Aims:
SOR is the only approved agent in uHCC, necessitating new options. LEN, an inhibitor of VEGFR1‒3, FGFR1‒4, PDGFRα, RET, and KIT, showed activity in uHCC in a phase 2 trial. We report a phase 3 trial of LEN vs SOR as first-line uHCC treatment.
Methods:
In this randomized, open-label, noninferiority study, pts had uHCC, ≥1 measurable target lesion, BCLC stage B or C, Child-Pugh A, ECOG PS≤1, and no prior systemic therapy. Pts were randomized 1:1 to LEN (weight ≥60 kg: 12 mg/day; <60 kg: 8 mg/day) or SOR 400 mg twice daily. Primary endpoint was OS. OS HR and its 95% CI were estimated with a stratified Cox proportional hazard model. The predefined noninferiority margin was 1.08. Secondary efficacy endpoints were PFS, TTP, and ORR by mRECIST. Type I error rates for secondary efficacy endpoints were controlled with a fixed sequence procedure at 2-sided α=0.05 after claiming OS noninferiority.
Results:
954 Pts enrolled (LEN: 478; SOR: 476). Efficacy outcomes appear in the table. TEAE incidence was similar in both arms, with the most common being hypertension (42%), diarrhea (39%), decreased appetite (34%), decreased weight (31%), and fatigue (30%). Median (range) treatment duration was 5.7 mos (0−35.0) for LEN and 3.7 mos (0.1−38.7) for SOR. 13% Of LEN-treated and 9% of SOR-treated pts discontinued due to adverse events. 33% Of LEN-treated and 39% of SOR-treated pts received second-line therapy.
Conclusions:
LEN is noninferior in OS and achieves statistically significant and clinically meaningful improvements in PFS, TTP, and ORR as first-line therapy for uHCC. TEAEs were consistent with the known LEN safety profile.
|
LEN |
SOR |
HR (95% CI) |
Median OS, mos |
13.6 |
12.3 |
0.92 (0.79−1.06) |
Median PFS, mos* |
7.4 |
3.7 |
0.66 (0.57−0.77) |
Median TTP, mos* |
8.9 |
3.7 |
0.63 (0.53−0.73) |
ORR, n (%)* |
115 |
44 (9) |
|
*P<0.00001