Trials / Active Not Recruiting

Active Not RecruitingNCT02867592

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

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
Phase: Phase 2
Study type: Interventional
Enrollment: 109 (actual)

Sponsor: National Cancer Institute (NCI) · NIH
Sex: All
Age: 2 Years – 30 Years
Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

Interventions

Type	Name	Description
DRUG	Cabozantinib	Given PO
DRUG	Cabozantinib S-malate	Given PO Note: Capsule formulation (Cometriq) not used in this trial.
OTHER	Pharmacological Study	Correlative studies

Timeline

Start date: 2017-05-18
Primary completion: 2021-06-30
Completion: 2026-06-27
First posted: 2016-08-16
Last updated: 2026-04-13
Results posted: 2022-11-28

Locations

142 sites across 1 country: United States

Regulatory

FDA-regulated drug study

Source: ClinicalTrials.gov record NCT02867592. Inclusion in this directory is not an endorsement.