Trial Outcomes & Findings for Cabozantinib S-malate in Treating Patients With Metastatic Pheochromocytomas or Paragangliomas That Cannot Be Removed by Surgery (NCT NCT02302833)
NCT ID: NCT02302833
Last Updated: 2025-07-31
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome timeframe
6 months
Results posted on
2025-07-31
Participant Flow
Participant milestones
| Measure |
Cabozantinib
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
COMPLETED
|
17
|
|
Overall Study
NOT COMPLETED
|
3
|
Reasons for withdrawal
| Measure |
Cabozantinib
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Overall Study
Lost to Follow-up
|
3
|
Baseline Characteristics
Cabozantinib S-malate in Treating Patients With Metastatic Pheochromocytomas or Paragangliomas That Cannot Be Removed by Surgery
Baseline characteristics by cohort
| Measure |
Cabozantinib
n=20 Participants
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Age, Continuous
|
52 years
n=99 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
14 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
20 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
2 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
17 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Region of Enrollment
United States
|
20 participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 6 monthsOutcome measures
| Measure |
Cabozantinib
n=17 Participants
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Objective Response Rate
|
25 percentage of participants
Interval 7.3 to 52.0
|
Adverse Events
Cabozantinib
Serious events: 20 serious events
Other events: 20 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Cabozantinib
n=20 participants at risk
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
15.0%
3/20 • 9 years
|
|
Blood and lymphatic system disorders
Anemia
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
5.0%
1/20 • 9 years
|
|
Infections and infestations
Bronchial infection
|
5.0%
1/20 • 9 years
|
|
Cardiac disorders
Chest Pain
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Constipation
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Gastrointestinal fistula
|
5.0%
1/20 • 9 years
|
|
Cardiac disorders
Heart Failure
|
5.0%
1/20 • 9 years
|
|
Cardiac disorders
Heart Failure, Death
|
5.0%
1/20 • 9 years
|
|
General disorders
Hypersomnia
|
5.0%
1/20 • 9 years
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • 9 years
|
|
Cardiac disorders
Left Ventricular systolic dysfunction
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder-other, Specify
|
5.0%
1/20 • 9 years
|
|
Blood and lymphatic system disorders
Myelodysplastic Syndrome, Death
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Nausea
|
5.0%
1/20 • 9 years
|
|
General disorders
Oral Hemorrhage
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Small Intestine obstruction
|
5.0%
1/20 • 9 years
|
|
Nervous system disorders
Stroke
|
5.0%
1/20 • 9 years
|
|
Surgical and medical procedures
Surgical and medical Procedures Other, Laminectomy, Tumor resection
|
5.0%
1/20 • 9 years
|
|
Surgical and medical procedures
Surgical and medical Procedures Other: Hip Replacement
|
5.0%
1/20 • 9 years
|
|
Surgical and medical procedures
Surgical and medical Procedures Other: Laminectomy Sacrum
|
5.0%
1/20 • 9 years
|
|
Vascular disorders
Thromboembolic Event
|
10.0%
2/20 • 9 years
|
|
Renal and urinary disorders
Urinary Tract Infection
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • 9 years
|
Other adverse events
| Measure |
Cabozantinib
n=20 participants at risk
Patients with advanced and progressive pheochromocytomas and paragangliomas meeting inclusion and exclusion criteria
Patients receive cabozantinib s-malate PO QD on days 1-28. Courses repeat every 4 weeks in the absence of disease progression or unacceptable toxicity.
Cabozantinib S-malate: Given PO
|
|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
15.0%
3/20 • 9 years
|
|
Gastrointestinal disorders
Abdominal pain
|
40.0%
8/20 • 9 years
|
|
Blood and lymphatic system disorders
Activated partial thromboplastin time prolonged
|
35.0%
7/20 • 9 years
|
|
General disorders
Agitation
|
5.0%
1/20 • 9 years
|
|
Investigations
Alanine aminotransferase increased
|
75.0%
15/20 • 9 years
|
|
Investigations
Alkaline phosphatase increased
|
55.0%
11/20 • 9 years
|
|
General disorders
Allergic rhinitis
|
5.0%
1/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
40.0%
8/20 • 9 years
|
|
Investigations
ALT increased
|
5.0%
1/20 • 9 years
|
|
General disorders
Anal fistula
|
5.0%
1/20 • 9 years
|
|
General disorders
Anal pain
|
5.0%
1/20 • 9 years
|
|
Blood and lymphatic system disorders
Anemia
|
90.0%
18/20 • 9 years
|
|
Metabolism and nutrition disorders
Anorexia
|
65.0%
13/20 • 9 years
|
|
Psychiatric disorders
Anxiety
|
20.0%
4/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
45.0%
9/20 • 9 years
|
|
Gastrointestinal disorders
Ascites
|
5.0%
1/20 • 9 years
|
|
Investigations
Aspartate aminotransferase increased
|
80.0%
16/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Atelectasis
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
45.0%
9/20 • 9 years
|
|
Gastrointestinal disorders
Bloating
|
35.0%
7/20 • 9 years
|
|
Blood and lymphatic system disorders
Blood and lymphatic system disorders
|
35.0%
7/20 • 9 years
|
|
Investigations
Blood bilirubin increased
|
10.0%
2/20 • 9 years
|
|
Eye disorders
Blurred vision
|
10.0%
2/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Bone pain
|
10.0%
2/20 • 9 years
|
|
General disorders
Bruising
|
25.0%
5/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Burn
|
5.0%
1/20 • 9 years
|
|
General disorders
Buttock pain
|
5.0%
1/20 • 9 years
|
|
Nervous system disorders
Cerebrospinal fluid leakage
|
5.0%
1/20 • 9 years
|
|
General disorders
Chills
|
5.0%
1/20 • 9 years
|
|
Psychiatric disorders
Concentration impairment
|
20.0%
4/20 • 9 years
|
|
Psychiatric disorders
Confusion
|
5.0%
1/20 • 9 years
|
|
Eye disorders
Conjunctivitis
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Constipation
|
60.0%
12/20 • 9 years
|
|
General disorders
Cough
|
35.0%
7/20 • 9 years
|
|
Investigations
Creatinine increased
|
30.0%
6/20 • 9 years
|
|
Metabolism and nutrition disorders
Dehydration
|
5.0%
1/20 • 9 years
|
|
Psychiatric disorders
Depression
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Diarrhea
|
75.0%
15/20 • 9 years
|
|
General disorders
Dizziness
|
40.0%
8/20 • 9 years
|
|
General disorders
Dry mouth
|
40.0%
8/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
55.0%
11/20 • 9 years
|
|
General disorders
Dysgeusia
|
65.0%
13/20 • 9 years
|
|
General disorders
Dyspepsia
|
5.0%
1/20 • 9 years
|
|
General disorders
Dysphagia
|
15.0%
3/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
20.0%
4/20 • 9 years
|
|
Ear and labyrinth disorders
Ear and labyrinth disorders
|
10.0%
2/20 • 9 years
|
|
General disorders
Edema limbs
|
35.0%
7/20 • 9 years
|
|
General disorders
Electrocardiogram QT corrected interval prolonged
|
10.0%
2/20 • 9 years
|
|
Endocrine disorders
Endocrine disorders
|
15.0%
3/20 • 9 years
|
|
Vascular disorders
Epistaxis
|
5.0%
1/20 • 9 years
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
5.0%
1/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Erythema multiforme
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Extraocular muscle paresis
|
5.0%
1/20 • 9 years
|
|
Eye disorders
Eye disorders
|
15.0%
3/20 • 9 years
|
|
Eye disorders
Eye pain
|
10.0%
2/20 • 9 years
|
|
General disorders
Fall
|
10.0%
2/20 • 9 years
|
|
General disorders
Fatigue
|
95.0%
19/20 • 9 years
|
|
General disorders
Fecal incontinence
|
5.0%
1/20 • 9 years
|
|
General disorders
Fever
|
10.0%
2/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Flatulence
|
15.0%
3/20 • 9 years
|
|
General disorders
Flu like symptoms
|
20.0%
4/20 • 9 years
|
|
Vascular disorders
Flushing
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Gastroesophageal reflux disease
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders
|
25.0%
5/20 • 9 years
|
|
General disorders
General disorders and administration site conditions
|
65.0%
13/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
GERD
|
5.0%
1/20 • 9 years
|
|
General disorders
Hand and foot syndrome
|
5.0%
1/20 • 9 years
|
|
Nervous system disorders
Headache
|
60.0%
12/20 • 9 years
|
|
Cardiac disorders
Heart Failure
|
5.0%
1/20 • 9 years
|
|
Renal and urinary disorders
Hematuria
|
10.0%
2/20 • 9 years
|
|
General disorders
Hoarseness
|
35.0%
7/20 • 9 years
|
|
General disorders
Hot flashes
|
5.0%
1/20 • 9 years
|
|
Investigations
Hypercalcemia
|
5.0%
1/20 • 9 years
|
|
Investigations
Hyperglycemia
|
75.0%
15/20 • 9 years
|
|
General disorders
Hyperhidrosis
|
55.0%
11/20 • 9 years
|
|
Investigations
Hyperkalemia
|
10.0%
2/20 • 9 years
|
|
Investigations
Hypertension
|
95.0%
19/20 • 9 years
|
|
Investigations
Hyperthyroidism
|
5.0%
1/20 • 9 years
|
|
Investigations
Hyperuricemia
|
5.0%
1/20 • 9 years
|
|
Investigations
Hypoalbuminemia
|
35.0%
7/20 • 9 years
|
|
Investigations
Hypocalcemia
|
15.0%
3/20 • 9 years
|
|
Investigations
Hypoglycemia
|
10.0%
2/20 • 9 years
|
|
Investigations
Hypokalemia
|
15.0%
3/20 • 9 years
|
|
Investigations
Hypomagnesemia
|
40.0%
8/20 • 9 years
|
|
Investigations
Hyponatremia
|
20.0%
4/20 • 9 years
|
|
Investigations
Hypophosphatemia
|
20.0%
4/20 • 9 years
|
|
General disorders
Hypotension
|
5.0%
1/20 • 9 years
|
|
Endocrine disorders
Hypothyroidism
|
65.0%
13/20 • 9 years
|
|
Immune system disorders
Immune system disorders
|
5.0%
1/20 • 9 years
|
|
Investigations
INR increased
|
20.0%
4/20 • 9 years
|
|
Psychiatric disorders
Insomnia
|
30.0%
6/20 • 9 years
|
|
Investigations
Investigations
|
75.0%
15/20 • 9 years
|
|
Reproductive system and breast disorders
Irregular menstruation
|
5.0%
1/20 • 9 years
|
|
General disorders
Irritability
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Laryngeal inflammation
|
5.0%
1/20 • 9 years
|
|
General disorders
LDH
|
5.0%
1/20 • 9 years
|
|
General disorders
Libido decreased
|
5.0%
1/20 • 9 years
|
|
Investigations
Lipase increased
|
20.0%
4/20 • 9 years
|
|
Investigations
Lymphocyte count decreased
|
65.0%
13/20 • 9 years
|
|
Nervous system disorders
Memory impairment
|
15.0%
3/20 • 9 years
|
|
Metabolism and nutrition disorders
Metabolism and nutrition disorders
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Mucosal infection
|
10.0%
2/20 • 9 years
|
|
Gastrointestinal disorders
Mucositis oral
|
65.0%
13/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness right-sided
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness upper limb
|
5.0%
1/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal and connective tissue disorder
|
50.0%
10/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
65.0%
13/20 • 9 years
|
|
Blood and lymphatic system disorders
Myelodysplastic syndrome
|
5.0%
1/20 • 9 years
|
|
General disorders
Nail ridging
|
5.0%
1/20 • 9 years
|
|
General disorders
Nasal congestion
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Nausea
|
75.0%
15/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
10.0%
2/20 • 9 years
|
|
Nervous system disorders
Nervous system disorders
|
10.0%
2/20 • 9 years
|
|
Investigations
Neutrophil Abs Increased
|
5.0%
1/20 • 9 years
|
|
Investigations
Neutrophil count decreased
|
10.0%
2/20 • 9 years
|
|
General disorders
Non-cardiac chest pain
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Oral dysesthesia
|
10.0%
2/20 • 9 years
|
|
General disorders
Pain
|
65.0%
13/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
45.0%
9/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
65.0%
13/20 • 9 years
|
|
Cardiac disorders
Palpitations
|
40.0%
8/20 • 9 years
|
|
General disorders
Paresthesia
|
40.0%
8/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Paronychia
|
10.0%
2/20 • 9 years
|
|
Eye disorders
Periorbital edema
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Pharyngeal mucositis
|
5.0%
1/20 • 9 years
|
|
General disorders
Photophobia
|
5.0%
1/20 • 9 years
|
|
General disorders
Photosensitivity
|
5.0%
1/20 • 9 years
|
|
Investigations
Platelet count decreased
|
45.0%
9/20 • 9 years
|
|
Investigations
Platelet Count increased
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonitis
|
5.0%
1/20 • 9 years
|
|
Investigations
Proteinuria
|
65.0%
13/20 • 9 years
|
|
Psychiatric disorders
Psychiatric disorders
|
10.0%
2/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
30.0%
6/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
15.0%
3/20 • 9 years
|
|
General disorders
Rectal fistula
|
5.0%
1/20 • 9 years
|
|
Renal and urinary disorders
Renal and urinary disorders
|
10.0%
2/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory, thoracic and mediastinal disorders
|
30.0%
6/20 • 9 years
|
|
Musculoskeletal and connective tissue disorders
Right hip cramp
|
5.0%
1/20 • 9 years
|
|
General disorders
Scalp pain
|
5.0%
1/20 • 9 years
|
|
Investigations
Serum amylase increased
|
15.0%
3/20 • 9 years
|
|
Cardiac disorders
Sinus Bradycardia
|
5.0%
1/20 • 9 years
|
|
Cardiac disorders
Sinus tachycardia
|
25.0%
5/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin and subcutaneous tissue disorders
|
75.0%
15/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin hyperpigmentation
|
5.0%
1/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin hypopigmentation
|
5.0%
1/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin induration
|
25.0%
5/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin infection
|
5.0%
1/20 • 9 years
|
|
Skin and subcutaneous tissue disorders
Skin ulceration
|
5.0%
1/20 • 9 years
|
|
General disorders
Somnolence
|
5.0%
1/20 • 9 years
|
|
General disorders
Sore throat
|
15.0%
3/20 • 9 years
|
|
Nervous system disorders
Stroke
|
5.0%
1/20 • 9 years
|
|
Surgical and medical procedures
Surgical and medical procedures
|
10.0%
2/20 • 9 years
|
|
Vascular disorders
Thromboembolic event
|
10.0%
2/20 • 9 years
|
|
General disorders
Toothache
|
25.0%
5/20 • 9 years
|
|
Investigations
U prot/Creat increased
|
5.0%
1/20 • 9 years
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory infection
|
10.0%
2/20 • 9 years
|
|
Renal and urinary disorders
Urinary frequency
|
5.0%
1/20 • 9 years
|
|
Renal and urinary disorders
Urinary incontinence
|
5.0%
1/20 • 9 years
|
|
Renal and urinary disorders
Urinary tract infection
|
10.0%
2/20 • 9 years
|
|
Renal and urinary disorders
Urine color change
|
5.0%
1/20 • 9 years
|
|
Vascular disorders
Vasculitis
|
5.0%
1/20 • 9 years
|
|
Metabolism and nutrition disorders
Vitamin D Deficiency
|
5.0%
1/20 • 9 years
|
|
Gastrointestinal disorders
Vomiting
|
40.0%
8/20 • 9 years
|
|
Eye disorders
Watering eyes
|
5.0%
1/20 • 9 years
|
|
Investigations
WBC count increased
|
5.0%
1/20 • 9 years
|
|
General disorders
Weight loss
|
85.0%
17/20 • 9 years
|
|
Investigations
White blood cell decreased
|
30.0%
6/20 • 9 years
|
Additional Information
Dr. Camilo Jimenez
The University of Texas MD Anderson Cancer Center
Phone: (713) 792-2841
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place