A Proof-of-Concept Study of Topical AC-203 in Patients With Bullous Pemphigoid
Status:
Terminated
Trial end date:
2019-01-22
Target enrollment:
Participant gender:
Summary
Bullous pemphigoid (BP) is a chronic, inflammatory, subepidermal, autoimmune blistering
disease which mainly develops in the elderly, with onset usually in the late 70s and a
substantial increase in incidence in people older than 80 years. If untreated, it can persist
for months or years, with periods of spontaneous remissions and exacerbations. It has been
found that blisters and sera of BP patients contain abnormally high levels of
pro-inflammatory cytokines such as interleukin-6 (IL-6) and IL-8. Recently, it also has been
demonstrated that NLRP3 (NACHT, LRR and PYD domains-containing protein 3) inflammasome
components (the NLRP3-caspase-1-IL-18 axis) were significantly up-regulated in peripheral
blood mononuclear cells from BP patients and positively correlated with disease activity.
AC-203 is a topical formulation of an oral modulator of inflammasome and IL-1beta pathways.
In vitro studies have demonstrated that AC-203 significantly reduced secretion of IL-6 and
moderately reduced IL-8 secretion in HaCaT cells treated with specific anti-BP180 IgG. This
study is designed to test the safety, tolerability, efficacy, and pharmacokinetics of AC-203
ointment (vs. a topical steroid comparator representing standard of care) ointment in
subjects with BP.