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Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor

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ClinicalTrials.gov Identifier: NCT01068769
Recruitment Status : Completed
First Posted : February 15, 2010
Results First Posted : February 24, 2014
Last Update Posted : August 24, 2020
Sponsor:
Collaborators:
Brigham and Women's Hospital
Massachusetts General Hospital
Fox Chase Cancer Center
Oregon Health and Science University
Bayer
Information provided by (Responsible Party):
Suzanne George, MD, Dana-Farber/Brigham and Women's Cancer Center

Study Type Interventional
Study Design Allocation: N/A;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Gastrointestinal Stromal Tumor
Intervention Drug: regorafenib
Enrollment 34
Recruitment Details Participants were enrolled at three sites between February and December, 2010.
Pre-assignment Details  
Arm/Group Title Regorafenib
Hide Arm/Group Description Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
Period Title: Overall Study
Started 34
Completed 33
Not Completed 1
Reason Not Completed
Ineligible             1
Arm/Group Title Regorafenib
Hide Arm/Group Description Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
Overall Number of Baseline Participants 33
Hide Baseline Analysis Population Description
Eligible patients were included in the baseline analysis population
Age, Continuous  
Median (Full Range)
Unit of measure:  Years
Number Analyzed 33 participants
56
(25 to 76)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 33 participants
Female
14
  42.4%
Male
19
  57.6%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 33 participants
33
1.Primary Outcome
Title Clinical Benefit as Defined by the Composite of Complete Response, Partial Response and Stable Disease Lasting 16 Weeks or More Per RECIST 1.1 as a Measure of Disease Control
Hide Description The composite of complete response, partial response, and stable disease lasting 16 weeks or more per RECIST 1.1 as a measure of disease control. This is for target lesions. Complete response is disappearance of all target lesions and partial response is >+30% decrease in the sum of the longest diameter of target lesions. Stable disease is neither shrinkage by greater than or equal to 30% of the sum of the longest diameter of target lesions or the increase of lesions by greater than or equal to 20% of the sum of the longest diameter of target lesions. Progressive disease is considered an increase of the sum of the longest diameter of target lesions by greater than or equal to 20%.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Regorafenib
Hide Arm/Group Description:
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
Overall Number of Participants Analyzed 33
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
75
(61 to 91)
2.Secondary Outcome
Title Progression-free Survival (PFS)
Hide Description Progression-free survival is defined as the duration of time from start of study drug administration to time of objective disease progression or death due to any cause, whichever comes first. Progression is evaluated every 8 weeks using Response Criteria for Solid Tumors (RECIST) 1.1. Objective disease progression is defined as a 20% increase in the sum of the longest diameter of target lesion(s).
Time Frame From date of enrollment until date of first documented progression or date of death from any cause, whichever came first
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Regorafenib
Hide Arm/Group Description:
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
Overall Number of Participants Analyzed 33
Median (95% Confidence Interval)
Unit of Measure: months
10.0
(8.3 to 14.9)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Regorafenib
Hide Arm/Group Description Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
All-Cause Mortality
Regorafenib
Affected / at Risk (%)
Total   --/-- 
Hide Serious Adverse Events
Regorafenib
Affected / at Risk (%)
Total   31/33 (93.94%) 
Cardiac disorders   
Acute coronary syndrome  1  1/33 (3.03%) 
Gastrointestinal disorders   
Abdominal pain  1  4/33 (12.12%) 
Colitis  1  8/33 (24.24%) 
Diarrhea  1  3/33 (9.09%) 
Nausea  1  2/33 (6.06%) 
Vomiting  1  1/33 (3.03%) 
Gastrointestinal disorders - Other, specify  1  3/33 (9.09%) 
General disorders   
Fatigue  1  2/33 (6.06%) 
Investigations   
Blood bilirubin increased  1  1/33 (3.03%) 
CD4 lymphocytes decreased  1  1/33 (3.03%) 
Lipase increased  1  2/33 (6.06%) 
Lymphocyte count decreased  1  1/33 (3.03%) 
Neutrophil count decreased  1  1/33 (3.03%) 
Serum amylase increased  1  1/33 (3.03%) 
Weight loss  1  2/33 (6.06%) 
Metabolism and nutrition disorders   
Anorexia  1  1/33 (3.03%) 
Hyperuricemia  1  2/33 (6.06%) 
Hyponatremia  1  1/33 (3.03%) 
Hypophosphatemia  1  6/33 (18.18%) 
Musculoskeletal and connective tissue disorders   
Back pain  1  2/33 (6.06%) 
Buttock pain  1  2/33 (6.06%) 
Myalgia  1  2/33 (6.06%) 
Pain in extremity  1  2/33 (6.06%) 
Psychiatric disorders   
Insomnia  1  2/33 (6.06%) 
Skin and subcutaneous tissue disorders   
Palmar-plantar erythrodysesthesia syndrome  1  12/33 (36.36%) 
Pruritus  1  1/33 (3.03%) 
Rash maculo-papular  1  4/33 (12.12%) 
Vascular disorders   
Hypertension  1  13/33 (39.39%) 
Thromboembolic event  1  1/33 (3.03%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Regorafenib
Affected / at Risk (%)
Total   33/33 (100.00%) 
Blood and lymphatic system disorders   
Leukocytosis  1  3/33 (9.09%) 
Anemia  1  9/33 (27.27%) 
Blood and lymphatic system disorders - Other, specify  1  3/33 (9.09%) 
Endocrine disorders   
Hyperthyroidism  1  3/33 (9.09%) 
Hypothyroidism  1  5/33 (15.15%) 
Eye disorders   
Eye disorders - Other, specify  1  3/33 (9.09%) 
Gastrointestinal disorders   
Bloating  1  2/33 (6.06%) 
Oral dysesthesia  1  6/33 (18.18%) 
Abdominal distension  1  4/33 (12.12%) 
Abdominal pain  1  8/33 (24.24%) 
Colitis  1  3/33 (9.09%) 
Constipation  1  9/33 (27.27%) 
Diarrhea  1  23/33 (69.70%) 
Dry mouth  1  3/33 (9.09%) 
Dyspepsia  1  8/33 (24.24%) 
Flatulence  1  7/33 (21.21%) 
Mucositis oral  1  14/33 (42.42%) 
Nausea  1  13/33 (39.39%) 
Oral hemorrhage  1  2/33 (6.06%) 
Oral pain  1  3/33 (9.09%) 
Stomach pain  1  2/33 (6.06%) 
Vomiting  1  9/33 (27.27%) 
Gastrointestinal disorders - Other, specify  1  19/33 (57.58%) 
General disorders   
Chills  1  2/33 (6.06%) 
Edema limbs  1  2/33 (6.06%) 
Fatigue  1  26/33 (78.79%) 
Pain  1  2/33 (6.06%) 
Infections and infestations   
Mucosal infection  1  2/33 (6.06%) 
Pharyngitis  1  2/33 (6.06%) 
Skin infection  1  2/33 (6.06%) 
Infections and infestations - Other, specify  1  2/33 (6.06%) 
Investigations   
Alanine aminotransferase increased  1  2/33 (6.06%) 
Alkaline phosphatase increased  1  3/33 (9.09%) 
Aspartate aminotransferase increased  1  6/33 (18.18%) 
Blood bilirubin increased  1  4/33 (12.12%) 
Creatinine increased  1  2/33 (6.06%) 
Lipase increased  1  6/33 (18.18%) 
Neutrophil count decreased  1  2/33 (6.06%) 
Platelet count decreased  1  4/33 (12.12%) 
Serum amylase increased  1  3/33 (9.09%) 
Weight loss  1  7/33 (21.21%) 
White blood cell decreased  1  3/33 (9.09%) 
Metabolism and nutrition disorders   
Anorexia  1  13/33 (39.39%) 
Dehydration  1  4/33 (12.12%) 
Hyperglycemia  1  2/33 (6.06%) 
Hyperkalemia  1  2/33 (6.06%) 
Hypermagnesemia  1  2/33 (6.06%) 
Hypoalbuminemia  1  2/33 (6.06%) 
Hypocalcemia  1  5/33 (15.15%) 
Hypomagnesemia  1  6/33 (18.18%) 
Hypophosphatemia  1  10/33 (30.30%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  10/33 (30.30%) 
Myalgia  1  16/33 (48.48%) 
Pain in extremity  1  5/33 (15.15%) 
Musculoskeletal and connective tissue disorder - Other, specify  1  3/33 (9.09%) 
Nervous system disorders   
Dizziness  1  2/33 (6.06%) 
Dysgeusia  1  8/33 (24.24%) 
Headache  1  15/33 (45.45%) 
Renal and urinary disorders   
Urinary frequency  1  2/33 (6.06%) 
Respiratory, thoracic and mediastinal disorders   
Hoarseness  1  18/33 (54.55%) 
Laryngeal inflammation  1  2/33 (6.06%) 
Sore throat  1  4/33 (12.12%) 
Dyspnea  1  4/33 (12.12%) 
Skin and subcutaneous tissue disorders   
Alopecia  1  10/33 (30.30%) 
Dry skin  1  3/33 (9.09%) 
Palmar-plantar erythrodysesthesia syndrome  1  18/33 (54.55%) 
Pruritus  1  3/33 (9.09%) 
Rash acneiform  1  3/33 (9.09%) 
Rash maculo-papular  1  4/33 (12.12%) 
Scalp pain  1  3/33 (9.09%) 
Skin hyperpigmentation  1  2/33 (6.06%) 
Skin and subcutaneous tissue disorders - Other, specify  1  4/33 (12.12%) 
Vascular disorders   
Flushing  1  2/33 (6.06%) 
Hot flashes  1  3/33 (9.09%) 
Hypertension  1  12/33 (36.36%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Suzanne George
Organization: Dana-Farber Cancer Institute
Phone: 617-632-5204
EMail: sgeorge2@partners.org
Layout table for additonal information
Responsible Party: Suzanne George, MD, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier: NCT01068769    
Other Study ID Numbers: 09-400
First Submitted: February 12, 2010
First Posted: February 15, 2010
Results First Submitted: January 9, 2014
Results First Posted: February 24, 2014
Last Update Posted: August 24, 2020