Background
Chemotherapy-induced peripheral neuropathy (CIPN) is the main dose-limiting factor for oxaliplatin, affecting quality-of-life and function. Severity of acute neurotoxicity (paraesthesias or dysesthesias) predicts CIPN. We evaluated whether ibudilast (an oral phosphodiesterase inhibitor) could reduce the severity of acute neurotoxicity and CIPN.
Methods
A randomised phase 2 placebo-controlled trial evaluating ibudilast 30mg PO bd, started 2 days before oxaliplatin, in participants treated with FOLFOX or CAPOX for early or metastatic CRC. Primary outcome: severity of acute neurotoxicity (Oxaliplatin Acute Symptom Questionnaire [OASQ] score, higher score worse). Key secondary outcomes: CIPN, adverse events. Comparisons are ibudilast vs placebo, with 2-sided p-values and 95% CI.
Results
We recruited 100 patients at 11 sites, November 2021 to July 2025: 51 assigned ibudilast, 49 placebo. Median age 62 (IQR 49-70); 57 early CRC (3-mo OX planned in 17, 6-mo in 40), 43 metastatic CRC.
Acute neurotoxicity: Mean OASQ score day 3 of week 4 CAPOX and week 5 FOLFOX: 7.1 vs 6.6, (difference 0.5, 95%CI -2.8, 3.9, p=0.8). Scores worsened over time but were similar between groups.
CIPN: FACT-GOG-Ntx-total, Ntx-subscale, Total Neuropathy Scores, Grooved Pegboard, worsened over time but were similar between groups. Ntx-subscale scores (lower score worse) 6-months after completing oxaliplatin were 32.4 vs 32.6; mixed model difference over time -0.06, p=0.966.
Mean number of oxaliplatin cycles: 7 in both adjuvant and metastatic settings. Median time to oxaliplatin discontinuation: ibudilast 3.7 vs placebo 4.8, p=0.36. Only 11% completed 3-months of oxaliplatin without dose modification: 3 (5.9%) vs 8 (16.3%).
Proportion of participants adhering to study treatment: ibudilast 68% vs placebo 73%. The frequency and severity of adverse events appeared similar between groups.
Conclusions
We found insufficient evidence to warrant further study of ibudilast for oxaliplatin-induced neuropathy. It was feasible to run a high-quality, multi-site, placebo-controlled, randomised trial with comprehensive assessment of neurotoxicity.