TWi Biotechnology, Inc. is conducting an international phase II/III study to evaluate the efficacy and safety of AC-203 (diacerein 1% ointment) for the treatment of lesions associated with generalized epidermolysis bullosa simplex (EBS). This genetic skin disorder is characterized by skin fragility and recurrent blister formation, primarily caused by mutations in keratins 5 and 14.
Common subtypes of EBS include localized, intermediate, and severe EBS, based on the clinical manifestations; intermediate and severe EBS are collectively known as generalized EBS. The mutated keratin proteins disrupt the keratin 5/14 network and may result in keratin aggregates, triggering inflammation and exacerbating EBS. Targeting both keratin aggregates and inflammation is considered a potential therapeutic approach in EBS.
AC-203 is a topical formulation of diacerein, which is known to inhibit inflammation. Diacerein has demonstrated its ability in reducing keratin aggregates in keratinocytes derived from severe EBS. AC-203 shows promise in reducing the clinical severity of EBS lesions. In the Phase II DELIVERS study (CCP-020-301), AC-203 was assessed in patients with different EBS subtypes, including patients carrying non-keratin mutations. In that trial, after once daily topical application of AC-203 for 8 weeks, the clinical severity of EBS had notably improved in the severe EBS subgroup. The subsequent long-term extension study, comprising up to two 8-week cycles of treatment, further confirmed that AC-203 was generally safe and well-tolerated.
In the EBShield study, approximately 80 patients with severe or intermediate EBS subtypes will receive either AC-203 or vehicle ointment, applied topically once daily to all identified EBS lesions and surrounding skin for 8 weeks, followed by 8 weeks off study drug. Patients who complete this portion of the study may be eligible to continue into an open-label extension portion of the study and receive AC-203 for 24 weeks. Overall, each patient may participate in the full study for a range of 40 weeks to 44 weeks.
- Male and female patient at least 6 months of age.
- Patient has been clinically diagnosed with severe EBS or intermediate EBS.
- Patient has a documented autosomal dominant genetic mutation in either the keratin 14 (KRT14) or keratin 5 (KRT5) gene.
- Patient has EBS skin lesions on 5% or more of their body surface area, excluding their hands and feet, and the overall lesion severity is rated as moderate to severe by the investigator.
- Patient and/or their legal representative has/have been informed, has/have read and understood the patient information/informed consent form, and has/have given written informed consent.
- Patient and their caregiver must be able and willing to follow study procedures and instructions.
- Please click here for detailed entry criteria, including exclusion criteria.
If you have any questions or are interested in participating in this clinical trial, please contact:
Sandy Lin
- Email: twib-ebs@twibiotech.com
Locations:
Please click the link below for information on where the study is being conducted and corresponding contact details.
