Results from a randomized phase II study to evaluate the safety and efficacy of acetyl-L-carnitine in the prevention of sagopilone-induced peripheral neuropathy (REASON).

9116 Background: Sagopilone, a novel, fully synthetic epothilone, has shown significant activity in a broad range of preclinical tumor models and in phase II studies in ovarian cancer (OC) and castrate-refractory prostate cancer (CRPC). Peripheral neuropathy (PN) is the most clinically relevant toxicity of sagopilone. Several studies have suggested that acetyl-L-carnitine (ALC) may prevent chemotherapy-induced PN. The REASON study is the first randomized, prospective, placebo-controlled study to investigate the safety, efficacy, and pharmacokinetics of sagopilone plus ALC for the prevention of sagopilone-induced PN. METHODS Patients (pts) with advanced refractory or relapsed OC or CRPC and no evidence of neuropathy received sagopilone (16 mg/m2 i.v. over 3 h every 3 wk) with either ALC (1,000 mg p.o. 3 times a day) or placebo (PBO) for ≤6 cycles until 1 month after the last sagopilone infusion. Primary endpoint was the incidence of PN determined by pt and investigator assessment; electromyography was performed if grade 2 PN occurred. RESULTS From August 2008, 150 pts were enrolled over 11 months (98 with OC, 52 with PC; 75 pts ALC, 75 pts PBO). Evaluable pts received ≥1 treatment cycle (n=121). Median duration of treatment (4 cycles, ALC; 5 cycles, PBO) and overall incidence of PN were similar between treatment groups. Incidence of high-grade PN was lower in the ALC group in both tumor types and statistically lower in OC pts (p=0.04). PN onset occurred within the first treatment cycle for 60% of ALC and 52% of PBO pts, with a similar median duration (grades 1-4 PN: 184 days, ALC; 191 days, PBO). There was no notable difference in discontinuations due to adverse events between the 2 groups (20 ALC, 23 PBO). CONCLUSIONS No significant difference in overall PN incidence was observed between treatment groups. However, in OC pts the incidence of high-grade PN was significantly lower in the ALC group compared with the PBO group. [Table: see text].