A Phase 2 interventional study of AC-203 and Vehicle in Inherited Epidermolysis Bullosa, sponsored by TWi Biotechnology, Inc.. Completed at 2 sites in Taiwan. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2019-04-16.
Sponsored by TWi Biotechnology, Inc. · Phase 2, Interventional, and Treatment
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.
Is male, or is female and meets all the following criteria:
Exclusion Criteria:
AC-203 1% ointment, QD
Drug: AC-203
Vehicle ointment, QD
Drug: Vehicle
The investigational product is formulated as 1% topical ointment
Vehicle-only control study medication is the same formulation as investigational product without active ingredient
Percentage change in lesion surface area from baseline by treatment
Time frame: 2, 4, 5, 6, 8, 12 Weeks
Percentage change in blister number from baseline by treatment
Time frame: 2, 4, 5, 6, 8, 12 Weeks
Proportion of subjects with at least 40% reduction in blister number from baseline by treatment
Time frame: 2, 4, 5, 6, 8, 12 Weeks
Pruritus assessment scale changes from baseline by treatment
100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)
Time frame: 2, 4, 5, 6, 8, 12 Week
Pain assessment scale changes from baseline by treatment
100-mm line (anchored at 0 mm for no pruritus, 100 mm for worst possible pruritus)
Time frame: 2, 4, 5, 6, 8, 12 Weeks
IL-1beta concentrations and changes from baseline
Time frame: 8 Weeks
hsCRP concentrations and changes from baseline
Time frame: 8 Weeks
This study is completed, as verified in Apr 2019. You cannot join it, but the record below documents what was studied.
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TWi Biotechnology, Inc.