Phase 2 Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
Nationwide Children's Hospital
Summary
This study will expand the types of pediatric cancers being evaluated for response to cabozantinib. The current COG study is restricted to Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, Wilms tumor, and a handful of uncommon tumors. The proposed study will extend this evaluation to tumors that have been shown to either express known targets of cabozantinib or with preclinical evidence of efficacy, including specifically neuroblastomas. These tumors have high morbidity and mortality, particularly in the relapse setting, and few or no proven therapeutic options. As such, evaluation of cabozantinib in these studies is warranted. The study hypothesizes that use of cabozantinib in patients with ultra-high-risk pediatric solid tumors with minimal disease burden, as defined in the inclusion criteria below, can prevent and/or slow recurrent tumor formation in pediatric solid tumors and thereby significantly extend the period of disease control and/or induce a durable cure.
Description
The study hypothesizes that use of cabozantinib in patients with ultra-high-risk pediatric solid tumors who have achieved and maintained (for at least 4 weeks) a "best response" to their most recent line of therapy, as defined in the inclusion criteria below, can prevent and/or slow recurrent tumor formation in pediatric solid tumors, improve one-year progression-free survival by 20% as compared to historical controls, and also improve longer-term progression-free and overall survival without significant impact on quality of life. We also hypothesize, in an exploratory aim, that there will be…
Eligibility
- Age range
- 1–40 years
- Sex
- All
- Healthy volunteers
- No
3.2 Patient Eligibility Criteria: 3.2.1 Assessment criteria for eligibility, based on disease burden at start of most recent line of therapy and corresponding response (patients must meet one of the following criteria in order to be deemed eligible for enrollment): 1. Patients previously with measurable disease, at start of most recent line of therapy, AND without evaluable disease (or for whom assessment of potential evaluable-only disease would not be considered standard of care) are eligible based on "measurable disease response" only in Section 3.1.2.a.i. 2. Patients previously with eval…
Interventions
- DrugCabozantinib
Enrolled patients will be treated with cabozantinib maleate, tablet formulation, using the recommended Phase 2 dose of 40 mg/m2/day to a maximum of 420 mg/week. Treatment will be administered in 28- day cycles.
Locations (6)
- Children's Hospital of Alabama/UABBirmingham, Alabama
- Children's Hospital of ColoradoAurora, Colorado
- Children's National Medical CenterWashington D.C., District of Columbia
- Children's Hospital at MontefioreThe Bronx, New York
- Nationwide Children's HospitalColumbus, Ohio
- Primary Children's HospitalSalt Lake City, Utah