Inherited Epidermolysis Bullosa Clinical Trial
Official title:
A Double-blind, Intra-individual Comparison, Proof-of-concept Trial of Topical AC-203 in Patients With Inherited Epidermolysis Bullosa
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.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT01294241 -
Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
|
Phase 2 | |
| Recruiting |
NCT05954416 -
FARD (RaDiCo Cohort) (RaDiCo-FARD)
|