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)

Y. C. Kim, J. Y. Yang, J. S. Yoon, S. J. Jo, Hyo Hyun Ahn, K. H. Song, D. Y. Lee, K. Y. Chung, Y. H. Won, Il-Hwan Kim

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3 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)836-843
Number of pages8
JournalBritish Journal of Dermatology
Volume179
Issue number4
DOIs
Publication statusPublished - 2018 Oct 1

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Phase IV Clinical Trials
Actinic Keratosis
Scalp
Extremities
Gels
Research Personnel
Safety
Confidence Intervals
Cicatrix
Population
Therapeutics
Skin
Hyperpigmentation
Pigmentation
Quality of Life
3-ingenyl angelate
Pain

ASJC Scopus subject areas

  • Dermatology

Cite this

@article{5a3e26efdc8a43868450841741637a12,
title = "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)",
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.",
author = "Kim, {Y. C.} and Yang, {J. Y.} and Yoon, {J. S.} and Jo, {S. J.} and Ahn, {Hyo Hyun} and Song, {K. H.} and Lee, {D. Y.} and Chung, {K. Y.} and Won, {Y. H.} and Il-Hwan Kim",
year = "2018",
month = "10",
day = "1",
doi = "10.1111/bjd.16368",
language = "English",
volume = "179",
pages = "836--843",
journal = "British Journal of Dermatology",
issn = "0007-0963",
publisher = "Wiley-Blackwell",
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}

TY - JOUR

T1 - 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)

AU - Kim, Y. C.

AU - Yang, J. Y.

AU - Yoon, J. S.

AU - Jo, S. J.

AU - Ahn, Hyo Hyun

AU - Song, K. H.

AU - Lee, D. Y.

AU - Chung, K. Y.

AU - Won, Y. H.

AU - Kim, Il-Hwan

PY - 2018/10/1

Y1 - 2018/10/1

N2 - 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.

AB - 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.

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U2 - 10.1111/bjd.16368

DO - 10.1111/bjd.16368

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