The purpose of this clinical trial is to evaluate the efficacy and safety of regorafenib as a subsequent therapy for patients with hepatocellular carcinoma (HCC) who have failed prior lenvatinib treatment. This investigational study aims to assess the therapeutic benefits and safety profile of regorafenib in patients whose disease has progressed following the use of lenvatinib, a targeted therapy for hepatocellular carcinoma
Lenvatinib is currently recognized as a standard first-line treatment for advanced hepatocellular carcinoma (HCC), having demonstrated non-inferiority to sorafenib in a phase 3 randomized clinical trial. It is also utilized as a second-line option following the failure of immunotherapy-based regimens. However, unlike sorafenib, there is a lack of prospective data regarding subsequent therapies following lenvatinib failure, which poses significant challenges in clinical decision-making. Regorafenib has demonstrated clinical efficacy and significant survival benefits compared to placebo in the RESORCE trial as a second-line treatment after sorafenib for Child-Pugh class A patients. Nevertheless, evidence supporting its use specifically in patients who have failed lenvatinib remains insufficient. This multicenter, single-arm, phase 2 study is designed to evaluate the efficacy and safety of regorafenib as a subsequent therapy for patients with unresectable HCC who have experienced disease progression or unacceptable toxicity during prior treatment with lenvatinib. Enrolled participants will receive regorafenib orally. The starting dose for Cycle 1 will be determined by the baseline Child-Pugh classification (160 mg once daily for Child-Pugh A; 120 mg once daily for Child-Pugh B). From Cycle 2 onwards, patients will maintain a standard regimen of 3 weeks on and 1 week off at the maximum tolerated dose. Treatment will continue until disease progression, unacceptable toxicity, withdrawal of consent, or study termination. The study will assess key efficacy endpoints including progression-free survival (PFS), overall survival (OS), objective response rate (ORR), and disease control rate (DCR), alongside comprehensive safety evaluations.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
"Participants with unresectable hepatocellular carcinoma who have failed prior lenvatinib treatment will receive oral regorafenib. The starting dose for Cycle 1 is 160 mg once daily for patients with Child-Pugh class A, and 120 mg once daily for patients with Child-Pugh class B. From Cycle 2 onwards, regorafenib will be administered at the maximum tolerated dose on a schedule of 3 weeks on and 1 week off (28-day cycle). Treatment continues until disease progression, unacceptable toxicity, withdrawal of consent, or study termination.
Asan Medical Center
Seoul, South Korea
progression free survival
Tumor assessments every 12 weeks (±7 days) after treatment discontinuation without documented progression until progression, death, or end of follow-up, whichever occurs first.
Time frame: From date of first dose until the date of first documented disease progression or death from any cause, whichever occurs first, assessed up to 36 months.
Overall survival
Survival follow-up assessments are conducted every 12 weeks (±7 days) after treatment discontinuation due to disease progression or withdrawal until death or end of follow-up.
Time frame: From date of first dose until the date of first documented disease progression or death from any cause, whichever occurs first, assessed up to 36 months.
time to progression
Baseline and scheduled tumor imaging assessments every 12 weeks (±7 days) thereafter (date of documented PD per RECIST v1.1).
Time frame: From treatment initiation until first documented progression or start of subsequent systemic anticancer therapy, assessed up to 36 months.
objective response rate
Assessment of Best Overall Response (CR/PR) at scheduled tumor imaging time points (every 12 weeks ±7 days) from treatment initiation until first PD or start of subsequent systemic anticancer therapy.
Time frame: From treatment initiation until first documented progression or start of subsequent systemic anticancer therapy, assessed up to 36 months.
disease control rate
Assessment of Best Overall Response (CR/PR/SD) at scheduled tumor imaging time points (every 12 weeks ±7 days) from treatment initiation until first PD or start of subsequent systemic anticancer therapy.
Time frame: From treatment initiation until first documented progression or start of subsequent systemic anticancer therapy, assessed up to 36 months.
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Masking
NONE
Enrollment
24
Number of Participants with Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Adverse events will be graded according to the NCI CTCAE version 5.0.
Time frame: From first dose of study drug until 30 days after last dose, assessed up to 24 months.