A double-blind study of topically applied 5% amlexanox in the treatment of aphthous ulcers.

A double-blind trial of amlexanox (C16H14N2O4) was carried out in 32 patients with recurrent oral aphthous ulcerations. During the treatment period, which lasted for 3 days, patients received either placebo topical paste or 5% amlexanox paste. The paste was applied by the investigator twice per day for 3 days and once on the fourth day. Efficacy was assessed by the following parameters: 1) pain measured by the patients marking a 15-cm line between poles connoting no pain versus severe pain; 2) erythema evaluated by the investigator on a four-point scale ranging from none to strong; 3) size determined by investigator measurement of the perpendicular dimensions of the ulcer; and 4) an investigator's improvement scale consisting of six rank-ordered points from -1 for worsening of the ulcer with respect to previously described criteria to +4 when the ulcer had healed completely. All evaluations were based on a comparison with the day 1 visit of the patient. Outcomes for patients receiving the active ingredient were superior on all four criteria of effectiveness. Group differences for all criteria but pain reduction were statistically significant (P < .05). No side effects were reported. It was concluded that amlexanox is effective in reducing aphthous ulcer erythema, pain, and lesional size.