Trial Outcomes & Findings for Vismodegib and Gemcitabine Hydrochloride in Treating Patients With Advanced Pancreatic Cancer (NCT NCT01195415)
NCT ID: NCT01195415
Last Updated: 2017-08-07
Results Overview
The median percentage of CD44+CD24+ESA+ cells from needle biopsy were calculated at baseline and at 3 weeks using FACS. The difference between the two time points was calculated.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
25 participants
Primary outcome timeframe
3 weeks
Results posted on
2017-08-07
Participant Flow
Participant milestones
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
STARTED
|
25
|
|
Overall Study
COMPLETED
|
23
|
|
Overall Study
NOT COMPLETED
|
2
|
Reasons for withdrawal
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Overall Study
No repeat biopsy due to progression
|
2
|
Baseline Characteristics
Vismodegib and Gemcitabine Hydrochloride in Treating Patients With Advanced Pancreatic Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 Participants
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Age, Continuous
|
65 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
15 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 3 weeksPopulation: Of the 25 patients enrolled, only 22 were evaluable for the primary endpoint.
The median percentage of CD44+CD24+ESA+ cells from needle biopsy were calculated at baseline and at 3 weeks using FACS. The difference between the two time points was calculated.
Outcome measures
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=22 Participants
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Median Percent at Baseline and 3 Weeks in CD44+/ CD24+/ ESA+ Cells From Needle Biopsy Calculated Using FACS
Baseline Median % of CD44+/CD24+/ESA+ CSCs
|
4.79 percentage of CD44+/ CD24+/ ESA+ cells
Interval 0.43 to 37.2
|
|
Median Percent at Baseline and 3 Weeks in CD44+/ CD24+/ ESA+ Cells From Needle Biopsy Calculated Using FACS
3 Weeks Post Tx Median % of CD44+/CD24+/ESA+ CSCs
|
3.09 percentage of CD44+/ CD24+/ ESA+ cells
Interval 0.74 to 45.9
|
SECONDARY outcome
Timeframe: Up to 4 weeksPopulation: All treated patients were evaluable.
The number of participants with either a complete response (CR) or a partial response (PR) will be calculated. A CR is defined as the disappearance of all target lesions. A PR is defined as at least a 30% decrease in the sum of the diameters of target lesions.
Outcome measures
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 Participants
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
The Number of Participants With an Objective Best Response (CR + PR)
|
5 participants
|
SECONDARY outcome
Timeframe: Up to 24 monthsMedian progression free survival was calculated for all treated patients. Assessed using the Kaplan-Meier method. The 95% confidence interval for this estimate will be computed using the Greenwood's formula.
Outcome measures
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 Participants
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Median Progression Free Survival
|
2.8 months
Interval 1.4 to 4.7
|
SECONDARY outcome
Timeframe: Up to 4 weeksOutcome measures
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 Participants
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Percentage of Treated Patients Experiencing Grade 3+ Toxicity Per National Cancer Institute Common Toxicity Criteria (CTC) Version 3.0
|
56 percentage of patients
|
Adverse Events
Treatment (Vismodegib, Gemcitabine Hydrochloride)
Serious events: 16 serious events
Other events: 25 other events
Deaths: 1 deaths
Serious adverse events
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 participants at risk
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Febrile Neutropenia
|
4.0%
1/25 • Number of events 1
|
|
Gastrointestinal disorders
Abdominal pain
|
8.0%
2/25 • Number of events 2
|
|
Gastrointestinal disorders
Duodenal stenosis
|
4.0%
1/25 • Number of events 1
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
4.0%
1/25 • Number of events 1
|
|
Gastrointestinal disorders
Upper gastrointestinal hemorrhage
|
4.0%
1/25 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
4.0%
1/25 • Number of events 1
|
|
General disorders
Fatigue
|
4.0%
1/25 • Number of events 1
|
|
General disorders
Fever
|
4.0%
1/25 • Number of events 1
|
|
General disorders
General disorders and administration site conditions - Other
|
8.0%
2/25 • Number of events 2
|
|
General disorders
Multi-organ failure
|
4.0%
1/25 • Number of events 1
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other
|
4.0%
1/25 • Number of events 1
|
|
Investigations
Aspartate aminotransferase increased
|
4.0%
1/25 • Number of events 1
|
|
Investigations
Blood bilirubin increased
|
4.0%
1/25 • Number of events 1
|
|
Metabolism and nutrition disorders
Anorexia
|
4.0%
1/25 • Number of events 1
|
|
Metabolism and nutrition disorders
Dehydration
|
12.0%
3/25 • Number of events 3
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
4.0%
1/25 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
8.0%
2/25 • Number of events 2
|
|
Nervous system disorders
Cognitive disturbance
|
4.0%
1/25 • Number of events 1
|
|
Renal and urinary disorders
Urinary tract obstruction
|
4.0%
1/25 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
4.0%
1/25 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
4.0%
1/25 • Number of events 1
|
|
Surgical and medical procedures
Surgical and medical procedures - Other
|
4.0%
1/25 • Number of events 1
|
|
Vascular disorders
Hypotension
|
8.0%
2/25 • Number of events 2
|
|
Vascular disorders
Superior vena cava syndrome
|
4.0%
1/25 • Number of events 1
|
|
Vascular disorders
Thromboembolic event
|
4.0%
1/25 • Number of events 1
|
|
General disorders
Death
|
4.0%
1/25 • Number of events 1
|
Other adverse events
| Measure |
Treatment (Vismodegib, Gemcitabine Hydrochloride)
n=25 participants at risk
Patients receive vismodegib PO QD on days 1-28 and gemcitabine hydrochloride IV over 30 minutes on days 1, 8, and 15 (beginning in course 2). Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
68.0%
17/25 • Number of events 29
|
|
Cardiac disorders
Chest pain - cardiac
|
8.0%
2/25 • Number of events 2
|
|
Ear and labyrinth disorders
Tinnitus
|
12.0%
3/25 • Number of events 3
|
|
Ear and labyrinth disorders
Vertigo
|
8.0%
2/25 • Number of events 2
|
|
Eye disorders
Photophobia
|
8.0%
2/25 • Number of events 2
|
|
Gastrointestinal disorders
Abdominal distension
|
16.0%
4/25 • Number of events 5
|
|
Gastrointestinal disorders
Abdominal pain
|
52.0%
13/25 • Number of events 19
|
|
Gastrointestinal disorders
Ascites
|
12.0%
3/25 • Number of events 4
|
|
Gastrointestinal disorders
Constipation
|
28.0%
7/25 • Number of events 9
|
|
Gastrointestinal disorders
Diarrhea
|
32.0%
8/25 • Number of events 8
|
|
Gastrointestinal disorders
Dry mouth
|
12.0%
3/25 • Number of events 3
|
|
Gastrointestinal disorders
Dyspepsia
|
12.0%
3/25 • Number of events 3
|
|
Gastrointestinal disorders
Flatulence
|
8.0%
2/25 • Number of events 2
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
8.0%
2/25 • Number of events 2
|
|
Gastrointestinal disorders
Gastroparesis
|
8.0%
2/25 • Number of events 2
|
|
Gastrointestinal disorders
Nausea
|
56.0%
14/25 • Number of events 16
|
|
Gastrointestinal disorders
Vomiting
|
52.0%
13/25 • Number of events 15
|
|
General disorders
Chills
|
12.0%
3/25 • Number of events 3
|
|
General disorders
Edema limbs
|
28.0%
7/25 • Number of events 8
|
|
General disorders
Fatigue
|
72.0%
18/25 • Number of events 28
|
|
General disorders
Fever
|
40.0%
10/25 • Number of events 14
|
|
General disorders
General disorders and administration site conditions - Other
|
8.0%
2/25 • Number of events 2
|
|
General disorders
Non-cardiac chest pain
|
8.0%
2/25 • Number of events 2
|
|
Hepatobiliary disorders
Hepatobiliary disorders - Other
|
8.0%
2/25 • Number of events 2
|
|
Infections and infestations
Papulopustular rash
|
12.0%
3/25 • Number of events 3
|
|
Investigations
Alanine aminotransferase increased
|
68.0%
17/25 • Number of events 22
|
|
Investigations
Aspartate aminotransferase increased
|
72.0%
18/25 • Number of events 24
|
|
Investigations
Blood bilirubin increased
|
32.0%
8/25 • Number of events 11
|
|
Investigations
CD4 lymphocytes decreased
|
12.0%
3/25 • Number of events 6
|
|
Investigations
Creatinine increased
|
12.0%
3/25 • Number of events 3
|
|
Investigations
Lipase increased
|
8.0%
2/25 • Number of events 2
|
|
Investigations
Lymphocyte count decreased
|
44.0%
11/25 • Number of events 12
|
|
Investigations
Neutrophil count decreased
|
32.0%
8/25 • Number of events 9
|
|
Investigations
Platelet count decreased
|
52.0%
13/25 • Number of events 18
|
|
Investigations
Weight gain
|
24.0%
6/25 • Number of events 6
|
|
Investigations
Weight loss
|
40.0%
10/25 • Number of events 12
|
|
Investigations
White blood cell decreased
|
40.0%
10/25 • Number of events 13
|
|
Metabolism and nutrition disorders
Anorexia
|
76.0%
19/25 • Number of events 24
|
|
Metabolism and nutrition disorders
Dehydration
|
20.0%
5/25 • Number of events 5
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
16.0%
4/25 • Number of events 4
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
72.0%
18/25 • Number of events 22
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
36.0%
9/25 • Number of events 11
|
|
Metabolism and nutrition disorders
Hypoalbuminemia
|
44.0%
11/25 • Number of events 16
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
52.0%
13/25 • Number of events 17
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
8.0%
2/25 • Number of events 2
|
|
Metabolism and nutrition disorders
Hyponatremia
|
76.0%
19/25 • Number of events 23
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
44.0%
11/25 • Number of events 14
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
16.0%
4/25 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
12.0%
3/25 • Number of events 3
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
5/25 • Number of events 6
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
28.0%
7/25 • Number of events 10
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
12.0%
3/25 • Number of events 3
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other
|
8.0%
2/25 • Number of events 2
|
|
Nervous system disorders
Dizziness
|
16.0%
4/25 • Number of events 4
|
|
Nervous system disorders
Dysgeusia
|
56.0%
14/25 • Number of events 16
|
|
Nervous system disorders
Headache
|
24.0%
6/25 • Number of events 6
|
|
Nervous system disorders
Somnolence
|
12.0%
3/25 • Number of events 3
|
|
Psychiatric disorders
Anxiety
|
28.0%
7/25 • Number of events 7
|
|
Psychiatric disorders
Confusion
|
12.0%
3/25 • Number of events 4
|
|
Psychiatric disorders
Depression
|
20.0%
5/25 • Number of events 5
|
|
Psychiatric disorders
Insomnia
|
12.0%
3/25 • Number of events 3
|
|
Renal and urinary disorders
Renal and urinary disorders - Other
|
8.0%
2/25 • Number of events 2
|
|
Renal and urinary disorders
Urinary frequency
|
8.0%
2/25 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
24.0%
6/25 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
28.0%
7/25 • Number of events 8
|
|
Respiratory, thoracic and mediastinal disorders
Hiccups
|
8.0%
2/25 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
Productive cough
|
20.0%
5/25 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
32.0%
8/25 • Number of events 10
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
12.0%
3/25 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
8.0%
2/25 • Number of events 2
|
|
Vascular disorders
Hypertension
|
8.0%
2/25 • Number of events 2
|
|
Vascular disorders
Hypotension
|
28.0%
7/25 • Number of events 8
|
Additional Information
Dr. Mark Zalupski, M.D.
University of Michigan Comprehensive Cancer Center
Phone: 734-615-3969
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60