Trial Outcomes & Findings for Pilot Study of Cabozantinib for Recurrent or Progressive Central Nervous System Tumors in Children (NCT NCT02885324)
NCT ID: NCT02885324
Last Updated: 2022-10-06
Results Overview
To assess the number of participants with best response of CR, PR, SD or PD. Response criteria are assessed based on the product of the longest diameter and its longest perpendicular diameter. Complete response (CR): Disappearance of all target lesions Partial response (PR): ≥50% decrease in the sum of the products of the two perpendicular diameters of all target lesions (up to 5), taking as reference the initial baseline measurements Stable disease (SD): Neither sufficient decrease in the sum of the products of the two perpendicular diameters of all target lesions to qualify for PR, nor sufficient increase in a single target lesion to qualify for PD. Progressive Disease (PD): The appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.
TERMINATED
PHASE2
4 participants
6 months
2022-10-06
Participant Flow
Participant milestones
| Measure |
Cabozantinib
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Pilot Study of Cabozantinib for Recurrent or Progressive Central Nervous System Tumors in Children
Baseline characteristics by cohort
| Measure |
Cabozantinib
n=4 Participants
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Age, Categorical
<=18 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 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
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
3 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
|
4 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 monthsTo assess the number of participants with best response of CR, PR, SD or PD. Response criteria are assessed based on the product of the longest diameter and its longest perpendicular diameter. Complete response (CR): Disappearance of all target lesions Partial response (PR): ≥50% decrease in the sum of the products of the two perpendicular diameters of all target lesions (up to 5), taking as reference the initial baseline measurements Stable disease (SD): Neither sufficient decrease in the sum of the products of the two perpendicular diameters of all target lesions to qualify for PR, nor sufficient increase in a single target lesion to qualify for PD. Progressive Disease (PD): The appearance of one or more new lesions and/or unequivocal progression of existing non-target lesions.
Outcome measures
| Measure |
Cabozantinib
n=4 Participants
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Number of Participants With Disease Response
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 monthsTo assess the number of participants who survived at 6 months.
Outcome measures
| Measure |
Cabozantinib
n=4 Participants
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Overall Survival
|
2 Participants
|
SECONDARY outcome
Timeframe: 6 monthsTo assess the number of participants who had progression free survival at 6 months.
Outcome measures
| Measure |
Cabozantinib
n=4 Participants
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Number of Participants With Progression Free Survival
|
0 Participants
|
Adverse Events
Cabozantinib
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Cabozantinib
n=4 participants at risk
Cabozantininb will be taken daily at a dose of 40 mg/m2. Drug cycles will last 28 days and be continuous for up to 12 months of therapy on study.
Cabozantinib: Subjects will receive Cabozantinib
|
|---|---|
|
Endocrine disorders
Hypothyroidism
|
75.0%
3/4 • 6 months
|
|
Gastrointestinal disorders
Abdominal pain
|
50.0%
2/4 • 6 months
|
|
Psychiatric disorders
Agitation
|
25.0%
1/4 • 6 months
|
|
Hepatobiliary disorders
Alanine aminotransferase increased
|
25.0%
1/4 • 6 months
|
|
Metabolism and nutrition disorders
Anorexia
|
50.0%
2/4 • 6 months
|
|
Psychiatric disorders
Anxiety
|
25.0%
1/4 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
25.0%
1/4 • 6 months
|
|
Hepatobiliary disorders
Aspartate aminotransferase increased
|
50.0%
2/4 • 6 months
|
|
Hepatobiliary disorders
Blood bilirubin increased
|
25.0%
1/4 • 6 months
|
|
Eye disorders
Blurred vision
|
25.0%
1/4 • 6 months
|
|
Psychiatric disorders
Confusion
|
25.0%
1/4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
25.0%
1/4 • 6 months
|
|
Psychiatric disorders
Depressed level of consciousness
|
25.0%
1/4 • 6 months
|
|
Gastrointestinal disorders
Diarrhea
|
50.0%
2/4 • 6 months
|
|
Eye disorders
Dry eye
|
25.0%
1/4 • 6 months
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
25.0%
1/4 • 6 months
|
|
Psychiatric disorders
Dysarthria
|
25.0%
1/4 • 6 months
|
|
General disorders
Dysgeusia
|
25.0%
1/4 • 6 months
|
|
Gastrointestinal disorders
Dyspepsia
|
25.0%
1/4 • 6 months
|
|
Psychiatric disorders
Dysphasia
|
25.0%
1/4 • 6 months
|
|
Blood and lymphatic system disorders
Epistaxis
|
25.0%
1/4 • 6 months
|
|
Eye disorders
diplopia
|
25.0%
1/4 • 6 months
|
|
Eye disorders
right visual field deficit
|
25.0%
1/4 • 6 months
|
|
General disorders
Fatigue
|
75.0%
3/4 • 6 months
|
|
General disorders
Gait disturbance
|
50.0%
2/4 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Generalized muscle weakness
|
25.0%
1/4 • 6 months
|
|
General disorders
Headache
|
50.0%
2/4 • 6 months
|
|
Blood and lymphatic system disorders
Hemoglobin increased
|
25.0%
1/4 • 6 months
|
|
General disorders
Hemorrhoids
|
25.0%
1/4 • 6 months
|
|
Hepatobiliary disorders
jaundice
|
25.0%
1/4 • 6 months
|
|
Cardiac disorders
Hypertension
|
25.0%
1/4 • 6 months
|
|
General disorders
Hypoxia
|
25.0%
1/4 • 6 months
|
|
Injury, poisoning and procedural complications
stubbed toe
|
25.0%
1/4 • 6 months
|
|
General disorders
Intracranial hemorrhage
|
25.0%
1/4 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Laryngeal inflammation
|
25.0%
1/4 • 6 months
|
|
General disorders
Lipase increased
|
25.0%
1/4 • 6 months
|
|
Psychiatric disorders
Memory impairment
|
25.0%
1/4 • 6 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
50.0%
2/4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
25.0%
1/4 • 6 months
|
|
Gastrointestinal disorders
Nausea
|
25.0%
1/4 • 6 months
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Progressive Disease
|
25.0%
1/4 • 6 months
|
|
Nervous system disorders
unable to coordinate movements
|
25.0%
1/4 • 6 months
|
|
Injury, poisoning and procedural complications
Pain in extremity
|
50.0%
2/4 • 6 months
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysethesia syndrome
|
25.0%
1/4 • 6 months
|
|
Renal and urinary disorders
Proteinuria
|
50.0%
2/4 • 6 months
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
50.0%
2/4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory failure
|
25.0%
1/4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
respiratory secretions
|
25.0%
1/4 • 6 months
|
|
Respiratory, thoracic and mediastinal disorders
subject hospitalized in acute respiratory failure
|
25.0%
1/4 • 6 months
|
|
Nervous system disorders
Seizure
|
25.0%
1/4 • 6 months
|
|
General disorders
Serum amylase increased
|
25.0%
1/4 • 6 months
|
|
Skin and subcutaneous tissue disorders
hair color change
|
50.0%
2/4 • 6 months
|
|
Skin and subcutaneous tissue disorders
Skin infection
|
25.0%
1/4 • 6 months
|
|
General disorders
Somnolence
|
25.0%
1/4 • 6 months
|
|
Gastrointestinal disorders
Stomach pain
|
25.0%
1/4 • 6 months
|
|
Renal and urinary disorders
Urinary retention
|
25.0%
1/4 • 6 months
|
|
General disorders
Voice alteration
|
25.0%
1/4 • 6 months
|
|
Gastrointestinal disorders
Vomiting
|
25.0%
1/4 • 6 months
|
|
Metabolism and nutrition disorders
Weight loss
|
25.0%
1/4 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place