feb 2018

• REC opinion

  Further Information Favourable Opinion