The following clinical trials represent a selection of key studies currently recruiting patients with primary liver cancers for inclusion in investigations of new therapies and new regimens of existing treatments for the disease. Each clinical trial description includes the NLM Identifier to be used as a reference with ClinicalTrials.gov. This information can help oncology practice managers and providers direct eligible patients to one of these trials.
1 Durvalumab plus Tremelimumab as First-Line Treatment for Advanced HCC
The purpose of this randomized, open-label, multicenter, global, phase 3 study is to assess the efficacy and safety of durvalumab (Imfinzi) plus tremelimumab (Imjudo) versus durvalumab monotherapy versus standard-of-care sorafenib (Nexavar) as first-line treatment for patients with advanced hepatocellular carcinoma (HCC). Patients aged ≥18 years with histopathologic confirmation of HCC, who have had no previous systemic therapy for HCC, who have Barcelona Clinic Liver Cancer (BCLC) stage B disease that is not eligible for locoregional therapy or stage C disease, who have Child-Pugh Score class A, and who have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 at enrollment may be eligible if other criteria are met. Eligible patients will be randomized to receive durvalumab alone; durvalumab plus tremelimumab in either of 2 regimens; or sorafenib alone.
The primary outcome measure is overall survival (OS) from the date of randomization until death due to any cause, assessed up to 4 years. Secondary outcome measures include time to objective tumor progression, progression-free survival (PFS), objective response rate (ORR), disease control rate (DCR), and duration of response (DOR), assessed up to 4 years. Other outcome measures include adverse events (AEs) from time of informed consent, throughout the treatment period, and up to the follow-up period of 4 years. The study plans to enroll 1504 participants throughout the United States and worldwide. For more information, contact the AstraZeneca Clinical Study Information Center at 1-877-240-9479 or
2 Lenvatinib plus Pembrolizumab in Combination with TACE in Incurable Nonmetastatic HCC
The purpose of this randomized, multicenter, double-blind, active-controlled, phase 3 study is to evaluate the safety and efficacy of lenvatinib (Lenvima) plus pembrolizumab (Keytruda) in combination with transarterial chemoembolization (TACE) versus TACE alone in the treatment of patients with incurable nonmetastatic HCC. Patients aged ≥18 years with a confirmed diagnosis of HCC that has localized to the liver and is not amenable to curative treatment, who have adequately controlled blood pressure with or without antihypertensive medications, and who have completed treatment for hepatitis C virus at least 1 month prior to the study start may be eligible if other criteria are met. Eligible patients will be randomized to receive lenvatinib 12 mg (for screening body weight ≥60 kg) or 8 mg (for screening body weight <60 kg) orally (PO) once daily for each 21-day cycle, plus intravenous (IV) pembrolizumab 400 mg once every 6 weeks and TACE for up to 2 years; or oral placebo once daily for each 21-day cycle plus IV placebo once every 6 weeks and TACE for up to 2 years.
The primary outcome measures include PFS per RECIST version 1.1 as assessed by blinded independent central review for up to 43 months, and OS from the time of randomization to death due to any cause for up to 95 months. Secondary outcome measures include PFS, ORR, DCR, DOR, and time to progression per RECIST version 1.1 and per modified RECIST as assessed by blinded independent central review; the percentage of participants who have at least 1 AE and at least 1 serious AE; the percentage of participants experiencing at least 1 hepatic event of clinical interest not due to disease progression or TACE; and the percentage of participants who discontinue the study drug as a result of an AE. The study plans to enroll 450 participants throughout the United States and worldwide. For more information, contact the recruiting sites director or Merck Sharp & Dohme at 1-888-577-8839 or
3 Futibatinib versus Gemcitabine plus Cisplatin as First-Line Treatment for Advanced CCA with FGFR2 Rearrangements
The purpose of this randomized, open-label, parallel 2-arm, phase 3 study is to evaluate the efficacy and safety of futibatinib (Lytgobi) versus gemcitabine plus cisplatin as first-line treatment for patients with advanced, metastatic, or recurrent unresectable intrahepatic cholangiocarcinoma (CCA) harboring FGFR2 gene rearrangements. Patients aged ≥18 years with histologically confirmed, locally advanced, metastatic, or recurrent unresectable intrahepatic CCA with FGFR2 rearrangements, who have radiographically measurable disease per RECIST version 1.1, who have received treatment for locally advanced disease with evidence of radiographic progression with measurable disease outside the previously treated lesions, and who have an ECOG performance status of 0 or 1 may be eligible if other criteria are met. Eligible patients will be randomized to receive futibatinib PO daily for each 21-day cycle, or cisplatin 25 mg/m2 IV plus gemcitabine 1000 mg/m2 IV on days 1 and 8 of each 21-day cycle.
The primary outcome measure is PFS from the date of randomization to the date of documented disease progression by independent central review per RECIST version 1.1 or the date of death, whichever comes first. Secondary outcome measures include ORR, DCR, OS, PFS per investigator assessment, and the safety and tolerability of treatment-emergent AEs. The study plans to enroll 216 participants throughout the United States and worldwide. For more information, contact Karim Benhadji, MD, at 1-609-250-7336, or e-mail
4 Pemigatinib versus Chemotherapy in Unresectable or Metastatic CCA with FGFR2 Rearrangements
The purpose of this randomized, open-label, multicenter, phase 3 study is to evaluate the efficacy and safety of pemigatinib (Pemazyre) versus gemcitabine plus cisplatin as first-line treatment for patients with unresectable or metastatic CCA with FGFR2 rearrangements. Patients aged ≥18 years with histologically or cytologically confirmed CCA that is previously untreated and considered unresectable and/or metastatic with documented FGFR2 rearrangements, who have radiographically measurable or evaluable disease per RECIST version 1.1, and who have an ECOG performance status of 0 or 1 may be eligible if other criteria are met. Eligible patients will be randomized to receive pemigatinib PO once daily for each 3-week cycle, or gemcitabine 1000 mg/m2 IV plus cisplatin 25 mg/m2 IV on days 1 and 8 of every 3-week cycle for up to 8 cycles.
The primary outcome measure is PFS per RECIST version 1.1 and assessed by an independent central review for up to 12 months. Secondary outcome measures include ORR, OS, DOR, DCR, the number of treatment-emergent AEs, and quality-of-life impact as assessed by the European Organisation for Research and Treatment of Cancer questionnaires. The study plans to enroll 434 participants throughout the United States and worldwide. For more information, contact Incyte Corporation at 1-855-463-3463 or
5 Durvalumab plus Tremelimumab with or without Lenvatinib in Combination with TACE in Locoregional HCC
The purpose of this randomized, open-label, multicenter, phase 3 trial is to assess the efficacy and safety of durvalumab (Imfinzi) plus tremelimumab (Imjudo) plus TACE with or without lenvatinib (Lenvima) versus TACE alone in the treatment of patients with locoregional HCC. Patients aged ≥18 years with no evidence of extrahepatic disease; whose disease is not amenable to curative surgery, transplantation, or curative ablation, but is amenable to TACE; who have Child-Pugh score class A; who have measurable disease per modified RECIST criteria; and who have an ECOG performance status of 0 or 1 at enrollment may be eligible if other criteria are met. Eligible patients will be randomized to receive either tremelimumab IV, durvalumab IV, lenvatinib PO, and TACE (arm A); tremelimumab IV, durvalumab IV, and TACE (arm B); or TACE alone (arm C).
The primary outcome measure is PFS from time of randomization until disease progression per RECIST version 1.1 or death due to any cause in arm A versus arm C. Secondary outcome measures include PFS in arm B versus arm C, OS in arm A versus arm C, and OS in arm B versus arm C. The study plans to enroll 525 participants throughout the United States and worldwide. For more information, contact the AstraZeneca Clinical Study Information Center at 1-877-240-9479 or
6 Cisplatin and Combination Chemotherapy in Children and Young Adults with Hepatoblastoma or Liver Cancer After Surgery
The purpose of this randomized, phase 2/3 trial is to evaluate the efficacy of combining surgery, cisplatin, and combination chemotherapy in the treatment of children and young adults with hepatoblastoma or HCC. Patients aged <30 years with newly diagnosed, histologically proven primary pediatric hepatic malignancies; who have an ECOG performance status of 0-2; who may have had surgical resection of the hepatic malignancy prior to enrollment; and who have not received other anticancer therapy for their current liver lesion may be eligible if other criteria are met. Eligible patients will be randomized into 15 groups that include watchful waiting, cisplatin IV with or without surgery, cisplatin IV with or without combination chemotherapy with or without surgery, and cisplatin IV with chemotherapy and sorafenib (Nexavar) in various combinations with or without surgery.
The primary outcome measures include event-free survival from time of randomization to the first failure event, defined as progression of existing disease or occurrence of disease at new sites, death due to any cause before disease progression, or diagnosis of malignant neoplasm; the percentage of patients with resectable tumors in the group assigned to receive cisplatin IV on day 1 every 14 days for up to 6 cycles; and the response rate of patients in the group assigned to receive cisplatin, chemotherapy, and sorafenib. Other outcome measures include failure-free survival; OS; the percentage of patients who have grade ≥3 AEs; the percentage of patients with chemotherapy-related cardiac, nephro-, and ototoxicity; and the percentage of patients whose disease is surgically resectable. The study plans to enroll 537 participants throughout the United States and worldwide. For more information, contact the study sites directly. The NLM identifier is NCT03533582.
7 Cabozantinib plus Atezolizumab versus Sorafenib in Patients with Advanced HCC Who Have Not Received Previous Systemic Therapy
The purpose of this randomized, open-label, multicenter, controlled, phase 3 study is to evaluate the safety and efficacy of cabozantinib (Cabometyx) plus atezolizumab (Tecentriq) versus the standard-of-care sorafenib (Nexavar) in patients with advanced HCC who have not received previous systemic anticancer therapy. Patients aged ≥18 years with a histologic, cytologic, or clinical diagnosis of HCC whose disease is not amenable to curative treatment or locoregional therapy, who have measurable disease per RECIST version 1.1, who have BCLC stage B or C disease, who have Child-Pugh score class A, and who have an ECOG performance status of 0 or 1 may be eligible if other criteria are met. Eligible patients will be randomized to receive cabozantinib 40 mg PO once daily plus atezolizumab 1200 mg IV once every 3 weeks (experimental arm); sorafenib 400 mg PO twice daily (control arm); or cabozantinib 60 mg PO once daily (single-agent arm).
The primary outcome measures include the duration of PFS and duration of OS in the experimental arm versus the control arm. The secondary outcome measure is the duration of PFS in the single-agent arm versus the control arm. The study plans to enroll 740 participants throughout the United States and worldwide. For more information, contact Exelixis Clinical Trials at 1-888-393-5494 or