NCT03468322

A Double-blind, Intra-individual Comparison, POC Trial of AC-203 in EB Patients

A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: TWi Biotechnology, Inc. · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

Inherited epidermolysis bullosa (EB) is a genetic skin disorder characterized by skin fragility and recurrent blister formation. More and more evidence has suggested that the skin lesions initially caused by genetic mutations may be further aggravated by inflammatory responses. Several reports showed successful alleviation of EB symptoms upon treatment with immunomodulatory therapies. Modulation of proinflammatory cytokine IL-1β has shown promising results in alleviating epidermolysis bullosa simplex (EBS), a major subtype of inherited EB, by downregulating IL-1β-mediated JNK/MAPK signaling pathway. This data further supports the potential of using cytokine modulators to treat EB. AC-203, a topical formulation, can inhibit the production and activity of IL-1β, down-regulate IL-1β receptors, and increase IL1β-receptor antagonist (IL1-Ra) expression. In addition, AC-203 has been reported to inhibit anti-BP180 autoantibody-induced IL-6/IL-8 upregulation in cultured keratinocytes and LPS-induced IL-6 upregulation in cultured macrophages. Furthermore, AC-203 was also found to inhibit the formation of NLRP3 inflammasome, which plays essential roles in induction of caspase-1-dependent pyroptosis and release of inflammatory cytokines IL-1β and IL-18. These studies demonstrated the cytokine modulatory properties of AC-203 and pointed out the possible application of AC-203 in a variety of inflammatory diseases. This study is designed to test the efficacy, safety, tolerability, and pharmacokinetics of AC-203 ointment (vs. placebo) in patients with inherited EB.

Conditions

• Inherited Epidermolysis Bullosa

Interventions

Type	Name	Description
DRUG	AC-203	The investigational product is formulated as 1% topical ointment
DRUG	Vehicle	Vehicle-only control study medication is the same formulation as investigational product without active ingredient

Timeline

• Start date: 2018-10-20
• Primary completion: 2019-04-09
• Completion: 2019-04-09
• First posted: 2018-03-16
• Last updated: 2019-04-16

Locations

2 sites across 1 country: Taiwan