Giles, F.J., Rodriguez, R., Weisdorf, D., Wingard, J.R., Martin, P.J., Fleming, T.R., et al. (2004). A phase III, randomized, double-blind, placebo-controlled study of iseganan for the reduction of stomatitis in patients receiving stomatotoxic chemotherapy. Leukemia Research, 28(6), 559–565.

3 ml of iseganan (9 mg) oral solution (n = 251) or placebo, administered as oral rinse six times daily; instructed to rinse mouth with water before each dose, swish study drug to coat all surfaces, gargle and retain for 2 mins, and swallow. (Expectorate if unable to swallow.)

Started study day 1 – within 1 day of chemo or TBI  for a minimum of 10 days, adm for 21 days unless neutrophil recovery with absence of OM.

Follow-up assessment 14 days after adm of study drug disc.
 

The study was comprised of 502 patients, with 251 blinded in each arm. The median age was 48/46.

> 7 yrs and scheduled to receive cytotoxic regimen associated with a >50% incidence of NCI CTC grade > 2 mucositis.
Randomized (stratified by study center and by stomatotoxic trt) strategy 1–non-ablative chemo, 2–ablative cytotoxic therapy followed by auto-SCT, or 3–ablative cytotoxic therapy followed by allogeneic bone marrow or peripheral blood SCT.
 

28 centers in the United States

Nov 2001 – June 2002

Multi site N = 502 large RCT

NCI CTC stomatitis grades, incidence of UOM in eight sites and opioid analgesic use.
Mouth pain and difficulty swallowing was assessed by use of questionnaires.
 

43% trt and 37% placebo patients did not have peak stomatitis, grade = 2, p = 0.182.

No significant difference in severity, incidence, peak mouth pain, peak difficulty swallowing, amountt of opiate, or adverse event type or incidence.
 

Iseganan–did not positively affect the severity of stomatitis or the rate of ulcerative oral mucositis.

H R. Redman and H Fuchs employed by IntraBiotics – probable supporter of study, as articulated in Trotti study.

Findings add to conflicting results in literature regarding efficacy impact of local antimicrobial trt as strategy to reduce severity of stomatitis or UOM.