Table of Contents
- Overview of the trials
- Trials in advanced liver cancer
- Trials in children and young adults
- Trials in other cancers
- Main endpoints studied
- Who may be able to join
- Study phases and what they mean
Overview of the trials
These studies look at Sorafenib as part of cancer treatment research, mainly in liver cancer and in some other cancer types.[1] The trials test Sorafenib alone as a standard treatment comparator, or together with other drugs, to see which approach gives better results.[1][2]
Most of the trials are interventional, which means researchers give a treatment and then measure the results.[1] The studies include both large Phase 3 trials and smaller Phase 1 or Phase 2 trials.[1][2]
Trials in advanced liver cancer
Several trials focus on hepatocellular carcinoma (HCC), which is the most common type of liver cancer.[1][2][4][6] Some studies include people with advanced HCC who have not had prior systemic therapy, meaning they have not yet received treatment that works throughout the body.[1][2][4]
In NCT04039607, the main question is whether nivolumab plus ipilimumab improves overall survival compared with standard care, which includes Sorafenib or lenvatinib.[1] In NCT03298451, researchers compare durvalumab alone or with tremelimumab against Sorafenib in people with unresectable advanced HCC.[2]
NCT04039607 has 763 participants and NCT03298451 has 1604 participants, so these are large studies designed to compare treatments in many people.[1][2] Another Phase 3 study, 2024-516479-34-00, compares cabozantinib plus atezolizumab with Sorafenib in advanced HCC and measures progression-free survival and overall survival.[4]
Study 2022-502948-13-00 looks at locally advanced or metastatic HCC in Child-Pugh A patients, which means the liver is still working well enough to meet the study rules.[6] This Phase 2 study tests whether livmoniplimab plus budigalimab can produce a confirmed complete or partial response before the next cancer treatment starts.[6]
Trials in children and young adults
The Paediatric Hepatic International Tumour Trial, NCT03017326, includes children and young people with hepatoblastoma and HCC.[5] Sorafenib appears in one treatment group for unresected HCC, where the study asks whether adding gemcitabine, oxaliplatin, and Sorafenib to cisplatin and doxorubicin improves outcomes.[5]
This is a Phase 3 study with 449 participants and several risk groups, including very low-risk, low-risk, intermediate-risk, high-risk, resected HCC, and unresected HCC.[5] The study also collects samples for biological and toxicity studies in some groups, which helps researchers learn more about the disease and treatment effects.[5]
Trials in other cancers
Not all Sorafenib studies are in liver cancer. Trial 2024-512887-77-00 studies advanced-stage malignancies with BRAF mutations, which are changes in a gene that can affect how cancer grows.[3] This Phase 1 study tests Sorafenib with trametinib to find the maximum tolerated dose and the recommended phase II dose.[3]
Trial 2026-525223-24-00 studies pediatric and young adult patients with high-risk osteosarcoma, including relapsed or refractory disease.[7] It compares mifamurtide with standard treatment containing Sorafenib and measures event-free survival, meaning the time until the first bad event such as death, progression, or relapse.[7]
One completed Phase 2 trial, 2023-509092-16-00, included older patients with acute myeloid leukemia, myelodysplastic syndrome, and chronic myelomonocyte leukemia.[5] In that study, Sorafenib was part of a precision therapy strategy, and the main outcome was cost-effectiveness measured through quality-adjusted life-years, or QALYs, which combine length of life and quality of life.[5]
Main endpoints studied
The most common endpoint is overall survival, which means how long people live after randomization, no matter what causes death.[1][2][4]
Other important endpoints include progression-free survival, which measures how long the cancer stays from getting worse, and event-free survival, which measures the time until a first major event such as progression, relapse, or death.[4][5][7]
Some studies measure tumor response using RECIST 1.1, a standard system for checking whether a tumor shrinks, stays stable, or grows.[6] Other studies look at response rates, maximum tolerated dose, recommended phase II dose, toxicity samples, or cost-effectiveness.[3][5]
Who may be able to join
Eligibility changes from study to study, but the trials here mainly include people with advanced HCC who have not had prior systemic therapy, people with unresectable liver cancer, children and young adults with hepatoblastoma or HCC, and patients with relapsed or refractory osteosarcoma.[1][2][5][6][7]
One study also includes older patients with AML, MDS, or CMML who are considered unfit for standard treatment.[5] Another requires Child-Pugh A liver function, which means the liver is still functioning well enough for the study rules.[6]
Study phases and what they mean
Phase 3 trials are the largest studies in this set and are usually designed to compare one treatment with another and confirm whether it works better or is safer.[1][2][4][5]
Phase 2 trials are smaller and often focus on how well a treatment works, which dose may be best, or whether a treatment strategy is worth testing further.[5][6][7]
Phase 1 trials are the earliest studies here and mainly look at safety and dose finding, such as the maximum tolerated dose and the dose recommended for later testing.[3]
