Trial Outcomes & Findings for Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor (NCT NCT01068769)
NCT ID: NCT01068769
Last Updated: 2020-08-24
Results Overview
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%.
COMPLETED
PHASE2
34 participants
2 years
2020-08-24
Participant Flow
Participants were enrolled at three sites between February and December, 2010.
Participant milestones
| Measure |
Regorafenib
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Overall Study
STARTED
|
34
|
|
Overall Study
COMPLETED
|
33
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
Regorafenib
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Overall Study
Ineligible
|
1
|
Baseline Characteristics
Regorafenib in Patients With Metastatic and/or Unresectable Gastrointestinal Stromal Tumor
Baseline characteristics by cohort
| Measure |
Regorafenib
n=33 Participants
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Age, Continuous
|
56 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
19 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
33 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 yearsThe 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%.
Outcome measures
| Measure |
Regorafenib
n=33 Participants
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
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
|
75 percentage of participants
Interval 61.0 to 91.0
|
SECONDARY outcome
Timeframe: From date of enrollment until date of first documented progression or date of death from any cause, whichever came firstProgression-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).
Outcome measures
| Measure |
Regorafenib
n=33 Participants
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Progression-free Survival (PFS)
|
10.0 months
Interval 8.3 to 14.9
|
Adverse Events
Regorafenib
Serious adverse events
| Measure |
Regorafenib
n=33 participants at risk
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Cardiac disorders
Acute coronary syndrome
|
3.0%
1/33
|
|
Gastrointestinal disorders
Abdominal pain
|
12.1%
4/33
|
|
Gastrointestinal disorders
Colitis
|
24.2%
8/33
|
|
Gastrointestinal disorders
Diarrhea
|
9.1%
3/33
|
|
Gastrointestinal disorders
Nausea
|
6.1%
2/33
|
|
Gastrointestinal disorders
Vomiting
|
3.0%
1/33
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
9.1%
3/33
|
|
General disorders
Fatigue
|
6.1%
2/33
|
|
Investigations
Blood bilirubin increased
|
3.0%
1/33
|
|
Investigations
CD4 lymphocytes decreased
|
3.0%
1/33
|
|
Investigations
Lipase increased
|
6.1%
2/33
|
|
Investigations
Lymphocyte count decreased
|
3.0%
1/33
|
|
Investigations
Neutrophil count decreased
|
3.0%
1/33
|
|
Investigations
Serum amylase increased
|
3.0%
1/33
|
|
Investigations
Weight loss
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Anorexia
|
3.0%
1/33
|
|
Metabolism and nutrition disorders
Hyperuricemia
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Hyponatremia
|
3.0%
1/33
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
18.2%
6/33
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
6.1%
2/33
|
|
Musculoskeletal and connective tissue disorders
Buttock pain
|
6.1%
2/33
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
6.1%
2/33
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.1%
2/33
|
|
Psychiatric disorders
Insomnia
|
6.1%
2/33
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
36.4%
12/33
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
3.0%
1/33
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.1%
4/33
|
|
Vascular disorders
Hypertension
|
39.4%
13/33
|
|
Vascular disorders
Thromboembolic event
|
3.0%
1/33
|
Other adverse events
| Measure |
Regorafenib
n=33 participants at risk
Regorafenib adminstered orally, 160 mg per day on days 1 through 21 of a 28 day cycle
|
|---|---|
|
Blood and lymphatic system disorders
Leukocytosis
|
9.1%
3/33
|
|
Blood and lymphatic system disorders
Anemia
|
27.3%
9/33
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders - Other, specify
|
9.1%
3/33
|
|
Endocrine disorders
Hyperthyroidism
|
9.1%
3/33
|
|
Endocrine disorders
Hypothyroidism
|
15.2%
5/33
|
|
Eye disorders
Eye disorders - Other, specify
|
9.1%
3/33
|
|
Gastrointestinal disorders
Bloating
|
6.1%
2/33
|
|
Gastrointestinal disorders
Oral dysesthesia
|
18.2%
6/33
|
|
Gastrointestinal disorders
Abdominal distension
|
12.1%
4/33
|
|
Gastrointestinal disorders
Abdominal pain
|
24.2%
8/33
|
|
Gastrointestinal disorders
Colitis
|
9.1%
3/33
|
|
Gastrointestinal disorders
Constipation
|
27.3%
9/33
|
|
Gastrointestinal disorders
Diarrhea
|
69.7%
23/33
|
|
Gastrointestinal disorders
Dry mouth
|
9.1%
3/33
|
|
Gastrointestinal disorders
Dyspepsia
|
24.2%
8/33
|
|
Gastrointestinal disorders
Flatulence
|
21.2%
7/33
|
|
Gastrointestinal disorders
Mucositis oral
|
42.4%
14/33
|
|
Gastrointestinal disorders
Nausea
|
39.4%
13/33
|
|
Gastrointestinal disorders
Oral hemorrhage
|
6.1%
2/33
|
|
Gastrointestinal disorders
Oral pain
|
9.1%
3/33
|
|
Gastrointestinal disorders
Stomach pain
|
6.1%
2/33
|
|
Gastrointestinal disorders
Vomiting
|
27.3%
9/33
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other, specify
|
57.6%
19/33
|
|
General disorders
Chills
|
6.1%
2/33
|
|
General disorders
Edema limbs
|
6.1%
2/33
|
|
General disorders
Fatigue
|
78.8%
26/33
|
|
General disorders
Pain
|
6.1%
2/33
|
|
Infections and infestations
Mucosal infection
|
6.1%
2/33
|
|
Infections and infestations
Pharyngitis
|
6.1%
2/33
|
|
Infections and infestations
Skin infection
|
6.1%
2/33
|
|
Infections and infestations
Infections and infestations - Other, specify
|
6.1%
2/33
|
|
Investigations
Alanine aminotransferase increased
|
6.1%
2/33
|
|
Investigations
Alkaline phosphatase increased
|
9.1%
3/33
|
|
Investigations
Aspartate aminotransferase increased
|
18.2%
6/33
|
|
Investigations
Blood bilirubin increased
|
12.1%
4/33
|
|
Investigations
Creatinine increased
|
6.1%
2/33
|
|
Investigations
Lipase increased
|
18.2%
6/33
|
|
Investigations
Neutrophil count decreased
|
6.1%
2/33
|
|
Investigations
Platelet count decreased
|
12.1%
4/33
|
|
Investigations
Serum amylase increased
|
9.1%
3/33
|
|
Investigations
Weight loss
|
21.2%
7/33
|
|
Investigations
White blood cell decreased
|
9.1%
3/33
|
|
Metabolism and nutrition disorders
Anorexia
|
39.4%
13/33
|
|
Metabolism and nutrition disorders
Dehydration
|
12.1%
4/33
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Hypermagnesemia
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
6.1%
2/33
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
15.2%
5/33
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
18.2%
6/33
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
30.3%
10/33
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
30.3%
10/33
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
48.5%
16/33
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
15.2%
5/33
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder - Other, specify
|
9.1%
3/33
|
|
Nervous system disorders
Dizziness
|
6.1%
2/33
|
|
Nervous system disorders
Dysgeusia
|
24.2%
8/33
|
|
Nervous system disorders
Headache
|
45.5%
15/33
|
|
Renal and urinary disorders
Urinary frequency
|
6.1%
2/33
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
54.5%
18/33
|
|
Respiratory, thoracic and mediastinal disorders
Laryngeal inflammation
|
6.1%
2/33
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
12.1%
4/33
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
12.1%
4/33
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
30.3%
10/33
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
9.1%
3/33
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
54.5%
18/33
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
9.1%
3/33
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
9.1%
3/33
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
12.1%
4/33
|
|
Skin and subcutaneous tissue disorders
Scalp pain
|
9.1%
3/33
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
6.1%
2/33
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other, specify
|
12.1%
4/33
|
|
Vascular disorders
Flushing
|
6.1%
2/33
|
|
Vascular disorders
Hot flashes
|
9.1%
3/33
|
|
Vascular disorders
Hypertension
|
36.4%
12/33
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place