A multicentre, open, investigator-initiated phase IV clinical trial to evaluate the efficacy and safety of ingenol mebutate gel, 0 center dot 015% on the face and scalp, and 0 center dot 05% on the trunk and extremities, in Korean patients with actinic keratosis (PERFECT)

Abstract

Background Ingenol mebutate gel is a novel, field-directed topical treatment for actinic keratosis (AK). Most pivotal studies have targeted Western populations. No clinical study has been conducted to investigate its efficacy and safety in Asian populations. ObjectivesPatients and methodsTo evaluate the efficacy and safety of ingenol mebutate gel for treating AK of face/scalp and trunk/extremities in a large Asian (Korean) population. In this multicentre, open-label, interventional, parallel-group, prospective phase IV study (PERFECT, trial registration no.: NCT02716714), the eligible patients were allocated into either the face/scalp or the trunk/extremities group, according to their selected treatment area location. After application of ingenol mebutate gel, the participants were followed up for 6months. The primary efficacy endpoint was complete clearance (CC) of AK lesions in the selected treatment area at day 57. Quality of life was evaluated using Skindex-29. Safety endpoints included local skin responses, scar, pigmentation, pain and adverse events. ResultsConclusionsIn total, 781% [95% confidence interval (CI) 6686-8692%] of subjects had CC at day 57, with 766% (95% CI 6431-8625%) in the face/scalp group and 889% (95% CI 5175-9972%) in the trunk/extremities group. Among them, CC was sustained in 889% (48 of 54, 95% CI 7737-9581%) at month 6. The local skin responses significantly increased 1day after the treatment compared with baseline, and decreased afterwards. Among the total subjects, 78% (6 of 77) had hyperpigmentation on the application area. Scars were not reported. Ingenol mebutate is effective for the treatment of AK in Asians, with tolerable safety profiles.