title: 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).
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 = 0.0131). Iseganan was well tolerated; no systemic absorption was detected. Iseganan is safe and may be effective in reducing UOM and its clinical sequelae.
authors.affiliation: Department of Leukemia, The University of Texas, M. D. Anderson Cancer Center, 1515 Holcombe Boulevard, Unit 428, Houston, TX 77030, USA. frankgiles@aol.com
authors.forename: Francis J
authors.initials: FJ
authors.lastname: Giles
authors.forename: Carole B
authors.initials: CB
authors.lastname: Miller
authors.forename: David D
authors.initials: DD
authors.lastname: Hurd
authors.forename: John R
authors.initials: JR
authors.lastname: Wingard
authors.forename: Thomas R
authors.initials: TR
authors.lastname: Fleming
authors.forename: Stephen T
authors.initials: ST
authors.lastname: Sonis
authors.forename: Williamson Z
authors.initials: WZ
authors.lastname: Bradford
authors.forename: Janis G
authors.initials: JG
authors.lastname: Pulliam
authors.forename: Elias J
authors.initials: EJ
authors.lastname: Anaissie
authors.forename: Roy A
authors.initials: RA
authors.lastname: Beveridge
authors.forename: Mark M
authors.initials: MM
authors.lastname: Brunvand
authors.forename: Paul J
authors.initials: PJ
authors.lastname: Martin
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000890:Anti-Infective Agents / Q000627:therapeutic use*; D023181:Antimicrobial Cationic Peptides; D000970:Antineoplastic Agents / Q000009:adverse effects; D002648:Child; D004311:Double-Blind Method; D006801:Humans; D007390:International Agencies; D008875:Middle Aged; D009061:Mouth Mucosa / Q000187:drug effects; D009369:Neoplasms / Q000188:drug therapy; D010455:Peptides; D010919:Placebos; D011506:Proteins / Q000627:therapeutic use*; D013280:Stomatitis / Q000139:chemically induced / Q000209:etiology / Q000517:prevention & control*
chemical_list: D000890:Anti-Infective Agents; D023181:Antimicrobial Cationic Peptides; D000970:Antineoplastic Agents; D010455:Peptides; D010919:Placebos; D011506:Proteins; C119872:antimicrobial peptide IB-367