Trial results for a Phase 2 study investigating pembrolizumab (Keytruda®) in hepatocellular carcinoma were posted on ClinicalTrials.gov on 2025-07-15. The study found statistically significant correlations between hepatocellular carcinoma (HCC) stiffness and overall survival (p=0.008), time to disease progression (p=0.009), and the number of intratumoral CD3+ T lymphocytes (p=0.01).
Background
Pembrolizumab (Keytruda®) was investigated as a second-line therapy for subjects with hepatocellular carcinoma (HCC) who either progressed on or after sorafenib or did not tolerate sorafenib.
Trial design
The study (NCT02940496) was a Phase 2 trial that enrolled 15 participants. It investigated pembrolizumab as a single agent in patients with BCLC Stage B Hepatocellular Carcinoma, BCLC Stage C Hepatocellular Carcinoma, Hepatitis C Infection, Refractory Liver Carcinoma, and Stage III Hepatocellular Carcinoma AJCC v7. Interventions included elbasvir/grazoprevir, pembrolizumab, and ribavirin.
Key results
The trial results posted for the ARM A Pembrolizumab (Q3W) group included measurements for HCC stiffness and its correlations:
- For the outcome "Correlation of HCC Stiffness to Overall Survival," the mean HCC stiffness was 0.12 kilopascals. A 2-sided t-test showed a correlation coefficient of 0.081 with a p-value of 0.008.
- For the outcome "Correlation of HCC Stiffness to Time To Disease Progression (TTP)," the mean HCC stiffness was 0.12 kilopascals. A 2-sided t-test showed a correlation coefficient of 0.88 with a p-value of 0.009.
- For the outcome "Correlation of HCC Stiffness to the Number of Intratumoral CD3+ T Lymphocytes," the mean HCC stiffness was 5.01 kilopascals (Standard Deviation: 1.91). A 2-sided t-test showed a correlation coefficient of 0.79 with a p-value of 0.01.
What this means
The statistically significant correlations observed in this Phase 2 trial suggest that HCC stiffness may serve as a relevant factor in predicting overall survival and time to disease progression in patients receiving pembrolizumab. The correlation with intratumoral CD3+ T lymphocytes also indicates a potential link between tumor microenvironment characteristics and treatment response or disease progression. These findings warrant further investigation into the role of HCC stiffness as a prognostic or predictive biomarker in hepatocellular carcinoma.
Source
The information regarding these trial results was obtained from ClinicalTrials.gov, a public database of clinical studies. The results for study NCT02940496, titled "Pembrolizumab (MK-3475) in Hepatocellular Carcinoma," were posted on 2025-07-15 on clinicaltrials.gov.