NALIRICC

Regimen

Experimental
Nanoliposomal irinotecan (nal-IRI) 70 mg/m2 (free base) d1 + leucovorin 400 mg/m2 + 5-FU 2400 mg/m2 46h infusion, q2w
Control
Leucovorin 400 mg/m2 + 5-FU 2400 mg/m2 q2w

Population

Advanced BTC (cholangiocarcinoma and gallbladder carcinoma) previously treated with gemcitabine-based therapy; ICC ~55%, ECC ~20%, GBC ~25%; Germany multicenter (AIO cooperative group); predominantly Caucasian patients

Key finding

NALIRICC failed to meet its primary OS endpoint (HR 0.68, p=0.074 — borderline but below significance threshold). This is a phase 2 trial with only 100 patients and limited statistical power; it is arguably underpowered to detect a modest OS difference reliably. The HR of 0.68 is numerically similar to ABC-06 FOLFOX effect, but given the 5FU/LV-only comparator (not FOLFOX), and the small sample size, the trial is inconclusive rather than definitively negative. The divergent results between NIFTY (positive PFS) and NALIRICC (negative OS) are best explained by: (1) different primary endpoints, (2) different populations (Korean vs German), and (3) statistical power differences (N=174 vs N=100).

Source: PMID 38870977

Timeline

    Guideline citations

    • NCCN BTC (p.6)