A Phase 2 interventional study of Cabozantinib and Cabozantinib S-malate in Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma and Central Nervous System Neoplasm, sponsored by National Cancer Institute (NCI). Active, not recruiting at 142 sites in United States. Open to participants aged 2 Years to 30 Years. Per ClinicalTrials.gov, last updated 2026-08-28.
Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment
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.
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.
94 studies on the registry are indexed under Adrenocortical Carcinoma; 26 are open to participants now.
This study's enrollment of 109 is above the median of 35 across 71 interventional studies indexed under Adrenocortical Carcinoma.
Browse Adrenocortical Carcinoma studies →National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patients must have recurrent or disease, or newly diagnosed disease with no known curative therapy or therapy proven to prolong survival with an acceptable quality of life; patients must have had histologic verification of one of the listed below at original diagnosis or at relapse:
Rare tumors
Pediatric solid tumors (including central nervous system [CNS] tumors) with known molecular alterations in the targets of XL184 (i.e., MET amplification, overexpression, activating mutation, MET translocation, MET exon skipping mutations, activating RET mutations, RET rearrangement, overexpression or activation of AXL); documentation of the alteration from a Clinical Laboratory Improvement Act (CLIA) certified laboratory will be required
Patients must have radiographically ; measurable disease is defined as the presence of at least one lesion on magnetic resonance imaging (MRI) or computed tomography (CT) scan that can be accurately measured with the longest diameter a minimum of 10 mm in at least one dimension (CT scan slice thickness no greater than 5 mm)
Note: The following do NOT qualify as measurable disease:
>= 2 weeks must have elapsed since local palliative radiation therapy (XRT) (small port); >= 6 weeks must have elapsed since treatment with therapeutic doses of M-Iodobenzylguanidine (MIBG); >= 3 months must have elapsed if prior craniospinal XRT was received, if >= 50% of the pelvis was irradiated, or if total-body irradiation (TBI) was received; >= 6 weeks must have elapsed if other substantial bone marrow irradiation was given
or radioisotope (GFR) >= 70 mL/min/1.73 m\^2 or a serum creatinine based on age/gender as follows:
2 to \< 6 years of age
6 to \< 10 years of age
10 to \< 13 years of age
13 to \< 16 years of age
>= 16 years of age
Exclusion Criteria:
Concomitant anticoagulation with oral anticoagulants (e.g., warfarin, direct thrombin, and Factor Xa inhibitors) or platelet inhibitors (e.g., clopidogrel) are prohibited
Patients who have had or are planning to have the following invasive procedures are not eligible:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Relapsed and refractory
Relapsed: the illness came back after treatment had worked. Refractory: it did not respond to treatment, or stopped responding while still on it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Leptomeningeal disease
Cancer that has spread to the leptomeninges, the thin layers covering the brain and spinal cord, and the fluid between them. It is not the same as brain metastases.
Criteria often treat it separately, and many studies leave it out even where treated brain metastases are allowed.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Reproductive potential
Able to have children. For women this usually means from the first period until menopause, unless permanently unable to through surgery; for men, unless permanently sterile. Read who the line is about — participants, women, men, or their partners.
Criteria about it usually ask for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
CYP3A (CYP3A4) inhibitors and inducers
CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.
Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Brain metastases
Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).
Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Drug: Cabozantinib · Drug: Cabozantinib S-malate · Other: Pharmacological Study
Given PO
Given PO Note: Capsule formulation (Cometriq) not used in this trial.
Also known as: BMS-907351, Cabometyx, Cometriq, XL 184, XL-184, XL184
Correlative studies
Objective Response (Non-Osteosarcoma Strata)
Will be assessed by Response Evaluation Criteria in Solid Tumors version 1.1. Response rates will be calculated as the percent of evaluable patients who are responders (Overall Best Response of Partial Response or Complete Response), and confidence intervals will be constructed accounting for the two-stage design.
Time frame: Up to the first 6 cycles of therapy
Objective Response (Osteosarcoma Stratum)
Will be assessed by the Response Evaluation Criteria in Solid Tumors version 1.1 and Disease Control (see section 9.3.2 of the ADVL1622 Protocol). Response + Disease Control rate will be calculated as the percent of evaluable patients who are responders or who met the definition of disease control, and confidence intervals will be constructed accounting for the two-stage design.
Time frame: Up to the first 6 cycles of therapy.
Percentage of Participants With Adverse Events
Will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Will report the percentage of patients within each disease stratum who experienced a grade 3 or higher toxicity with attribution of possible, probable, or definite while on protocol therapy or within 30 days of the last dose of therapy
Time frame: Up to 5 years (duration of protocol therapy plus 30 days after last dose of therapy)
Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Cmax
Day 1 PK peak concentration will be summarized by the mean and the standard deviation.
Time frame: Prior to dose, 2, 4, 8 and 20-28 hours after dose on day 1
Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Tmax
Day 1 PK time to peak concentration will be summarized by the mean and the standard deviation.
Time frame: Prior to dose, 2, 4, 8 and 20-28 hours after dose on day 1
Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: AUC
Day 1 PK area under the curve will be summarized by the mean and the standard deviation.
Time frame: Prior to dose, 2, 4, 8 and 20-28 hours after dose on day 1
Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Accumulation
PK accumulation will be summarized by the mean and the standard deviation.
Time frame: Cycle 1, Day 1 (20-28 hours after dose) and Cycle 1, Day 22
Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Half-life
PK half-life will be summarized by the mean and the standard deviation.
Time frame: Cycle 1, Day 1 (20-28 hours after dose) and Cycle 1, Day 22
Time to Progression (TTP)
Percent of patients not yet progressed at 1 year as estimated by the Kaplan-Meier method. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECISTv1.1), as a \>=20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Clinical progression was also counted as an event for this analysis.
Time frame: Up to 1 year
Progression Free Survival (PFS)
The 1-year Progression Free Survival will be estimated using Kaplan-Meier methodology. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECISTv1.1), as a \>=20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
Time frame: Up to 1 year
Overall Survival (OS)
The 1-year Overall Survival will be estimated using Kaplan-Meier methodology.
Time frame: Up to 1 year
Change in Immune Biomarkers
The association between the host immune system and response to cabozantinib-s-malate will be assessed in an exploratory manner. each biomarker will be correlated with the clinical outcomes of objective response and progression free survival.
Time frame: Baseline, day 1 (prior to dose) of cycles 2 and 3
| Milestone | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Started | 15 | 14 | 13 | 13 | 25 | 29 |
| Completed | 0 | 0 | 0 | 0 | 0 | 0 |
| Not completed | 15 | 14 | 13 | 13 | 25 | 29 |
| Withdrew: Adverse event | 3 | 0 | 2 | 1 | 1 | 5 |
| Withdrew: Death | 0 | 0 | 0 | 1 | 0 | 1 |
| Withdrew: Lack of efficacy | 8 | 10 | 8 | 8 | 12 | 17 |
| Withdrew: Physician decision | 1 | 2 | 2 | 0 | 5 | 1 |
| Withdrew: Protocol violation | 0 | 0 | 0 | 0 | 1 | 1 |
| Withdrew: Withdrawal by subject | 0 | 1 | 0 | 1 | 3 | 3 |
| Withdrew: Ineligible | 1 | 1 | 0 | 0 | 2 | 0 |
| Withdrew: No treatment | 1 | 0 | 0 | 0 | 0 | 0 |
| Withdrew: Still on therapy | 0 | 0 | 0 | 0 | 1 | 0 |
| Withdrew: Patient received concurrent anticancer or investigational therapy | 0 | 0 | 0 | 0 | 0 | 1 |
| Withdrew: Repeat eligibility studies (if required) are outside the parameters required for eligibility | 1 | 0 | 1 | 2 | 0 | 0 |
Will be assessed by Response Evaluation Criteria in Solid Tumors version 1.1. Response rates will be calculated as the percent of evaluable patients who are responders (Overall Best Response of Partial Response or Complete Response), and confidence intervals will be constructed accounting for the two-stage design.
| Percentage of patients | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors |
|---|---|---|---|---|---|
| Objective Response (Non-Osteosarcoma Strata) | 0 (0.0 to 24.71) | 0 (0.0 to 24.71) | 0 (0.0 to 24.71) | 0 (0.0 to 24.71) | 13.04 (2.78 to 33.59) |
Will be assessed by the Response Evaluation Criteria in Solid Tumors version 1.1 and Disease Control (see section 9.3.2 of the ADVL1622 Protocol). Response + Disease Control rate will be calculated as the percent of evaluable patients who are responders or who met the definition of disease control, and confidence intervals will be constructed accounting for the two-stage design.
| Percentage of patients | Osteosarcoma |
|---|---|
| Objective Response (Osteosarcoma Stratum) | 35.18 (20.16 to 48.27) |
Will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0. Will report the percentage of patients within each disease stratum who experienced a grade 3 or higher toxicity with attribution of possible, probable, or definite while on protocol therapy or within 30 days of the last dose of therapy
| Percentage of patients | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Percentage of Participants With Adverse Events | 61.54 (31.58 to 86.14) | 38.46 (13.86 to 68.42) | 61.54 (31.58 to 86.14) | 38.46 (13.86 to 68.42) | 60.87 (38.54 to 80.29) | 68.97 (49.17 to 84.72) |
Day 1 PK peak concentration will be summarized by the mean and the standard deviation.
| ng/mL | All Pharmacokinetic Patients |
|---|---|
| Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Cmax | 567 ± 379.0 |
Day 1 PK time to peak concentration will be summarized by the mean and the standard deviation.
| Hour | All Pharmacokinetic Patients |
|---|---|
| Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Tmax | 2.9 ± 1.0 |
Day 1 PK area under the curve will be summarized by the mean and the standard deviation.
| hr*ng/mL | All Pharmacokinetic Patients |
|---|---|
| Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: AUC | 7934 ± 4267.0 |
PK accumulation will be summarized by the mean and the standard deviation.
| Ratio | All Pharmacokinetic Patients |
|---|---|
| Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Accumulation | 6.6 ± 6.0 |
PK half-life will be summarized by the mean and the standard deviation.
| Hour | All Pharmacokinetic Patients |
|---|---|
| Pharmacokinetics (PK) Parameters of Cabozantinib S-malate: Half-life | 101 ± 100.1 |
Percent of patients not yet progressed at 1 year as estimated by the Kaplan-Meier method. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECISTv1.1), as a \>=20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions. Clinical progression was also counted as an event for this analysis.
| Percentage of patients | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Time to Progression (TTP) | 16.78 (2.67 to 41.51) | 0 (0 to 0) | 15.39 (2.48 to 38.78) | 23.08 (5.58 to 47.46) | 20.6 (5.89 to 41.42) | 21.25 (7.64 to 39.35) |
The 1-year Progression Free Survival will be estimated using Kaplan-Meier methodology. Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECISTv1.1), as a \>=20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions
| Percentage of patients | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Progression Free Survival (PFS) | 15.39 (2.48 to 38.78) | 0 (0 to 0) | 15.39 (2.48 to 38.78) | 23.08 (5.58 to 47.46) | 13.04 (3.27 to 29.72) | 14.71 (4.66 to 30.19) |
The 1-year Overall Survival will be estimated using Kaplan-Meier methodology.
| Percentage of patients | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Overall Survival (OS) | 53.85 (24.77 to 75.99) | 25 (6.01 to 50.48) | 74.59 (39.76 to 91.10) | 42.74 (15.90 to 67.50) | 34.78 (16.63 to 53.71) | 35.86 (18.96 to 53.13) |
The association between the host immune system and response to cabozantinib-s-malate will be assessed in an exploratory manner. each biomarker will be correlated with the clinical outcomes of objective response and progression free survival.
Results for this outcome have not been posted.
Collected over From start of treatment to 30 days after last dose, a median of 3.4 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Ewing's Sarcoma | 12/13 (92.3%) | 6/13 (46.2%) | 5/13 (38.5%) |
| Rhabdomyosarcoma | 10/13 (76.9%) | 2/13 (15.4%) | 7/13 (53.8%) |
| Non-Rhabdomyosarcoma Soft Tissue Sarcoma | 8/13 (61.5%) | 4/13 (30.8%) | 7/13 (53.8%) |
| Wilms Tumor | 10/13 (76.9%) | 6/13 (46.2%) | 7/13 (53.8%) |
| Rare Tumors | 18/23 (78.3%) | 10/23 (43.5%) | 13/23 (56.5%) |
| Osteosarcoma | 20/29 (69%) | 8/29 (27.6%) | 17/29 (58.6%) |
| Event | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| HypoxiaRespiratory, thoracic and mediastinal disorders | 0/13 | 0/13 | 0/13 | 3/13 | 0/23 | 0/29 |
| Infections and infestations - Other, specifyInfections and infestations | 0/13 | 0/13 | 0/13 | 2/13 | 0/23 | 0/29 |
| Pericardial effusionCardiac disorders | 2/13 | 0/13 | 0/13 | 0/13 | 0/23 | 0/29 |
| Disease progressionGeneral disorders | 0/13 | 0/13 | 0/13 | 2/13 | 2/23 | 0/29 |
| Aspartate aminotransferase increasedInvestigations | 0/13 | 0/13 | 0/13 | 0/13 | 3/23 | 0/29 |
| Abdominal painGastrointestinal disorders | 0/13 | 0/13 | 0/13 | 0/13 | 2/23 | 0/29 |
| Alanine aminotransferase increasedInvestigations | 0/13 | 0/13 | 0/13 | 0/13 | 2/23 | 0/29 |
| Blood bilirubin increasedInvestigations | 1/13 | 0/13 | 0/13 | 0/13 | 2/23 | 0/29 |
| DehydrationMetabolism and nutrition disorders | 0/13 | 0/13 | 0/13 | 1/13 | 2/23 | 0/29 |
| Flank painMusculoskeletal and connective tissue disorders | 0/13 | 0/13 | 0/13 | 0/13 | 2/23 | 0/29 |
| Event | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma |
|---|---|---|---|---|---|---|
| Lipase increasedInvestigations | 1/13 | 2/13 | 4/13 | 1/13 | 0/23 | 2/29 |
| Neutrophil count decreasedInvestigations | 3/13 | 0/13 | 0/13 | 1/13 | 4/23 | 2/29 |
| Alanine aminotransferase increasedInvestigations | 0/13 | 1/13 | 2/13 | 0/13 | 3/23 | 1/29 |
| DiarrheaGastrointestinal disorders | 2/13 | 0/13 | 1/13 | 0/13 | 0/23 | 1/29 |
| HypertensionVascular disorders | 0/13 | 2/13 | 1/13 | 0/13 | 3/23 | 2/29 |
| NauseaGastrointestinal disorders | 0/13 | 0/13 | 2/13 | 1/13 | 0/23 | 1/29 |
| Platelet count decreasedInvestigations | 1/13 | 0/13 | 0/13 | 2/13 | 1/23 | 0/29 |
| VomitingGastrointestinal disorders | 0/13 | 1/13 | 2/13 | 0/13 | 1/23 | 1/29 |
| Weight lossInvestigations | 0/13 | 1/13 | 2/13 | 1/13 | 0/23 | 1/29 |
| AnemiaBlood and lymphatic system disorders | 0/13 | 1/13 | 0/13 | 1/13 | 3/23 | 0/29 |
| Age, Categorical(Participants) | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma | Total |
|---|---|---|---|---|---|---|---|
| <=18 years | 7 | 6 | 8 | 11 | 22 | 17 | 71 |
| Between 18 and 65 years | 8 | 8 | 5 | 2 | 3 | 12 | 38 |
| >=65 years | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Continuous(years) | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma | Total |
|---|---|---|---|---|---|---|---|
| Median | 18.1 (7.3 to 26.8) | 19.3 (5.7 to 26.8) | 16.1 (10.1 to 26.3) | 14.1 (8.9 to 27.1) | 13.2 (5.6 to 19.5) | 16.8 (9.1 to 22.3) | 15.7 (5.6 to 27.1) |
| Sex: Female, Male(Participants) | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma | Total |
|---|---|---|---|---|---|---|---|
| Female | 5 | 5 | 8 | 10 | 15 | 10 | 53 |
| Male | 10 | 9 | 5 | 3 | 10 | 19 | 56 |
| Ethnicity (NIH/OMB)(Participants) | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma | Total |
|---|---|---|---|---|---|---|---|
| Hispanic or Latino | 0 | 2 | 2 | 1 | 8 | 8 | 21 |
| Not Hispanic or Latino | 15 | 12 | 10 | 11 | 15 | 20 | 83 |
| Unknown or Not Reported | 0 | 0 | 1 | 1 | 2 | 1 | 5 |
| Race (NIH/OMB)(Participants) | Ewing's Sarcoma | Rhabdomyosarcoma | Non-Rhabdomyosarcoma Soft Tissue Sarcoma | Wilms Tumor | Rare Tumors | Osteosarcoma | Total |
|---|---|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 | 0 | 0 | 0 | 0 |
| Asian | 0 | 2 | 1 | 2 | 2 | 1 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 | 0 | 1 | 1 | 2 |
| Black or African American | 1 | 1 | 3 | 1 | 2 | 6 | 14 |
| White | 11 | 10 | 7 | 8 | 14 | 16 | 66 |
| More than one race | 1 | 0 | 0 | 0 | 0 | 0 | 1 |
| Unknown or Not Reported | 2 | 1 | 2 | 2 | 6 | 5 | 18 |
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Studies of the drug tested here: Cabozantinib
National Cancer Institute (NCI) →