Trial Information

Lenalidomide as Second-line Treatment for Advanced Hepatocellular Carcinoma (HCC): a Phase II Clinical Trial

This is a single-arm, open-label phase II trial. Eligible patients must have histological or
clinical diagnosis of HCC, advanced tumors that are not amenable to loco-regional therapy,
documented progression with or intolerance to sorafenib-based treatment or other
anti-angiogenic therapy as first-line therapy for advanced HCC, ECOG performance status 0 or
1, Child-Pugh class A liver function, and measurable tumors (by RECIST 1.1). All enrolled
patients will receive lenalidomide, starting at 25 mg orally daily on days 1-21, every 4
weeks. Lenalidomide treatment will continue until objective disease progression, development
of unacceptable toxicity, or voluntary discontinuation. Dose titration will be done
according to the severity of adverse events. Tumor assessment will be done after 4 weeks and
8 weeks of treatment and every 8 weeks thereafter until objective disease progression. All
patients will receive DCE-MRI at baseline, on day 3 ± 1 day, and on day 14 ± 2 days. The
primary endpoint is the percentage of patients who are tumor progression-free (according to
RECIST 1.1) at 6 months after lenalidomide treatment. It is estimated that in the 2nd-line
setting, 20% or less patients will remain progression-free at 6 months with current
treatment, i.e., no standard treatment. Lenalidomide will be considered effective if the
percentage of patients who remain progression-free at 6 months can be increased to 40%. With
type I and type II errors of 0.05 and 0.1, respectively, 50 evaluable patients, i.e.,
patients who receive at least 4 weeks of study medication and receive the first scheduled
assessment of tumor response, will be required and the planned sample size will be 55
patients, assuming a 10% dropout rate. The study is expected to complete in 2 years.