Trial Outcomes & Findings for A Study of Axitinib in Advanced Carcinoid Tumors (NCT NCT01435122)
NCT ID: NCT01435122
Last Updated: 2018-10-02
Results Overview
Progression-free survival rate at 12 months. PFS: determined as the time from administration of the initial dose of axitinib until objective tumor progression using Response Evaluation Criteria In Solid Tumors (RECIST), or death. Progressive Disease (PD) Appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. Unequivocal progression should not normally trump target lesion status. It must be representative of overall disease status change, not a single lesion increase. Stable Disease (SD): Neither sufficient shrinkage to qualify for Partial Response (PR) nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
30 participants
Primary outcome timeframe
12 Months
Results posted on
2018-10-02
Participant Flow
Participants were enrolled at H. Lee Moffitt Cancer Center and Research Institute, Tampa, Florida and the UCSF Comprehensive Cancer Center, University of California, San Francisco, California, from October 2011 through January 2014.
Participant milestones
| Measure |
Axitinib Administration
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Overall Study
STARTED
|
30
|
|
Overall Study
COMPLETED
|
22
|
|
Overall Study
NOT COMPLETED
|
8
|
Reasons for withdrawal
| Measure |
Axitinib Administration
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Overall Study
Early withdrawal due to toxicity
|
8
|
Baseline Characteristics
A Study of Axitinib in Advanced Carcinoid Tumors
Baseline characteristics by cohort
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
16 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
14 Participants
n=5 Participants
|
|
Age, Continuous
|
64 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
17 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
13 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
30 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 MonthsPopulation: All participants.
Progression-free survival rate at 12 months. PFS: determined as the time from administration of the initial dose of axitinib until objective tumor progression using Response Evaluation Criteria In Solid Tumors (RECIST), or death. Progressive Disease (PD) Appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions. Unequivocal progression should not normally trump target lesion status. It must be representative of overall disease status change, not a single lesion increase. Stable Disease (SD): Neither sufficient shrinkage to qualify for Partial Response (PR) nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Outcome measures
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Rate of Progression Free Survival (PFS)
|
74.5 percentage of participants
|
PRIMARY outcome
Timeframe: Up to 36 MonthsPopulation: All participants.
Post follow-up progression free survival at time of analysis.
Outcome measures
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Median Progression Free Survival
|
26.7 months
Interval 11.4 to 35.1
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: All participants evaluable at time of analysis.
Tumor response rate using RECIST. Complete Response (CR): Disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response (PR): At least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters.
Outcome measures
| Measure |
Axitinib Administration
n=22 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Tumor Response Rate
Partial response
|
1 Participants
|
|
Tumor Response Rate
Stable disease
|
21 Participants
|
SECONDARY outcome
Timeframe: 24 monthsPopulation: All participants
Overall survival by Kaplan Meier, determined from the time of drug administration to death from any cause. The effect of an intervention is assessed by measuring the number of subjects survived or saved after that intervention over a period of time. The time starting from a defined point to the occurrence of a given event, for example death is called as survival time and the analysis of group data as survival analysis.
Outcome measures
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
24 Month Overall Survival (OS) Rate
|
74.3 percentage of participants
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: All participants.
Time to Treatment Failure: Time from administration of the initial dose of axitinib until study discontinuation for any reason (e.g., disease progression, toxicity, death, withdrawal of consent).
Outcome measures
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Time to Treatment Failure
|
9.6 months
Interval 5.5 to 12.0
|
SECONDARY outcome
Timeframe: 12 MonthsPopulation: All participants.
Grade 2 through 4 toxicities considered at least possibly related to treatment. Percentage of participants affected per category. Safety assessments will consist of monitoring and recording all adverse events and serious adverse events, the regular monitoring of hematology and blood chemistry parameters and regular physical examinations. Adverse events will be evaluated continuously throughout the study. Safety and tolerability will be assessed according to the National Institute of Health/National Cancer Institute (NIH/NCI) Common Terminology Criteria for Adverse Events version 4 (CTCAE v4) available at: http://ctep.cancer.gov/protocolDevelopment/electronic\_applications/ctc.htm.
Outcome measures
| Measure |
Axitinib Administration
n=30 Participants
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Occurrence of Possibly Related Adverse Events (AEs)
Hypertension
|
87 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Abdominal pain
|
17 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Confusion
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Insomnia
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Syncope
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Diarrhea
|
17 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Nausea
|
13 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Vomiting
|
7 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Mucositis
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Oral pain
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Headache
|
20 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Anxiety
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Intracranial hemorrhage
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Fatigue
|
27 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Weight loss
|
6 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Anorexia
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Myalgia
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Dehydration
|
10 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Hypothyroidism
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Increased transaminases
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Increased alkaline phosphatase
|
10 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Increased gamma-glutamyl transferase
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Urinary tract infection
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Right ventricular dysfunction
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Pain in extremity
|
3 percentage of participants
|
|
Occurrence of Possibly Related Adverse Events (AEs)
Hand-foot syndrome
|
2 percentage of participants
|
Adverse Events
Axitinib Administration
Serious events: 12 serious events
Other events: 30 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Axitinib Administration
n=30 participants at risk
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Cardiac disorders
Aortic valve disease
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Abdominal pain
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
Gastrointestinal disorders
Colitis
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Gastrointestinal disorders
Constipation
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Diarrhea
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Ileus
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Nausea
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Gastrointestinal disorders
Pancreatitis
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Rectal ulcer
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Small intestinal obstruction
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Gastrointestinal disorders
Vomiting
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
General disorders
Chills
|
3.3%
1/30 • Number of events 3 • 3 years, 10 months
|
|
General disorders
Fatigue
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
General disorders
Fever
|
3.3%
1/30 • Number of events 3 • 3 years, 10 months
|
|
Hepatobiliary disorders
Bile duct stenosis
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Investigations
Blood bilirubin increased
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Anorexia
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Dehydration
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Nervous system disorders
Intracranial hemorrhage
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Nervous system disorders
Lethargy
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Nervous system disorders
Nervous system disorders - Other, neurologic changes
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Nervous system disorders
Seizure
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Psychiatric disorders
Confusion
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
|
Renal and urinary disorders
Acute kidney injury
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Vascular disorders
Hypertension
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Vascular disorders
Hypotension
|
3.3%
1/30 • Number of events 1 • 3 years, 10 months
|
Other adverse events
| Measure |
Axitinib Administration
n=30 participants at risk
The investigational drug used in this study is axitinib, and is available as tablets.
|
|---|---|
|
Blood and lymphatic system disorders
Anemia
|
23.3%
7/30 • Number of events 7 • 3 years, 10 months
|
|
Endocrine disorders
Hypothyroidism
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
Eye disorders
Dry eye
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Gastrointestinal disorders
Abdominal pain
|
26.7%
8/30 • Number of events 16 • 3 years, 10 months
|
|
Gastrointestinal disorders
Anal mucositis
|
6.7%
2/30 • Number of events 6 • 3 years, 10 months
|
|
Gastrointestinal disorders
Anal pain
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Gastrointestinal disorders
Bloating
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
Gastrointestinal disorders
Constipation
|
23.3%
7/30 • Number of events 19 • 3 years, 10 months
|
|
Gastrointestinal disorders
Diarrhea
|
40.0%
12/30 • Number of events 27 • 3 years, 10 months
|
|
Gastrointestinal disorders
Dry mouth
|
13.3%
4/30 • Number of events 4 • 3 years, 10 months
|
|
Gastrointestinal disorders
Dyspepsia
|
10.0%
3/30 • Number of events 4 • 3 years, 10 months
|
|
Gastrointestinal disorders
Flatulence
|
10.0%
3/30 • Number of events 4 • 3 years, 10 months
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Other
|
10.0%
3/30 • Number of events 5 • 3 years, 10 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
16.7%
5/30 • Number of events 7 • 3 years, 10 months
|
|
Gastrointestinal disorders
Mucositis oral
|
16.7%
5/30 • Number of events 7 • 3 years, 10 months
|
|
Gastrointestinal disorders
Nausea
|
40.0%
12/30 • Number of events 19 • 3 years, 10 months
|
|
Gastrointestinal disorders
Oral pain
|
10.0%
3/30 • Number of events 6 • 3 years, 10 months
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Gastrointestinal disorders
Rectal pain
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
6/30 • Number of events 8 • 3 years, 10 months
|
|
General disorders
Edema limbs
|
10.0%
3/30 • Number of events 4 • 3 years, 10 months
|
|
General disorders
Fatigue
|
56.7%
17/30 • Number of events 32 • 3 years, 10 months
|
|
General disorders
General disorders and administration site conditions - Other, specify
|
10.0%
3/30 • Number of events 7 • 3 years, 10 months
|
|
General disorders
Non-cardiac chest pain
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
General disorders
Pain
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Infections and infestations
Urinary tract infection
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Investigations
Alanine aminotransferase increased
|
13.3%
4/30 • Number of events 5 • 3 years, 10 months
|
|
Investigations
Alkaline phosphatase increased
|
13.3%
4/30 • Number of events 8 • 3 years, 10 months
|
|
Investigations
Blood bilirubin increased
|
10.0%
3/30 • Number of events 5 • 3 years, 10 months
|
|
Investigations
Creatinine increased
|
13.3%
4/30 • Number of events 4 • 3 years, 10 months
|
|
Investigations
Platelet count decreased
|
26.7%
8/30 • Number of events 18 • 3 years, 10 months
|
|
Investigations
Weight loss
|
33.3%
10/30 • Number of events 10 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Anorexia
|
30.0%
9/30 • Number of events 9 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Dehydration
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
33.3%
10/30 • Number of events 22 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
13.3%
4/30 • Number of events 8 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hypocalcemia
|
6.7%
2/30 • Number of events 5 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hypoglycemia
|
10.0%
3/30 • Number of events 6 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
13.3%
4/30 • Number of events 6 • 3 years, 10 months
|
|
Metabolism and nutrition disorders
Hyponatremia
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
20.0%
6/30 • Number of events 10 • 3 years, 10 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
16.7%
5/30 • Number of events 6 • 3 years, 10 months
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
6.7%
2/30 • Number of events 4 • 3 years, 10 months
|
|
Nervous system disorders
Dizziness
|
16.7%
5/30 • Number of events 7 • 3 years, 10 months
|
|
Nervous system disorders
Headache
|
46.7%
14/30 • Number of events 21 • 3 years, 10 months
|
|
Nervous system disorders
Paresthesia
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Psychiatric disorders
Anxiety
|
13.3%
4/30 • Number of events 4 • 3 years, 10 months
|
|
Psychiatric disorders
Confusion
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Psychiatric disorders
Depression
|
13.3%
4/30 • Number of events 4 • 3 years, 10 months
|
|
Psychiatric disorders
Insomnia
|
23.3%
7/30 • Number of events 11 • 3 years, 10 months
|
|
Renal and urinary disorders
Proteinuria
|
13.3%
4/30 • Number of events 7 • 3 years, 10 months
|
|
Renal and urinary disorders
Renal and urinary disorders - Other
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Reproductive system and breast disorders
Reproductive system and breast disorders - Other
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
13.3%
4/30 • Number of events 4 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
6.7%
2/30 • Number of events 4 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Hoarseness
|
33.3%
10/30 • Number of events 12 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders - Other
|
10.0%
3/30 • Number of events 4 • 3 years, 10 months
|
|
Respiratory, thoracic and mediastinal disorders
Sore throat
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
10.0%
3/30 • Number of events 3 • 3 years, 10 months
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
16.7%
5/30 • Number of events 6 • 3 years, 10 months
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
6.7%
2/30 • Number of events 2 • 3 years, 10 months
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders - Other
|
10.0%
3/30 • Number of events 4 • 3 years, 10 months
|
|
Vascular disorders
Hematoma
|
6.7%
2/30 • Number of events 3 • 3 years, 10 months
|
|
Vascular disorders
Hypertension
|
86.7%
26/30 • Number of events 266 • 3 years, 10 months
|
Additional Information
Dr. Jonathan R. Strosberg
H. Lee Moffitt Cancer Center and Research Institute
Phone: 813-745-3636
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place