Phase II, Randomized, Double-Blind, Placebo-Controlled Study of Recombinant Human Intestinal Trefoil Factor Oral Spray for Prevention of Oral Mucositis in Patients With Colorectal Cancer Who Are Receiving Fluorouracil-Based Chemotherapy

Purpose This study evaluated the safety and efficacy of recombinant human intestinal trefoil factor (rhITF) administered as topical oral spray for prevention and treatment of chemotherapy-induced oral mucositis (OM). Patients and Methods Ninety-nine patients with colorectal cancer who had moderate to severe OM (WHO grade ≥ 2) in the first cycle of chemotherapy were randomly assigned to receive either placebo, rhITF 10 mg/mL (ie, low dose), or rhITF 80 mg/mL (ie, high dose) by oral spray (300 μL, eight times each day) for 14 consecutive days in the second chemotherapy cycle. Patients were assessed on days 1, 3, 5, 7, 10, 12, 14, and 21 (± 2 days for the last assessment) for safety and for OM incidence and severity. Results Treatment of patients at high risk for developing OM with low- or high-dose rhITF significantly reduced the amount of incidence (75% to 81%; low-dose rhITF P < .001; high-dose rhITF P = .002). Frequencies of WHO grade ≥ 2 OM in the placebo, low-dose rhITF, and high-dose rhITF groups were...