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

This study is ongoing, but not recruiting participants.
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
ClinicalTrials.gov Identifier:
NCT01068769
First received: February 12, 2010
Last updated: September 12, 2016
Last verified: September 2016
Results First Received: January 9, 2014  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Gastrointestinal Stromal Tumor
Intervention: Drug: regorafenib

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were enrolled at three sites between February and December, 2010.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Regorafenib Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle

Participant Flow:   Overall Study
    Regorafenib
STARTED   34 
COMPLETED   33 
NOT COMPLETED   1 
Ineligible                1 



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Eligible patients were included in the baseline analysis population

Reporting Groups
  Description
Regorafenib Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle

Baseline Measures
   Regorafenib 
Overall Participants Analyzed 
[Units: Participants]
 33 
Age 
[Units: Years]
Median (Full Range)
 56 
 (25 to 76) 
Gender 
[Units: Participants]
 
Female   14 
Male   19 
Region of Enrollment 
[Units: Participants]
 
United States   33 


  Outcome Measures
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1.  Primary:   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   [ Time Frame: 2 years ]

2.  Secondary:   Progression-free Survival (PFS)   [ Time Frame: From date of enrollment until date of first documented progression or date of death from any cause, whichever came first ]


  Serious Adverse Events


  Other Adverse Events
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Time Frame No text entered.
Additional Description No text entered.

Frequency Threshold
Threshold above which other adverse events are reported   5  

Reporting Groups
  Description
Regorafenib Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle

Other Adverse Events
    Regorafenib
Total, other (not including serious) adverse events   
# participants affected / at risk   33/33 (100.00%) 
Blood and lymphatic system disorders   
Leukocytosis † 1   
# participants affected / at risk   3/33 (9.09%) 
Anemia † 1   
# participants affected / at risk   9/33 (27.27%) 
Blood and lymphatic system disorders - Other, specify † 1   
# participants affected / at risk   3/33 (9.09%) 
Endocrine disorders   
Hyperthyroidism † 1   
# participants affected / at risk   3/33 (9.09%) 
Hypothyroidism † 1   
# participants affected / at risk   5/33 (15.15%) 
Eye disorders   
Eye disorders - Other, specify † 1   
# participants affected / at risk   3/33 (9.09%) 
Gastrointestinal disorders   
Bloating † 1   
# participants affected / at risk   2/33 (6.06%) 
Oral dysesthesia † 1   
# participants affected / at risk   6/33 (18.18%) 
Abdominal distension † 1   
# participants affected / at risk   4/33 (12.12%) 
Abdominal pain † 1   
# participants affected / at risk   8/33 (24.24%) 
Colitis † 1   
# participants affected / at risk   3/33 (9.09%) 
Constipation † 1   
# participants affected / at risk   9/33 (27.27%) 
Diarrhea † 1   
# participants affected / at risk   23/33 (69.70%) 
Dry mouth † 1   
# participants affected / at risk   3/33 (9.09%) 
Dyspepsia † 1   
# participants affected / at risk   8/33 (24.24%) 
Flatulence † 1   
# participants affected / at risk   7/33 (21.21%) 
Mucositis oral † 1   
# participants affected / at risk   14/33 (42.42%) 
Nausea † 1   
# participants affected / at risk   13/33 (39.39%) 
Oral hemorrhage † 1   
# participants affected / at risk   2/33 (6.06%) 
Oral pain † 1   
# participants affected / at risk   3/33 (9.09%) 
Stomach pain † 1   
# participants affected / at risk   2/33 (6.06%) 
Vomiting † 1   
# participants affected / at risk   9/33 (27.27%) 
Gastrointestinal disorders - Other, specify † 1   
# participants affected / at risk   19/33 (57.58%) 
General disorders   
Chills † 1   
# participants affected / at risk   2/33 (6.06%) 
Edema limbs † 1   
# participants affected / at risk   2/33 (6.06%) 
Fatigue † 1   
# participants affected / at risk   26/33 (78.79%) 
Pain † 1   
# participants affected / at risk   2/33 (6.06%) 
Infections and infestations   
Mucosal infection † 1   
# participants affected / at risk   2/33 (6.06%) 
Pharyngitis † 1   
# participants affected / at risk   2/33 (6.06%) 
Skin infection † 1   
# participants affected / at risk   2/33 (6.06%) 
Infections and infestations - Other, specify † 1   
# participants affected / at risk   2/33 (6.06%) 
Investigations   
Alanine aminotransferase increased † 1   
# participants affected / at risk   2/33 (6.06%) 
Alkaline phosphatase increased † 1   
# participants affected / at risk   3/33 (9.09%) 
Aspartate aminotransferase increased † 1   
# participants affected / at risk   6/33 (18.18%) 
Blood bilirubin increased † 1   
# participants affected / at risk   4/33 (12.12%) 
Creatinine increased † 1   
# participants affected / at risk   2/33 (6.06%) 
Lipase increased † 1   
# participants affected / at risk   6/33 (18.18%) 
Neutrophil count decreased † 1   
# participants affected / at risk   2/33 (6.06%) 
Platelet count decreased † 1   
# participants affected / at risk   4/33 (12.12%) 
Serum amylase increased † 1   
# participants affected / at risk   3/33 (9.09%) 
Weight loss † 1   
# participants affected / at risk   7/33 (21.21%) 
White blood cell decreased † 1   
# participants affected / at risk   3/33 (9.09%) 
Metabolism and nutrition disorders   
Anorexia † 1   
# participants affected / at risk   13/33 (39.39%) 
Dehydration † 1   
# participants affected / at risk   4/33 (12.12%) 
Hyperglycemia † 1   
# participants affected / at risk   2/33 (6.06%) 
Hyperkalemia † 1   
# participants affected / at risk   2/33 (6.06%) 
Hypermagnesemia † 1   
# participants affected / at risk   2/33 (6.06%) 
Hypoalbuminemia † 1   
# participants affected / at risk   2/33 (6.06%) 
Hypocalcemia † 1   
# participants affected / at risk   5/33 (15.15%) 
Hypomagnesemia † 1   
# participants affected / at risk   6/33 (18.18%) 
Hypophosphatemia † 1   
# participants affected / at risk   10/33 (30.30%) 
Musculoskeletal and connective tissue disorders   
Arthralgia † 1   
# participants affected / at risk   10/33 (30.30%) 
Myalgia † 1   
# participants affected / at risk   16/33 (48.48%) 
Pain in extremity † 1   
# participants affected / at risk   5/33 (15.15%) 
Musculoskeletal and connective tissue disorder - Other, specify † 1   
# participants affected / at risk   3/33 (9.09%) 
Nervous system disorders   
Dizziness † 1   
# participants affected / at risk   2/33 (6.06%) 
Dysgeusia † 1   
# participants affected / at risk   8/33 (24.24%) 
Headache † 1   
# participants affected / at risk   15/33 (45.45%) 
Renal and urinary disorders   
Urinary frequency † 1   
# participants affected / at risk   2/33 (6.06%) 
Respiratory, thoracic and mediastinal disorders   
Hoarseness † 1   
# participants affected / at risk   18/33 (54.55%) 
Laryngeal inflammation † 1   
# participants affected / at risk   2/33 (6.06%) 
Sore throat † 1   
# participants affected / at risk   4/33 (12.12%) 
Dyspnea † 1   
# participants affected / at risk   4/33 (12.12%) 
Skin and subcutaneous tissue disorders   
Alopecia † 1   
# participants affected / at risk   10/33 (30.30%) 
Dry skin † 1   
# participants affected / at risk   3/33 (9.09%) 
Palmar-plantar erythrodysesthesia syndrome † 1   
# participants affected / at risk   18/33 (54.55%) 
Pruritus † 1   
# participants affected / at risk   3/33 (9.09%) 
Rash acneiform † 1   
# participants affected / at risk   3/33 (9.09%) 
Rash maculo-papular † 1   
# participants affected / at risk   4/33 (12.12%) 
Scalp pain † 1   
# participants affected / at risk   3/33 (9.09%) 
Skin hyperpigmentation † 1   
# participants affected / at risk   2/33 (6.06%) 
Skin and subcutaneous tissue disorders - Other, specify † 1   
# participants affected / at risk   4/33 (12.12%) 
Vascular disorders   
Flushing † 1   
# participants affected / at risk   2/33 (6.06%) 
Hot flashes † 1   
# participants affected / at risk   3/33 (9.09%) 
Hypertension † 1   
# participants affected / at risk   12/33 (36.36%) 
Events were collected by systematic assessment
1 Term from vocabulary, CTCAE (4.0)



  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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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:  
Name/Title: Dr. Suzanne George
Organization: Dana-Farber Cancer Institute
phone: 617-632-5204
e-mail: sgeorge2@partners.org



Responsible Party: Suzanne George, MD, Dana-Farber/Brigham and Women's Cancer Center
ClinicalTrials.gov Identifier: NCT01068769     History of Changes
Other Study ID Numbers: 09-400
Study First Received: February 12, 2010
Results First Received: January 9, 2014
Last Updated: September 12, 2016
Health Authority: United States: Food and Drug Administration