Trial Outcomes & Findings for A Study of Regorafenib in Advanced Pancreatic Cancer Patients (NCT NCT02080260)
NCT ID: NCT02080260
Last Updated: 2022-04-21
Results Overview
16-week progression free survival was determined for each subject as a binary variable indicating whether or not the subject is alive and progression free at 16 weeks after treatment start, with progression defined radiographically using RECIST v1.1 or clinically based upon investigator assessment.
COMPLETED
PHASE2
20 participants
16 weeks after enrollment
2022-04-21
Participant Flow
Participant milestones
| Measure |
Single Arm, Regorafenib
Oral regorafenib in advanced previously treated pancreatic cancer.
regorafenib: Single agent drug therapy with regorafenib starting at 120mg per day for 3 weeks out 4, with dose adjustments based upon tolerance
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
19
|
Reasons for withdrawal
| Measure |
Single Arm, Regorafenib
Oral regorafenib in advanced previously treated pancreatic cancer.
regorafenib: Single agent drug therapy with regorafenib starting at 120mg per day for 3 weeks out 4, with dose adjustments based upon tolerance
|
|---|---|
|
Overall Study
Death
|
18
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
A Study of Regorafenib in Advanced Pancreatic Cancer Patients
Baseline characteristics by cohort
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
|
Age, Continuous
|
66.2 years
STANDARD_DEVIATION 8.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
14 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
6 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
8 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
12 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 16 weeks after enrollmentPopulation: Efficacy analyses were conducted on the population of subjects who began regorafenib treatment
16-week progression free survival was determined for each subject as a binary variable indicating whether or not the subject is alive and progression free at 16 weeks after treatment start, with progression defined radiographically using RECIST v1.1 or clinically based upon investigator assessment.
Outcome measures
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Number of Patients Progression Free and Surviving at 16 Weeks as a Percent of All Enrolled Subjects
|
2 Participants
|
SECONDARY outcome
Timeframe: From date of treatment start to date of progression or death, or censored as described above; assessed for approximately 3 years.Population: Efficacy analyses were conducted on the population of subjects who began regorafenib treatment
PFS is defined as duration of time from enrollment to the study to time of progression or death. Disease progression (PD) can be objectively determined as per RECIST v1.1 (Response Evaluation Criteria in Solid Tumors, where PD is defined as a 20% increase in the sum of the longest diseased of target lesions, or a measurable increase in non-target lesion, or the appearance of new lesions) or progression can be subjective as determined by the investigator. Evidence for subjective progressions must be documented in medical records. For surviving subjects who do not have documented PD, PFS will be censored at last radiologic assessment. For subjects who receive subsequent anti-cancer therapy prior to documented PD, PFS will be censored at last radiologic assessment prior to commencement of subsequent therapy. Subjects who experience a PFS event following an interval equal to two or more scheduled CT assessments will be censored at date of last assessment prior to first missed assessment.
Outcome measures
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Progression Free Survival
|
6.1 weeks
Interval 2.9 to 7.1
|
SECONDARY outcome
Timeframe: From date of treatment start to date of death, or censored as described above; assessed for approximately 3 years.Population: Efficacy analyses were conducted on the population of subjects who began regorafenib treatment
Overall survival is defined as the duration from enrollment date to the date of death from any cause. Subjects who are alive or lost to follow-up at the time of the analysis will be censored at the last known date they were alive.
Outcome measures
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Overall Survival
|
9.4 weeks
Interval 8.1 to 17.0
|
SECONDARY outcome
Timeframe: From enrollment to best response while on regorafenib; Subjects remained on treatment until disease progression or death or discontinuation from study or at least 28 days after last dose (subjects were on treatment for an average of 6 weeks)Population: Efficacy analyses were conducted on the population of subjects who began regorafenib treatment. Analysis of overall response rate was conducted on those patients in the efficacy population with measurable disease present at baseline.
Overall response will be determined as the best treatment response for each patient as a binary variable indicating whether or not the patient achieved a Complete Response (CR) or Partial Response (PR) as determined by RECIST v1.1 criteria. Per Response Evaluation Criteria in Solid Tumors Criteria (RECIST v1.1):Complete Response (CR) is the disappearance of all lesions (target and non target); Partial Response (PR) is at least 30% decrease in the sum of the diameters of target lesions from baseline and no new lesions or unequivocal progression in non target lesions from baseline; Stable Disease (SD) is neither sufficient shrinkage in target lesions to qualify for PR (less than 30% decrease) nor sufficient increase in target lesions (versus smallest sum of diameters) to qualify for PD (less than 20% increase), with no new lesions or unequivocal progression in non target lesions from baseline. For the purposes of response determination, confirmatory scan for CR and PR is not required.
Outcome measures
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Overall Response
|
1 Participants
|
SECONDARY outcome
Timeframe: From enrollment to best response while on regorafenib; Subjects remained on treatment until disease progression or death or discontinuation from study or at least 28 days after last dose (subjects were on treatment for an average of 6 weeks)Population: Efficacy analyses were conducted on the population of subjects who began regorafenib treatment
Disease control will be determined for each patient as a binary variable indicating whether or not the patient achieved a best overall best response of CR, PR, or stable disease as determined by RECIST v1.1 criteria. Per Response Evaluation Criteria in Solid Tumors Criteria (RECIST v 1.1): Complete Response (CR) is the disappearance of all lesions (target and non target); Partial Response (PR) is at least 30% decrease in the sum of the diameters of target lesions from baseline and no new lesions or unequivocal progression in non target lesions from baseline; Stable Disease (SD) is neither sufficient shrinkage in target lesions to qualify for PR (less than 30% decrease) nor sufficient increase in target lesions (versus smallest sum of diameters) to qualify for PD (less than 20% increase), with no new lesions or unequivocal progression in non target lesions from baseline.
Outcome measures
| Measure |
Single Arm, Regorafenib
n=20 Participants
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Disease Control
|
6 Participants
|
Adverse Events
Single Arm, Regorafenib
Serious adverse events
| Measure |
Single Arm, Regorafenib
n=20 participants at risk
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Death NOS
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Blood bilirubin increased
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Cardiac disorders
Chest pain - cardiac
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Psychiatric disorders
Confusion
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Dehydration
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Duodenal obstruction
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Fever
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Vascular disorders
Hypotension
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Infections and infestations - Other
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Nausea
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Stroke
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Vomiting
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Lung infection
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Non-cardiac chest pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
Other adverse events
| Measure |
Single Arm, Regorafenib
n=20 participants at risk
Oral Regorafenib
regorafenib: Single agent drug therapy with regorafenib
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
40.0%
8/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Blood and lymphatic system disorders
Leukocytosis
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Cardiac disorders
Sinus tachycardia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders - Other, specify
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Ear and labyrinth disorders
Hearing impaired
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Eye disorders
Eye pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Eye disorders
Floaters
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Abdominal distension
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Abdominal pain
|
40.0%
8/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Ascites
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Constipation
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Diarrhea
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Dry mouth
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Dyspepsia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Dysphagia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Hemorrhoids
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Mucositis oral
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Nausea
|
35.0%
7/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Oral hemorrhage
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Oral pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
5/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Chills
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Fatigue
|
60.0%
12/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Fever
|
25.0%
5/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Pain
|
25.0%
5/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Edema limbs
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
General disorders
Non-cardiac chest pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Hepatobiliary disorders
Hepatic failure
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Skin infection
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Urinary tract infection
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Device related infection
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Infections and infestations
Papulopustular rash
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Injury, poisoning and procedural complications
Fall
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Alanine aminotransferase increased
|
45.0%
9/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Alkaline phosphatase increased
|
45.0%
9/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Aspartate aminotransferase increased
|
60.0%
12/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Blood bilirubin increased
|
45.0%
9/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Creatinine increased
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Investigations - Other
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Lymphocyte count decreased
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Platelet count decreased
|
30.0%
6/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Weight loss
|
40.0%
8/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
White blood cell decreased
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Hemoglobin increased
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Investigations
Urine output decreased
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Anorexia
|
40.0%
8/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Dehydration
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypernatremia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
45.0%
9/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
50.0%
10/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
30.0%
6/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Chest wall pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
25.0%
5/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Concentration impairment
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Dizziness
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Dysgeusia
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Headache
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Nervous system disorders
Paresthesia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Psychiatric disorders
Anxiety
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Psychiatric disorders
Confusion
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Psychiatric disorders
Depression
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Psychiatric disorders
Insomnia
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Renal and urinary disorders
Urinary retention
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Renal and urinary disorders
Urine discoloration
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Renal and urinary disorders
Urinary tract pain
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
25.0%
5/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
20.0%
4/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
5.0%
1/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Vascular disorders
Hypertension
|
30.0%
6/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Vascular disorders
Hypotension
|
15.0%
3/20 • Baseline, during treatment, and 30 days after last dose.
|
|
Vascular disorders
Thromboembolic event
|
10.0%
2/20 • Baseline, during treatment, and 30 days after last dose.
|
Additional Information
Chair of Biostatistics Department
Levine Cancer Institute
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place