Trial Outcomes & Findings for Sorafenib in Children and Young Adults With Recurrent or Progressive Low-Grade Astrocytomas (NCT NCT01338857)
NCT ID: NCT01338857
Last Updated: 2017-02-17
Results Overview
To estimate the objective response rates to sorafenib in children and young adults with low-grade astrocytomas, including optic pathway gliomas.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
12 participants
Primary outcome timeframe
one year
Results posted on
2017-02-17
Participant Flow
Study opened to recruitment 4/13/2011 and ended on 4/24/2012. Recruitment took place at the NYU Hassenfeld Children's Center outpatient clinic.
Participants were not excluded from the trial before assignment to groups. Treatment was based on age and diagnosis of neurofibromatosis type I.
Participant milestones
| Measure |
Sorafenib (Nexavar)
Sorafenib will be administered orally BID (approximately every 12 hours). A cycle is 28 days w/o interruption between cycles. Patients may receive up to a total of 12 cycles provided that no off-protocol or off-study criteria are met.
Children/adolescents (\< 18 years of age, non-NF1): 200 mg/m2/dose PO twice daily (rounded to the nearest 50 mg increment) to a maximum of 400 mg PO twice daily
Adults (greater than or equal to 18 years of age, non-NF1): 400 mg PO twice daily
NF1 patients: 80mg/m2/dose PO 2x daily to max of 150mg PO 2x daily.
|
|---|---|
|
Overall Study
STARTED
|
12
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Sorafenib in Children and Young Adults With Recurrent or Progressive Low-Grade Astrocytomas
Baseline characteristics by cohort
| Measure |
Sorafenib (Nexavar)
n=12 Participants
Sorafenib will be administered orally BID (approximately every 12 hours). A cycle is 28 days w/o interruption between cycles. Patients may receive up to a total of 12 cycles provided that no off-protocol or off-study criteria are met.
Children/adolescents (\< 18 years of age, non-NF1): 200 mg/m2/dose PO twice daily (rounded to the nearest 50 mg increment) to a maximum of 400 mg PO twice daily
Adults (greater than or equal to 18 years of age, non-NF1): 400 mg PO twice daily
NF1 patients: 80mg/m2/dose PO 2x daily to max of 150mg PO 2x daily.
|
|---|---|
|
Age, Continuous
|
9.25 years
STANDARD_DEVIATION 3 • n=5 Participants
|
|
Gender
Female
|
6 Participants
n=5 Participants
|
|
Gender
Male
|
6 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
12 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: one yearPopulation: Study terminated and 1/12 participants completed and data was analyzed
To estimate the objective response rates to sorafenib in children and young adults with low-grade astrocytomas, including optic pathway gliomas.
Outcome measures
| Measure |
Sorafenib (Nexavar)
n=1 Participants
Sorafenib will be administered orally BID (approximately every 12 hours). A cycle is 28 days w/o interruption between cycles. Patients may receive up to a total of 12 cycles provided that no off-protocol or off-study criteria are met.
Children/adolescents (\< 18 years of age, non-NF1): 200 mg/m2/dose PO twice daily (rounded to the nearest 50 mg increment) to a maximum of 400 mg PO twice daily
Adults (greater than or equal to 18 years of age, non-NF1): 400 mg PO twice daily
NF1 patients: 80mg/m2/dose PO 2x daily to max of 150mg PO 2x daily.
|
|---|---|
|
Response Rate to Sorafenib
|
1 participants
|
PRIMARY outcome
Timeframe: MRIs performed after every 3rd 28-day cycle and off-studyPopulation: Study terminated and 1/12 participants completed and data was analyzed
Determination of tumor response (CR, PR, SD) will be defined based on the comparison of the baseline MRI performed at study entry to the subsequent MRI which demonstrated best response. PR will be defined by a \>15% decrease in tumor volume, as measured by 3D volumetric analysis.
Outcome measures
| Measure |
Sorafenib (Nexavar)
n=1 Participants
Sorafenib will be administered orally BID (approximately every 12 hours). A cycle is 28 days w/o interruption between cycles. Patients may receive up to a total of 12 cycles provided that no off-protocol or off-study criteria are met.
Children/adolescents (\< 18 years of age, non-NF1): 200 mg/m2/dose PO twice daily (rounded to the nearest 50 mg increment) to a maximum of 400 mg PO twice daily
Adults (greater than or equal to 18 years of age, non-NF1): 400 mg PO twice daily
NF1 patients: 80mg/m2/dose PO 2x daily to max of 150mg PO 2x daily.
|
|---|---|
|
Objective Response Rates
|
1 participants
|
Adverse Events
Sorafenib (Nexavar)
Serious events: 0 serious events
Other events: 12 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Sorafenib (Nexavar)
n=12 participants at risk
Sorafenib will be administered orally BID (approximately every 12 hours). A cycle is 28 days w/o interruption between cycles. Patients may receive up to a total of 12 cycles provided that no off-protocol or off-study criteria are met.
Children/adolescents (\< 18 years of age, non-NF1): 200 mg/m2/dose PO twice daily (rounded to the nearest 50 mg increment) to a maximum of 400 mg PO twice daily
Adults (greater than or equal to 18 years of age, non-NF1): 400 mg PO twice daily
NF1 patients: 80mg/m2/dose PO 2x daily to max of 150mg PO 2x daily.
|
|---|---|
|
Investigations
ALT increase
|
41.7%
5/12 • Number of events 6
|
|
Gastrointestinal disorders
anal hemorrhage
|
8.3%
1/12 • Number of events 1
|
|
Blood and lymphatic system disorders
anemia
|
16.7%
2/12 • Number of events 2
|
|
Metabolism and nutrition disorders
anorexia
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
ascites
|
8.3%
1/12 • Number of events 1
|
|
Investigations
AST elevation
|
66.7%
8/12 • Number of events 9
|
|
Gastrointestinal disorders
blood in stool
|
8.3%
1/12 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
cold symptoms (runny nose)
|
8.3%
1/12 • Number of events 1
|
|
Eye disorders
conjunctivitis
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
constipation
|
25.0%
3/12 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
cough
|
25.0%
3/12 • Number of events 3
|
|
Metabolism and nutrition disorders
decreased appetite
|
16.7%
2/12 • Number of events 2
|
|
Gastrointestinal disorders
diarrhea
|
58.3%
7/12 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
dry skin
|
50.0%
6/12 • Number of events 7
|
|
Gastrointestinal disorders
emesis
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
epistaxis
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
erythema
|
16.7%
2/12 • Number of events 5
|
|
Eye disorders
eye pain
|
8.3%
1/12 • Number of events 1
|
|
General disorders
fatigue
|
33.3%
4/12 • Number of events 5
|
|
General disorders
fever
|
8.3%
1/12 • Number of events 1
|
|
Vascular disorders
flushing (facial)
|
16.7%
2/12 • Number of events 2
|
|
Gastrointestinal disorders
gastric hemorrhage
|
8.3%
1/12 • Number of events 1
|
|
Investigations
GGT
|
8.3%
1/12 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
hand-foot skin reaction
|
33.3%
4/12 • Number of events 7
|
|
Nervous system disorders
headache
|
25.0%
3/12 • Number of events 3
|
|
Metabolism and nutrition disorders
hyperglycemia
|
33.3%
4/12 • Number of events 9
|
|
Skin and subcutaneous tissue disorders
hyperkeratotic eruptions
|
8.3%
1/12 • Number of events 4
|
|
Metabolism and nutrition disorders
hypernatremia
|
16.7%
2/12 • Number of events 2
|
|
Skin and subcutaneous tissue disorders
hyperpigmentation
|
8.3%
1/12 • Number of events 1
|
|
Vascular disorders
hypertension
|
16.7%
2/12 • Number of events 2
|
|
Metabolism and nutrition disorders
hypocalcemia
|
16.7%
2/12 • Number of events 3
|
|
Metabolism and nutrition disorders
hypoglycemia
|
16.7%
2/12 • Number of events 2
|
|
Metabolism and nutrition disorders
hypokalemia
|
25.0%
3/12 • Number of events 5
|
|
Metabolism and nutrition disorders
hyponatremia
|
41.7%
5/12 • Number of events 10
|
|
Metabolism and nutrition disorders
hypophosphatemia
|
41.7%
5/12 • Number of events 6
|
|
Skin and subcutaneous tissue disorders
hypopigmentation
|
8.3%
1/12 • Number of events 1
|
|
Vascular disorders
hypotension
|
8.3%
1/12 • Number of events 1
|
|
Nervous system disorders
intracranial pressure
|
8.3%
1/12 • Number of events 1
|
|
Nervous system disorders
lethargy
|
16.7%
2/12 • Number of events 2
|
|
Investigations
lymphocyte count decreased
|
16.7%
2/12 • Number of events 2
|
|
Investigations
lymphopenia
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
mucositis
|
8.3%
1/12 • Number of events 1
|
|
Musculoskeletal and connective tissue disorders
muscle weakness-right sided
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
nausea
|
16.7%
2/12 • Number of events 2
|
|
Investigations
neutrophil count decreased
|
25.0%
3/12 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
papulopustular rash
|
8.3%
1/12 • Number of events 1
|
|
Investigations
platelet count decreased
|
16.7%
2/12 • Number of events 2
|
|
Respiratory, thoracic and mediastinal disorders
postnasal drip
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
pruritus
|
8.3%
1/12 • Number of events 1
|
|
Eye disorders
ptosis (CN III)
|
8.3%
1/12 • Number of events 1
|
|
Investigations
PTT
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
rash
|
83.3%
10/12 • Number of events 13
|
|
Respiratory, thoracic and mediastinal disorders
respiratory failure
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
skin hyperpigmentation
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
skin hypopigmentation
|
8.3%
1/12 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
skin ulceration (nasal)
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
sore throat
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
tooth pain
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
upper respiratory infection
|
8.3%
1/12 • Number of events 1
|
|
Infections and infestations
UTI
|
8.3%
1/12 • Number of events 1
|
|
Injury, poisoning and procedural complications
vascular access complication
|
8.3%
1/12 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
voice alteration
|
8.3%
1/12 • Number of events 1
|
|
Gastrointestinal disorders
vomiting
|
41.7%
5/12 • Number of events 6
|
|
Eye disorders
watery and itchy eyes
|
8.3%
1/12 • Number of events 1
|
|
Investigations
white blood cell decreased
|
16.7%
2/12 • Number of events 2
|
Additional Information
Matthias A. Karajannis, MD
New York University Langone Medical Center
Phone: 212-263-9630
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place