Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors

Hepatocellular Carcinoma Clinical Trial

Official title: Phase 2 Trial of XL184 (Cabozantinib) an Oral Small-Molecule Inhibitor of Multiple Kinases, in Children and Young Adults With Refractory Sarcomas, Wilms Tumor, and Other Rare Tumors

Status Active, not recruiting

Clinical Trial Summary

This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed. Cabozantinib-s-malate may stop the growth of tumor cells by blocking some of the enzymes needed for tumor growth and tumor blood vessel growth.

Clinical Trial Description

PRIMARY OBJECTIVES:

I. To determine the objective response rate (complete response + partial response) of cabozantinib-s-malate (XL184) in children and young adults with Ewing sarcoma, rhabdomyosarcoma, non-rhabdomyosarcoma soft tissue sarcoma, and Wilms tumor.

II. To estimate whether XL184 therapy either improves the disease control rate at 4 months in patients with recurrent measurable osteosarcoma as compared to a historical Childrens Oncology Group (COG) experience or produces an objective response rate.

SECONDARY OBJECTIVES:

I. To further define XL184 related toxicities in pediatric, adolescent and young adult patients.

II. To further define XL184 pharmacokinetics in the pediatric and adolescent patients.

III. To estimate 1-year time to progression, progression free survival (PFS) and overall survival for each stratum, and if feasible to compare to historical controls.

EXPLORATORY OBJECTIVES:

I. To assess the effect of XL184 on patients' immune cell subsets. II. To obtain tumor tissue (snap frozen, formalin-fixed and paraffin-embedded [FFPE] blocks, or unstained slides) from diagnosis, recurrence, or both, for possible future studies.

OUTLINE:

Patients receive cabozantinib-s-malate orally (PO) on a continuous dosing schedule using a dosing nomogram on days 1-28. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up at 30 days, every 6 months for 1 year and then annually for up to 5 years. ;

Related Conditions & MeSH terms

• Adrenal Cortical Carcinoma
• Adrenocortical Carcinoma
• Alveolar Soft Part Sarcoma
• Carcinoma
• Carcinoma, Hepatocellular
• Carcinoma, Neuroendocrine
• Carcinoma, Renal Cell
• Central Nervous System Neoplasm
• Central Nervous System Neoplasms
• Clear Cell Sarcoma of Soft Tissue
• Ewing Sarcoma
• Hepatoblastoma
• Hepatocellular Carcinoma
• Neoplasms
• Nervous System Neoplasms
• Osteosarcoma
• Recurrence
• Recurrent Alveolar Soft Part Sarcoma
• Recurrent Ewing Sarcoma
• Recurrent Hepatoblastoma
• Recurrent Hepatocellular Carcinoma
• Recurrent Kidney Wilms Tumor
• Recurrent Malignant Solid Neoplasm
• Recurrent Osteosarcoma
• Recurrent Primary Malignant Central Nervous System Neoplasm
• Recurrent Renal Cell Carcinoma
• Recurrent Rhabdomyosarcoma
• Recurrent Soft Tissue Sarcoma
• Recurrent Thyroid Gland Medullary Carcinoma
• Refractory Ewing Sarcoma
• Refractory Hepatoblastoma
• Refractory Hepatocellular Carcinoma
• Refractory Malignant Solid Neoplasm
• Refractory Osteosarcoma
• Refractory Primary Central Nervous System Neoplasm
• Refractory Primary Malignant Central Nervous System Neoplasm
• Refractory Renal Cell Carcinoma
• Refractory Rhabdomyosarcoma
• Refractory Soft Tissue Sarcoma
• Renal Cell Carcinoma
• Rhabdomyosarcoma
• Sarcoma
• Sarcoma, Alveolar Soft Part
• Sarcoma, Clear Cell
• Sarcoma, Ewing
• Soft Tissue Sarcoma
• Solid Neoplasm
• Thyroid Diseases
• Thyroid Gland Medullary Carcinoma
• Thyroid Neoplasms
• Wilms Tumor

• NCT number: NCT02867592
• Study type: Interventional
• Source: National Cancer Institute (NCI)
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: May 18, 2017
• Completion date: September 21, 2024