CuraGen has reported preliminary top-line results from its Phase II randomized,
placebo-controlled clinical trial evaluating a single-dose of velafermin for
the prevention of oral mucositis in patients receiving high-dose chemotherapy,
with or without total-body irradiation, prior to autologous bone marrow transplantation.
Analysis of this dose-ranging study showed a reduction in oral mucositis incidence
in one of the three velafermin dose arms compared to placebo, although the primary
endpoint, as evaluated by a predefined dose trend analysis did not reach statistical
The trial was a randomized, double-blind, placebo-controlled trial evaluating a single administration of either placebo or one of three doses of velafermin given to patients one day after bone marrow transplantation. The primary analysis of the study examined a linear trend in dose across the 0.03 mg/kg, 0.1 mg/kg, and 0.2 mg/kg velafermin arms compared to placebo. An analysis of each individual dose group versus placebo was also conducted, as well as the evaluation of secondary endpoints including duration of Grade 3 and 4 oral mucositis, use of narcotic pain medications, time to neutrophil engraftment, and the incidence and duration of diarrhea.