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New treatment option for Neuroblastoma
Official title A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
ClinicalTrials.gov ID: NCT05135975
What this study is testing
What is Cabozantinib?
Cabozantinib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for neuroblastoma.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 40
You may be able to join if
- 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...
- Patients previously with measurable disease, at start of most recent line of therapy, AND without evaluable disease (or for whom assessment of...
- Patients previously with evaluable disease at start of most recent line of therapy, AND without measurable disease at that time are eligible based on...
- Patients previously with measurable AND evaluable disease at start of most recent line of therapy must meet BOTH "measurable disease" and "evaluable...
- Stratum A: Solid tumors with 1-year PFS ≤25% (target enrollment 36 patients)
See the full eligibility criteria
Who can join
- 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...
- 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)...
- Patients previously with evaluable disease at start of most recent line of therapy, AND without measurable disease at that time are eligible based on "evaluable disease" definition alone only in Section 3.1.2.a.ii.
- Patients previously with measurable AND evaluable disease at start of most recent line of therapy must meet BOTH "measurable disease" and "evaluable disease" definitions under Section 3.1.2.a. 3.2.2. Disease Strata...
- Stratum A: Solid tumors with 1-year PFS ≤25% (target enrollment 36 patients)
- Neuroblastoma BR1 at the end of frontline therapy with residual disease (less than complete response (CR), including MIBG-avid stable disease as per Section 3.1.2.a.ii,.or \>5% bone marrow involvement) but not...
- Neuroblastoma BR2 or later.
- Osteosarcoma, BR2 or later
- Ewing sarcoma, BR2 or later
- Rhabdomyosarcoma, with positive surgical margins OR with primary extremity tumor with \>2 metastases at diagnosis, BR1 or later
- Rhabdomyosarcoma, alveolar subtype or fusion-positive subtype, BR2 or later
- Rhabdomyosarcoma, embryonal subtype, Group 4 at original diagnosis, BR2 or later
- Desmoplastic small round blue cell tumor, BR2 or later
- Any other soft tissue sarcoma, BR2 or later
- Wilms tumor, diffuse anaplasia histology, any stage, BR2 or later
- Wilms tumor, any histology with relapse within 12 months of diagnosis, who have received prior treatment with doxorubicin, and intraabdominal recurrence, BR2 or later
- Wilms tumor, BR3 or later
- Other solid tumor with 1-year PFS ≤ 20% can be discussed with study chair for eligibility
- Stratum B: Metastatic Ewing sarcoma with metastases not undergoing complete metastatectomy, BR1 (target enrollment 20 patients)
- Stratum C: Metastatic osteosarcoma after frontline therapy, BR1 (target enrollment 20 patients)
- Stratum D: Metastatic fusion-positive rhabdomyosarcoma after frontline therapy, BR1 (target enrollment 10 patients) 3.2.3 for Eligibility:
- Age: ≥ 18 months of age and \<40 years of age at time of study enrollment
- Performance level: Patients must have a Lansky or Karnofsky performance status score of ≥ 50, corresponding to ECOG categories 0, 1 or 2. Use Karnofsky for patients \> 16 years of age and Lansky for patients ≤ 16 years...
- Patient Body Surface Area (BSA): Patients must be ≥0.35 m2 in BSA, using the Mosteller formula, BSA = (((Height in cm) \ (Weight in kg))/ 3600)½ within two weeks of study enrollment
- Prior therapy: patients must have recovered from the acute toxic effects of prior therapy, with the following time specifications:
- Myelosuppressive chemotherapy: Patients must not have received myelosuppressive chemotherapy within 3 weeks of enrollment on study (6 weeks if prior therapy included nitrosourea)
- Other medicinal anti-cancer agents: Patients must not have received non-myelosuppressive anticancer agents, including any type of small molecule kinase inhibitor, within 14 days of enrollment on study
- Biological anticancer therapy (including antibody therapy or cellular therapy): Patients must not have received biological anticancer therapy within 21 days of enrollment on study
- Radiation therapy: Patients must not have received external beam radiation therapy to sites outside of the lungs within 2 weeks of study enrollment, external beam radiation therapy to sites within the lungs within 4...
- Myeloablative therapy: Patients must not have received myeloablative therapy within 2 months of study enrollment, must not have received a blood stem cell/marrow infusion within 3 weeks of study enrollment, and must...
- Bone Marrow Function: Patients must have adequate bone marrow function at time of study enrollment, as defined as:
- Absolute neutrophil count (ANC) ≥1000/mcL; patients cannot have received filgrastim, pegfilgrastim or equivalent biosimilar within 14 days of study enrollment
- Platelet count ≥ 100,000/mcL; patients can receive no more than 15 mL/kg of platelet transfusions per week at time of enrollment to meet the parameters; patients can receive a TPO agonist (e.g., eltrombopag or...
- Hemoglobin ≥ 8.0 g/dL; patients can receive no more than 10 mL/kg of packed red blood cells (PRBCs)/week transfused at time of enrollment on therapy to meet the parameters; patients may receive erythropoietin or...
- Patients with residual bone marrow metastases at end of most recent line of therapy must have stable disease or better at two bone marrow evaluations at least 4 weeks apart, with the second marrow assessment at least 4...
- Renal Function: Patient must meet criteria for both a. and b. below to have adequate renal function, within 2 weeks of study enrollment
- Creatinine clearance or radioisotope GFR ≥ 70 mL/min/1.73 m2, as per institutional standard testing OR serum creatinine based on age/gender as listed in protocol:
- Urine protein: ≤ 30 mg/dl in urinalysis (clean catch recommended), equivalent to ≤ 1+ on dipstick OR quantitative urine protein \ 30 mg/dL urine protein (or \> 1+ on dipstick), a 24-hour quantitative urine protein...
- Hepatic function: Patient must meet ALL of the below criteria, within 14 days of study enrollment, to have adequate hepatic function:
- Total bilirubin \< 2x institutional upper limit of normal (ULN) for age
- ALT\<5x ULN
- Serum albumin \>2.7 g/dL
- Cardiovascular Function: Patients must have adequate cardiovascular function as defined as:
- No significant arrhythmias, strokes, transient ischemic attacks, or myocardial infarction within 6 months of study enrollment
- QTc ≤ 480 msec within 7 days of study enrollment (calculated using Bazett calculation or Fridericia calculation as per institutional standard of care; whichever calculation is used for eligibility must be used for all...
- Blood pressure ≤ 95th percentile for age, height, and gender for patients \<18 years of age (78), or BP ≤140/90 for patients ≥18 years of age. At time of enrollment, patients may be on one antihypertensive agent at a...
- Pancreatic function: Patient must have adequate pancreatic function, as defined by a serum lipase \<2x ULN
- Neurologic function: Patients with defined seizures who are on a stable anti- convulsant regimen using drugs that do not induce hepatic metabolizing enzymes for at least 4 weeks are eligible for enrollment Recommended...
- Lung integrity: Patients must not have had any invasive pulmonary procedure (including bronchoalveolar lavage, lung biopsy, transbronchial biopsy, or thoracotomy) or pneumothorax within 4 weeks of enrollment on study.
- Surgeries or trauma:
- Patients must not have had any major surgical procedures, laparoscopic procedures, sepsis, shock, or physical trauma requiring hospitalization within 4 weeks of enrollment on study. The primary surgeon of any major...
- Patients must not have had a central line or subcutaneous port placement, revision, or removal (excluding a peripherally inserted central catheter (PICC)) within 7 days of study enrollment. Advise patients with a...
- Patients must not have had a core or fine needle biopsy within 7 days of study enrollment.
- Any surgical wounds or incisions must be healed, as determined by treating physician, prior to enrollment on study.
- Bone marrow aspiration and/or biopsy are not considered surgical procedures for the purpose of this study.
- Patients must be able to swallow tablets intact. Tablets cannot be cut or crushed.
- Sexually active fertile people and their partners must agree to use medically accepted methods of contraception (eg, barrier methods, including male condom, female condom, or diaphragm with spermicidal gel) during the...
- Patient or legal guardian must be capable of understanding and complying with the protocol requirements and must have signed the informed consent document.
- Patient must be able to start study treatment no later than 12 weeks after end of prior therapy, where 1 week = 7 days.
- Patient must be enrolled on study within 14 days of qualifying radiographic imaging studies demonstrating best response as per Section 3.1.2.
- Patient must be able to start study treatment no later than 7 days from study enrollment. SEE PROTOCOL FOR ADDITIONAL
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Aurora, Colorado, United States
- Washington D.C., District of Columbia, United States
- The Bronx, New York, United States
- Columbus, Ohio, United States
- Salt Lake City, Utah, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 months to 40 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Aurora, Colorado, United States; Washington D.C., District of Columbia, United States; The Bronx, New York, United States; Columbus, Ohio, United States; Salt Lake City, Utah, United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.