PEP005 (ingenol mebutate) gel for the topical treatment of superficial basal cell carcinoma: results of a randomized phase IIa trial.

Abstract

OBJECTIVES:

To evaluate the safety of two applications of PEP005 (ingenol mebutate) gel in superficial basal cell carcinoma. Efficacy was a secondary end-point.

METHODS:

Randomized, vehicle-controlled, phase IIa study conducted at eight private dermatology clinics in Australia. A total of 60 patients with histologically confirmed superficial basal cell carcinoma (lesion size, 4-15 mm) were randomized to treatment on days 1 and 2 (Arm A) or days 1 and 8 (Arm B) and, within each arm, to ingenol mebutate gel, 0.0025%, 0.01% or 0.05%, or vehicle gel. The main outcome measures were the incidence and severity of adverse events and local skin responses in Arms A and B; lesion clearance at day 85 was a secondary measure.

RESULTS:

The incidence of adverse events was low. One patient treated with ingenol mebutate gel, 0.05% in Arm A experienced severe flaking/scaling/dryness extending beyond the application site. Non-severe, potentially treatment-related events included erythema extending beyond the application site, application-site pain and headache in two patients each. Six patients in Arm A had one or more severe local skin responses. Efficacy appeared to be dose-related and there was a trend towards higher clinical and histological lesion clearance rates in Arm A compared with Arm B. Histological clearance occurred in five of eight patients (63%) randomized to ingenol mebutate gel, 0.05% in Arm A.

CONCLUSIONS:

Two applications of ingenol mebutate gel, 0.05%, are safe and have efficacy in patients with superficial basal cell carcinoma.

• Authors: Greg Siller, Robert Rosen, Michael Freeman, Peter Welburn, Janelle Katsamas, Steven M Ogbourne
• Journal: The Australasian journal of dermatology (Australas J Dermatol) Vol. 51 Issue 2 Pg. 99-105 (May 2010) ISSN: 1440-0960 [Electronic] Australia
• PMID: 20546215 (Publication Type: Clinical Trial, Phase II, Journal Article, Randomized Controlled Trial, Research Support, Non-U.S. Gov't)

Chemical References

• 3-ingenyl angelate
• Antineoplastic Agents
• Diterpenes
• Gels

Topics

• Administration, Topical
• Adult
• Aged
• Aged, 80 and over
• Antineoplastic Agents (administration & dosage, adverse effects)
• Carcinoma, Basal Cell (drug therapy, pathology)
• Diterpenes (administration & dosage, adverse effects)
• Dose-Response Relationship, Drug
• Female
• Gels
• Headache (chemically induced)
• Humans
• Male
• Middle Aged
• Pain (chemically induced)
• Remission Induction
• Skin Diseases (chemically induced)
• Skin Neoplasms (drug therapy, pathology)
• Treatment Outcome