Symptoms and General Pathology

All Conditions

Neoplasms

Rare Diseases

Recurrence

Sarcoma

Hemangiosarcoma

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Vascular Tissue

Soft Tissue Sarcoma

Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

regorafenib

Mark Agulnik, M.D.

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.

Daily Oral Regorafenib for Chemotherapy-Refractory, Metastatic and Locally Advanced Angiosarcoma

Multicenter, Open-Label Phase II Study of Daily Oral Regorafenib for Chemotherapy-Refractory, Metastatic and Locally Advanced Angiosarcoma

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.

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.

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.

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.

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

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

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

Bayer

National Cancer Institute (NCI)

Northwestern University

Due to data collection methods, some serious adverse event (SAEs) data maybe captured within the other adverse event data information. There are patients currently on treatment and these sections will be updated with complete information at the study completion. SAEs for any patients that were screenfails (i.e., signed consent but never started study treatment) have been removed from these results. These patients were not included in the participant flow and baseline characteristics section.

Patients receive regorafenib PO QD on days 1-21. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

regorafenib: Given PO

Treatment (Regorafenib)

Age, Customized

Sex: Female, Male

Ethnicity (NIH/OMB)

Race (NIH/OMB)

United States

Region of Enrollment

Type of disease

This includes patients that started study treatment. Any patients who signed consent but never initiated treatment (screenfails) are not included.

Mary Mulcahy, MD

8 Patients were not included due to the following reasons: progressive disease, patient refused further treatment (withdrawal of consent for further treatment), toxicity. This was due to data having either missing time points or "cycle 1" or "Cycle 1: follow up" missing from the dataset at the time of the analysis.

Progression-Free Survival (PFS) at 4 Months

3 months

6 months

Progression-Free Survival (PFS) at 3 and 6 Months

Median Progression-free Survival (PFS)

Overall Survival

Complete Response

Partial Response

Response Rate

Stable Disease

Rate of Tumor Control

Blood and lymphatic system disorders

Endocrine disorders

Eye disorders

Gastrointestinal disorders

General disorders

Infections and infestations

Injury, poisoning and procedural complications

Metabolism and nutrition disorders

Investigations

musculoskeletal and connective tissue disorders

Neoplasms benign, malignant and unspecified

Nervous system disorders

Psychiatric disorders

Renal and urinary disorders

Respiratory, thoracic and mediastinal disorders

Skin and subcutaneous tissue disorders

Vascular disorders

At the time of data pull (12/02/2019) for this outcome measure two patients remained on treatment. Any subsequent AEs determined to be related to treatment for these patients is not included here.

Spots Global Cancer Trial Database for Daily Oral Regorafenib for Chemotherapy-Refractory, Metastatic and Locally Advanced Angiosarcoma

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

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