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

Br J Dermatol. 2018 Oct;179(4):836-843. doi: 10.1111/bjd.16368. Epub 2018 May 14.

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.

Objectives: To evaluate the efficacy and safety of ingenol mebutate gel for treating AK of face/scalp and trunk/extremities in a large Asian (Korean) population.

Patients and methods: 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 6 months. 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.

Results: In total, 78·1% [95% confidence interval (CI) 66·86-86·92%] of subjects had CC at day 57, with 76·6% (95% CI 64·31-86·25%) in the face/scalp group and 88·9% (95% CI 51·75-99·72%) in the trunk/extremities group. Among them, CC was sustained in 88·9% (48 of 54, 95% CI 77·37-95·81%) at month 6. The local skin responses significantly increased 1 day after the treatment compared with baseline, and decreased afterwards. Among the total subjects, 7·8% (6 of 77) had hyperpigmentation on the application area. Scars were not reported.

Conclusions: Ingenol mebutate is effective for the treatment of AK in Asians, with tolerable safety profiles.

Publication types

  • Clinical Trial, Phase IV
  • Multicenter Study

MeSH terms

  • Administration, Topical
  • Adult
  • Aged
  • Aged, 80 and over
  • Diterpenes / administration & dosage*
  • Diterpenes / adverse effects
  • Extremities
  • Facial Dermatoses / drug therapy*
  • Facial Dermatoses / psychology
  • Female
  • Follow-Up Studies
  • Humans
  • Hyperpigmentation / chemically induced
  • Hyperpigmentation / epidemiology
  • Keratosis, Actinic / drug therapy*
  • Keratosis, Actinic / psychology
  • Male
  • Middle Aged
  • Prospective Studies
  • Quality of Life
  • Republic of Korea
  • Scalp Dermatoses / drug therapy*
  • Scalp Dermatoses / psychology
  • Torso
  • Treatment Outcome

Substances

  • 3-ingenyl angelate
  • Diterpenes