Wilms tumor (nephroblastoma) is the most common kidney cancer in children, typically diagnosed in those aged 3 to 5, and accounts for about 5% of all childhood cancers. Overall survival rates now exceed 90% for localized disease thanks to combined surgery, chemotherapy, and sometimes radiation — representing one of the great successes of pediatric oncology. Research now focuses on curing more children while reducing the long-term side effects of treatment.
What's actually going on in research
Surgery to remove the affected kidney, combined with vincristine and actinomycin D chemotherapy, is highly effective for standard-risk disease. Doxorubicin and radiation are added for higher-risk histology and stages. The Children's Oncology Group (COG) and SIOP international consortium continue to run trials to determine the minimum effective treatment for low-risk patients — to spare them late effects such as infertility, heart damage, and secondary cancers — while intensifying therapy for high-risk diffuse anaplastic and relapsed disease.
Treatment de-escalation
Trials are identifying low-risk patients — including very young children and those with small, favorable-histology tumors — who can be safely treated with less chemotherapy or radiation to preserve long-term health.
Diffuse anaplastic Wilms treatment
High-risk diffuse anaplastic Wilms tumor has poorer outcomes; trials are testing intensified chemotherapy regimens, targeted agents, and vincristine-irinotecan combinations to improve survival.
Relapsed Wilms tumor therapy
Novel approaches including anti-GD2 immunotherapy, antibody-drug conjugates, and AURKA inhibitors are in early trials for children with relapsed or refractory Wilms tumor who have exhausted standard options.
What to know before you search
Eligibility is based on tumor stage, histology (favorable vs anaplastic), patient age, and prior treatment — bilateral disease has separate trial pathways.
What types of trials are currently open
- Treatment reduction trials — Identifying low-risk patients who can safely receive less chemotherapy or avoid radiation without compromising cure.
- High-risk histology trials — Testing intensified regimens and targeted drugs for diffuse anaplastic and blastemal-predominant Wilms tumor.
- Relapse treatment trials — Evaluating immunotherapy, antibody-drug conjugates, and novel drugs for relapsed or refractory nephroblastoma.
- Bilateral Wilms trials — Studying nephron-sparing surgery and chemotherapy sequencing to preserve kidney function in bilateral disease.
- Long-term survivor studies — Tracking late effects of treatment including cardiac, fertility, and secondary cancer risks in childhood Wilms survivors.
Recently added Wilms Tumor trials
Selective Antigen Specific T Cells and CAR T Cells in Subjects With Relapsed/Refractory Embryonal Tumors (SABRE)
This is a phase I dose-escalation study to determine the safety and feasibility of autologous CAR-TA T cells (B7-H3 CAR+ T cells administered with DNR-PRAME Tumor Antigen-specific T cells) following lymphodepleting chemotherapy in participants with relapsed/refractory rhabdomyosarcoma, Ewing sarcoma, neuroblastoma and Wilms tumor. Patients will be enrolled to one of three planned dose levels with B7-H3 CAR T cell dose determined based on the percentage of B7-H3 transduced cells (B7-H3+ population of cells), and dTBRII-transduced PRAME TA-specific T cell dose based on the total cell population. Both doses will be based on the recipient's body weight. The safety of the CAR-TA T cell product will be evaluated and the maximum tolerated dose (MTD) will be determined. The safety endpoint will be assessed by monitoring for dose limiting toxicities for 28 days following CAR-TA T cell administration.
Immunotherapy for Solid Tumor Malignancies in Pediatrics Using Interleukin-15 and -21 Armored Glypican-3-specific Chimeric Antigen Receptor T Cells
This Phase 1, open-label, non-randomized study will enroll pediatric and young adult subjects with relapsed or refractory non-central nervous system (CNS) malignant solid tumors expressing glypican-3 (GPC3) to examine the safety, feasibility, and efficacy of administering T cell products derived from peripheral blood mononuclear cells (PBMC) that have been genetically modified to co-express a GPC3-specific chimeric antigen receptor (CAR), interleukin (IL)-15 and IL-21 as well as the inducible caspase 9 (iC9) suicide gene (SC-CAR.GPC3xIL15.21 T cells). A child or young adult meeting all eligibility criteria and meeting none of the exclusion criteria will have a blood sample collected, which will be used to bioengineer the CAR T cells targeting their tumor.
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