Ongoing Clinical Trials for Hepatoblastoma
This article provides information about 3 ongoing clinical trials for hepatoblastoma, a rare form of liver cancer that primarily affects children. These trials are testing various chemotherapy combinations and new targeted therapies across multiple European countries, offering treatment options for patients at different stages of the disease.
Clinical trial locations
- Austria
- Belgium
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Czechia
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Denmark
- France
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Germany
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Greece
- Hungary
- Ireland
- Netherlands
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Norway
- Poland
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on Doxorubicin, Fluorouracil, and Oxaliplatin for Children and Adolescents with Primary Malignant Liver Cancer
- Spain
- Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
- Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
- Sweden
Study of drug combinations including cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin and sorafenib for children with hepatoblastoma or hepatocellular carcinoma
This trial focuses on testing various combinations of chemotherapy medications for children and young adults aged 30 years or younger with liver cancer. The study evaluates different treatment strategies depending on the risk level of each patient’s disease.
Who can participate: Children and young adults up to 30 years old with either a clinical or confirmed diagnosis of hepatoblastoma or hepatocellular carcinoma are eligible. Participants must have adequate heart function, normal blood counts including white blood cells above 0.75 x 10⁹/L and platelets above 75 x 10⁹/L, and good kidney function. Normal levels of potassium, magnesium, and calcium are required. Women of childbearing age must have a negative pregnancy test and agree to use effective contraception during the trial.
Who cannot participate: Patients under 6 months or over 30 years old at diagnosis, those who have already received chemotherapy for liver cancer, those with other types of cancer, and patients with severe heart or kidney problems are excluded. Pregnant or breastfeeding women, patients with uncontrolled infections, known allergies to study medications, or those unable to follow study procedures cannot join. Participation in other clinical trials within 30 days before enrollment is also not permitted.
What the trial involves: The study uses a risk-based approach, dividing patients into groups A through F based on disease stage. Treatment ranges from monitoring only for very low-risk patients to intensive chemotherapy combinations for high-risk cases. The medications being tested include cisplatin, carboplatin, doxorubicin, fluorouracil, vincristine, etoposide, irinotecan, gemcitabine, oxaliplatin, and sorafenib. Most treatments are given through intravenous infusion, though some may be taken orally. The trial monitors how well the cancer responds to treatment, tracks survival outcomes, and carefully watches for side effects, particularly concerning heart, kidney, and hearing function.
Study on the Safety and Effectiveness of Patritumab Deruxtecan for Children with Relapsed or Refractory Hepatoblastoma and Rhabdomyosarcoma
This trial investigates a new targeted therapy called patritumab deruxtecan for children whose liver cancer has returned after treatment or has not responded to previous therapies. The study also includes children with rhabdomyosarcoma, another type of cancer.
Who can participate: Children with confirmed advanced or metastatic hepatoblastoma or rhabdomyosarcoma who have experienced disease progression after at least one prior treatment are eligible. There must be no satisfactory alternative treatment options available. Patients must have recovered from previous treatment side effects to a mild level, though those with well-managed hormone-related side effects or moderate nerve damage may still qualify. Children with hepatitis B can participate if they are receiving antiviral therapy and have an undetectable viral load. Those with a history of hepatitis C infection are eligible if their viral load is undetectable.
Who cannot participate: Patients with cancer types not specified in the study, those who have had severe allergic reactions to similar medications, and patients with uncontrolled or serious medical conditions are excluded. Pregnant or breastfeeding patients, those participating in another clinical trial simultaneously, and patients who have not recovered from major surgery or significant injury cannot join. Active infections requiring treatment, history of drug or alcohol abuse that could affect study participation, and inability to comply with study procedures or follow-up visits are also grounds for exclusion.
What the trial involves: The study tests patritumab deruxtecan, an antibody-drug conjugate that targets a specific protein called HER3 on cancer cells. Once attached, it delivers a powerful anti-cancer agent directly into the cancer cell. The medication is given through intravenous infusion. The trial is conducted in two parts: the first focuses on determining the safest dose, while the second evaluates how well the treatment controls the cancer. Participants receive additional medications to manage symptoms and side effects, including dexamethasone and anti-nausea medications. Throughout the study, patients are closely monitored for side effects and cancer response through regular assessments, blood tests, and imaging scans.
Study on Doxorubicin, Fluorouracil, and Oxaliplatin for Children and Adolescents with Primary Malignant Liver Cancer
This Polish trial aims to improve treatment outcomes for children and adolescents with primary malignant liver cancer by tailoring therapy based on disease stage, reducing side effects while maintaining cure rates, and selecting optimal surgical approaches.
Who can participate: Children and adolescents under 30 years old with clinical signs of hepatoblastoma or hepatocellular carcinoma are eligible. Both males and females can participate. Patients must have a confirmed liver tumor and must sign informed consent to participate in the study, agreeing after being fully informed about the trial.
Who cannot participate: Patients with different types of cancer other than hepatoblastoma and hepatocellular carcinoma, those outside the specified age range, and patients unable to follow study procedures are excluded. Other medical conditions that might interfere with the study, pregnancy or breastfeeding, recent participation in another clinical trial, and allergies to study medications are also exclusion factors. Patients with a history of substance abuse, serious infections or illnesses that could affect results, or recent or planned surgery during the study period cannot participate.
What the trial involves: The study uses multiple chemotherapy medications including doxorubicin hydrochloride, fluorouracil, oxaliplatin, cisplatin, sorafenib tosilate, irinotecan hydrochloride, etoposide, gemcitabine hydrochloride, vinorelbine, carboplatin, and sodium thiosulfate. Most medications are administered intravenously, with the exception of sorafenib, which is taken orally. Patients are classified into treatment groups based on disease stage, and treatment is individualized accordingly. The study monitors event-free survival, overall survival, adherence to surgical guidelines, treatment toxicity, hearing loss, tumor response, and whether tumors can be surgically removed. Regular evaluations assess treatment effectiveness and side effects. Surgical intervention may be considered if the tumor becomes removable after chemotherapy. Follow-up care continues after treatment completion to monitor recovery and check for cancer recurrence.
Summary
Three clinical trials are currently recruiting patients with hepatoblastoma across Europe. The trials reflect different treatment approaches: risk-adapted chemotherapy for newly diagnosed patients, targeted therapy for relapsed or refractory disease, and tailored treatment strategies combining chemotherapy with surgical planning.
Two trials are available in multiple European countries, with the largest study spanning 10 countries including Czechia, Germany, Belgium, France, Austria, Norway, Spain, Poland, Netherlands, and Ireland. A second multi-country trial operates in Belgium, France, Germany, Czechia, Hungary, Denmark, Spain, Sweden, Netherlands, and Greece. One trial is specific to Poland. This wide geographical distribution provides access to experimental treatments for patients across Europe.
Common chemotherapy agents appear across multiple trials, including cisplatin, doxorubicin, fluorouracil, and oxaliplatin. The largest study offers the most comprehensive approach with risk-based treatment strategies ranging from observation only for very low-risk patients to intensive multi-drug combinations for high-risk disease. For patients whose cancer has returned or not responded to initial treatment, the patritumab deruxtecan trial offers a novel targeted therapy approach.
All trials emphasize careful monitoring of treatment effects and side effects, particularly concerning heart, kidney, and hearing function, which can be affected by these powerful medications. The studies aim to improve survival rates while minimizing treatment-related complications, representing ongoing efforts to advance care for this rare childhood cancer.
