A Phase 3 interventional study of Biospecimen Collection and Cabozantinib S-malate in Functioning Pancreatic Neuroendocrine Tumor, Intermediate Grade Lung Neuroendocrine Neoplasm and Locally Advanced Digestive System Neuroendocrine Neoplasm, sponsored by National Cancer Institute (NCI). Active, not recruiting at 432 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-26.
Sponsored by National Cancer Institute (NCI) · Phase 3, Interventional, and Treatment
Among 59 studies in Adenoma, Islet Cell with a site in United States — 13 open to participants now, 21 under way, 4 of them phase 3. Adenoma, Islet Cell · United States.
Among the 59 studies in Adenoma, Islet Cell with a site in United States, this one is the 5th largest by enrollment of the 51 interventional ones that give a count, is one of 2 that started in 2018, is one of 3 in phase 3, reaches primary completion later than 0 of the 20 other live ones, and was the 36th registered.
113 studies on the registry are indexed under Adenoma, Islet Cell; 30 are open to participants now.
Browse Adenoma, Islet Cell studies →13 of the 59 studies in Adenoma, Islet Cell with a site in United States are open now. Recruiting first, the newest first:
Children's Hospital of Philadelphia · Sites in United States
Ages 18+
RayzeBio, Inc. · Sites in United States, Canada
Ages 18+
Anusha Kalbasi · Sites in United States
Ages 18–75
Exelixis · Sites in United States, Australia, Austria and 15 more countries
Ages 18+
Rezolute · Sites in United States, France, Netherlands and 2 more countries
Ages 18+
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 24 Adenoma, Islet Cell studies recruiting now, 17 (71%) set no upper age limit and 2 (8%) stop at 75 or younger; 2 take children under 18; 3 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
59 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 18 · Phase 2 29 · Phase 3 5
First in 1978; this study’s year is marked. 2 started in 2018.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 298 is above the median of 38 across 76 interventional studies indexed under Adenoma, Islet Cell.
Median enrollment 39 across the 51 interventional studies, 173 across the 4 observational. Of the 33 that completed or were terminated, 15 (45%) have results posted.
This tab as a report to print or save as PDF → With an account.
13 of 59 have an industry lead sponsor. The 10 most frequent:
National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 412 completed or terminated interventional studies of FDA-regulated products, 374 (91%) have results posted.
National Cancer Institute (NCI) has 4 studies filed under Adenoma, Islet Cell — fewer than ten, so there is no comparison to draw.
12 of the studies in Adenoma, Islet Cell with a site in United States that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05477576Phase 3RayzeBio, Inc.Study of RYZ101 Compared With SOC in Pts w Inoperable SSTR+ Well-differentiated GEP-NET That Has Progressed Following 177Lu-SSA Therapy | 2026-12 |
| NCT06881992Phase 3RezoluteA Phase 3 Study of Ersodetug in Patients With Tumor Hyperinsulinism | 2027-09 |
| NCT06663072University of ChicagoStudy to Test the Combination of Fulvestrant With Lu-DOTATATE for Advanced Pancreatic Neuroendocrine Tumors | 2026-11 |
| NCT05687123National Cancer Institute (NCI)Testing the Addition of Sunitinib Malate to Lutetium Lu 177 Dotatate (Lutathera) in Pancreatic Neuroendocrine Tumors | 2026-12-14 |
| NCT06148636David BushnellA Safety Study of 212Pb-VMT-alpha-NET in Patients With Neuroendocrine Tumors | 2026-12-31 |
Add all 12 to your calendar (.ics) → With an account.
Among the 59 studies in Adenoma, Islet Cell with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
This phase III trial studies cabozantinib to see how well it works compared with placebo in treating patients with neuroendocrine or carcinoid tumors that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of the body (advanced). Cabozantinib is a chemotherapy drug known as a tyrosine kinase inhibitor, and it targets specific tyrosine kinase receptors, that when blocked, may slow tumor growth.
PRIMARY OBJECTIVES:
I. To determine whether cabozantinib (cabozantinib S-malate) can significantly improve progression-free survival (PFS) compared to placebo in patients with advanced pancreatic neuroendocrine tumors (NET) whose disease has progressed after prior therapy.
II. To determine whether cabozantinib can significantly improve progression-free survival (PFS) compared to placebo in patients with advanced carcinoid tumors whose disease has progressed after prior therapy.
SECONDARY OBJECTIVES:
I. To determine whether cabozantinib can significantly improve overall survival (OS) compared to placebo in patients with advanced pancreatic NET whose disease has progressed after prior therapy.
II. To determine whether cabozantinib can significantly improve overall survival (OS) compared to placebo in patients with advanced carcinoid tumors whose disease has progressed after prior therapy.
III. To evaluate safety and tolerability of cabozantinib versus placebo in patients with advanced pancreatic NET using Common Terminology Criteria for Adverse Events (CTCAE) and Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE).
IV. To evaluate safety and tolerability of cabozantinib versus placebo in patients with advanced carcinoid tumors using CTCAE and PRO-CTCAE.
V. To evaluate the overall radiographic response rate of cabozantinib versus placebo in patients with advanced pancreatic NET whose disease has progressed after prior therapy.
VI. To evaluate the overall radiographic response rate of cabozantinib versus placebo in patients with advanced carcinoid tumors whose disease has progressed after prior therapy.
OTHER OBJECTIVE:
I. Results of the primary analysis will be examined for consistency, while taking into account the stratification factors and/or covariates of baseline quality of life (QOL) and fatigue.
QUALITY OF LIFE SUBSTUDY OBJECTIVE:
I. To compare overall quality of life, disease-related symptoms, and other domains between the two treatment groups (cabozantinib versus [vs.] placebo) within each cohort of patients (pancreatic NET vs. carcinoid tumor). (Quality of Life Substudy Objective - A021602-HO1)
POPULATION PHARMACOKINETICS SUBSTUDY OBJECTIVE:
I. To describe the population pharmacokinetic and exposure-response relationships of cabozantinib in patients with advanced neuroendocrine tumors. (Population Pharmacokinetics Substudy Objective - A021602-PP1)
OUTLINE: Patients are randomized to 1 of 2 arms.
ARM I: Patients receive cabozantinib S-malate orally (PO) once daily (QD) on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood and urine sample collection, and computed tomography (CT), magnetic resonance imaging (MRI), and/or x-ray imaging during screening and on study.
ARM II: Patients receive placebo PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood and urine sample collection, and CT, MRI, and/or x-ray imaging during screening and on study. Patients may crossover to receive cabozantinib S-malate at the time of disease progression.
After completion of study treatment, patients are followed up every 12 weeks until disease progression or start of new anticancer therapy, and then every 6 months until 8 years after registration.
Inclusion Criteria:
Documentation of Disease:
Histologic Documentation: Well- or moderately-differentiated neuroendocrine tumors of pancreatic and non-pancreatic (i.e. carcinoid) origin by local pathology
Tumor Site: Histological documentation of neuroendocrine tumor of pancreatic, gastrointestinal (GI), lung, thymus, other, or unknown primary site; GI, lung, thymus, other, and unknown primary NETs will enroll in the carcinoid tumor cohort of the study
Prior Treatment
Patient History
Concomitant Medications
Not pregnant and not nursing
Total =\< 1.5 x ULN
Potassium within normal limits (WNL)
Phosphorus WNL
Calcium WNL
Magnesium WNL
TSH WNL
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.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
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 line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
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.
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 US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
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.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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.
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.
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.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
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.
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.
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).
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.
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.
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.
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.
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.
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.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
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.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
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.
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.
CTCAE grade
The US National Cancer Institute’s scale of how severe an adverse event is — any unwanted medical event during a study, such as a symptom, an illness or an abnormal test result, whether or not a treatment caused it. Whether it did is judged separately.
“Side effects of earlier treatment resolved to grade 1 or less” means they have settled to mild or gone.
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.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
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.
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.
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.
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.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and 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.
Postmenopausal
Periods have stopped for good: usually no period for 12 months in a row without another medical reason, sometimes confirmed by a blood test (FSH).
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.
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.
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.
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.
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.
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.
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.
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.
Patients receive cabozantinib S-malate PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood and urine sample collection, and CT, MRI, and/or x-ray imaging during screening and on study.
Procedure: Biospecimen Collection · Drug: Cabozantinib S-malate · Procedure: Computed Tomography · Procedure: Magnetic Resonance Imaging · Other: Quality-of-Life Assessment · Procedure: X-Ray Imaging
Patients receive placebo PO QD on days 1-28 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Patients also undergo blood and urine sample collection, and CT, MRI, and/or x-ray imaging during screening and on study. Patients may crossover to receive cabozantinib S-malate at the time of disease progression.
Procedure: Biospecimen Collection · Procedure: Computed Tomography · Procedure: Magnetic Resonance Imaging · Other: Placebo Administration · Other: Quality-of-Life Assessment · Procedure: X-Ray Imaging
Undergo blood and urine sample collection
Also known as: Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Given PO
Also known as: BMS-907351, Cabometyx, Cometriq, XL 184, XL-184, XL184
Undergo CT
Also known as: CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography, Computerized axial tomography (procedure), Computerized Tomography, Computerized Tomography (CT) scan, CT, CT Scan, Diagnostic CAT Scan, Diagnostic CAT Scan Service Type, tomography
Undergo MRI
Also known as: Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan, Medical Imaging, Magnetic Resonance / Nuclear Magnetic Resonance, MR, MR Imaging, MRI, MRI Scan, MRIs, NMR Imaging, NMRI, Nuclear Magnetic Resonance Imaging, sMRI, Structural MRI
Given PO
Ancillary studies
Also known as: Quality of Life Assessment
Undergo x-ray
Also known as: Conventional X-Ray, Diagnostic Radiology, Medical Imaging, X-Ray, Plain film radiographs, Radiographic Imaging, Radiographic imaging procedure (procedure), Radiography, RG, Static X-Ray, X-Ray
Progression-free Survival (PFS)
Will be assessed per Response Evaluation Criteria in Solid Tumors 1.1 determined by retrospective independent central review. Will be compared between treatment arms using the stratified log rank test at one-sided level 0.025. The stratification factors will be used for the analysis. The hazard ratio (HR) for PFS will be estimated using a stratified Cox proportional hazards model, and the 95% confidence interval (CI) for the HR will be provided. Results from an unstratified analysis will also be provided. Kaplan-Meier methodology will be used to estimate the median PFS for each treatment arm, and Kaplan-Meier curves will be produced. Brookmeyer Crowley methodology will be used to construct the 95% CI for the median PFS for each treatment arm.
Time frame: 36 months
Overall Survival (OS)
The analyses for OS will follow intent-to-treat (ITT) principle and will be conducted separately within each cohort (pancreatic neuroendocrine tumor \[NET\] and carcinoid tumor). The distribution of OS will be estimated using the method of Kaplan-Meier. The median OS, along with the 95% CI, will be estimated by the two treatment groups. Overall survival will be compared between treatment arms using the stratified log-rank test at a one-sided cumulative 2.5% level of significance. The stratified Cox regression will be used to estimate the HR of OS, along with the 95% CI. A hierarchical approach will be used to control for family-wise type-I error rate, therefore OS will be formally statistically tested only if the primary efficacy endpoint, PFS, is statistically significantly different between the two treatment groups.
Time frame: 60 months
Number of Patients Experiencing Grade 3+ Adverse Events (AEs) Graded According to the Common Terminology Criteria for Adverse Events (CTCAE) and Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE)
For CTCAE data, the frequency tables will be reviewed to determine the patterns. The overall adverse event rates will be compared between treatment groups using Chi-square test (or Fisher's exact test if the data in contingency table is sparse). PRO-CTCAE data will, at minimum, be analyzed similarly to CTCAE data. The initial analysis of each PRO-CTCAE item will use all available scores in an analysis which mirrors the approach used for the CTCAE data. Supplemental analysis will use model-based multiple imputation incorporating baseline patient characteristics and physician-rated performance status. CTCAE data may be incorporated as auxiliary data into multiple imputation models for AEs which are captured by both PRO-CTCAE and CTCAE. Results from supplemental analysis will be descriptively compared to the results of the initial analysis to assess the robustness of results to missing data. Additional analyses of PRO-CTCAE data beyond those specified above may be undertaken.
Time frame: 60 months
Radiographic Response Rate
Will be defined as the proportion of patients in each arm whose best response is either complete response (CR) or partial response (PR). Radiographic response rate for both cohorts: the analyses for confirmed radiographic response rate will follow the ITT principle and will be conducted separately within each cohort (pancreatic NET and carcinoid tumor). The proportion of patients with either confirmed CR or confirmed PR as their best response will be estimated using point estimates and 95% confidence intervals. Radiographic response rate will be compared between treatment arms using the 2-sample z-test to compare sample proportion at a one-sided 2.5% level of significance.
Time frame: 36 months
| Milestone | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Started | 134 | 69 | 64 | 31 |
| Initiated treatment | 132 | 67 | 63 | 31 |
| Completed | 21 | 7 | 14 | 2 |
| Not completed | 113 | 62 | 50 | 29 |
| Withdrew: Adverse event | 34 | 9 | 10 | 0 |
| Withdrew: Death | 6 | 3 | 0 | 0 |
| Withdrew: Withdrawal by subject | 7 | 4 | 5 | 4 |
| Withdrew: Progressive disease | 55 | 41 | 29 | 24 |
| Withdrew: Alternative treatment | 6 | 1 | 1 | 0 |
| Withdrew: Other disease | 1 | 1 | 3 | 0 |
| Withdrew: Withdrawal prior to initiation | 2 | 2 | 1 | 0 |
| Withdrew: Physician decision | 1 | 1 | 1 | 1 |
| Withdrew: Non-compliance | 1 | 0 | 0 | 0 |
Will be assessed per Response Evaluation Criteria in Solid Tumors 1.1 determined by retrospective independent central review. Will be compared between treatment arms using the stratified log rank test at one-sided level 0.025. The stratification factors will be used for the analysis. The hazard ratio (HR) for PFS will be estimated using a stratified Cox proportional hazards model, and the 95% confidence interval (CI) for the HR will be provided. Results from an unstratified analysis will also be provided. Kaplan-Meier methodology will be used to estimate the median PFS for each treatment arm, and Kaplan-Meier curves will be produced. Brookmeyer Crowley methodology will be used to construct the 95% CI for the median PFS for each treatment arm.
| months | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Progression-free Survival (PFS) | 8.4 (7.6 to 12.7) | 3.9 (3.0 to 5.7) | 13.8 (9.2 to 18.5) | 4.4 (3.0 to 5.9) |
The analyses for OS will follow intent-to-treat (ITT) principle and will be conducted separately within each cohort (pancreatic neuroendocrine tumor \[NET\] and carcinoid tumor). The distribution of OS will be estimated using the method of Kaplan-Meier. The median OS, along with the 95% CI, will be estimated by the two treatment groups. Overall survival will be compared between treatment arms using the stratified log-rank test at a one-sided cumulative 2.5% level of significance. The stratified Cox regression will be used to estimate the HR of OS, along with the 95% CI. A hierarchical approach will be used to control for family-wise type-I error rate, therefore OS will be formally statistically tested only if the primary efficacy endpoint, PFS, is statistically significantly different between the two treatment groups.
| months | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Overall Survival (OS) | 21.9 (18.8 to 30.4) | 19.7 (14.2 to 30.0) | 40.0 (31.3 to NA) | 31.1 (23.4 to NA) |
For CTCAE data, the frequency tables will be reviewed to determine the patterns. The overall adverse event rates will be compared between treatment groups using Chi-square test (or Fisher's exact test if the data in contingency table is sparse). PRO-CTCAE data will, at minimum, be analyzed similarly to CTCAE data. The initial analysis of each PRO-CTCAE item will use all available scores in an analysis which mirrors the approach used for the CTCAE data. Supplemental analysis will use model-based multiple imputation incorporating baseline patient characteristics and physician-rated performance status. CTCAE data may be incorporated as auxiliary data into multiple imputation models for AEs which are captured by both PRO-CTCAE and CTCAE. Results from supplemental analysis will be descriptively compared to the results of the initial analysis to assess the robustness of results to missing data. Additional analyses of PRO-CTCAE data beyond those specified above may be undertaken.
| Participants | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Number of Patients Experiencing Grade 3+ Adverse Events (AEs) Graded According to the Common Terminology Criteria for Adverse Events (CTCAE) and Patient-Reported Outcomes Version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) | 104 | 47 | 49 | 22 |
Will be defined as the proportion of patients in each arm whose best response is either complete response (CR) or partial response (PR). Radiographic response rate for both cohorts: the analyses for confirmed radiographic response rate will follow the ITT principle and will be conducted separately within each cohort (pancreatic NET and carcinoid tumor). The proportion of patients with either confirmed CR or confirmed PR as their best response will be estimated using point estimates and 95% confidence intervals. Radiographic response rate will be compared between treatment arms using the 2-sample z-test to compare sample proportion at a one-sided 2.5% level of significance.
| Participants | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Radiographic Response Rate | 7 | 0 | 12 | 0 |
Collected over 60 months. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | 60/134 (44.8%) | 89/134 (66.4%) | 134/134 (100%) |
| Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | 37/69 (53.6%) | 41/69 (59.4%) | 69/69 (100%) |
| Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | 21/64 (32.8%) | 49/64 (76.6%) | 64/64 (100%) |
| Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | 11/31 (35.5%) | 21/31 (67.7%) | 31/31 (100%) |
| Event | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| HypertensionVascular disorders | 30/134 | 5/69 | 16/64 | 8/31 |
| FatigueGeneral disorders | 15/134 | 7/69 | 10/64 | 4/31 |
| Abdominal painGastrointestinal disorders | 9/134 | 5/69 | 4/64 | 4/31 |
| Thromboembolic eventVascular disorders | 2/134 | 2/69 | 8/64 | 0/31 |
| DiarrheaGastrointestinal disorders | 15/134 | 5/69 | 4/64 | 0/31 |
| Lymphocyte count decreasedInvestigations | 13/134 | 1/69 | 2/64 | 0/31 |
| HyperglycemiaMetabolism and nutrition disorders | 0/134 | 2/69 | 5/64 | 3/31 |
| Small intestinal obstructionGastrointestinal disorders | 2/134 | 1/69 | 1/64 | 3/31 |
| Mucositis oralGastrointestinal disorders | 4/134 | 1/69 | 5/64 | 0/31 |
| NauseaGastrointestinal disorders | 3/134 | 1/69 | 5/64 | 2/31 |
| Event | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) |
|---|---|---|---|---|
| Aspartate aminotransferase increasedInvestigations | 96/134 | 17/69 | 50/64 | 15/31 |
| FatigueGeneral disorders | 103/134 | 42/69 | 48/64 | 17/31 |
| Alanine aminotransferase increasedInvestigations | 89/134 | 15/69 | 49/64 | 12/31 |
| DiarrheaGastrointestinal disorders | 82/134 | 31/69 | 40/64 | 10/31 |
| Platelet count decreasedInvestigations | 74/134 | 8/69 | 24/64 | 6/31 |
| HypertensionVascular disorders | 65/134 | 27/69 | 31/64 | 11/31 |
| HyperglycemiaMetabolism and nutrition disorders | 50/134 | 25/69 | 26/64 | 15/31 |
| Mucositis oralGastrointestinal disorders | 48/134 | 6/69 | 26/64 | 2/31 |
| NauseaGastrointestinal disorders | 53/134 | 13/69 | 21/64 | 8/31 |
| Palmar-plantar erythrodysesthesia syndrmSkin and subcutaneous tissue disorders | 47/134 | 5/69 | 24/64 | 5/31 |
| Age, Continuous(years) | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | Total |
|---|---|---|---|---|---|
| Median | 66 (28 to 86) | 66 (30 to 82) | 59.5 (29 to 79) | 64 (39 to 79) | 66 (28 to 86) |
| Sex: Female, Male(Participants) | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | Total |
|---|---|---|---|---|---|
| Female | 74 | 31 | 27 | 13 | 145 |
| Male | 60 | 38 | 37 | 18 | 153 |
| Ethnicity (NIH/OMB)(Participants) | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | Total |
|---|---|---|---|---|---|
| Hispanic or Latino | 8 | 9 | 2 | 2 | 21 |
| Not Hispanic or Latino | 125 | 56 | 61 | 26 | 268 |
| Unknown or Not Reported | 1 | 4 | 1 | 3 | 9 |
| Race (NIH/OMB)(Participants) | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | Total |
|---|---|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 1 | 0 | 1 |
| Asian | 3 | 1 | 4 | 0 | 8 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 1 | 0 | 1 |
| Black or African American | 9 | 7 | 3 | 3 | 22 |
| White | 115 | 55 | 54 | 25 | 249 |
| More than one race | 0 | 0 | 1 | 0 | 1 |
| Unknown or Not Reported | 7 | 6 | 0 | 3 | 16 |
| Region of Enrollment(participants) | Arm Ia (Cabozantinib S-malate, Extra-pancreatic NET Cohort [epNET]) | Arm IIa (Placebo, Extra-pancreatic NET Cohort [epNET]) | Arm Ib (Cabozantinib S-malate, Pancreatic NET Cohort [pNET]) | Arm IIb (Placebo, Pancreatic NET Cohort [pNET]) | Total |
|---|---|---|---|---|---|
| United States | 134 | 69 | 64 | 31 | 298 |
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Ran from Oct 2018 to primary completion in Aug 2023, 57 months.
Results were first posted in Oct 2024, 1 year 1 month after primary completion.
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Studies of the drug tested here: Cabozantinib
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