A Phase 2 interventional study of regorafenib in Adult Angiosarcoma, Recurrent Adult Soft Tissue Sarcoma and Stage III Adult Soft Tissue Sarcoma, sponsored by Northwestern University. Completed at 7 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-04-25.
Sponsored by Northwestern University · Phase 2, Interventional, and Treatment
The purpose of this study is to see whether a drug called regorafenib might be effective in treating angiosarcoma. This study is for patients who have angiosarcoma that has gotten worse after they received chemotherapy. Regorafenib is a type of drug called a kinase inhibitor. Regorafenib interferes with how some kinase proteins work. Some of these kinases in cancer cells might normally help the cancer cells grow or form new blood vessels that could feed a growing tumor. By blocking these proteins, regorafenib may help stop the growth of certain cancers.
PRIMARY OBJECTIVES:
I. To define the progression-free survival (PFS) at 4 months with daily oral regorafenib (160 mg) in previously treated locally advanced/metastatic angiosarcoma patients
SECONDARY OBJECTIVES:
I. Progression-free rate at 3 and 6 months. II. Progression-free survival. III. Overall survival (up to 5 years). IV. Response rate (by Response Evaluation Criteria in Solid Tumors [RECIST] version [v] 1.1).
V. Rate and duration of tumor control (complete response [CR] + partial response [PR] + stable disease [SD]).
VI. Safety/tolerability of regorafenib.
OUTLINE:
Patients receive regorafenib orally (PO) once daily (QD) on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months for up to 5 years.
99 studies on the registry are indexed under Hemangiosarcoma; 16 are open to participants now.
This study's enrollment of 31 is below the median of 64 across 83 interventional studies indexed under Hemangiosarcoma.
Browse Hemangiosarcoma studies →Northwestern University is the lead sponsor of 1,423 studies on the registry; 198 are open to participants now.
Of its 105 completed or terminated interventional studies of FDA-regulated products, 78 (74%) 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.
Exclusion Criteria:
Active or cardiac disease including:
Therapeutic anticoagulation with Vitamin-K antagonists (e.g., warfarin) or with heparins and heparinoids; NOTE: Prophylactic anticoagulation as described below is allowed:
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.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
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.
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.
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 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.
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.
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.
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 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).
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.
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).
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.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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 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.
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.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
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.
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.
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.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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 regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
Drug: regorafenib
Given PO
Also known as: BAY 73-4506, multikinase inhibitor BAY 73-4506, Stivarga
Progression-Free Survival (PFS) at 4 Months
The progression-free survival (PFS) at 4 months will be defined as the number of patients with progression absent at 4 months divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first and will be estimated using Kaplan-Meier methods and reported as a survival probability. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
Time frame: At 4 months (of treatment)
Progression-Free Survival (PFS) at 3 and 6 Months
The progression-free survival (PFS) at 3 and 6 months will be defined as the number of patients with progression absent at 3 months and 6 months divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first and will be estimated using Kaplan-Meier methods and reported as a survival probability. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
Time frame: Assessed at 3 months and 6 months
Median Progression-free Survival (PFS)
The median progression-free rate (PFR) will be defined as the number of patients with progression absent divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first for up to 5 years and will be estimated using Kaplan-Meier methods. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
Time frame: The duration of time from start of treatment until time of progression, up to 5 years
Overall Survival
Overall survival will be defined as the time from start of treatment until death from any cause and will be estimated using Kaplan-Meier methods and reported as a survival probability. Patients that are alive at the time of data analysis will be censored at the date of known survival status.
Time frame: From start of treatment up to 5 years
Response Rate
Imaging (such as a CT scan) will be done at baseline (within 4 weeks before the first dose) then after every 2 cycles while on treatment for tumor response evaluation. Response will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Best response of each patient will be used and responses will be defined as the following: Complete response (CR): Disappearance of all target lesions Partial response (PR): \> 30% decrease in the baseline sum diameters of target lesions
Time frame: At baseline and after every 2 cycles, up to 12 cycles where one cycle is 28 days
Rate of Tumor Control
Imaging will be used at baseline then after every 2 cycles while on treatment to measure tumor size and response to treatment, the data collected from all patients on study will be used to calculate the rate and duration of tumor control. Response will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Response and stable disease will be defined as the following: Complete response (CR): Disappearance of all target lesions Partial response (PR): \> 30% decrease in the baseline sum diameters of target lesions Stable disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study
Time frame: At baseline and after every 2 cycles, up to 12 cycles where one cycle is 28 days
Number of Grade 1, 2, 3, 4, and 5 Related Adverse Events Observed During Study Treatment (Defined by CTCAE v 4.0)
Overall worst grade related toxicity (number of patients) was collected from the start of treatment until 30 days post the last treatment where patients were treated until progressive disease or unacceptable toxicity or patient withdrawal of treatment. All Adverse events that were determined to be at least possibly related to treatment are reported. All adverse events will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. The severity of an AE is graded as follows: Mild (grade 1): the event causes discomfort without disruption of normal daily activities. Moderate (grade 2): the event causes discomfort that affects normal daily activities. Severe (grade 3): the event makes the patient unable to perform normal daily activities or significantly affects his/her clinical status. Life-threatening (grade 4): the patient was at risk of death at the time of the event. Fatal (grade 5): the event caused death
Time frame: From treatment initiation though 30 days post the last treatment for a max of 12 cycles where one cycle is 28 days
The study was opened for enrollment March 12, 2014 with the first patient starting treatment June 13, 2014. A total of 31 patients were treated under the protocol. The study was permanently suspended to further enrollment August 5, 2019.
| Milestone | Treatment (Regorafenib) |
|---|---|
| Started | 31 |
| Attempted 1st cycle | 31 |
| Attempted 2nd cycle | 23 |
| Completed | 23 |
| Not completed | 8 |
| Withdrew: Adverse event | 2 |
| Withdrew: Withdrawal by subject | 4 |
| Withdrew: Progressive disease | 2 |
| Milestone | Treatment (Regorafenib) |
|---|---|
| Started | 23 |
| Assessed for first response | 23 |
| Went on to cycle 3 and beyond | 13 |
| Completed | 13 |
| Not completed | 10 |
| Withdrew: Progressive disease | 10 |
| Milestone | Treatment (Regorafenib) |
|---|---|
| Started | 31 |
| Completed | 4 |
| Not completed | 27 |
| Withdrew: Death | 24 |
| Withdrew: Withdrawal by subject | 3 |
The progression-free survival (PFS) at 4 months will be defined as the number of patients with progression absent at 4 months divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first and will be estimated using Kaplan-Meier methods and reported as a survival probability. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
| percentage of patients alive | Treatment (Regorafenib) |
|---|---|
| Progression-Free Survival (PFS) at 4 Months | 52.17 (35.28 to 77.16) |
The progression-free survival (PFS) at 3 and 6 months will be defined as the number of patients with progression absent at 3 months and 6 months divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first and will be estimated using Kaplan-Meier methods and reported as a survival probability. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
| percentage patients alive | Treatment (Regorafenib) |
|---|---|
| 3 months | 56.52 (39.5 to 80.89) |
| 6 months | 47.83 (31.21 to 73.29) |
The median progression-free rate (PFR) will be defined as the number of patients with progression absent divided by the total number of evaluable study patients. This will be measured from start of treatment until time of progression or death, whichever occurs first for up to 5 years and will be estimated using Kaplan-Meier methods. Progression will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Progression will be defined as: Progressive disease (PD): \> 20% increase in the sum diameters of target lesions, taking as reference the smallest sum diameters while on study. The sum diameters must also demonstrate an absolute increase of 5 mm. The appearance of one or more new lesions also qualifies as PD.
| months | Treatment (Regorafenib) |
|---|---|
| Median Progression-free Survival (PFS) | 5.49 (1.84 to 9.53) |
Overall survival will be defined as the time from start of treatment until death from any cause and will be estimated using Kaplan-Meier methods and reported as a survival probability. Patients that are alive at the time of data analysis will be censored at the date of known survival status.
| months | Treatment (Regorafenib) |
|---|---|
| Overall Survival | 14.06 (9.76 to 22.24) |
Imaging (such as a CT scan) will be done at baseline (within 4 weeks before the first dose) then after every 2 cycles while on treatment for tumor response evaluation. Response will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Best response of each patient will be used and responses will be defined as the following: Complete response (CR): Disappearance of all target lesions Partial response (PR): \> 30% decrease in the baseline sum diameters of target lesions
| participants | Treatment (Regorafenib) |
|---|---|
| Complete Response | 2 |
| Partial Response | 2 |
Imaging will be used at baseline then after every 2 cycles while on treatment to measure tumor size and response to treatment, the data collected from all patients on study will be used to calculate the rate and duration of tumor control. Response will be evaluated using the international criteria proposed by the Response Evaluation Criteria in Solid Tumors (RECIST) v 1.1 Committee. Changes in only the largest diameter (unidimensional measurement) of the tumor lesions are used in RECIST v 1.1. Response and stable disease will be defined as the following: Complete response (CR): Disappearance of all target lesions Partial response (PR): \> 30% decrease in the baseline sum diameters of target lesions Stable disease (SD): Neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study
| patients | Treatment (Regorafenib) |
|---|---|
| Complete Response | 2 |
| Partial Response | 2 |
| Stable Disease | 9 |
Overall worst grade related toxicity (number of patients) was collected from the start of treatment until 30 days post the last treatment where patients were treated until progressive disease or unacceptable toxicity or patient withdrawal of treatment. All Adverse events that were determined to be at least possibly related to treatment are reported. All adverse events will be graded according to the NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0. The severity of an AE is graded as follows: Mild (grade 1): the event causes discomfort without disruption of normal daily activities. Moderate (grade 2): the event causes discomfort that affects normal daily activities. Severe (grade 3): the event makes the patient unable to perform normal daily activities or significantly affects his/her clinical status. Life-threatening (grade 4): the patient was at risk of death at the time of the event. Fatal (grade 5): the event caused death
| Participants | Treatment (Regorafenib) |
|---|---|
| Blood and lymphatic system disorders — Grade 1 | 5 |
| Blood and lymphatic system disorders — Grade 2 | 3 |
| Blood and lymphatic system disorders — Grade 3 | 3 |
| Blood and lymphatic system disorders — Grade 4 | 0 |
| Blood and lymphatic system disorders — Number of patients who didn't experience any grade | 20 |
| Endocrine disorders — Grade 1 | 1 |
| Endocrine disorders — Grade 2 | 0 |
| Endocrine disorders — Grade 3 | 0 |
| Endocrine disorders — Grade 4 | 0 |
| Endocrine disorders — Number of patients who didn't experience any grade | 30 |
| Eye disorders — Grade 1 | 2 |
| Eye disorders — Grade 2 | 0 |
| Eye disorders — Grade 3 | 0 |
| Eye disorders — Grade 4 | 0 |
| Eye disorders — Number of patients who didn't experience any grade | 29 |
| Gastrointestinal disorders — Grade 1 | 14 |
| Gastrointestinal disorders — Grade 2 | 9 |
| Gastrointestinal disorders — Grade 3 | 3 |
| Gastrointestinal disorders — Grade 4 | 0 |
| Gastrointestinal disorders — Number of patients who didn't experience any grade | 5 |
| General disorders — Grade 1 | 11 |
| General disorders — Grade 2 | 8 |
| General disorders — Grade 3 | 4 |
| General disorders — Grade 4 | 0 |
| General disorders — Number of patients who didn't experience any grade | 8 |
| Infections and infestations — Grade 1 | 1 |
| Infections and infestations — Grade 2 | 2 |
| Infections and infestations — Grade 3 | 0 |
| Infections and infestations — Grade 4 | 0 |
| Infections and infestations — Number of patients who didn't experience any grade | 28 |
| Injury, poisoning and procedural complications — Grade 1 | 0 |
| Injury, poisoning and procedural complications — Grade 2 | 1 |
| Injury, poisoning and procedural complications — Grade 3 | 0 |
| Injury, poisoning and procedural complications — Grade 4 | 0 |
| Injury, poisoning and procedural complications — Number of patients who didn't experience any grade | 30 |
| Metabolism and nutrition disorders — Grade 1 | 10 |
| Metabolism and nutrition disorders — Grade 2 | 4 |
| Metabolism and nutrition disorders — Grade 3 | 5 |
| Metabolism and nutrition disorders — Grade 4 | 0 |
| Metabolism and nutrition disorders — Number of patients who didn't experience any grade | 12 |
| Investigations — Grade 1 | 14 |
| Investigations — Grade 2 | 2 |
| Investigations — Grade 3 | 3 |
| Investigations — Grade 4 | 1 |
| Investigations — Number of patients who didn't experience any grade | 11 |
| musculoskeletal and connective tissue disorders — Grade 1 | 7 |
| musculoskeletal and connective tissue disorders — Grade 2 | 3 |
| musculoskeletal and connective tissue disorders — Grade 3 | 1 |
| musculoskeletal and connective tissue disorders — Grade 4 | 0 |
| musculoskeletal and connective tissue disorders — Number of patients who didn't experience any grade | 20 |
| Neoplasms benign, malignant and unspecified — Grade 1 | 1 |
| Neoplasms benign, malignant and unspecified — Grade 2 | 0 |
| Neoplasms benign, malignant and unspecified — Grade 3 | 0 |
| Neoplasms benign, malignant and unspecified — Grade 4 | 0 |
| Neoplasms benign, malignant and unspecified — Number of patients who didn't experience any grade | 30 |
| Nervous system disorders — Grade 1 | 11 |
| Nervous system disorders — Grade 2 | 2 |
| Nervous system disorders — Grade 3 | 1 |
| Nervous system disorders — Grade 4 | 0 |
| Nervous system disorders — Number of patients who didn't experience any grade | 17 |
| Psychiatric disorders — Grade 1 | 3 |
| Psychiatric disorders — Grade 2 | 1 |
| Psychiatric disorders — Grade 3 | 2 |
| Psychiatric disorders — Grade 4 | 0 |
| Psychiatric disorders — Number of patients who didn't experience any grade | 25 |
| Renal and urinary disorders — Grade 1 | 0 |
| Renal and urinary disorders — Grade 2 | 1 |
| Renal and urinary disorders — Grade 3 | 0 |
| Renal and urinary disorders — Grade 4 | 0 |
| Renal and urinary disorders — Number of patients who didn't experience any grade | 30 |
| Respiratory, thoracic and mediastinal disorders — Grade 1 | 9 |
| Respiratory, thoracic and mediastinal disorders — Grade 2 | 1 |
| Respiratory, thoracic and mediastinal disorders — Grade 3 | 1 |
| Respiratory, thoracic and mediastinal disorders — Grade 4 | 0 |
| Respiratory, thoracic and mediastinal disorders — Number of patients who didn't experience any grade | 20 |
| Skin and subcutaneous tissue disorders — Grade 1 | 14 |
| Skin and subcutaneous tissue disorders — Grade 2 | 6 |
| Skin and subcutaneous tissue disorders — Grade 3 | 1 |
| Skin and subcutaneous tissue disorders — Grade 4 | 0 |
| Skin and subcutaneous tissue disorders — Number of patients who didn't experience any grade | 10 |
| Vascular disorders — Grade 1 | 3 |
| Vascular disorders — Grade 2 | 8 |
| Vascular disorders — Grade 3 | 4 |
| Vascular disorders — Grade 4 | 0 |
| Vascular disorders — Number of patients who didn't experience any grade | 16 |
Collected over Adverse events were collected for each patient from the initiation of treatment up until 30 days post last dose of treatment for a maximum of 12 cycles for any patient (where 1 Cycle = 28 days).. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment (Regorafenib) | 24/31 (77.4%) | 15/31 (48.4%) | 31/31 (100%) |
| Event | Treatment (Regorafenib) |
|---|---|
| Disease progressionNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/31 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 1/31 |
| Pleural EffusionRespiratory, thoracic and mediastinal disorders | 1/31 |
| Intractable painGeneral disorders | 1/31 |
| Vascular DisorderVascular disorders | 1/31 |
| Intra-abdominal hemorrhageGastrointestinal disorders | 1/31 |
| Peripheral sensory neuropathyNervous system disorders | 1/31 |
| FeverGeneral disorders | 1/31 |
| NauseaGastrointestinal disorders | 1/31 |
| NauseaGastrointestinal disorders | 1/31 |
| Event | Treatment (Regorafenib) |
|---|---|
| FatigueGeneral disorders | 23/31 |
| HypertensionVascular disorders | 17/31 |
| DiarrheaGastrointestinal disorders | 16/31 |
| NauseaGastrointestinal disorders | 16/31 |
| Lymphocyte count decreasedInvestigations | 16/31 |
| HypoalbuminemiaMetabolism and nutrition disorders | 16/31 |
| AnemiaBlood and lymphatic system disorders | 14/31 |
| AnorexiaMetabolism and nutrition disorders | 13/31 |
| HypocalcemiaMetabolism and nutrition disorders | 13/31 |
| ConstipationGastrointestinal disorders | 12/31 |
This includes patients that started study treatment. Any patients who signed consent but never initiated treatment (screenfails) are not included.
| Age, Customized(years) | Treatment (Regorafenib) |
|---|---|
| Mean | 64.32 ± 14.58 |
| Sex: Female, Male(Participants) | Treatment (Regorafenib) |
|---|---|
| Female | 16 |
| Male | 15 |
| Ethnicity (NIH/OMB)(Participants) | Treatment (Regorafenib) |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 31 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | Treatment (Regorafenib) |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 3 |
| White | 27 |
| More than one race | 0 |
| Unknown or Not Reported | 1 |
| Region of Enrollment(participants) | Treatment (Regorafenib) |
|---|---|
| United States | 31 |
| Type of disease(Participants) | Treatment (Regorafenib) |
|---|---|
| Metastatic | 21 |
| Locally advanced/Unresectable | 9 |
| Not reported | 1 |
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Studies of the drug tested here: Regorafenib