Actively Recruiting
Phase 2 Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
Led by Nationwide Children's Hospital · Updated on 2025-08-12
86
Participants Needed
6
Research Sites
178 weeks
Total Duration
On this page
Sponsors
N
Nationwide Children's Hospital
Lead Sponsor
E
Exelixis
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying cabozantinib as a maintenance treatment to prevent progression or recurrence in children and young adults with ultra-high-risk pediatric solid tumors. These tumors include neuroblastoma, Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, Wilms tumor, and other rare tumors that express targets of cabozantinib or show preclinical evidence of response. The study aims to improve progression-free survival and overall survival while maintaining quality of life in patients who have achieved their best response to prior therapy. Participants will receive cabozantinib tablets at a dose of 40 mg/m2 per day, up to a maximum of 420 mg per week, given in 28-day cycles for up to one year. This approach targets the critical period within 12 months after last therapy when recurrence risk is highest. The study expands current research by evaluating cabozantinib in a wider range of pediatric solid tumors and testing its use as a maintenance therapy during remission to potentially extend disease control. Throughout the study, patients will be monitored for progression-free survival at one year as the primary outcome, with additional assessments of overall survival, duration of response, and safety over several years. Participants must meet strict eligibility criteria including age, performance status, organ function, and recovery from prior treatments. The study involves regular clinical evaluations, laboratory tests, imaging, and assessment of adverse events to ensure safety and measure treatment effects over time.
CONDITIONS
Brief Title
A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age at least 18 months and less than 40 years at study enrollment
- Lansky or Karnofsky performance status score of 50 or higher
- Body surface area of at least 0.35 m2 measured within two weeks of enrollment
- Recovery from prior therapies with specified waiting periods before enrollment
- Adequate bone marrow function meeting specific blood count criteria
- Adequate renal function with normal creatinine clearance and urine protein levels
- Adequate liver function with total bilirubin, ALT, and serum albumin within defined limits
- Adequate cardiovascular function including no significant recent heart events and controlled blood pressure
- Adequate pancreatic function with normal serum lipase
- Stable neurologic function if on anticonvulsants with allowed medications
- No recent invasive pulmonary procedures or pneumothorax within 4 weeks
- No recent major surgeries, trauma, or needle biopsies within specified timeframes
- Ability to swallow tablets intact without crushing or cutting
- Agreement to use medically accepted contraception if sexually active and fertile
- Capability to understand and comply with study requirements and provide consent
- Ability to start study treatment within 12 weeks after prior therapy and within 7 days of enrollment
- Enrollment within 14 days of qualifying imaging studies showing best response
You will not qualify if you...
- Receiving myelosuppressive chemotherapy within 3 weeks (6 weeks if nitrosourea) before enrollment
- Receiving non-myelosuppressive anticancer agents within 14 days before enrollment
- Receiving biological anticancer therapy within 21 days before enrollment
- Receiving external beam radiation at certain sites within 2 to 6 weeks before enrollment
- Receiving myeloablative therapy within 2 months before enrollment
- Receiving blood stem cell or marrow infusion within 3 weeks before enrollment
- Filgrastim, pegfilgrastim, or equivalent use within 14 days before enrollment
- Unstable bone marrow metastases or worsening marrow disease
- QTc interval greater than 480 msec after repeated testing
- Blood pressure above defined limits or unstable on antihypertensive therapy
- Use of enzyme-inducing anticonvulsant medications
- Recent invasive pulmonary procedures or pneumothorax within 4 weeks
- Recent major surgery, sepsis, shock, or trauma requiring hospitalization within 4 weeks
- Recent central line or port placement or revision within 7 days before enrollment
- Recent core or fine needle biopsy within 7 days before enrollment
- Unhealed surgical wounds or incisions at enrollment
- Inability to swallow tablets intact
- Pregnancy at time of enrollment
- Inability to comply with protocol or provide informed consent
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 12 months
Participants receive cabozantinib daily in 28-day cycles as a maintenance therapy to prevent tumor progression or recurrence for up to one year.
Trial Site Locations
Total: 6 locations
1
Children's Hospital of Alabama/UAB
Birmingham, Alabama, United States, 35233
Actively Recruiting
2
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
4
Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
Actively Recruiting
5
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
6
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
Actively Recruiting
Research Team
M
Madeline Richardson, MA
P
Parth Patel, MPH, CCRP
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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