Trial Outcomes & Findings for TKI 258 in Von Hippel-Lindau Syndrome (VHL) (NCT NCT01266070)
NCT ID: NCT01266070
Last Updated: 2017-02-13
Results Overview
Most frequent \& Most Serious Adverse Events: Safety of treatment with Dovitinib for 6 months in participants with VHL who have a measurable hemangioblastoma undergoing surveillance evaluated by toxicity scored using Common Toxicity Criteria (CTC) Version 4.0.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
6 participants
Primary outcome timeframe
Every 2 cycles (approximately 8 weeks) for 6 months
Results posted on
2017-02-13
Participant Flow
Recruitment Period: November 16, 2012 to January 30, 2014. All recruitment done at The University of Texas MD Anderson Cancer Center.
Participant milestones
| Measure |
Dovitinib
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
Overall Study
STARTED
|
6
|
|
Overall Study
COMPLETED
|
2
|
|
Overall Study
NOT COMPLETED
|
4
|
Reasons for withdrawal
| Measure |
Dovitinib
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Protocol Violation
|
1
|
Baseline Characteristics
TKI 258 in Von Hippel-Lindau Syndrome (VHL)
Baseline characteristics by cohort
| Measure |
Dovitinib
n=6 Participants
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
Age, Continuous
|
44 years
n=39 Participants
|
|
Gender
Female
|
1 Participants
n=39 Participants
|
|
Gender
Male
|
5 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
United States
|
6 participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Every 2 cycles (approximately 8 weeks) for 6 monthsMost frequent \& Most Serious Adverse Events: Safety of treatment with Dovitinib for 6 months in participants with VHL who have a measurable hemangioblastoma undergoing surveillance evaluated by toxicity scored using Common Toxicity Criteria (CTC) Version 4.0.
Outcome measures
| Measure |
Dovitinib
n=6 Participants
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Alanine aminotransferase increased
|
6 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Alkaline phosphatase increased
|
6 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Aspartate aminotransferase increased
|
6 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Diarrhea
|
5 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Dyspepsia
|
3 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
GGT Increased
|
5 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Hyperglycemia
|
4 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Hyperkalemia
|
4 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Nausea
|
6 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Rash maculo-papular
|
6 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
White Blood cell decreased
|
5 Participants
|
|
Most Frequent & Most Serious Adverse Events: Safety of Dovitinib for 6 Months
Mucositis oral
|
4 Participants
|
SECONDARY outcome
Timeframe: Every 2 cycles (approximately 8 weeks) for 6 monthsEfficacy of treatment with dovitinib for 6 months in patients with VHL who have a measurable lesion evaluated by response using RECIST (Response Evaluation Criteria in Solid Tumors): Complete Response, Partial Response, Progressive Disease or Stable Disease.
Outcome measures
| Measure |
Dovitinib
n=6 Participants
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
Number of VHL Participants With Response at 6 Months
|
0 Participants
|
Adverse Events
Dovitinib
Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Dovitinib
n=6 participants at risk
Dovitinib oral 500 mg/day on a 5 day on, 2 day off schedule (Days 1-5, 8-12, 15-19, and 22-26) of each 28-day cycle
|
|---|---|
|
General disorders
Abdominal pain
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Psychiatric disorders
Agitation
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Alanine aminotransferase increased
|
100.0%
6/6 • Number of events 12 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Alkaline phosphatase increased
|
100.0%
6/6 • Number of events 7 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Psychiatric disorders
Anxiety
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Aspartate aminotransferase increased
|
83.3%
5/6 • Number of events 5 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Eye disorders
Blurred vision
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Cholesterol high
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Creatinine increased
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Psychiatric disorders
Depression
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Gastrointestinal disorders
Diarrhea
|
100.0%
6/6 • Number of events 12 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Gastrointestinal disorders
Dyspepsia
|
50.0%
3/6 • Number of events 3 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
33.3%
2/6 • Number of events 3 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Reproductive system and breast disorders
Erectile dysfunction
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Infections and infestations
Eye infection
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
General disorders
Fatigue
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
GGT increased
|
83.3%
5/6 • Number of events 5 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Nervous system disorders
Headache
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
66.7%
4/6 • Number of events 4 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Metabolism and nutrition disorders
Hyperkalemia
|
66.7%
4/6 • Number of events 4 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Vascular disorders
Hypertension
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Metabolism and nutrition disorders
Hypertriglyceridemia
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Metabolism and nutrition disorders
Hypomagnesemia
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Lipase increased
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Lymphocyte count decreased
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Gastrointestinal disorders
Mucositis oral
|
66.7%
4/6 • Number of events 4 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Gastrointestinal disorders
Nausea
|
100.0%
6/6 • Number of events 9 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Neutrophil count decreased
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
General disorders
Pain (Dorsum of Left Foot)
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
General disorders
Pain (Tooth Pain)
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrome
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Psychiatric disorders
Personality change
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Platelet count decreased
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Skin and subcutaneous tissue disorders
Rash acneiform
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
100.0%
6/6 • Number of events 13 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
Serum amylase increased
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Infections and infestations
Tooth infection
|
16.7%
1/6 • Number of events 1 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Infections and infestations
Urinary tract infection
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
2/6 • Number of events 2 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
|
Investigations
White blood cell decreased
|
83.3%
5/6 • Number of events 5 • Adverse events collected through 28 day treatment cycle, up to 6 months.
|
Additional Information
Eric Jonasch, MD/Professor, Genitourinary Medical Oncology
The University of Texas (UT) MD Anderson Cancer Center
Phone: 713-792-7734
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place