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

Participant Flow

Recruitment Details	Participants were enrolled at three sites between February and December, 2010.
Pre-assignment Details


Arm/Group Title	Regorafenib
Arm/Group Description	Regorafenib adminstered orally, 160...
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

Baseline Characteristics

Arm/Group Title		Regorafenib
Arm/Group Description		Regorafenib adminstered orally, 160...
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
Baseline Analysis Population Description		...
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
United States		33

Outcome Measures

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
Description	The composite of complete response, partial response, and stable disea...
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

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Regorafenib
Arm/Group Description:	Regorafenib adminstered orally, 160...
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)
Description	Progression-free survival is defined as the duration of time from star...
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

Outcome Measure Data

Analysis Population Description

[Not Specified]


Arm/Group Title	Regorafenib
Arm/Group Description:	Regorafenib adminstered orally, 160...
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)

Adverse Events

Time Frame	[Not Specified]
Adverse Event Reporting Description	[Not Specified]

Arm/Group Title	Regorafenib
Arm/Group Description	Regorafenib adminstered orally, 160...
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	--/--
Serious Adverse Events 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)
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)

Limitations and Caveats

[Not Specified]

More Information

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

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Ben-Ami E, Barysauskas CM, von Mehren M, Heinrich MC, Corless CL, Butrynski JE, Morgan JA, Wagner AJ, Choy E, Yap JT, Van den Abbeele AD, Solomon SM, Fletcher JA, Demetri GD, George S. Long-term follow-up results of the multicenter phase II trial of regorafenib in patients with metastatic and/or unresectable GI stromal tumor after failure of standard tyrosine kinase inhibitor therapy. Ann Oncol. 2016 Sep;27(9):1794-9. doi: 10.1093/annonc/mdw228. Epub 2016 Jul 1.

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

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