A Phase III, Randomized, Double-blind, Placebo-controlled, Multinational Trial of Iseganan for the Prevention of Oral Mucositis in Patients Receiving Stomatotoxic Chemotherapy (PROMPT-CT Trial)

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Abstract
Microfloral invasion and colonization of oral cavity mucosal tissues contribute to the pathophysiology of ulcerative oral mucositis (UOM). Iseganan is an analog of Protegrin-1, a naturally occurring peptide with broad-spectrum microbicidal activity. A randomized, double-blind, placebo-controlled study was conducted to evaluate iseganan in preventing UOM after stomatotoxic therapy. Patients received an oral rinse of iseganan 9 mg or placebo, swished/swallowed 6 times daily, starting with stomatotoxic therapy and continuing for 21–28 days. One hundred sixty three and 160 patients, respectively, were randomized to receive iseganan or placebo. One hundred and two patients (32%) were affected by a drug dispensing error, caused by a flawed computerized allocation system. Among all 323 patients, analyzed according to randomization assignment, 43% and 33% of iseganan and placebo patients, respectively, did not develop UOM (P=0.067). On an 11-point scale, iseganan patients experienced less mouth pain (3.0 and 3.8 {P=0.041}), throat pain (3.8 and 4.6 {P=0.048}), and difficulty swallowing (3.9 and 4.7 {P=0.074}), compared to placebo patients. On the 5-point NCI CTC scale, iseganan patients experienced lower stomatitis scores (1.6 and 2.0 {P=0.013}). Iseganan was well tolerated; no systemic absorption was detected. Iseganan is safe and may be effective in reducing UOM and its clinical sequelae.